표준 관리 인자 IX 단백질(FIX) 대체 요법으로 예방을 받는 B형 혈우병 성인 남성 참가자의 질병 특성을 평가하는 임상시험 (Honey-B)
표준 치료 FIX 대체 요법으로 예방을 받는 혈우병 B 참가자의 질병 특성을 평가하기 위한 전향적 연구
This study is focused on males who have Hemophilia B and who need regular preventive treatment with factor IX protein (FIX) replacement therapy to prevent and also to control their bleeding events. The aim of the study is to gather at least 6 months of information on bleeding events for each individual participant while they continue to use their usual FIX replacement therapy. There is no experimental treatment being tested in this study. The study is informational, and part of a larger program to understand and treat Hemophilia B with a potential experimental new therapy in the future. There is no obligation to agree to taking part in this future study.
The study is looking to answer several other research questions to help understand each participant's individual disease characteristics, including:
- How often to use FIX replacement therapy, both on a regular basis (prophylaxis) and as needed to treat bleeding events
- Measurement of FIX activity (factor IX is a clotting factor) by different laboratories using different types of tests in Hemophilia B participants
- Possible complications from the FIX replacement therapy the patient receives (usual standard of care will continue to be used)
- How quality of life is affected by Hemophilia B
- How joint health is affected by Hemophilia B
- How often the participant visits the emergency room, urgent care center, physician's office, hospital, or has a telemedicine visit as a result of bleeding events
- Whether the body makes antibodies (a protein produced by the body's immune system) against the FIX replacement therapy you receive, which could make the drug less effective or could lead to side effects
연구 개요
상태
상태
정황
정황
개입 / 치료
개입 / 치료
연구 유형
연구 유형
등록 (실제)
등록
연락처 및 위치
연구 장소
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Frankfurt, 독일, 60590
- Klinikum der Johann Wolfgang Goethe-Universitaet Frankfurt
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Hamburg, 독일, 20246
- University Hospital Hamburg Eppendorf
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Bavaria
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Regensburg, Bavaria, 독일, 93053
- University Hospital of Regensburg
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Colorado
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Aurora, Colorado, 미국, 80045
- University of Colorado Hemophilia and Thrombosis Center
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Connecticut
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New Haven, Connecticut, 미국, 06510
- Yale HTC
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District of Columbia
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Washington D.C., District of Columbia, 미국, 20057
- Georgetown University Medical Center
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Florida
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Gainesville, Florida, 미국, 32610
- University of Florida
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Illinois
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Chicago, Illinois, 미국, 60612
- Rush University Medical Center
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Indiana
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Indianapolis, Indiana, 미국, 46260
- Indiana Hemophilia and Thrombosis Center
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Michigan
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Ann Arbor, Michigan, 미국, 48109
- University of Michigan
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Ohio
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Columbus, Ohio, 미국, 43205
- Nationwide Children's Hospital
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Texas
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Houston, Texas, 미국, 77030
- University of Texas Health Science Center at Houston
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London, 영국, SE1 7EH
- St. Thomas' Hospital
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London, 영국, E1 1FR
- Barts Health NHS Trust, Royal London Hospital
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Hampshire
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Southampton, Hampshire, 영국, SO16 6YD
- Southampton General Hospital
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Ontario
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Hamilton, Ontario, 캐나다, L8N 3Z5
- McMaster University Medical Centre - Hamilton Health Sciences
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Quebec
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Montreal, Quebec, 캐나다, H4A 3J1
- Mcgill University Health Center (MUHC)
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참여기준
자격 기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
샘플링 방법
연구 인구
Male patients with Hemophilia B with a history of FIX:C ≤2% or a documented genotype known to produce severe hemophilia B on current FIX Replacement Therapy prophylaxis
설명
Key Inclusion Criteria:
- Previous experience with FIX therapy (≥50 documented exposure days to a FIX protein product such as recombinant, plasma-derived or extended half-life FIX product) with a current stable prophylaxis regimen for >2 months prior to enrollment and intention to use FIX replacement therapy for the duration of the study
- No known hypersensitivity to FIX replacement product
- Willing to be contacted about a potential future clustered regularly interspaced short palindromic repeats (CRISPR)-based Factor 9 (F9) gene insertion clinical trial in which they may have the opportunity to screen for enrollment
Key Exclusion Criteria:
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History of any coagulation disorder; requires anticoagulant therapy
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Lack of adherence with documentation of bleeds and/or prophylaxis replacement therapy administration in the opinion of the investigator, based on medical history
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History of FIX inhibitor (clinical or laboratory-based assessment) on 2 or more occasions, as defined in the protocol
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Bethesda inhibitor titer greater than the upper limit of normal (ULN) at screening
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Any detectable pre-existing antibodies to the Adeno-associated virus serotype 8 (AAV8) capsid; as measured by an assay at prescreening, as defined in the protocol
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Is positive for hepatitis B or C at screening, as defined in protocol
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If any of the following pre-existing diagnoses are documented:
- Cholestatic liver disease
- Liver cirrhosis
- Portal hypertension; or
- Splenomegaly; or
- Hepatic encephalopathy
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History of arterial or venous thrombo-embolic events, as defined in the protocol
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History of clinically significant cardiovascular, respiratory, hepatic, renal (including nephrotic syndrome), gastrointestinal (including protein-losing enteropathy), endocrine, hematological (including thrombophilia), psychiatric, or neurological disease, as assessed by the investigator that may confound the results of the study or poses an additional risk to the participant by study participation
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Previously received of any AAV-gene based therapy with a marketed gene therapy or in a clinical trial or intent to receive approved or investigational AAV-gene based therapy during the study period
NOTE: Other Inclusion/Exclusion Protocol Defined Criteria Apply
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 관찰 모델: COHORT
- 시간 관점: PROSPECTIVE
그룹/코호트 수
코호트 및 개입
그룹/코호트그룹/코호트 |
개입 / 치료개입 / 치료 |
|---|---|
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Cohort 1
Male participants with hemophilia B on current FIX Replacement Therapy prophylaxis or a documented genotype known to produce severe hemophilia B |
No study treatment will be administered in this study. |
연구는 무엇을 측정합니까?
주요 결과 측정
주요 결과 측정
결과 측정 |
기간 |
|---|---|
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연간 출혈율(ABR)
기간: 최소 26주 최대 96주
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최소 26주 최대 96주
|
2차 결과 측정
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
1단계 및 발색 검정에 의한 예방 FIX 대체 요법 참가자의 FIX:C 차이
기간: 연구가 끝날 때까지 약 96주
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연구가 끝날 때까지 약 96주
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예방 FIX 대체 요법 참가자의 실험실에 의한 1단계 분석과 발색 기질 분석 간의 FIX:C 차이
기간: 연구가 끝날 때까지 약 96주
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연구가 끝날 때까지 약 96주
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예방 FIX 대체 요법 참가자의 분석(1단계 분석 및 발색 기질 분석)에 의한 실험실 간 FIX:C의 차이
기간: 연구가 끝날 때까지 약 96주
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연구가 끝날 때까지 약 96주
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FIX 대체 요법의 연간 이용률(IU/kg)
기간: 최대 96주
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최대 96주
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부작용(AE) 발생률
기간: 연구가 끝날 때까지 약 96주
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연구가 끝날 때까지 약 96주
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AE의 심각도
기간: 연구가 끝날 때까지 약 96주
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연구가 끝날 때까지 약 96주
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아데노 관련 바이러스(AAV) 캡시드 단백질에 대한 항체 검출
기간: 최대 96주
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최대 96주
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FIX functional (coagulant) activity (FIX:C) in participants on prophylaxis FIX replacement therapy
기간: Through the end of the study, approximately 96 Weeks
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FIX:C includes pre-dose and average activity |
Through the end of the study, approximately 96 Weeks
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Hemophilia Quality of Life (Haemo-Qol-A) total and individual domain scores
기간: Up to 96 Weeks
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The Haemo-QoL-A is a self-reported Hemophilia-specific health-related quality of life questionnaire which consists of 41 questions covering 6 domains (Physical Functioning, Role Functioning, Worry, Consequences of Bleeding, Emotional Impact, and Treatment Concerns). Items are answered on a 6-point Likert-type scale, ranging from 0 (None of the time) to 5 (All of the time). Higher scores mean better health-related quality of life or less impairment. |
Up to 96 Weeks
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Hemophilia Activities List (HAL) total and individual scores
기간: Up to 96 Weeks
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HAL measures of the impact of Hemophilia on functional abilities in adults. The questionnaire consists of 42 items across 7 domains. Each item is rated on a scale of 1 (Impossible) to 6 (Never). Higher scores represent lower levels of activity limitations. |
Up to 96 Weeks
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European Quality of Life 5 Dimensions 3-levels (EQ-5D-3L) score
기간: Up to 96 Weeks
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EQ-5D-3L health questionnaire is a participant/relative answered questionnaire scoring 5 dimensions - mobility, self-care, usual activities, pain/discomfort and anxiety/depression. the status of each dimension had 3 possible responses (1 =no problem, 2 some problem 3 =severe problems) in the relevant health dimension. Higher score indicated a worsening health condition. |
Up to 96 Weeks
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Hemophilia Joint Health Score (HJHS) total and individual scores per joint
기간: Up to 96 Weeks
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The examination involves a physical assessment of elbow, knee, and ankle joints. Individual joints are scored based on duration and presence of swelling, joint pain, flexibility, muscle atrophy, strength, and overall gait. The HJHS is a validated 11-item scoring tool based on radiologic and clinical evaluation, sensitive to detect early signs and minor changes. HJHS ranges from 0 to 124. Higher values in the HJHS represent worse situation for the participant. |
Up to 96 Weeks
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Annualized medically attended visit (MAV) rate for any reason
기간: Up to 96 Weeks
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The annual rate of MAVs including emergency room (ER) visits, urgent care center visits, physician's office visits, telemedicine visits, or hospitalizations related to bleeds treated on-demand with FIX replacement therapy |
Up to 96 Weeks
|
공동 작업자 및 조사자
수사관
수사관
- 연구 책임자: Clinical Trial Management, Regeneron Pharmaceuticals
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
연구 시작
기본 완료 (추정된)
기본 완료
연구 완료 (추정된)
연구 완료
연구 등록 날짜
최초 제출
최초 제출
QC 기준을 충족하는 최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
처음 게시됨
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
마지막 업데이트 게시됨
QC 기준을 충족하는 마지막 업데이트 제출
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
키워드
추가 관련 MeSH 약관
기타 연구 ID 번호
기타 연구 ID 번호
- R0000-HEMB-2187
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
IPD 계획 설명
All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
IPD 공유 기간
When Regeneron has
- received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication or has globally discontinued development of the product for all indications on or after April 2020 and has no plans for future development
- made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry)
- the legal authority to share the data, and
- ensured the ability to protect participant privacy
IPD 공유 액세스 기준
Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf
IPD 공유 지원 정보 유형
- 연구_프로토콜
- 수액
- ICF
- ANALYTIC_CODE
- CSR
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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. .