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Gene therapy trial hopes to tackle muscle disease

NCT ID NCT06844214

Recruiting now Disease control Sponsor: Sanofi Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a gene therapy called SAR446268 for people aged 10 to 55 with myotonic dystrophy type 1. The therapy is given once through an IV and aims to reduce harmful DMPK RNA and improve muscle function. The trial has two parts: first, finding the safest dose in a small group, then testing that dose in more participants. It will last about two years per person.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
SAR446268 (gene therapy given by IV infusion)
What this could lead to
If successful, this could lead to a treatment that slows or improves muscle function in people with myotonic dystrophy type 1.
What could go wrong
This is an early phase 1/2 trial with only 32 participants, so safety and effectiveness are not yet proven. Gene therapies can have unexpected side effects, and the long-term risks are unknown.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

myotonic dystrophy myotonic dystrophy type 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

MYOTONIC DYSTROPHY

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Columbia University Medical Center - Neurological Institute, 710 W. 168th, 2nd floor, suite 204 - Site Number : 8400003

    RECRUITING

    New York, New York, 10032, United States

  • Hospital Italiano de Buenos Aires, Juan Domingo Peron 4190 - Site Number: 0320001

    RECRUITING

    Buenos Aires, 1181, Argentina

  • Investigational Site Number : 0360001

    RECRUITING

    Brisbane, Queensland, 4029, Australia

  • Investigational Site Number : 3760002

    RECRUITING

    Ramat Gan, 5262100, Israel

  • Investigational Site Number : 8260002

    RECRUITING

    Newcastle upon Tyne, NE7 7DN, United Kingdom

  • The Montreal Neurological Institute and Hospital, 3801 rue University - Site Number: 1240001

    RECRUITING

    Montreal, Quebec, H3A 2B4, Canada

  • University of Florida, 2004 Mowry Road - Site Number: 8400005

    RECRUITING

    Gainesville, Florida, 32601, United States

  • University of South Florida - Neuromuscular Research, 13330 USF Laurel Drive - Site Number: 8400001

    RECRUITING

    Tampa, Florida, 33612, United States

  • Virginia Commonwealth University Medical Center- Site Number : 8400006

    RECRUITING

    Richmond, Virginia, 23219, United States

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