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FVIII 억제제가 없는 경증 또는 중등도 혈우병 A 환자에서 Emicizumab의 안전성, 효능, 약동학 및 약력학을 평가하기 위한 연구 (HAVEN 6)

2026년 8월 5일 업데이트: Hoffmann-La Roche

FVIII 억제제가 없는 경증 또는 중등도 혈우병 A 환자에서 Emicizumab의 안전성, 효능, 약동학 및 약력학을 평가하기 위한 다기관, 공개 라벨 연구

이는 인자 VIII(FVIII)에 대한 억제제가 없는 경증 또는 중등도 혈우병 A 참가자를 대상으로 에미시주맙의 안전성, 효능, 약동학 및 약력학을 평가하기 위해 설계된 다기관, 공개 라벨, 단일군 연구입니다.

연구 개요

상태

완전한

정황

개입 / 치료

연구 유형

중재적

등록 (실제)

73

단계

  • 3단계

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 장소

  • 남아프리카
      • Johannesburg, 남아프리카, 2193
        • Charlotte Maxeke Johannesburg Hospital
  • 네덜란드
      • Amsterdam, 네덜란드, 1105 AZ
        • Amsterdam UMC Location AMC
  • 독일
      • Bonn, 독일, 53127
        • Universitatsklinikum Bonn
      • München, 독일, 80336
        • Klinikum der Universität München, Campus Innenstadt
  • 미국
    • California
      • Los Angeles, California, 미국, 90027
        • Childrens Hospital LA
    • Georgia
      • Atlanta, Georgia, 미국, 30308
        • Hemophilia of Georgia Center for Bleeding & Clotting Disorders
    • Indiana
      • Indianapolis, Indiana, 미국, 46260
        • Indiana Hemophilia & Thrombosis center
    • Michigan
      • Ann Arbor, Michigan, 미국, 48109
        • University of Michigan, C.S. Mott Children's Hospital
    • Washington
      • Seattle, Washington, 미국, 98101
        • Washington Institute for Coagulation
  • 벨기에
      • Brussels, 벨기에, 1200
        • Cliniques Universitaires St-Luc
      • Leuven, 벨기에, 3000
        • UZ Leuven Gasthuisberg
  • 스페인
      • Madrid, 스페인, 28046
        • Hospital Universitario La Paz
      • Seville, 스페인, 41013
        • Hospital Universitario Virgen Del Rocio
  • 영국
      • Cardiff, 영국, CF14 4XW
        • Cardiff and Vale NHS Trust
      • London, 영국, NW3 2QG
        • Royal Free Hospital
      • London, 영국, WC1N 3HR
        • Great Ormond Street Hospital for Children NHS Foundation Trust
  • 캐나다
    • Alberta
      • Edmonton, Alberta, 캐나다, T6G 1Z1
        • Kaye Edmonton Clinic
    • Newfoundland and Labrador
      • St. John's, Newfoundland and Labrador, 캐나다, A1B 3V6
        • Eastern Health - General Hospital
  • 폴란드
      • Warsaw, 폴란드, 02-776
        • Instytut Hematologii i Transfuzjologii
  • 프랑스
      • Bron, 프랑스, 69677
        • Hopital Cardio-vasculaire Louis Pradel
      • Le Kremlin-Bicêtre, 프랑스, 94275
        • CH de Bicêtre
      • Paris, 프랑스, 75015
        • Groupe Hospitalier Necker Enfants Malades

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

  • 어린이
  • 성인
  • 고령자

건강한 자원 봉사자를 받아들입니다

아니

설명

포함 기준:

  • 경미한 진단(FVIII 수준 >5% ~
  • 무게 ≥3kg(kg)
  • 연구자 평가에 근거한 예방 조치의 필요성
  • 억제제에 대한 음성 테스트(즉,
  • 문서화된 억제제 없음(즉,
  • 등록 전 마지막 24주 동안의 예방적 또는 간헐적 FVIII 치료 및 출혈 횟수에 대한 세부 사항 문서화
  • 적절한 혈액학적 간 및 신장 기능
  • 가임 여성의 경우: 치료 기간 동안 및 연구 약물의 최종 투여 후 최소 24주 동안 금욕을 유지하거나 피임법(프로토콜에 정의됨)을 사용하는 데 동의

제외 기준:

  • 경증 또는 중등도 선천성 혈우병 A 이외의 유전성 또는 후천성 출혈 장애
  • 검사자의 판단에 따라 스크리닝 전 48주 이내에 불법 약물 또는 알코올 남용 이력
  • 혈전색전증 또는 혈전색전증 징후에 대한 이전(지난 12개월 이내) 또는 현재 치료
  • 현재 출혈 또는 혈전증의 위험을 증가시킬 수 있는 기타 상태
  • 단클론 항체 요법 또는 에미시주맙 주사 성분과 관련된 임상적으로 유의미한 과민증의 병력
  • 에미시주맙 로딩 용량 단계 동안 계획된 수술(5주차부터 에미시주맙 참가자의 수술은 허용됨)
  • CD4 수치가 있는 알려진 HIV 감염
  • 수반되는 질병, 상태, 스크리닝 평가 또는 실험실 검사에서의 유의미한 이상 또는 연구 수행을 방해할 수 있는 치료, 또는 연구자의 의견으로는 참가자에게 연구 약물을 투여하는 데 허용할 수 없는 추가 위험을 제기하는 치료
  • 다음 중 하나의 수령: 사전 에미시주맙 예방을 제외하고 마지막 약물 투여의 5 반감기 이내에 혈우병 출혈의 위험을 치료하거나 감소시키기 위한 연구 약물; 지난 30일 이내 또는 반감기 5일 이내의 비혈우병 관련 시험용 약물 중 더 짧은 기간 또는 현재 투여 중이거나 투여 예정인 기타 임상시험용 약물
  • 조사자의 의견으로는 연구 프로토콜을 준수할 수 없음
  • 임신 중이거나 모유 수유 중이거나 연구 기간 동안 임신을 계획 중인 여성(가임 여성은 연구 약물 투여 시작 전 7일 이내에 혈청 임신 검사 결과가 음성이어야 함)

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 해당 없음
  • 중재 모델: 단일 그룹 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: 에미시주맙
제8인자(FVIII) 억제제가 없는 경증 및 중등도 혈우병 A 참가자는 에미시주맙 부하 용량 요법을 받은 후 참가자가 선호하는 3가지 유지 용량 요법 중 하나를 받도록 등록됩니다.
의약품: 에미시주맙
체중 1kg당 3밀리그램(mg/kg)의 에미시주맙 4회 부하 용량을 4주 동안 주 1회(QW) 피하(SC) 투여한 후 참가자는 다음 3가지 유지 SC 용량 요법 중 하나를 선호합니다: 1.5mg /kg QW, 2주에 한 번(Q2W) 3 mg/kg 또는 4주에 한 번(Q4W) 6 mg/kg.
다른 이름들:
  • 헴리브라
  • RO5534262
  • RG6013
  • 에이스910

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
치료된 출혈에 대한 모델 기반 연간 출혈 비율
기간: 첫 번째 에미시주맙 투여일부터 에미시주맙 치료 최소 52주까지 (중앙값[범위, 최소-최대] 효능 기간: 55.64[8.6-89.9]주)
효능 기간 동안 처리된 출혈의 수는 다양한 추적 시간을 설명하는 음의 이항 회귀 모델을 사용하여 연간 출혈률(ABR)로 추정되었습니다. 치료된 출혈은 "출혈 치료"로 보고된 혈우병 약물이 바로 뒤따른 출혈로 정의되었습니다. 72시간 규칙이 시행되었습니다. 첫 번째 출혈에 대한 마지막 치료로부터 72시간 이내에 두 번째 출혈이 발생한 경우 동일한 유형 및 동일한 해부학적 위치에서 두 번의 출혈이 한 번의 출혈로 계산되었습니다. 수술/시술로 인한 출혈은 제외되었습니다.
첫 번째 에미시주맙 투여일부터 에미시주맙 치료 최소 52주까지 (중앙값[범위, 최소-최대] 효능 기간: 55.64[8.6-89.9]주)
Mean Calculated ABR for Treated Bleeds
기간: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Median Calculated ABR for Treated Bleeds
기간: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)

2차 결과 측정

결과 측정
측정값 설명
기간
치료된 자연 출혈에 대한 평균 계산된 연간 출혈률
기간: 첫 번째 에미시주맙 투여일부터 에미시주맙 치료 최소 52주까지 (중앙값[범위, 최소-최대] 효능 기간: 55.64[8.6-89.9]주)
유효성 기간 동안 처리된 자연 출혈의 수는 다음 공식을 사용하여 각 참가자에 대해 연간으로 환산한 계산된 연간 출혈률(ABR)로 여기에 표시됩니다. ABR = (유효 기간 동안의 출혈 수/일수) x 365.25 . 치료된 자발 출혈은 외상이나 절차/수술과 같은 다른 알려진 기여 요인이 없는 치료된 출혈("출혈 치료"로 보고된 혈우병 약물이 바로 뒤따르는 출혈)으로 정의되었습니다. 72시간 규칙이 시행되었습니다. 첫 번째 출혈에 대한 마지막 치료로부터 72시간 이내에 두 번째 출혈이 발생한 경우 동일한 유형 및 동일한 해부학적 위치에서 두 번의 출혈이 한 번의 출혈로 계산되었습니다. 수술/시술로 인한 출혈은 제외되었습니다.
첫 번째 에미시주맙 투여일부터 에미시주맙 치료 최소 52주까지 (중앙값[범위, 최소-최대] 효능 기간: 55.64[8.6-89.9]주)
치료된 자연 출혈에 대해 계산된 연간 출혈률 중앙값
기간: 첫 번째 에미시주맙 투여일부터 에미시주맙 치료 최소 52주까지 (중앙값[범위, 최소-최대] 효능 기간: 55.64[8.6-89.9]주)
유효성 기간 동안 처리된 자연 출혈의 수는 다음 공식을 사용하여 각 참가자에 대해 연간으로 환산한 계산된 연간 출혈률(ABR)로 여기에 표시됩니다. ABR = (유효 기간 동안의 출혈 수/일수) x 365.25 . 치료된 자발 출혈은 외상이나 절차/수술과 같은 다른 알려진 기여 요인이 없는 치료된 출혈("출혈 치료"로 보고된 혈우병 약물이 바로 뒤따르는 출혈)으로 정의되었습니다. 72시간 규칙이 시행되었습니다. 첫 번째 출혈에 대한 마지막 치료로부터 72시간 이내에 두 번째 출혈이 발생한 경우 동일한 유형 및 동일한 해부학적 위치에서 두 번의 출혈이 한 번의 출혈로 계산되었습니다. 수술/시술로 인한 출혈은 제외되었습니다.
첫 번째 에미시주맙 투여일부터 에미시주맙 치료 최소 52주까지 (중앙값[범위, 최소-최대] 효능 기간: 55.64[8.6-89.9]주)
17주차에 Emicizumab 선호도 조사를 통해 평가한 바와 같이 Emicizumab SC 치료, 이전 혈우병 IV 치료를 선호하거나 선호하지 않는 참가자 비율
기간: 17주차
에미시주맙 선호도 조사는 피하(SC) 에미시주맙, 정맥내(IV) 인자 VIIII(FVIII) 또는 선호하지 않는 치료에 대한 참가자의 선호도를 기록하기 위해 스폰서가 개발한 목적에 맞는 설문지입니다. 95% 신뢰 구간은 Pearson-Clopper 방법을 사용하여 계산되었습니다.
17주차
17주차에 Emicizumab 선호도 조사를 통해 평가한 바와 같이 Emicizumab SC 치료, 자녀의 이전 혈우병 IV 치료를 선호하거나 선호하지 않는 간병인의 비율
기간: 17주차
Emicizumab 선호도 조사는 피하(SC) emicizumab, 정맥(IV) 인자 VIIII(FVIII) 또는 선호하지 않는 치료에 대한 간병인의 선호도를 기록하기 위해 스폰서가 개발한 목적에 맞는 설문지입니다. 95% 신뢰 구간은 Pearson-Clopper 방법을 사용하여 계산되었습니다.
17주차
시간 경과에 따른 일일 평균 최대 활동 기간의 기준선에서 변경
기간: 기준선(1-2주) 및 13주(12-13주 기간), 25주(24-25주 기간), 37주(36-37주 기간) 및 49주(48-49주 기간)
신체 활동 평가를 위해 5세 이상의 참가자는 연구 기간 동안 지정된 2주 동안 매일 지속적으로(24시간/일) 손목에 연구 가속도 측정 장치를 착용하도록 지시받았습니다. 참가자는 연구 기간 동안 지정된 각 2주 기간 중 최소 8일 동안 매일 지속적으로(≥8시간/일) 연구 장치를 착용한 경우 신체 활동 평가를 준수하는 것으로 간주되었습니다. 특정 시점에서 이 준수 기준에 도달하지 못한 경우 참가자는 준수에 도달하지 못한 지정된 2주 기간의 분석에 포함되지 않았습니다. 일일 측정은 14일 시점에 걸쳐 평균을 냈습니다. 활동 횟수는 장치에서 측정한 가속도의 척도입니다. 일일 최대 활동 시간은 하루 중 중간에서 격렬한 활동의 ​​합계(분 단위)로 정의되었습니다.
기준선(1-2주) 및 13주(12-13주 기간), 25주(24-25주 기간), 37주(36-37주 기간) 및 49주(48-49주 기간)
시간 경과에 따른 평균 일일 걸음 수의 기준선에서 변경
기간: 기준선(1-2주) 및 13주(12-13주), 25주(24-25주), 37주(36-37주) 및 49주(48-49주)
신체 활동 평가를 위해 5세 이상의 참가자는 연구 기간 동안 지정된 2주 동안 매일 지속적으로(24시간/일) 손목에 연구 가속도 측정 장치를 착용하도록 지시받았습니다. 참가자는 연구 기간 동안 지정된 각 2주 기간 중 최소 8일 동안 매일 지속적으로(≥8시간/일) 연구 장치를 착용한 경우 신체 활동 평가를 준수하는 것으로 간주되었습니다. 특정 시점에서 이 준수 기준에 도달하지 못한 경우 참가자는 준수에 도달하지 못한 지정된 2주 기간의 분석에 포함되지 않았습니다. 일일 측정은 14일 시점에 걸쳐 평균을 냈습니다. 활동 횟수는 장치에서 측정한 가속도의 척도입니다.
기준선(1-2주) 및 13주(12-13주), 25주(24-25주), 37주(36-37주) 및 49주(48-49주)
Model-Based ABR for All Bleeds
기간: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of all bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Mean Calculated ABR for All Bleeds
기간: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Median Calculated ABR for All Bleeds
기간: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Model-Based ABR for Treated Joint Bleeds
기간: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of treated joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Mean Calculated ABR for Treated Joint Bleeds
기간: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Median Calculated ABR for Treated Joint Bleeds
기간: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Model-Based ABR for Treated Target Joint Bleeds
기간: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of treated target joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Mean Calculated ABR for Treated Target Joint Bleeds
기간: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Median Calculated ABR for Treated Target Joint Bleeds
기간: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Model-Based ABR for Treated Spontaneous Bleeds
기간: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
The number of treated spontaneous bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times. A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Bleeds
기간: Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Bleeds
기간: Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Bleeds
기간: Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Model-Based ABR for All Bleeds
기간: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of all bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Mean Calculated ABR for All Bleeds
기간: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy: Median Calculated ABR for All Bleeds
기간: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Joint Bleeds
기간: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Joint Bleeds
기간: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Joint Bleeds
기간: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Target Joint Bleeds
기간: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated target joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Target Joint Bleeds
기간: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Target Joint Bleeds
기간: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Spontaneous Bleeds
기간: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated spontaneous bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times. A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Spontaneous Bleeds
기간: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated spontaneous bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Spontaneous Bleeds
기간: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
The number of treated spontaneous bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
CATCH Questionnaire for Adult Participants: Change From Baseline in the Daily Activity Risk Perception (RP) and Impact Domain Scores Over Time
기간: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and Study completion (SC)/Early discontinuation (ED) (up to approximately 274 weeks)
Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, the daily activity RP and impact domain has 48 items with each item scored on 4 or 5 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation. The transformed scores range from 0-100 scale for both the RP and Impact. Higher scores indicating higher perceived risk of having a bleed while doing daily activities and higher impact of hemophilia on daily activities.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and Study completion (SC)/Early discontinuation (ED) (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Change From Baseline in the Social Activity RP and Impact Domain Scores Over Time
기간: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, the social activity RP and impact domain has 22 items with each item scored on 4 or 5 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation. The transformed scores range from 0-100 scale for both the RP and Impact. Higher scores indicating higher perceived risk of having a bleed while doing social activities and higher impact of hemophilia on social activities.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Change From Baseline in the Recreational Activity RP and Impact Domain Scores Over Time
기간: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, the recreational activity RP and impact domain has 32 items and a larger bank of additional items with each item scored on 4 or 5 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation. The transformed scores range from 0-100 scale for both the RP and Impact. Higher scores indicating higher perceived risk of having a bleed while doing recreational activities and higher impact of hemophilia on recreational activities.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Change From Baseline in the Work Impact (WI) and College Impact (CI) Domains Score Over Time
기간: WI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks); CI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, work and college impact has 10 items and the items were scored on 4 or 5 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated impact of hemophilia on work and college activities.
WI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks); CI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Change From Baseline in the Preoccupation Domain Score Over Time
기간: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, preoccupation has 10 items which were scored on 4 or 5 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated greater preoccupation related to hemophilia.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Change From Baseline in the Treatment Burden Domain Score Over Time
기간: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, treatment burden has 8 items which were scored on 4 or 5 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated greater perceived burden of the hemophilia treatment.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain Associated With a Bleed Over Time
기간: Baseline, Weeks 25, 49, 61, 85, 121, 133, 157 and 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, the pain domain consisting of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine. The data reported here are from participants who answered the following question: "Did you get a bleed in the past 7 days?" If the response was yes, participants were asked to rate their pain by selecting the number that best described the pain associated with the bleed. Data is reported only for categories/timepoints with non-zero values.
Baseline, Weeks 25, 49, 61, 85, 121, 133, 157 and 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain in Target Joints Over Time
기간: Baseline, Weeks 1, 13, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, the pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine. The data reported here are from participants who answered the following question "Do you have target joints?" (If yes, please rate your pain by picking the number that best describes your pain in your target joint(s) over the past 7 days). Data is reported only for categories/timepoints with non-zero values.
Baseline, Weeks 1, 13, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain at Its Worst Over Time
기간: Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine. The data reported here are from participants who answered the following question "Please rate your pain by picking the number that best describes your pain at its WORST over the past 7 days". Data is reported only for categories/timepoints with non-zero values.
Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain at Its Least Over Time
기간: Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine. The data reported here are from participants who answered the following question "Please rate your pain by picking the number that best describes your pain at its LEAST over the past 7 days." Data is reported only for categories/timepoints with non-zero values.
Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain at Its Least on the Average Over Time
기간: Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years). The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain. Of this, the pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine. The data reported here are from participants who answered the following question "Please rate your pain by picking the number that best describes your pain at its average over the past 7 days". Data is reported only for categories/timepoints with non-zero values.
Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Daily Activity RP and Impact Domain Score Over Time
기간: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years). The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain). Of this, daily activity RP and impact has 38 items with each item scored on 3 or 4 points ordinal scale. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicating higher perceived risk of having a bleed while doing daily activities and higher impact of hemophilia on daily activities.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Social Activity Risk Perception and Impact Domain Score Over Time
기간: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years). The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain). Of this, social activity RP and impact has 16 items which were scored on 3 or 4 points ordinal scale. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated higher perceived risk of having a bleed while doing social activities and higher impact of hemophilia on social activities.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Recreational Activity RP and Impact Domain Score Over Time
기간: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years). The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain. Of this, recreational activity RP & impact has 34 items + larger bank of additional items. The items were scored on 3 or 4 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated higher perceived risk of having a bleed while doing recreational activities and higher impact of hemophilia on recreational activities.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Pediatric Participants: Change From Baseline in the School Impact Domain Score Over Time
기간: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years). The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain. Of this, school impact has 11 items and the items were scored on 3 or 4 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated higher impact of hemophilia on school activities.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Preoccupation Domain Score Over Time
기간: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years). The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain. Of this, preoccupation has 3 items which were scored on 3 or 4 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated greater preoccupation related to hemophilia.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Treatment Burden Domain Score Over Time
기간: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years). The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain. Of this, treatment burden has 7 items which were scored on 3 or 4 points ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated lower perceived burden of the hemophilia treatment.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Pediatric Participants: Number of Participants by Responses to Their Level of Hurt Associated With a Bleed Over Time
기간: Baseline, Weeks 13, 25, 37, 49, 61, 85, 97, 109, 121, 133, 145 and 157
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years). The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain). Of this, pain domain (Hurt) consists of two items, assessed using an 11-point numeric rating scale where 0 = no hurt and 10 = a lot of hurt. The data reported here are from participants who answered the following question "Did you get a bleed in the past 7 days? ( If yes, how much did the bleed hurt?)." Data is reported only for categories/timepoints with non-zero values.
Baseline, Weeks 13, 25, 37, 49, 61, 85, 97, 109, 121, 133, 145 and 157
CATCH Questionnaire for Pediatric Participants: Number of Participants by Responses to Their Level of Hurt at Its Worst Over Time
기간: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145,157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years). The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain. Of this, pain domain (Hurt) consists of two items, assessed using an 11-point numeric rating scale where 0 = no hurt and 10 = a lot of hurt. The data reported here are from participants who answered the following question "What is the WORST that your body has hurt over the past 7 days". Data is reported only for categories/timepoints with non-zero values.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145,157, 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Caregivers: Change From Baseline in the Preoccupation Domain Score Over Time
기간: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on caregivers of pediatric participants (aged <8 years). The caregiver version has 2 domains (preoccupation and treatment burden). Of this, preoccupation has 13 items with each of the items were scored on 4 or 5 ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated greater preoccupation related to hemophilia.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH Questionnaire for Caregivers: Change From Baseline in the Treatment Burden Domain Score Over Time
기간: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
CATCH was used to measure the effect of hemophilia and its treatment on caregivers of pediatric participants (aged <8 years). The caregiver version has 2 domains (preoccupation and treatment burden). Of this, treatment burden has 8 items which were scored on 4 or 5 ordinal scales. Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation. The transformed scores range from 0-100 scale. Higher scores indicated greater perceived burden of the hemophilia treatment.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
Change From Baseline in Hemophilia Joint Health Scores Over Time
기간: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
The HJHS measures joint health of the joints (knees, ankles, and elbows) most commonly affected by bleeding in hemophilia. The HJHS 2.1 provides joint specific total scores which are added to obtain the sum of joints totals and also a global gait score. These two scores are then added to obtain HJHS total score. The minimum score per joint is 0, the maximum score is 20. In addition, Gait is scored on a scale from 0 to 4 based on the number of skills that are not within the normal limits. The total score is the sum of scores across all six joints plus the gait score (range from 0 to 124, with 0 being normal and 124 being the most severe disease). A higher score indicates worse joint health.
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
Menstrual Bleed Questionnaire (MBQ) for Female Participants of Childbearing Potential: Change From Baseline in the MBQ Total Score Over Time
기간: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and145
The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL). The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item). The MBQ score ranges from 0 to 75. Higher scores indicate a worse quality of life and more severe symptoms.
Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and145
MBQ for Female Participants of Childbearing Potential: Change From Baseline in the Heaviness Subscale Score Over Time
기간: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137 (up to approximately 274 weeks)
The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL). The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item). The total score for heaviness subscale ranges from 0 to 29. Higher score indicates more heavy bleeding.
Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137 (up to approximately 274 weeks)
MBQ for Female Participants of Childbearing Potential: Change From Baseline in the QoL Subscale Score Over Time
기간: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL). The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item). The total score for QoL subscale ranges from 0 to 37. Higher scores indicate a worse QoL.
Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
MBQ for Female Participants of Childbearing Potential: Change From Baseline in the Irregularity Subscale Score Over Time
기간: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL). The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item). The total score for irregularity subscale ranges from 0 to 6. Higher score indicates more irregularity.
Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
MBQ for Female Participants of Childbearing Potential: Change From Baseline in the Pain Subscale Score Over Time
기간: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137
The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL). The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item). The total score for pain subscale ranges from 0 to 3. Higher score indicates severe pain.
Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137
Menstruation Diary With the Pictorial Blood Assessment Chart (PBAC) for Female Participants of Childbearing Potential: PBAC Scores Over Time
기간: Baseline and monthly (on days of menstruation) until Study Completion (up to approximately 247 weeks)
The Menstruation Diary used a PBAC, which has shown good correlation with menstrual blood loss. The PBAC is a chart that records the occurrence and size of clots, and the number of episodes of heavy bleeding (flooding), as well as depicts the amount of blood loss during a cycle.The PBAC records pad and tampon use (as either light [1 point], medium [5 points], or heavy [10 points] flow), clots (small [1 point] or large [5 points]), and flooding episodes (1 point each) which can be recorded as many times as necessary any day of the month. The PBAC is scored from 0 (no bleeding) onwards, with a score of >100 defining abnormal coagulation and heavy menstrual bleeding (corresponds to >80ml of blood loss per menstrual cycle).
Baseline and monthly (on days of menstruation) until Study Completion (up to approximately 247 weeks)
Number of Participants With at Least One Adverse Event by Severity, According to the World Health Organization (WHO) Toxicity Grading Scale
기간: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. As per WHO toxicity grading, Grade 1= mild, 2= moderate, 3=severe, 4=life threatening.
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
Number of Participants With Adverse Events Leading to Study Drug Discontinuation
기간: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
Number of Participants With at Least One Thromboembolic Event
기간: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. The following signs and symptoms were recognized as potential thromboembolism (i.e., dyspnea, chest pain, leg pain, or swelling; or if in the head, headache, numbness in the face, eye pain or swelling, or vision impairment).
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
Number of Participants With at Least One Event of Thrombotic Microangiopathy
기간: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. TMA is used to describe a group of disorders with clinical features of microangiopathic hemolytic anemia, thrombocytopenia, and organ damage that can include the kidneys, gastrointestinal system, central nervous system, etc.
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
Number of Participants With at Least One Injection-Site Reaction by Severity, According to the WHO Toxicity Grading Scale
기간: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Injection-site reactions are defined as AEs related that occur within 24 hours after study drug administration and are judged to be related to the study drug injection. Local injection-site reactions included erythema, hematoma, rash, discomfort, pain, and pruritus and were mostly of mild and moderate intensity. As per WHO toxicity grading, Grade 1= mild, 2= moderate, 3=severe, 4=life threatening.
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
Number of Participants With at Least One Severe Hypersensitivity, Anaphylaxis, and Anaphylactoid Event
기간: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
Number of Participants With at Least One Shifts in Clinical Laboratory Parameters From Baseline WHO Toxicity Scale Grade 0-2 to Post-baseline Grade 3 or 4
기간: Up to approximately 274 weeks
An abnormal laboratory value was defined as a laboratory test result outside of the normal range for hematology or serum chemistry parameters. The WHO toxicity grading scale, which ranges from Grades 1 to 4 (least severe to most severe, respectively; Grade 0 is within normal range), was used for assessing the severity of laboratory abnormalities and adverse events (WHO 2003). Not every laboratory abnormality qualified as an adverse event; an abnormality was reported as an adverse event if it met any of the following criteria: was accompanied by clinical symptoms; resulted in a change in study treatment (e.g., dosage modification, treatment interruption or discontinuation); resulted in a medical intervention or a change in concomitant therapy; or was clinically significant in the investigator's judgment.
Up to approximately 274 weeks
Number of Participants With at Least One Adverse Events of Changes From Baseline in Vital Signs
기간: Up to approximately 274 weeks
The number of participants with adverse events of changes from baseline in vital signs is reported here. Vital signs measurements consisted of heart and respiratory rate, temperature, and systolic and diastolic blood pressures, with an abnormal vital sign value being outside of the normal range. An abnormal vital sign result was reported as an adverse event if it met any of the following criteria: was accompanied by clinical symptoms; resulted in a change in study treatment (e.g., dosage modification, treatment interruption or discontinuation); resulted in a medical intervention or a change in concomitant therapy; or was clinically significant in the investigator's judgment. All of the adverse events reported here were assessed independently by the investigator as not related to treatment with emicizumab.
Up to approximately 274 weeks
Change From Baseline in Electrocardiogram (ECG) Parameters Over Time: QT, QTcB, QTcF, RR, PR, and QRS Intervals
기간: Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
Change From Baseline in Heart Rate Over Time, as Measured by ECG
기간: Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
Plasma Trough Concentration (Ctrough) of Emicizumab Over Time
기간: Pre-dose at Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and 181 (up to approximately 274 weeks)
Pre-dose at Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and 181 (up to approximately 274 weeks)
Number of Participants With Anti-Drug Antibodies Against Emicizumab at Baseline and Post-Baseline
기간: Baseline, Weeks 5, 13, 25, 33, 41, 49, 61, 73, 85, 97, 109, 121, 133, 145, 169, 181 and SC/ED (up to approximately 274 weeks)
Participants were considered to be ADA positive if they were ADA negative at baseline but develop an ADA response following study drug administration (treatment-induced ADA response), or if they were ADA positive at baseline and the titer of one or more post-baseline samples is at least 4-fold greater than the titer of the baseline sample (treatment-enhanced ADA response). The sum of all visits has been reported here.
Baseline, Weeks 5, 13, 25, 33, 41, 49, 61, 73, 85, 97, 109, 121, 133, 145, 169, 181 and SC/ED (up to approximately 274 weeks)
Number of Participants Who Develop Anti-FVIII Inhibitors Over Time
기간: Up to approximately 274 weeks
Central laboratory measurement of anti-FVIII inhibitory antibodies (inhibitors) was performed using a Chromogenic Bethesda Assay (CBA). Participants who develop anti-FVIII inhibitors (titer ≥ 0.6 Bethesda units per milliliter [BU/mL]) following study drug administration were summarized.
Up to approximately 274 weeks

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

Hoffmann-La Roche

수사관

  • 연구 책임자: Clinical Trials, Hoffmann-La Roche

간행물 및 유용한 링크

연구에 대한 정보 입력을 담당하는 사람이 자발적으로 이러한 간행물을 제공합니다. 이것은 연구와 관련된 모든 것에 관한 것일 수 있습니다.

일반 간행물

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (실제)

2020년 2월 10일

기본 완료 (실제)

2021년 10월 30일

연구 완료 (실제)

2025년 12월 19일

연구 등록 날짜

최초 제출

2019년 11월 7일

QC 기준을 충족하는 최초 제출

2019년 11월 7일

처음 게시됨 (실제)

2019년 11월 12일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 8월 27일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 8월 5일

마지막으로 확인됨

2026년 8월 1일

추가 정보

이 연구와 관련된 용어

키워드

추가 관련 MeSH 약관

기타 연구 ID 번호

  • BO41423
  • 2019 (미국 NIH 보조금/계약: Chief Medical Office (CMO) Alberta Health Services)
  • 2023 (미국 NIH 보조금/계약: GRAMMY Museum Foundation)
  • 2019-002179-32 (EudraCT 번호)
  • 2023-506610-52-00 (레지스트리 식별자: EU CT Number)

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

예

IPD 계획 설명

자격을 갖춘 연구원은 요청 플랫폼(www.vivli.org)을 통해 개별 환자 수준 데이터에 대한 액세스를 요청할 수 있습니다. 적격 연구에 대한 Roche의 기준에 대한 자세한 내용은 여기(https://vivli.org/ourmember/roche/)에서 확인할 수 있습니다.

Roche의 임상 연구 정보 공유에 대한 글로벌 정책 및 관련 임상 연구 문서에 대한 액세스 요청 방법에 대한 자세한 내용은 여기(https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm)를 참조하십시오.

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

예

미국 FDA 규제 기기 제품 연구

아니

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

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