Overview
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.
Description
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.
Eligibility
Inclusion Criteria:
- Males or females ≥ 5 years of age
- Clinical diagnosis of RP with confirmed genetic diagnosis of autosomal dominant RHO mutation
- Clinical Diagnosis of Syndromic or Non-Syndromic RP with/without confirmed genetic diagnosis of any other RP associated mutation (except AD-NR2E3)
- BCVA ≤ 75 letters and ≥25 letters as measured by an ETDRS chart
- Visual field of >5° in any meridian as measured by a III4e isopter or equivalent
- Able to perform a Luminance LDNA at certain light intensity at the Screening visit
- Presence of photoreceptors as determined by SD-OCT
Exclusion Criteria:
- Subject lacks evidence of outer nuclear layer
- Previous treatment with a gene-therapy or cell therapy product or treatment with any investigational drug or ocular device within one year.
- History of any corticosteroid contraindication, corticosteroid related IOP spikes or uncontrolled glaucoma.
- Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months.
- Active ocular/intraocular infection, any history of rhegmatogenous retinal detachment or Current retinal detachment or retinal implant.
- Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception