Contact SCGE




Gene Therapy Trial Report

Summary

Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B

NCTID NCT01505062 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Usher Syndrome, Retinitis Pigmentosa
Disease Ontology Term DOID:0070655
Compound Name SAR421869
Compound Alias UshStat
Compound Description EIAV-CMV-MYO7A-WPRE
Sponsor Sanofi
Funder Type Industry
Recruitment Status
Terminated
Enrollment Count 9 (ACTUAL)
Results Posted View Results

Therapy Information

Target Gene/Variant MYO7A
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Subretinal
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type EIAV
Editor Type none
Dose 1 1.4E5 TU/eye (n=3)
Dose 2 4.7E5 TU/eye (n=3)
Dose 3 1.4E6 TU/eye (n=3)
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2012-01-04
Completion Date 2019-08-16
Last Update 2022-04-28

Participation Criteria

Eligible Age >=6 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL

Locations

No.of Trial Sites 2
Locations United States,France

Regulatory Information

Has US IND True
FDA Designations
Recent Updates Study suspended 2017, terminated 2019; product was discontinued due to risk/benefit profile, license returned to Oxford Biomedica

Resources/Links

Clinical Publications
News and Press Releases
Preclinical Publications
Related NCTID