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

Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age16-70
SponsorSarepta Therapeutics, Inc.

About this trial

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.

Eligibility criteria

Qualifiers

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

Disqualifiers

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

Trial design

Treatments tested in this trial

  • SRP-1001 for Injection
  • Placebo

Treatment groups

60 Participants
are divided into 2 treatment groups

Sponsors and collaborators