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
Public ClinicalTrials.gov record NCT06844214. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 1/Phase 2 Open-label Single Arm Study With Dose Escalation (Part A), and Dose Expansion (Part B) Parts to Evaluate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants 10 to 55 Years Old With Non-congenital Myotonic Dystrophy Type 1
Study identification
- NCT ID
- NCT06844214
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 32 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 10 Years to 55 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jul 22, 2025
- Primary completion
- Feb 27, 2029
- Completion
- Apr 26, 2032
- Last update posted
- Apr 27, 2026
2025 – 2032
United States locations
- U.S. sites
- 4
- U.S. states
- 3
- U.S. cities
- 4
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Florida, 2004 Mowry Road - Site Number: 8400005 | Gainesville | Florida | 32601 | Recruiting |
| University of South Florida - Neuromuscular Research, 13330 USF Laurel Drive - Site Number: 8400001 | Tampa | Florida | 33612 | Recruiting |
| Columbia University Medical Center - Neurological Institute, 710 W. 168th, 2nd floor, suite 204 - Site Number : 8400003 | New York | New York | 10032 | Recruiting |
| Virginia Commonwealth University Medical Center- Site Number : 8400006 | Richmond | Virginia | 23219 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 5 non-U.S. sites.
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About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT06844214, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Apr 27, 2026 · Synced May 18, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT06844214 live on ClinicalTrials.gov.