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Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1

StatusRecruiting
PhasePhase 1/2
Started2024-06-19
View on ClinicalTrials.gov ↗
Trial flagged as At Risk
Primary completion moved at least 24 months later Dec 2026 → Dec 31, 2028
Primary endpoint reworded 1 of 1 changed
See other at-risk trials from Sarepta Therapeutics, Inc.

Amendment history

2026-08-07
Study Identification, Study Status, Sponsor/Collaborators, Study Description, Conditions, Arms and Interventions, Outcome Measures, Eligibility, Contacts/Locations v11
Primary endpoint(s) modified1 entry, revised
WasNumber of Participants With Treatment-Emergent Adverse Events (TEAEs) Over Time Through End of Study (EOS)
NowNumber of Participants With Treatment-Emergent Adverse Events Over Time Through End of Study
Sponsor changed: Arrowhead Pharmaceuticals → Sarepta Therapeutics, Inc.+1 characters
WasArrowhead Pharmaceuticals
NowSarepta Therapeutics, Inc.
Primary completion pushed: 2026-12 → 2028-12-31
Completion pushed: 2026-12 → 2028-12-31
Start date2024-02-22→2024-06-19
Study sites17→16
InterventionsARO-DUX4 for Injection (Drug), Placebo (Drug)→Placebo (Drug), SRP-1001 for Injection (Drug)
Eligibility criteria+645 characters
Key Inclusion Criteria: Genetically confirmed FSHD1 based on Screeningscreening 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 o [...] Must have eligible lower extremity muscle for biopsy as dete [...] ed from MRI by a central reader A 12-lead electrocardiog [...]
Secondary endpoints10 entries, revised
Pharmacokinetics (PK) of AROSRP-DUX41001: Maximum Observed Plasma Concentration (Cmax) PK of AROSRP-DUX41001: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24) PK of AROSRP-DUX41001: Area Under the Plasma Concentration Versus Time Curve from [...] the Last Quantifiable Plasma Concentration (AUClast) PK of AROSRP-DUX41001: Area Under the Plasma Concentration Versus Time from Zero to Infinity (AUCinf) PK of AROSRP-DUX41001: Terminal Elimination Half-Life (t1/2) PK of AROSRP-DUX41001: Systemic Clearance (CL) PK of AROSRP-DUX41001: Volume of Distribution (Vss) PK of A [...] [...]
Study title+4 characters
Study of AROSRP-DUX41001 in Adult and Adolescent PatientsParticipants With Facioscapulohumeral Muscular Dystrophy Type 1
2026-02-04
Study Status v10
Start date2024-02-26→2024-02-22
2025-10-02
Study Identification, Study Status, Eligibility, Contacts/Locations v9
Primary completion date2026-11→2026-12
Completion date2026-11→2026-12
Study sites5→17
Study title+15 characters
Study of ARO-DUX4 in Adult and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1
2025-08-15
Study Status v8
Primary completion date2025-05→2026-11
Completion date2025-09→2026-11
2025-03-23
Study Status, Study Design v7
PhasePhase 1→Phase 1/2
Show 6 earlier versions
2024-10-22
Study Status, Study Description, Study Design, Outcome Measures, Contacts/Locations v6
Primary completion date2025-04→2025-05
Completion date2025-06→2025-09
Enrollment target52→60
Study sites3→5
Secondary endpointsdetails revised at 10 of 10 entries
2024-07-29
Study Status, Outcome Measures, Eligibility, Contacts/Locations v5
Study sites1→3
Eligibility criteriarevised
[...] not donate sperm during the study from Day 1 until at least 12weeks12 weeks following the end of study or last dose of study medication [...] tension Severe cardiovascular disease History of thrombolic eve4ntsevents Platelet count less that the lower limit of normal at Scree [...]
Secondary endpoints10 entries, revised
PK of ARO-DUX4: Area Under the Plasma Concentration Versus Time from Zero to Infinity (AUCinf)
2024-03-12
Study Status, Contacts/Locations v4
Re-verified, no change to tracked fields
2024-03-05
Not Yet Recruiting→Recruiting Study Status, Contacts/Locations v3
Trial statusNot Yet Recruiting→Recruiting
Start dateestimated 2024-02→confirmed 2024-02-26
Study sitesdetails revised at 1 of 1 site
2024-02-02
Study Status v2
Start date2024-01→2024-02
2024-01-30
Study Status, Contacts/Locations v1
Study sites0→1
2023-11-13
Original filing
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.
Trial Details
NCT Number NCT06131983
Lead Sponsor Sarepta Therapeutics, Inc.
Conditions Muscular Dystrophy, Facioscapulohumeral
Enrollment 60 participants
Start Date 2024-06-19
Primary Completion 2028-12-31 (estimated)
Study Completion 2028-12-31 (estimated)
Updated on ClinicalTrials.gov 2026-08-11