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
Study 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.
Eligibility
Sex
ALL
Min age
16 Years
Max age
70 Years
Healthy volunteers
No
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
Primary outcome measure(s)
Number of Participants With Treatment-Emergent Adverse Events Over Time Through End of Study — Part 1: Up to Day 90; Part 2: Up to Day 360
Trial sites (16)
Facility
City
Region
Status
Liverpool Hospital
Liverpool
New South Wales
Recruiting
Wesley Research Institute
Auchenflower
Queensland
Recruiting
Sunshine Coast University Hospital
Birtinya
Queensland
Recruiting
Alfred Health
Melbourne
Victoria
Recruiting
University of Calgary
Calgary
Alberta
Not Yet Recruiting
University of Alberta
Edmonton
Alberta
Recruiting
McGill University
Montreal
Quebec
Recruiting
Universitätsklinikum München
München
Germany
Recruiting
Neurologische Universitätsklinik Am Rku
Ulm
Germany
Recruiting
Centro Clinico Nemo Milann
Milan
Italy
Recruiting
Fondazione Policlinico Universitario A. Gemelli
Roma
Italy
Recruiting
Centre For Human Drug Research
Leiden
Netherlands
Recruiting
New Zealand Clinical Research
Auckland
New Zealand
Recruiting
Hospital Universitario Vall d'Hebron
Barcelona
Spain
Recruiting
Hospital Universitario Ramon y Cajal
Madrid
Spain
Recruiting
Hospital Universitari l Politecnic La Fe de Valencia
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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