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Gene Therapy Trial Report

Summary

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


NCTID NCT06388200 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Retinitis Pigmentosa
Disease Ontology Term DOID:0110399
Compound Name OCU400
Sponsor Ocugen
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 140 (ACTUAL)
Results Posted Not Available

Therapy Information


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
Delivery System Viral transduction
Vector Type AAV5
Editor Type none
Dose 1 5E19 vg/eye
Dose 2 1E10 vg/eye
Dose 3 5E10 vg/eye
Dose 4 Phase 3 dose: 2.5E10 vg/eye

Study Record Dates


Current Stage Phase3
Submit Date 2024-04-09
Completion Date 2027-02-12
Last Update 2026-03-05

Participation Criteria


Eligible Age >=3 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1. Males or females ≥ 3 years of age 2. Confirmed genetic diagnosis of autosomal dominant RHO mutation with clinical diagnosis of RP 3. Clinical Diagnosis of Syndromic or Non-Syndromic RP with/without confirmed genetic diagnosis of any other RP associated mutation (except AD-NR2E3) 4. BCVA ≤ 80 letters and ≥25 letters as measured by an ETDRS chart 5. Visual field of \>5° in any meridian as measured by a III4e isopter or equivalent 6. Able to perform a Luminance LDNA at certain light intensity at the Screening visit 7. Presence of photoreceptors as determined by SD-OCT Exclusion Criteria: 1. Subject lacks evidence of outer nuclear layer 2. Previous treatment with a gene-therapy or cell therapy product or treatment with any investigational drug or ocular device within one year. 3. History of any corticosteroid contraindication, corticosteroid related IOP spikes or uncontrolled glaucoma. 4. Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months. 5. Active ocular/intraocular infection, any history of rhegmatogenous retinal detachment or Current retinal detachment or retinal implant. 6. Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 17
Locations Canada,United States

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation, Regenerative Medicine Advanced Therapy
Recent Updates Potential BLA/MAA filings by mid-2026

Resources/Links