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

a study on Retinitis Pigmentosa

Summary

Eligibility
for people ages 8 years and up (full criteria)
Location
at La Jolla, California and other locations
Dates
study started
completion around

Description

Summary

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.

Official Title

A Phase 3, Multi-Center, Randomized Study to Assess The Efficacy, Safety and Tolerability of Subretinal OCU400 Gene Therapy for the Treatment of Retinitis Pigmentosa

Details

A total of one hundred and fifty (150) RP participants will be enrolled in this study- 75 in each of either two arms, RHO arm (n=75) or Gene agnostic arm (n=75). RHO arm will only enroll participants with confirmed genetic diagnosis of mutation in RHO gene; whereas Gene Agnostic arm will enroll RP Participants based on clinical diagnosis of RP and a confirmed genetic diagnosis with a gene associated with RP.

Subjects in each arm will be randomized into treatment (N=50) and control groups (N=25). Subjects in the treatment group will receive a sequential, bilateral sub-retinal injection of OCU400 if both eyes meet inclusion criteria. Control or untreated group subjects will receive OCU400 subretinal injection after completion of 12-month follow-up.

Keywords

Retinitis Pigmentosa, Modified Gene therapy, RHO, RP, NR2E3, Subretinal Injection, Retinitis, Sub-Retinal Administration of OCU400-301

Eligibility

You can join if…

Open to people ages 8 years and up

  1. Males or females ≥ 8 years of age
  2. Clinical diagnosis of RP with confirmed genetic diagnosis of autosomal dominant RHO mutation or any other mutation associated with RP.
  3. BCVA ≤ 75 letters and ≥25 letters as measured by an ETDRS chart.
  4. Visual field of >5° in any meridian as measured by a III4e isopter or equivalent.
  5. Able to perform a Luminance LDNA at certain light intensity at the Screening visit.
  6. Presence of photoreceptors as determined by SD-OCT

You CAN'T join if...

  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 corticosteroid related IOP spikes or uncontrolled glaucoma.
  4. Absence of large regions of sensitivity in the pericentral and peripheral retinal regions
  5. Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months.
  6. Active ocular/intraocular infection, any history of rhegmatogenous retinal detachment or Current retinal detachment or retinal implant.
  7. Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception

Locations

  • University of Southern Califormia accepting new patients
    La Jolla California 92093 United States
  • University of Southern California, Roski Eye Insitute accepting new patients
    Los Angeles California 90033 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Ocugen
ID
NCT06388200
Phase
Phase 3 Retinitis Pigmentosa Research Study
Study Type
Interventional
Participants
Expecting 150 study participants
Last Updated
Contact us about this trial