얼굴견갑상완근이영양증 1형 성인 환자를 대상으로 한 ARO-DUX4 연구
2026년 10월 1일 업데이트: Sarepta Therapeutics, Inc.
안면견갑상완근이영양증 1형 성인 환자를 대상으로 ARO-DUX4의 안전성, 내약성, 약동학, 약력학을 평가하기 위한 1/2a상 용량 증량 연구
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of SRP-1001 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1). In Part 1 of the study, participants will receive one dose of SRP-1001 or placebo. In Part 2 of the study, participants will receive 4 doses of SRP-1001 or placebo. Participants who complete Part 1 will have the option to re-screen and re-randomize into Part 2. All participants will undergo pre- and post-dose magnetic imaging resonance (MRI)-guided muscle biopsies (a total of 2 biopsies). Participants who complete Part 1 and enroll in Part 2 will be required to undergo an additional screening biopsy. Participants completing Part 1 or Part 2 may have the option to continue to receive drug in an open-label extension study or may be eligible to participate in later-stage clinical studies.
연구 개요
연구 유형
중재적
등록 (추정된)
60
단계
- 2 단계
- 1단계
연락처 및 위치
이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.
연구 장소
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Leiden, 네덜란드, 2333
- Centre For Human Drug Research
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Auckland, 뉴질랜드, 1010
- New Zealand Clinical Research
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München, 독일, 80336
- Universitätsklinikum München
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Ulm, 독일, 89081
- Neurologische Universitätsklinik Am Rku
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Barcelona, 스페인, 08035
- Hospital Universitario Vall d'Hebron
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Madrid, 스페인, 28034
- Hospital Universitario Ramon y Cajal
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Valencia, 스페인, 46026
- Hospital Universitari l Politecnic La Fe de Valencia
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Milan, 이탈리아, 20162
- Centro Clinico Nemo Milann
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Roma, 이탈리아, 00168
- Fondazione Policlinico Universitario A. Gemelli
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Alberta
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Calgary, Alberta, 캐나다, T2N4Z6
- University of Calgary
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Edmonton, Alberta, 캐나다, T6G2G4
- University of Alberta
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Quebec
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Montreal, Quebec, 캐나다, H3A2B4
- McGill University
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New South Wales
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Liverpool, New South Wales, 호주, 2170
- Liverpool Hospital
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Queensland
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Auchenflower, Queensland, 호주, 4066
- Wesley Research Institute
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Birtinya, Queensland, 호주, 4575
- Sunshine Coast University Hospital
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Victoria
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Melbourne, Victoria, 호주, 3004
- Alfred Health
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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
공부할 수 있는 나이
16년 (어린이, 성인, 고령자)
건강한 자원 봉사자를 받아들입니다
아니
연구 대상 성별
모두
설명
Key Inclusion Criteria:
- Genetically confirmed FSHD1 based on screening evaluation or source verifiable medical record
- Clinical severity score between 3 and 8 (scale, 0 to 10)
- Must have an eligible lower extremity muscle for biopsy as determined from MRI by a central reader, with muscle fat fraction ≥10% and less than approximately 40%
- Males or nonpregnant, nonlactating females ≥18 years of age who do not plan to become pregnant during the study, with an upper age limit of ≤70 years
- Able and willing to provide written informed consent prior to the performance of any study specific procedures
- Participants with a body mass index (BMI) between 18.0 and 35.0 kilograms/square meter, inclusive. A participant with FSHD1 and a BMI outside this range may be allowed into the study at the discretion of the principal investigator.
- Must have eligible lower extremity muscle for biopsy as determined from MRI by a central reader
- A 12-lead electrocardiogram at screening with no abnormalities that may compromise participant's safety in the study
- Participants of childbearing potential and their partners must use highly effective contraception during the study and for at least 9 months following the end of study or last dose of study medication, whichever is later. Males must not donate sperm during the study from Day 1 until at least 9 months following the end of study or last dose of study medication, whichever is later.
Key Exclusion Criteria:
- Human immunodeficiency virus (HIV) infection as shown by presence of anti-HIV antibody (seropositive) at screening
- Seropositive for hepatitis B or hepatitis C at screening
- Uncontrolled hypertension
- Severe cardiovascular disease
- History of thrombolic events
- Platelet count less that the lower limit of normal at screening
- History or presence of: a hypercoagulable state, nephrotic range proteinuria, antiphospholipid antibody syndrome, myeloproliferative disease, inability to ambulate, use of hormone-based contraceptives.
- Any contraindication to muscle biopsy or MRI
Note: additional inclusion/exclusion criteria may apply per protocol
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: 무작위
- 중재 모델: 병렬 할당
- 마스킹: 네 배로
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
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실험적: SRP-1001
SRP-1001 for injection |
Single or multiple doses of SRP-1001 by intravenous (IV) infusion
다른 이름들:
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위약 비교기: Placebo
Sodium chloride (0.9%) |
Calculated volume to match active treatment by IV infusion |
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
기간 |
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Number of Participants With Treatment-Emergent Adverse Events Over Time Through End of Study
기간: Part 1: Up to Day 90; Part 2: Up to Day 360
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Part 1: Up to Day 90; Part 2: Up to Day 360
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2차 결과 측정
결과 측정 |
기간 |
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PK of SRP-1001: Maximum Observed Plasma Concentration (Cmax)
기간: Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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PK of SRP-1001: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24)
기간: Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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PK of SRP-1001: Area Under the Plasma Concentration Versus Time Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)
기간: Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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PK of SRP-1001: Area Under the Plasma Concentration Versus Time from Zero to Infinity (AUCinf)
기간: Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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PK of SRP-1001: Terminal Elimination Half-Life (t1/2)
기간: Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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PK of SRP-1001: Systemic Clearance (CL)
기간: Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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PK of SRP-1001: Volume of Distribution (Vss)
기간: Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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PK of SRP-1001: Recovery of Unchanged Drug in Urine Over 0-24 Hours (Amount Excreted: Ae)
기간: Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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PK of SRP-1001: Fraction of Drug Excreted in Urine as Percent of IV Dose (Fe)
기간: Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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PK of SRP-1001: Renal Clearance (CLr)
기간: Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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공동 작업자 및 조사자
여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.
수사관
- 연구 책임자: Medical Director, Sarepta Therapeutics, Inc.
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)
2024년 6월 19일
기본 완료 (추정된)
2028년 12월 31일
연구 완료 (추정된)
2028년 12월 31일
연구 등록 날짜
최초 제출
2023년 11월 8일
QC 기준을 충족하는 최초 제출
2023년 11월 13일
처음 게시됨 (실제)
2023년 11월 15일
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
2026년 10월 5일
QC 기준을 충족하는 마지막 업데이트 제출
2026년 10월 1일
마지막으로 확인됨
2026년 10월 1일
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
- SRP-1001-101
- 2023-509748-89-00 (씨티스)
- 2023 (미국 NIH 보조금/계약)
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
아니요
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
예
미국 FDA 규제 기기 제품 연구
아니
미국에서 제조되어 미국에서 수출되는 제품
예
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. .