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Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy
Sponsor: Sarepta Therapeutics, Inc.
Summary
This is a phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple ascending doses of SRP-1003 compared to placebo in male and female participants with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of SRP-1003 or placebo.
Official title: A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DM1 (SRP-1003) in Subjects With Type 1 Myotonic Dystrophy Who Are ≥18 to ≤ 65 Years
Key Details
Gender
All
Age Range
18 Years - 65 Years
Study Type
INTERVENTIONAL
Enrollment
78
Start Date
2024-03-04
Completion Date
2026-12-31
Last Updated
2026-08-11
Healthy Volunteers
No
Conditions
Interventions
SRP-1003 IV Infusion
SRP-1003 by IV infusion
Placebo IV Infusion
0.9% sodium chloride (NaCl) calculated volume to match active treatment by IV infusion
SRP-1003 SC Injection
SRP-1003 by SC injection(s)
Placebo SC Injection
0.9% NaCl calculated volume to match active treatment by SC injection(s)
Locations (35)
Liverpool Hospital
Liverpool, New South Wales, Australia
Sunshine Coast University Hospital
Birtinya, Queensland, Australia
Royal Brisbane And Women's Hospital
Herston, Queensland, Australia
Alfred Health
Melbourne, Victoria, Australia
Nucleus Network Brisbane Clinic
Herston, Australia
Mater Hospital Brisbane
South Brisbane, Australia
Universitair Ziekenhuis Gent
Ghent, Belgium
UZ Leuven
Leuven, Belgium
University Of Calgary
Calgary, Canada
University Of Alberta
Edmonton, Canada
McGill University
Montreal, Canada
University Hospital of Angers
Angers, France
Centre Hospitalier Universitaire De Bordeaux
Bordeaux, France
Centre Hospitalier Universitaire De Lille
Lille, France
CHU de Nice - Hôpital Pasteur 2
Nice, France
Assistance Publique Hôpitaux De Paris
Paris, France
Klinikum der Universität München AöR
Munich, Germany
Deutsches Zentrum für Neurodegenerative Erkrankungen e.V.
Ulm, Germany
Fondazione Serena Onlus - Centro Clinico Nemo Brescia
Gussago, Italy
Centro Clinico Nemo (Milan)
Milan, Italy
Azienda Ospedaliera Universitaria Pisana (AOUP)
Pisa, Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, Italy
New Zealand Clinical Research Christchurch
Christchurch, New Zealand
Hospital Universitario Torrecárdenas
Almería, Spain
Hospital Universitari Vall D Hebron
Barcelona, Spain
Hospital Universitario Infanta Sofia
Madrid, Spain
China Medical University Hospital
Taichung, Taiwan
National Taiwan University Hospital
Taipei, Taiwan
Taipei Veterans General Hospital
Taipei, Taiwan
Songklanagarind Hospital
Hat Yai, Changwat Songkhla, Thailand
Siriraj Hospital
Bangkok, Thailand
Lampang Hospital
Lampang, Thailand
The National Hospital for Neurology and Neurosurgery, NHS Foundation Trust
London, United Kingdom
Nottingham University Hospitals NHS Trust
Nottingham, United Kingdom
Southampton General Hospital, NHS Foundation Trust
Southampton, United Kingdom