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Clinical Trials in the UK / NCT06138743
Recruiting Phase 1/2

Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy

NCT06138743 · tracked via the Priya Life Science UK tracker
Sponsor
Sarepta Therapeutics, Inc.
Phase
Phase 1/2
Started
2024-03-04
Last updated
2026-08-11

Condition(s) studied

Myotonic Dystrophy 1

Investigational drug(s) / intervention(s)

SRP-1003 IV InfusionPlacebo IV InfusionSRP-1003 SC InjectionPlacebo SC Injection

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)

Study 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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
65 Years
Healthy volunteers
No
Key Inclusion Criteria: * Genetically confirmed diagnosis of DM1 * Clinician-assessed signs of DM1 including clinically apparent myotonia * Onset of DM1 symptoms occurred after the age of 12 years * Walk for at least 10 meters independently at screening * Participants of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of study or last dose of study drug, whichever is later. Participants must not donate sperm or eggs during the study and for at least 90 days following the end of study or last dose of study drug whichever is later. Key Exclusion Criteria: * Inadequately controlled diabetes * Confirmed diagnosis of congenital DM1 * Uncontrolled hypertension * History of tibialis anterior (TA) biopsy within 3 months of Day 1 or planning to undergo TA biopsies during the study period * Clinically significant cardiac, liver or renal disease * Human immunodeficiency virus infection (seropositive) at screening * Seropositive for hepatitis B or hepatitis C at screening * Untreated or poorly controlled epilepsy * Treatment with anti-myotonia medication within a period of 5 half-lives of the medication prior to screening. * Abnormal coagulation parameters at screening including platelet count, international normalized ratio, prothrombin time, and activated partial thromboplastin time Note: Additional inclusion/exclusion criteria may apply per protocol

Primary outcome measure(s)

Trial sites (35)

FacilityCityRegionStatus
Liverpool Hospital Liverpool New South Wales Recruiting
Sunshine Coast University Hospital Birtinya Queensland Recruiting
Royal Brisbane And Women's Hospital Herston Queensland Recruiting
Alfred Health Melbourne Victoria Recruiting
Nucleus Network Brisbane Clinic Herston Australia Recruiting
Mater Hospital Brisbane South Brisbane Australia Recruiting
Universitair Ziekenhuis Gent Ghent Belgium Recruiting
UZ Leuven Leuven Belgium Recruiting
University Of Calgary Calgary Canada Recruiting
University Of Alberta Edmonton Canada Recruiting
McGill University Montreal Canada Recruiting
University Hospital of Angers Angers France Recruiting
Centre Hospitalier Universitaire De Bordeaux Bordeaux France Recruiting
Centre Hospitalier Universitaire De Lille Lille France Recruiting
CHU de Nice - Hôpital Pasteur 2 Nice France Recruiting
Assistance Publique Hôpitaux De Paris Paris France Recruiting
Klinikum der Universität München AöR Munich Germany Recruiting
Deutsches Zentrum für Neurodegenerative Erkrankungen e.V. Ulm Germany Recruiting
Fondazione Serena Onlus - Centro Clinico Nemo Brescia Gussago Italy Recruiting
Centro Clinico Nemo (Milan) Milan Italy Recruiting
Azienda Ospedaliera Universitaria Pisana (AOUP) Pisa Italy Recruiting
Fondazione Policlinico Universitario Agostino Gemelli IRCCS Roma Italy Recruiting
New Zealand Clinical Research Christchurch Christchurch New Zealand Recruiting
Hospital Universitario Torrecárdenas Almería Spain Recruiting
Hospital Universitari Vall D Hebron Barcelona Spain Recruiting
Hospital Universitario Infanta Sofia Madrid Spain Recruiting
China Medical University Hospital Taichung Taiwan Recruiting
National Taiwan University Hospital Taipei Taiwan Recruiting
Taipei Veterans General Hospital Taipei Taiwan Recruiting
Songklanagarind Hospital Hat Yai Changwat Songkhla Recruiting
Siriraj Hospital Bangkok Thailand Recruiting
Lampang Hospital Lampang Thailand Recruiting
The National Hospital for Neurology and Neurosurgery, NHS Foundation Trust London United Kingdom Recruiting
Nottingham University Hospitals NHS Trust Nottingham United Kingdom Recruiting
Southampton General Hospital, NHS Foundation Trust Southampton United Kingdom Recruiting

More Sarepta Therapeutics, Inc. trials in the UK

Other trials for the same condition

Official registry record

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.

View NCT06138743 on ClinicalTrials.gov ↗ ← All trials in the UK