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Clinical Trial Report

Gene Therapy Trial Report

Summary

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria


NCTID NCT05972629 (View at clinicaltrials.gov)
Description
Indication Phenylketonuria
Compound Name SAR444836
Sponsor Sanofi
Funder Type Industry
Status
Recruiting
Enrollment Count 32

Therapy Information


Target Gene/Variant PAH
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV SNY001
Editor Type
Dose 1 Undisclosed dose 1
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2023-06-23
Completion Date 2027-07-31
Last Update 2024-11-01

Participation Criteria


Eligible Age 18 Years - 65 Years
Standard Ages Adult, Older adult
Eligible Sex ALL

Locations


No.of Trial Sites 5
Locations Turkey,United States,Israel

Regulatory Information


Has US IND True
Recent Updates Deprioritized by Sponsor

Resources/Links