Find Myotonic Dystrophy Type 2 Clinical Trials Near You
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
Status: Recruiting
Location: See all (35) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 65
Healthy Volunteers: f
View:
• 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.
Locations
Other Locations
Australia
Sunshine Coast University Hospital
RECRUITING
Birtinya
Nucleus Network Brisbane Clinic
RECRUITING
Herston
Royal Brisbane And Women's Hospital
RECRUITING
Herston
Liverpool Hospital
RECRUITING
Liverpool
Alfred Health
RECRUITING
Melbourne
Mater Hospital Brisbane
RECRUITING
South Brisbane
Belgium
Universitair Ziekenhuis Gent
RECRUITING
Ghent
UZ Leuven
RECRUITING
Leuven
Canada
University Of Calgary
RECRUITING
Calgary
University Of Alberta
RECRUITING
Edmonton
McGill University
RECRUITING
Montreal
France
University Hospital of Angers
RECRUITING
Angers
Centre Hospitalier Universitaire De Bordeaux
RECRUITING
Bordeaux
Centre Hospitalier Universitaire De Lille
RECRUITING
Lille
CHU de Nice - Hôpital Pasteur 2
RECRUITING
Nice
Assistance Publique Hôpitaux De Paris
RECRUITING
Paris
Germany
Klinikum der Universität München AöR
RECRUITING
Munich
Deutsches Zentrum für Neurodegenerative Erkrankungen e.V.
RECRUITING
Ulm
Italy
Fondazione Serena Onlus - Centro Clinico Nemo Brescia