Ireland
--:--IST
Enrolling by invitation Phase 1/2

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

NCT06138743 · tracked via the Priya Life Science UK tracker
Phase
Phase 1/2
Started
2024-03-04
Last updated
2026-10-05

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)

  • Number of Participants with Treatment-emergent Adverse Events Over Time Through End of Study (EOS) — Single-dose phase (Part 1): Up to Day 90 (EOS); multiple-dose phase (Part 2): Up to Day 180 (EOS)

Trial sites (35)

FacilityCityRegionStatus
Liverpool Hospital Liverpool New South Wales
Sunshine Coast University Hospital Birtinya Queensland
Royal Brisbane And Women's Hospital Herston Queensland
Alfred Health Melbourne Victoria
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
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
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