RecruitingPhase 1Phase 2NCT06844214

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 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


Sponsor

Sanofi

Enrollment

32 participants

Start Date

Jul 23, 2025

Study Type

INTERVENTIONAL

Conditions

Summary

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 55 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1). The purpose of this study is to evaluate the safety and efficacy of SAR446268 in knocking down dystrophia myotonica protein kinase (DMPK) messenger ribonucleic acid (mRNA) levels and improving neuromuscular function in DM1 participants receiving a single intravenous (IV) administration of SAR446268. The study consists of a dose escalation part (Part A) during which single ascending doses of SAR446268 will be evaluated in 3 distinct cohorts and an optional fourth dose cohort. Once a safe and effective dose is identified, additional participants will be treated in Part B, the dose expansion phase of the study. The study duration will be 112 weeks (approximately 2 years) for each participant in Parts A and B respectively and includes an optional pre-screening period, approximately 8-week screening phase and a 104-week follow-up period post-SAR446268 administration.


Eligibility

Min Age: 10 YearsMax Age: 55 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a biological treatment called SAR446268 for people with myotonic dystrophy. The study is currently recruiting participants at 9 locations.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

BIOLOGICALSAR446268

Pharmaceutical form: Solution for infusion; Route of administration: IV infusion


Locations(9)

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

Gainesville, Florida, United States

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

Tampa, Florida, United States

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

New York, New York, United States

Virginia Commonwealth University Medical Center- Site Number : 8400006

Richmond, Virginia, United States

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

Buenos Aires, Argentina

Investigational Site Number : 0360001

Brisbane, Queensland, Australia

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

Montreal, Quebec, Canada

Investigational Site Number : 3760002

Ramat Gan, Israel

Investigational Site Number : 8260002

Newcastle upon Tyne, United Kingdom

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NCT06844214


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