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

Part of paid clinical trials in Gainesville, Florida.

Sponsor
Sanofi
Study ID
NCT06844214
Phase
PHASE1/PHASE2
Status
Recruiting
Apply to this trial

Conditions

  • Myotonic Dystrophy

Eligibility Criteria

Sex
ALL
Age
10 Years - 55 Years
Healthy Volunteers
Not accepted

Interventions

  • SAR446268 — BIOLOGICAL
    Pharmaceutical form: Solution for infusion; Route of administration: IV infusion

Study Details

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.

Key Dates

Start date
Jul 23, 2025
Status verified
May 2026
Primary completion
Feb 28, 2029
Completion
Apr 27, 2032

Study Design

Enrollment
32 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: SAR446268
    Participants will receive a single dose of SAR446268 on Day 1

Primary Outcome Measure

Part A and Part B: Incidence of treatment-emergent adverse events (TEAEs) following SAR446268 administration [ Time Frame: Baseline to Week 52 ]

Central Contacts

  • Trial Transparency email recommended (Toll free for US & Canada)
    800-633-1610
    [email protected]

Locations (4)

FacilityCityStateZIPSite coordinators
University of Florida, 2004 Mowry Road - Site Number: 8400005GainesvilleFlorida32601
Melissa Lewis
[email protected]
352-273-8219
Dr. Subramony (PRINCIPAL_INVESTIGATOR)
University of South Florida - Neuromuscular Research, 13330 USF Laurel Drive - Site Number: 8400001TampaFlorida33612
Sruthi Kommi Reddy
[email protected]
Dr. Tuan Vu (PRINCIPAL_INVESTIGATOR)
Columbia University Medical Center - Neurological Institute, 710 W. 168th, 2nd floor, suite 204 - Site Number : 8400003New YorkNew York10032
Jorge Cabrera
[email protected]
212-305-7462
Dr. Christina Ulane (PRINCIPAL_INVESTIGATOR)
Virginia Commonwealth University Medical Center- Site Number : 8400006RichmondVirginia23219-

Find similar trials in Gainesville, FL

By research site
University of Florida, 2004 Mowry Road· Gainesville, FLUniversity of South Florida - Neuromuscular Research, 13330 USF Laurel Drive· Tampa, FLColumbia University Medical Center - Neurological Institute, 710 W. 168th, 2nd floor, suite 204· New York, NYVirginia Commonwealth University Medical Center· Richmond, VA

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