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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
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.