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

Phase 2RecruitingNCT06131983

Sarepta Therapeutics, Inc.60 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Muscular Dystrophy, Facioscapulohumeral

Interventions

Eligibility

Sex: ALLMin age: 16 YearsMax age: 70 Years

Medical Language ↔ Plain English

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

Locations (16)

Liverpool Hospital

Liverpool, New South Wales, Australia

RECRUITING

Wesley Research Institute

Auchenflower, Queensland, Australia

RECRUITING

Sunshine Coast University Hospital

Birtinya, Queensland, Australia

RECRUITING

Alfred Health

Melbourne, Victoria, Australia

RECRUITING

University of Calgary

Calgary, Alberta, Canada

NOT_YET_RECRUITING

University of Alberta

Edmonton, Alberta, Canada

RECRUITING

McGill University

Montreal, Quebec, Canada

RECRUITING

Universitätsklinikum München

München, Germany

RECRUITING

Neurologische Universitätsklinik Am Rku

Ulm, Germany

RECRUITING

Centro Clinico Nemo Milann

Milan, Italy

RECRUITING

...and 6 more locations

Outcomes

Primary Outcomes

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