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SAR446268

Phase 1

Myotonic Dystrophy | Monoclonal antibody | Neurology |Sanofi|Last Updated: Jun 1, 2026

Success Probability

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment32

FDA Designations

FAST_TRACKORPHAN_DRUG

Clinical trial landscape

SAR446268 · 1 trial · 1 indication

Phase 1 1
NCT06844214A 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 1Myotonic Dystrophy
RECRUITING32 Analytics
PHASE1RECRUITING
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
Myotonic Dystrophy
NCT0684421432
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Study Endpoints

Primary Endpoints

Part A and Part B: Incidence of treatment-emergent adverse events (TEAEs) following SAR446268 administration
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
Weeks 12 and 52

Secondary Endpoints

Part A: Change in 10-meter walk-run test from baseline to Weeks 26 and 52 following SAR446268 administration
Baseline to Week 26 and 52
Part A: Change in myotonia from baseline to Weeks 26 and 52 following SAR446268 administration as measured by the hand opening time (middle finger)
Baseline to Week 26 and 52
Part A: Change in bilateral hand grip test from baseline to Weeks 26 and 52 following SAR446268 administration
Baseline to Week 26 and 52
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
SAR446268EXPERIMENTALParticipants will receive a single dose of SAR446268 on Day 1

Interventions

NameTypeDescription
SAR446268BIOLOGICALPharmaceutical form: Solution for infusion; Route of administration: IV infusion
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Eligibility Criteria

Age Range10 Years to 55 Years
SexALL
Healthy VolunteersNo
Study Sites9

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 55 years of age inclusive, at the time of signing the informed consent. * For Part B, participants must be as follows: * 10 to 17 years...

Countries:United StatesArgentinaAustraliaCanadaIsraelUnited Kingdom
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Frequently asked questions about SAR446268

What is SAR446268 used for?

SAR446268 is an investigational monoclonal antibody being developed for myotonic dystrophy, specifically non-congenital myotonic dystrophy type 1. It is currently in Phase 1 clinical development and has received Fast Track and Orphan Drug designations from the FDA.

How does SAR446268 work?

SAR446268 is a monoclonal antibody designed to target the underlying pathology of myotonic dystrophy type 1. While the specific molecular target is not disclosed, as a monoclonal antibody it is intended to bind to a specific protein or molecule involved in the disease process.

Who is developing SAR446268?

SAR446268 is being developed by Sanofi, a global biopharmaceutical company listed on the stock exchange under the ticker symbol SNY. The drug is currently in Phase 1 clinical trials for the treatment of myotonic dystrophy.

What phase is SAR446268 in?

SAR446268 is in Phase 1 clinical development. It is an investigational drug and has not yet been approved by regulatory authorities. The ongoing Phase 1 trial is recruiting participants to evaluate the safety, tolerability, and efficacy of the drug.

What clinical trials is SAR446268 in?

SAR446268 is being studied in a Phase 1 clinical trial registered as NCT06844214. This open-label, uncontrolled study is recruiting 32 participants aged 10 to 55 years with non-congenital myotonic dystrophy type 1. The trial is being conducted in the United States, Argentina, Australia, Canada, Israel, and the United Kingdom.

Is SAR446268 FDA approved?

SAR446268 is not FDA approved. It is an investigational drug currently in Phase 1 clinical trials. The FDA has granted it Fast Track and Orphan Drug designations, which are intended to expedite development and review for serious conditions like myotonic dystrophy.