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
상태
완전한
정황
정황
개입 / 치료
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
연구 유형
중재적
등록 (실제)
등록
120
단계
단계
- 3단계
연락처 및 위치
이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.
연구 장소
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Gauteng
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Johannesburg, Gauteng, 남아프리카, 2193
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Western Cape
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Cape Town, Western Cape, 남아프리카, 7925
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Utrecht, 네덜란드, 3584 CX
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North Rhine-westphalia
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Bonn, North Rhine-westphalia, 독일, 53127
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Arizona
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Phoenix, Arizona, 미국, 85016
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California
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Sacramento, California, 미국, 95817
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Colorado
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Aurora, Colorado, 미국, 80045
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Georgia
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Atlanta, Georgia, 미국, 30322
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Hawaii
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Honolulu, Hawaii, 미국, 96826
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Indiana
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Indianapolis, Indiana, 미국, 46260
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Louisiana
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New Orleans, Louisiana, 미국, 70112
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Michigan
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East Lansing, Michigan, 미국, 48823
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Pennsylvania
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Pittsburgh, Pennsylvania, 미국, 15213
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Washington
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Seattle, Washington, 미국, 98104
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Bruxelles, 벨기에, 1200
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Leuven, 벨기에, 3000
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Sao Paulo
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Campinas, Sao Paulo, 브라질, 13083-878
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Malmö, 스웨덴, 20502
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Stockholm, 스웨덴, 17176
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Dublin, 아일랜드, D12 N512
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Cambridgeshire
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Cambridge, Cambridgeshire, 영국, CB2 0QQ
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Greater London
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London, Greater London, 영국, E1 1BB
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London, Greater London, 영국, SE1 7EH
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Hampshire
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Basingstoke, Hampshire, 영국, RG24 9NA
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Florence, 이탈리아, 50134
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Milano, 이탈리아, 20122
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Karnataka
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Bangalore, Karnataka, 인도, 560034
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Maharashtra
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Pune, Maharashtra, 인도, 411004
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Tamil Nadu
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Vellore, Tamil Nadu, 인도, 632004
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Aichi-Ken
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Nagoya-Shi, Aichi-Ken, 일본, 466-8550
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Fukuoka-Ken
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Kitakyushu, Fukuoka-Ken, 일본, 807-8555
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Kanagawa-Ken
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Kawasaki, Kanagawa-Ken, 일본, 216-8511
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Nara-Ken
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Kashihara-shi, Nara-Ken, 일본, 634-8522
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Tokyo-To
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Shinjuku-ku, Tokyo-To, 일본, 160-0023
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Tokyo, Tokyo-To, 일본, 167-8515
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Beijingshì
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Beijing, Beijingshì, 중국, 100005
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Guangdongsheng
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Guangzhou, Guangdongsheng, 중국, 510515
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Shànghaishì
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Shanghai, Shànghaishì, 중국, 200025
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Tianjinshì
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Tianjing, Tianjinshì, 중국, 300020
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Ontario
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Toronto, Ontario, 캐나다, M5B 1W8
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Quebec
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Montreal, Quebec, 캐나다, H3T 1C5
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Lodz, 폴란드, 93-510
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Bouches-Du-Rhône
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Marseille, Bouches-Du-Rhône, 프랑스, 13385
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South Australia
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Adelaide, South Australia, 호주, 5000
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Victoria
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Parkville, Victoria, 호주, 3052
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Western Australia
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Murdoch, Western Australia, 호주, 6150
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Perth, Western Australia, 호주, 6008
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Hong Kong, 홍콩
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New Territories
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Hong Kong, New Territories, 홍콩
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참여기준
연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.
자격 기준
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)
팔의 수
2
무기와 개입
참가자 그룹 / 팔참가자 그룹 / 팔 |
개입 / 치료개입 / 치료 |
|---|---|
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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.
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Administered as specified in the treatment arm.
다른 이름들:
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실험적: Prophylaxis
Weekly prophylaxis, individualized prophylaxis or personalized prophylaxis available.
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Administered as specified in the treatment arm.
다른 이름들:
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연구는 무엇을 측정합니까?
주요 결과 측정
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Number of Participants With Any Positive Inhibitor Development
기간: Approximately 5 years
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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.
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Approximately 5 years
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2차 결과 측정
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Annualized Bleeding Rate (ABR)
기간: Approximately 5 years
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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.
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Approximately 5 years
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Annualized Spontaneous Joint Bleeding Episodes
기간: Approximately 5 years
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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.
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Approximately 5 years
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Total Number of Exposure Days (EDs)
기간: Approximately 5 years
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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.
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Approximately 5 years
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Annualized rFIXFc Consumption (International Units Per Kilogram [IU/kg])
기간: Approximately 5 years
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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.
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Approximately 5 years
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Physicians' Global Assessment of Participant's Response to rFIXFc Regimen Using a 4-Point Scale
기간: Approximately 5 years
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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.
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Approximately 5 years
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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
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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.
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Approximately 5 years
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공동 작업자 및 조사자
여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.
수사관
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.
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)
연구 시작
2011년 12월 8일
기본 완료 (실제)
기본 완료
2017년 10월 1일
연구 완료 (실제)
연구 완료
2017년 10월 1일
연구 등록 날짜
최초 제출
최초 제출
2011년 8월 19일
QC 기준을 충족하는 최초 제출
QC 기준을 충족하는 최초 제출
2011년 8월 29일
처음 게시됨 (추정)
처음 게시됨
2011년 8월 30일
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
마지막 업데이트 게시됨
2020년 12월 19일
QC 기준을 충족하는 마지막 업데이트 제출
QC 기준을 충족하는 마지막 업데이트 제출
2020년 12월 16일
마지막으로 확인됨
마지막으로 확인됨
2018년 11월 1일
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
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
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NCT06896526아직 모집하지 않음그룹 B 연쇄상 구균 감염 | 그룹 B 연쇄상 구균 감염, 후기 발병 | 그룹 B 연쇄상 구균 감염, 조기 발병 | 침습성 그룹 B 연쇄상 구균 질병 | 그룹 B 연쇄상 구균 | 그룹 B 연쇄상구균(GBS) 질병
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NCT02534935완전한
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NCT02370160완전한난치성 B-혈통 백혈병 | 재발된 B 계통 백혈병 | 난치성 B 계통 림프종 | 재발된 B 계통 림프종
rFIXFc에 대한 임상 시험
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NCT01440946완전한