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

Recruiting Phase 1 Phase 2 Interventional Study
Myotonic Dystrophy
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
10 – 55
Sex
Any
Trial phase
Phase 1/2
Study type
Interventional
Purpose
Treatment
Participants needed
32 (estimated)
Sponsor
Sanofi · Industry
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About This Trial
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) lev…
Trial Locations
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Eligibility Criteria
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…
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800-633-1610

contact-us@sanofi.com

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