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)
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.
| Facility | City | Region | Status |
|---|---|---|---|
| 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 |
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