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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 50 Years of Age With Non-congenital Myotonic Dystrophy Type 1
NCTID
NCT06844214
(View at clinicaltrials.gov)
Description
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 50 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 4th 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 110 weeks (approximately 2 years) for each participant in Parts A and B respectively and includes a 6-week screening phase and a 104-week follow-up period post-SAR446268 administration.
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Development Status
Active
Indication
Myotonic Dystrophy
Disease Ontology Term
DOID:11722
Compound Name
SAR446268
Sponsor
Sanofi
Funder Type
Industry
Recruitment Status
Not yet recruiting
Enrollment Count
32
Results Posted
Not Available
Therapy Information
Target Gene/Variant
DMPK
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Gene inactivation
Route of Administration
Intravenous
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV.SAN011
Editor Type
Dose 1
3 cohort dose escalation, undisclosed concentrations
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2025-02-19
Completion Date
2030-02-28
Last Update
2025-03-12
Participation Criteria
Eligible Age
10 Years - 50 Years
Standard Ages
Child, Adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
Locations
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Resources/Links
Resources/Links
No External Links Available.