A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1
Start Date
7/23/2025
Completion Date
4/27/2032
Summary
This is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 55 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1). The purpose of this study is to evaluate the safety and efficacy of SAR446268 in knocking down dystrophia myotonica protein kinase (DMPK) messenger ribonucleic acid (mRNA) levels and improving neuromuscular function in DM1 participants receiving a single intravenous (IV) administration of SAR446268. The study consists of a dose escalation part (Part A) during which single ascending doses of SAR446268 will be evaluated in 3 distinct cohorts and an optional fourth dose cohort. Once a safe and effective dose is identified, additional participants will be treated in Part B, the dose expansion phase of the study. The study duration will be 112 weeks (approximately 2 years) for each participant in Parts A and B respectively and includes an optional pre-screening period, approximately 8-week screening phase and a 104-week follow-up period post-SAR446268 administration.
Detailed Description
Each participant meeting the eligibility criteria for each of the study parts will receive a single dose administration of SAR446268.
Eligibility Criteria
Age Range: 10 years to 55 years
Interventions
SAR446268
Conditions
Locations
University of Florida, 2004 Mowry Road - Site Number: 8400005
Gainesville, Florida 32601
United States
University of South Florida - Neuromuscular Research, 13330 USF Laurel Drive - Site Number: 8400001
Tampa, Florida 33612
United States
Columbia University Medical Center - Neurological Institute, 710 W. 168th, 2nd floor, suite 204 - Site Number : 8400003
New York, New York 10032
United States
Virginia Commonwealth University Medical Center- Site Number : 8400006
Richmond, Virginia 23219
United States
Hospital Italiano de Buenos Aires, Juan Domingo Peron 4190 - Site Number: 0320001
Buenos Aires, 1181
Argentina
Investigational Site Number : 0360001
Brisbane, Queensland 4029
Australia
The Montreal Neurological Institute and Hospital, 3801 rue University - Site Number: 1240001
Montreal, Quebec H3A 2B4
Canada
Investigational Site Number : 3760002
Ramat Gan, 5262100
Israel
Investigational Site Number : 8260002
Newcastle upon Tyne, NE7 7DN
United Kingdom