A형 또는 B형 혈우병을 앓고 있는 1세 이상 12세 미만 소아과 남성 대상자의 Fitusiran 예방법 (ATLAS-PEDS)
ATLAS-PEDS: A형 또는 B형 혈우병을 앓고 있는 1세에서 12세 미만의 남성 소아 피험자를 대상으로 피투시란 예방에 대한 공개 라벨, 다국가 연구
Primary Objective:
- To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to <12 years of age) with severe hemophilia A or B
Secondary Objectives:
- To characterize the safety and tolerability
- To determine fitusiran plasma concentrations at selected time points
연구 개요
상세 설명
The estimated total time on study is up to 256 weeks for participants who roll over into the extension study and up to 280 weeks for participants who do not roll over into the extension study (due to the requirement for up to an additional 6 months of follow-up for monitoring of AT levels).
연구 유형
등록 (실제)
단계
- 2 단계
- 3단계
연락처 및 위치
연구 장소
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California
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Los Angeles, California, 미국, 90027
- Children's Hospital Los Angeles Site Number : 8400002
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New Jersey
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Hackensack, New Jersey, 미국, 07601
- Hackensack University Medical Center- Site Number : 8400008
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Ohio
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Cleveland, Ohio, 미국, 44106
- University Hospitals of Cleveland- Site Number : 8400007
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Pennsylvania
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Hershey, Pennsylvania, 미국, 17033
- Penn State Milton S. Hershey Medical Center- Site Number : 8400006
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Madrid, Comunidad de
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Madrid, Madrid, Comunidad de, 스페인, 28046
- Investigational Site Number : 7240002
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Florence, 이탈리아, 50134
- Investigational Site Number : 3800002
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Milan, 이탈리아, 20122
- Investigational Site Number : 3800001
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Bangalore, 인도, 560034
- Investigational Site Number : 3560006
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Mumbai, 인도, 400 022
- Investigational Site Number : 3560002
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Pune-411011, 인도, 411 011
- Investigational Site Number : 3560001
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Vellore, 인도, 632004
- Investigational Site Number : 3560004
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Ontario
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Hamilton, Ontario, 캐나다, L8N 3Z5
- Investigational Site Number : 1240001
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Ottawa, Ontario, 캐나다, K1H 8L1
- Investigational Site Number : 1240002
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Adana, 터키 (Türkiye), 01130
- Investigational Site Number : 7920001
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Istanbul, 터키 (Türkiye), 34093
- Investigational Site Number : 7920002
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Izmir, 터키 (Türkiye), TR-35100
- Investigational Site Number : 7920003
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참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
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Male, aged 1 to <12 years at the time of enrollment.
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Severe hemophilia A or B (Factor VIII (FVIII) <1% or Factor IX (FIX) ≤2%)
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Participants must have inhibitory antibodies to FVIII or FIX and must meet one of the following Nijmegen-modified Bethesda assay results criteria:
- Inhibitor titer of ≥0.6 BU/mL at screening, OR
- Inhibitor titer of <0.6 BU/mL at screening with medical record evidence of 2 consecutive titers ≥0.6 BU/mL, OR
- Inhibitor titer of <0.6 BU/mL at screening with medical record evidence of 1 inhibitor titer ≥0.6 BU/mL and a history of anamnestic response or severe allergic reaction (anaphylaxis or nephrotic syndrome)
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Adequate peripheral venous access, as determined by the Investigator, to allow the blood draws required by the study protocol
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Weight requirements at the time of enrollment: 8 to <45 kg
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Willing and able to comply with the study requirements and to provide signed written informed consent obtained from parent(s)/legal guardian (hereinafter the "parent") and written or oral assent obtained from participant, per local and national requirements
Exclusion Criteria:
Participants are excluded from the study if any of the following criteria apply:
- Known co-existing bleeding disorders other than hemophilia A or B
- Antithrombin (AT) activity <60% at Screening
- Co-existing thrombophilic disorder
- Clinically significant liver disease
- Active Hepatitis C virus infection
- Acute or chronic Hepatitis B virus infection
- Acute Hepatitis A or hepatitis E infection
- HIV positive with a CD4 count of <400 cells/μL
- History of arterial or venous thromboembolism, unrelated to an indwelling venous access
- Inadequate renal function
- History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc)
- Subjects with central or peripheral indwelling catheters, with history of venous access complications leading to hospitalization and/or systemic anticoagulation therapy.
- History of intolerance to subcutaneous (SC) injection(s)
- Use of emicizumab (Hemlibra®) within 6 months prior to screening
- Any other conditions or comorbidities that would make the patient unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgment
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
공부 계획
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 |
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실험적: Fitusiran
Participants will receive a selected dose of fitusiran at regular intervals, as per study protocol |
Pharmaceutical form:Solution for injection-Route of administration:Subcutaneous
다른 이름들:
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연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Plasma antithrombin (AT) activity levels
기간: Day 1 to the AT analysis time point at the optimal therapeutic dose (approximately 256 weeks)
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Characterize the AT activity at the optimal therapeutic dose |
Day 1 to the AT analysis time point at the optimal therapeutic dose (approximately 256 weeks)
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Number of participants reported with adverse events
기간: Up to 280 weeks (up to 256 weeks of treatment + up to 24 weeks of AT follow up)
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Number of participants reported with treatment-emergent adverse events (TEAEs) |
Up to 280 weeks (up to 256 weeks of treatment + up to 24 weeks of AT follow up)
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Fitusiran plasma concentrations
기간: Day 1 and Day 85
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Plasma samples will be collected for measurement of plasma concentrations of fitusiran at specific time points post dose on Day 1 and pre dose on Day 85 |
Day 1 and Day 85
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공동 작업자 및 조사자
수사관
- 연구 책임자: Clinical Sciences & Operations, Sanofi
간행물 및 유용한 링크
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (실제)
연구 완료 (실제)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
- EFC15467
- 2019-000679-18 (EudraCT 번호)
- U1111-1223-4368 (레지스트리 식별자: ICTRP)
- 2024-512501-76 (레지스트리 식별자: CTIS)
- 2019 (미국 NIH 보조금/계약)
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
IPD 계획 설명
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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