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Clinical Trial Report

Gene Therapy Trial Report

Summary

Study to Assess the Safety and Efficacy of OCU410ST for Stargardt Disease

NCTID NCT05956626 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Stargardt Disease
Disease Ontology Term DOID:0050817
Compound Name OCU410ST
Sponsor Ocugen
Funder Type Industry
Recruitment Status
Recruiting
Enrollment Count 42
Results Posted Not Available

Therapy Information

Target Gene/Variant RORA
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Subretinal
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV5
Editor Type none
Dose 1 3.75E10 vg/mL
Dose 2 7.5E10 vg/mL
Dose 3 2.25E11 vg/mL (Maximum Tolerated Dose)
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2023-06-30
Completion Date 2025-10-28
Last Update 2025-03-20

Participation Criteria

Eligible Age 6 Years - 65 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL

Locations

No.of Trial Sites 6
Locations United States

Regulatory Information

Has US IND True
FDA Designations Orphan Drug Designation
Recent Updates Reached an alignment with FDA on Phase 2/3 pivotal confirmatory clinical trial

Resources/Links

News and Press Releases
Preclinical Publications