이 페이지는 자동 번역되었으며 번역의 정확성을 보장하지 않습니다. 참조하십시오 영문판 원본 텍스트의 경우.

Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B (B-YOND)

2020년 12월 16일 업데이트: Bioverativ Therapeutics Inc.

An Open-Label, Multicenter, Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor IX Fusion Protein (rFIXFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia B

The primary objective of the study is to evaluate the long-term safety of rFIXFc in participants with hemophilia B.

The secondary objective of this study is to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes.

연구 개요

상태

완전한

정황

개입 / 치료

상세 설명

Participants will follow either a prophylaxis or on-demand regimen. The starting dose in this study will be determined by the clinical profile of the patient in the preceding studies, B-LONG 998HB102 (NCT01027364) and Kids B-LONG study 9HB02PED (NCT01440946)

연구 유형

중재적

등록 (실제)

120

단계

  • 3단계

연락처 및 위치

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

연구 장소

  • 남아프리카
    • Gauteng
      • Johannesburg, Gauteng, 남아프리카, 2193
        • Research Site
    • Western Cape
      • Cape Town, Western Cape, 남아프리카, 7925
        • Research Site
  • 네덜란드
      • Utrecht, 네덜란드, 3584 CX
        • Research Site
  • 독일
    • North Rhine-westphalia
      • Bonn, North Rhine-westphalia, 독일, 53127
        • Research Site
  • 미국
    • Arizona
      • Phoenix, Arizona, 미국, 85016
        • Research Site
    • California
      • Sacramento, California, 미국, 95817
        • Research Site
    • Colorado
      • Aurora, Colorado, 미국, 80045
        • Research Site
    • Georgia
      • Atlanta, Georgia, 미국, 30322
        • Research Site
    • Hawaii
      • Honolulu, Hawaii, 미국, 96826
        • Research Site
    • Indiana
      • Indianapolis, Indiana, 미국, 46260
        • Research Site
    • Louisiana
      • New Orleans, Louisiana, 미국, 70112
        • Research Site
    • Michigan
      • East Lansing, Michigan, 미국, 48823
        • Research Site
    • Pennsylvania
      • Pittsburgh, Pennsylvania, 미국, 15213
        • Research Site
    • Washington
      • Seattle, Washington, 미국, 98104
        • Research Site
  • 벨기에
      • Bruxelles, 벨기에, 1200
        • Research Site
      • Leuven, 벨기에, 3000
        • Research Site
  • 브라질
    • Sao Paulo
      • Campinas, Sao Paulo, 브라질, 13083-878
        • Research Site
  • 스웨덴
      • Malmö, 스웨덴, 20502
        • Research Site
      • Stockholm, 스웨덴, 17176
        • Research Site
  • 아일랜드
      • Dublin, 아일랜드, D12 N512
        • Research Site
  • 영국
    • Cambridgeshire
      • Cambridge, Cambridgeshire, 영국, CB2 0QQ
        • Research Site
    • Greater London
      • London, Greater London, 영국, E1 1BB
        • Research Site
      • London, Greater London, 영국, SE1 7EH
        • Research Site
    • Hampshire
      • Basingstoke, Hampshire, 영국, RG24 9NA
        • Research Site
  • 이탈리아
      • Florence, 이탈리아, 50134
        • Research Site
      • Milano, 이탈리아, 20122
        • Research Site
  • 인도
    • Karnataka
      • Bangalore, Karnataka, 인도, 560034
        • Research Site
    • Maharashtra
      • Pune, Maharashtra, 인도, 411004
        • Research Site
    • Tamil Nadu
      • Vellore, Tamil Nadu, 인도, 632004
        • Research Site
  • 일본
    • Aichi-Ken
      • Nagoya-Shi, Aichi-Ken, 일본, 466-8550
        • Research Site
    • Fukuoka-Ken
      • Kitakyushu, Fukuoka-Ken, 일본, 807-8555
        • Research Site
    • Kanagawa-Ken
      • Kawasaki, Kanagawa-Ken, 일본, 216-8511
        • Research Site
    • Nara-Ken
      • Kashihara-shi, Nara-Ken, 일본, 634-8522
        • Research Site
    • Tokyo-To
      • Shinjuku-ku, Tokyo-To, 일본, 160-0023
        • Research Site
      • Tokyo, Tokyo-To, 일본, 167-8515
        • Research Site
  • 중국
    • Beijingshì
      • Beijing, Beijingshì, 중국, 100005
        • Research Site
    • Guangdongsheng
      • Guangzhou, Guangdongsheng, 중국, 510515
        • Research Site
    • Shànghaishì
      • Shanghai, Shànghaishì, 중국, 200025
        • Research Site
    • Tianjinshì
      • Tianjing, Tianjinshì, 중국, 300020
        • Research Site
  • 캐나다
    • Ontario
      • Toronto, Ontario, 캐나다, M5B 1W8
        • Research Site
    • Quebec
      • Montreal, Quebec, 캐나다, H3T 1C5
        • Research Site
  • 폴란드
      • Lodz, 폴란드, 93-510
        • Research Site
  • 프랑스
    • Bouches-Du-Rhône
      • Marseille, Bouches-Du-Rhône, 프랑스, 13385
        • Research Site
  • 호주
    • South Australia
      • Adelaide, South Australia, 호주, 5000
        • Research Site
    • Victoria
      • Parkville, Victoria, 호주, 3052
        • Research Site
    • Western Australia
      • Murdoch, Western Australia, 호주, 6150
        • Research Site
      • Perth, Western Australia, 호주, 6008
        • Research Site
  • 홍콩
      • Hong Kong, 홍콩
        • Research Site
    • New Territories
      • Hong Kong, New Territories, 홍콩
        • Research Site

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

연구 대상 성별

남성

설명

Key Inclusion Criteria:

  • Subjects who have completed studies 998HB102 (NCT01027364) or 9HB02PED (NCT01440946) or other studies with rFIXFc
  • Ability to understand the purposes & risks of the study and provide signed and dated informed consent.

Key Exclusion Criteria:

  • High-titer inhibitor (>/=5.00 BU/mL)

NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.

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: 무작위화되지 않음
  • 중재 모델: 병렬 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: On-Demand
The individual dose of rFIXFc to treat bleeding episodes will be based on participant's clinical condition, type and severity of the bleeding event, and if indicated, Factor IX peak (recovery) levels.
생물학적: rFIXFc
Administered as specified in the treatment arm.
다른 이름들:
  • 알프로릭스
  • BIIB029
  • 응고 인자 IX(재조합) Fc 융합 단백질
실험적: Prophylaxis
Weekly prophylaxis, individualized prophylaxis or personalized prophylaxis available.
생물학적: rFIXFc
Administered as specified in the treatment arm.
다른 이름들:
  • 알프로릭스
  • BIIB029
  • 응고 인자 IX(재조합) Fc 융합 단백질

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Number of Participants With Any Positive Inhibitor Development
기간: Approximately 5 years
An inhibitor test result greater than or equal to (>=)0.6 Bethesda units per milliliter (BU/mL), confirmed on 2 separate samples drawn 2 to 4 weeks apart, was considered positive. Both tests were to be performed by the central laboratory using the Nijmegen-modified Bethesda Assay. Data was summarized by treatment regimen for participants from Study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from Study 9HB02PED per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Approximately 5 years

2차 결과 측정

결과 측정
측정값 설명
기간
Annualized Bleeding Rate (ABR)
기간: Approximately 5 years
ABR is annualized number of bleeding episodes per participant per year. Bleeding episodes were classified as spontaneous if participant records bleeding event when there is no known contributing factor such as definite trauma/antecedent strenuous activity and classified as traumatic if participant records bleeding event when there is known reason for bleed. ABR=(Number of bleeding episodes during efficacy period/number of days during efficacy period)*365.25. Efficacy period reflects sum of all intervals of time during which participants were treated with rFIXFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals. ABR was summarized by treatment regimen for participants from study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from study 9HB02PED per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Approximately 5 years
Annualized Spontaneous Joint Bleeding Episodes
기간: Approximately 5 years
Bleeding episodes were classified as spontaneous if participant records a bleeding event when there is no known contributing factor such as definite trauma/antecedent strenuous activity. In addition, location of bleed (joint, internal, skin/mucosa or muscle) were also collected. Annualized spontaneous joint bleeding episodes=(Number of spontaneous joint bleeding episodes during efficacy period/number of days during efficacy period)*365.25. Efficacy period reflects sum of all intervals of time during which participants were treated with rFIXFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals. Bleeding episodes were summarized by treatment regimen for participants from study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from study 9HB02PED as per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Approximately 5 years
Total Number of Exposure Days (EDs)
기간: Approximately 5 years
An exposure day is a 24-hour period in which one or more rFIXFc injections are given. The total number of days of exposure to rFIXFc were summarized by treatment regimen for participants from study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from study 9HB02PED as per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Approximately 5 years
Annualized rFIXFc Consumption (International Units Per Kilogram [IU/kg])
기간: Approximately 5 years
Annualized consumption = (total international unit per kilogram [IU/kg] of study treatment received during the efficacy period / total number of days during the efficacy period) multiplied by 365.25. Efficacy period reflects sum of all intervals of time during which participants were treated with rFIXFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals. Annualized consumption was summarized by treatment regimen for participants from study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from study 9HB02PED as per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Approximately 5 years
Physicians' Global Assessment of Participant's Response to rFIXFc Regimen Using a 4-Point Scale
기간: Approximately 5 years
Participants were assessed for response to their rFIXFc regimen using following 4-point scale: 1=Excellent: bleeding episodes responded to less than or equal to (<=)usual number of injections or dose of rFIXFc or rate of breakthrough bleeding during prophylaxis was <= that usually observed; 2=Effective: most bleeding episodes responded to same number of injections and dose, but some required more injections or higher doses, or there was minor increase in rate of breakthrough; 3=Partially Effective: bleeding episodes most often required more injections and/or higher doses than expected or adequate breakthrough bleeding prevention during prophylaxis required more frequent injections and/or higher doses and 4=Ineffective: routine failure to control hemostasis/hemostatic control require additional agents. Total number of scale responses =total count of scale responses for all participants; multiple responses per participant including those at scheduled and unscheduled visits are counted.
Approximately 5 years
Participant's Assessment of Response (Excellent or Good Response) to rFIXFc Injections for the Treatment of Bleeding Episodes Using a 4-Point Scale
기간: Approximately 5 years
Using eDiary, participant received rating for treatment response to any bleeding episode (BE) using 4-point scale- 1=Excellent: Abrupt pain relief and/or improvement in signs of bleeding within approximately (approx.) 8 hours (h) after initial injection (inj.); 2=Good: Definite pain relief and/or improvement in signs of bleeding within approx. 8h after an injection, but possibly requiring more than 1 injection after 24-48h for complete resolution; 3=Moderate: Probable/slight beneficial effect within 8h after initial injection and requires more than 1 injection and 4=None: No improvement, or condition worsens within approx. 8h after initial injection. This assessment was to be made approx. 8 to 12h from time the injection was given to treat BE and prior to any additional doses of rFIXFc given for same bleeding episode. Percentages are based on the number of bleeding episodes for which a response (excellent or good) was provided for the first injection during the efficacy period.
Approximately 5 years

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

Bioverativ Therapeutics Inc.

수사관

  • 연구 책임자: Medical Director, Bioverativ Therapeutics Inc.

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)

2011년 12월 8일

기본 완료 (실제)

2017년 10월 1일

연구 완료 (실제)

2017년 10월 1일

연구 등록 날짜

최초 제출

2011년 8월 19일

QC 기준을 충족하는 최초 제출

2011년 8월 29일

처음 게시됨 (추정)

2011년 8월 30일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2020년 12월 19일

QC 기준을 충족하는 마지막 업데이트 제출

2020년 12월 16일

마지막으로 확인됨

2018년 11월 1일

추가 정보

이 연구와 관련된 용어

키워드

추가 관련 MeSH 약관

기타 연구 ID 번호

  • 9HB01EXT
  • 2011-003075-11

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. .

중증 혈우병 B에 대한 임상 시험

rFIXFc에 대한 임상 시험

유사한 임상시험 검색

스폰서 및 공동 작업자

건강 상태

약물 개입

CROs by country

CROs in Bulgaria

정황

희귀 질병

약물 개입

식이 보충제

스폰서 / 협력자

위치

3
구독하다