NCT06844214 · Sanofi
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
(BrAAVe)
What this study is about
This is a Phase 1/Phase 2 where both patients and doctors know the treatment given single treatment group$1, conducted at multiple hospitals, and multinational study with SAR446268 for treatment of male and female participants 10 to 50 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1).
View original scientific description
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 50 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1).
Interventions
BIOLOGICAL
SAR446268
Pharmaceutical form: Solution for infusion; Route of administration: IV infusion
Primary outcome measures
Part A and Part B: Incidence of treatment-emergent adverse events (TEAEs) following SAR446268 administration
Time frame: Baseline to Week 52
Number of TEAEs post-SAR446268 administration
Part B: Proportion of participants with at least 40% DMPK mRNA knockdown in muscle biopsy at Weeks 12 and 52 following SAR446268 administration
Time frame: Weeks 12 and 52
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Participants are eligible to be included in the study only if all of the following criteria apply:
- For Part A, participants must be 18 to 50 years of age inclusive, at the time of signing the informed consent.
- For Part B, participants must be as follows:
- 10 to 17 years of age inclusive, at the time of signing the informed consent or,
- 18 to 50 years of age inclusive, at the time of signing the informed consent.
- Participants with non-congenital onset DM1
- Participants presenting with signs of DM1 including myotonia and muscle weakness, as diagnosed previously by a clinician based on medical history.
- Participants with genetic diagnosis of DM1 \[cytosine-thymine-guanine (CTG) repeat length ≥50 in one allele from medical history\]
- Participants who can walk independently for at least 10 meters at screening (orthoses and ankle braces allowed).
- Participants who have been classified according to cardiac risk by the Investigator as:
- Moderate risk p
Where
- Gainesville, Florida
- Tampa, Florida
- New York, New York
- Richmond, Virginia
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 1, 2026 · Source of record for eligibility and locations