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

Phase 3Active Not RecruitingNCT06388200

Ocugen140 enrolled

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 140 subjects. Study has completed enrollment of all 140 subjects.

A total of one hundred and forty (140) RP participants will be enrolled in this study into RHO arm or Gene agnostic arm. 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 and control groups with a 2:1 ratio. 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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

SINGLE

Enrollment

140

Conditions

Retinitis Pigmentosa

Interventions

Sub-Retinal Administration of OCU400-301GENETIC

Sub-Retinal Administration of OCU400-301

Eligibility

Sex: ALLMin age: 3 Years

Medical Language ↔ Plain English

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

Locations (17)

Associated Retina Consultants

Phoenix, Arizona, United States

University of Southern Califormia

La Jolla, California, United States

Outcomes

Primary Outcomes

Change in functional vision from baseline to week 52 in pooled analysis when study eyes in treatment group were compared to study eyes in untreated control

Change in functional vision from baseline to week 52 in pooled analysis from retinitis pigmentosa subjects when study eyes in treatment group were compared to study eyes in untreated control, as measured by the ability of a study participant to navigate through a maze in Luminance Dependent Navigation Assessment (LDNA)

Time frame: 52 weeks

Secondary Outcomes

Change in functional vision from baseline to week 52 in all the treated eyes from RP subjects (study eyes + fellow treated eyes) when compared to all the eyes (study eyes and fellow eyes) in untreated control group, as measured by the ability of a study participant to navigate through a maze in Luminance Dependent Navigation Assessment (LDNA).

Time frame: 52 weeks

Change from Baseline in visual function in patients with retinitis pigmentosa when treatment group were compared to untreated controls

Change from Baseline in visual function in patients with retinitis pigmentosa when treatment group were compared to untreated controls in all RP subjects, as assessed by binocular low luminance visual acuity when using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score at Week 52.

Time frame: 52 weeks

Change from baseline in LDNA Lux-level results in treated subjects when compared to the untreated controls

Change from baseline in LDNA Lux-level results in all RP subjects in treated subjects when compared to the untreated controls at weeks 12, 24, 36, and 52

Time frame: 52 weeks

Number of participants with change in visual function in Gene Agnostic Arm as assessed by LLVA letter scores

Data from ClinicalTrials.gov

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