A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa

NCTID	NCT06388200 (View at clinicaltrials.gov)
Description	This is a Phase 3 study to Assess the Efficacy, Safety and Tolerability of OCU400 in patients with retinitis pigmentosa (RP) associated with RHO mutations and patients with any other RP associated mutation with a clinical phenotype of RP. This is a multicenter, assessor blinded and randomized study which will enroll 150 subjects. (Show More)
Development Status	Active
Indication	Retinitis Pigmentosa
Disease Ontology Term	DOID:0110399
Compound Name	OCU400
Sponsor	Ocugen
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	150
Results Posted	Not Available

Target Gene/Variant	NR2E3
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	2.5E10 vg/eye
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	15
Locations	Canada,United States

Has US IND	True
FDA Designations
Recent Updates	OCU400 remains on track to meet 1H 2025 recruitment completion and potential BLA/MAA filings by mid-2026