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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
Development Status Inactive
Indication Phenylketonuria (PKU)
Disease Ontology Term DOID:9281
Compound Name SAR444836
Sponsor Sanofi
Funder Type Industry
Recruitment Status
Recruiting
Enrollment Count 32
Results Posted Not Available

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 none
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 2025-03-27

Participation Criteria


Eligible Age 18 Years - 65 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 9
Locations Argentina,Turkey,United States,Brazil,Israel

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Deprioritized by Sponsor

Resources/Links