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
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
Recruiting · NCT06844214 · Registry updated 2026-09-21Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| 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 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Phase 1 / Phase 2 | 2026-09-21 | NCT06844214 |
1 results · Page 1 of 1
Organizations
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| Organizations | Linked trials |
|---|---|
| Sanofi | 254 |
7 results · Page 1 of 1
Research sites
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| Research sites | City | Country | Linked trials |
|---|---|---|---|
| Columbia University Medical Center - Neurological Institute, 710 W. 168th, 2nd floor, suite 204 - Site Number : 8400003 | New York | United States | 1 |
| Hospital Italiano de Buenos Aires, Juan Domingo Peron 4190 - Site Number: 0320001 | Buenos Aires | Argentina | 1 |
| Penn Medicine: University of Pennsylvania Health System- Site Number : 8400009 | Philadelphia | United States | 1 |
| The Montreal Neurological Institute and Hospital, 3801 rue University - Site Number: 1240001 | Montreal | Canada | 1 |
| University of Florida, 2004 Mowry Road - Site Number: 8400005 | Gainesville | United States | 1 |
| University of South Florida - Neuromuscular Research, 13330 USF Laurel Drive - Site Number: 8400001 | Tampa | United States | 1 |
| Virginia Commonwealth University Medical Center- Site Number : 8400006 | Richmond | United States | 1 |