A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 50 Years of Age With Non-congenital Myotonic Dystrophy Type 1

Recruiting

Phase 1 — Early testing in a small group of people (usually 20–80) to evaluate safety, determine safe dosage, and identify side effects.

Trial locations(8 sites)

United States

University of Florida, 2004 Mowry Road - Site Number: 8400005, Gainesville, Florida

University of South Florida - Neuromuscular Research, 13330 USF Laurel Drive - Site Number: 8400001, Tampa, Florida

Columbia University Medical Center - Neurological Institute, 710 W. 168th, 2nd floor, suite 204 - Site Number : 8400003, New York, New York

Argentina

Hospital Italiano de Buenos Aires, Juan Domingo Peron 4190 - Site Number: 0320001, Buenos Aires

Australia

Investigational Site Number : 0360001, Brisbane, Queensland

Canada

The Montreal Neurological Institute and Hospital, 3801 rue University - Site Number: 1240001, Montreal, Quebec

Israel

Investigational Site Number : 3760002, Ramat Gan

United Kingdom

Investigational Site Number : 8260002, Newcastle upon Tyne