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