Recruiting
Phase 1
Phase 2

ARO-DUX4

Sponsor:

Sarepta Therapeutics, Inc.

Code:

NCT06131983

Conditions

Muscular Dystrophy, Facioscapulohumeral

Eligibility Criteria

Sex: All

Age: 16 - 70

Healthy Volunteers: Not accepted

Interventions

SRP-1001 for Injection

Placebo

Study Details

Brief summary:

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.

Conditions

Muscular Dystrophy, Facioscapulohumeral

Study ID

NCT06131983

Start date

Jun 19, 2024

Status verified date

Aug, 2026

Completion date

Dec 31, 2028

Anticipated

Primary completion date

Dec 31, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 16 - 70

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

60 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: SRP-1001

SRP-1001 for injection

placebo comparator: Placebo

Sodium chloride (0.9%)

Interventions

SRP-1001 for Injection

Single or multiple doses of SRP-1001 by intravenous (IV) infusion

Placebo

Calculated volume to match active treatment by IV infusion

Primary outcome measure

  • Number of Participants With Treatment-Emergent Adverse Events Over Time Through End of Study [ Time Frame: Part 1: Up to Day 90; Part 2: Up to Day 360 ]

Central Contacts and Locations

Central contacts

Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4

1-888-SAREPTA (1-888-727-3782)SareptAlly@sarepta.com

Locations

University of Alberta

Recruiting

Edmonton, Alberta, Canada, T6G2G4

McGill University

Recruiting

Montreal, Quebec, Canada, H3A2B4

More Information

Sponsor

Sarepta Therapeutics, Inc.

Last update posted

Aug 11, 2026

Last verified

Aug, 2026

Keywords

  • Facio-Scapulo-Humeral Muscular Dystrophy
  • SRP-1001
  • ARO-DUX4

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Sarepta Therapeutics, Inc. on 2026-08-11.