Clinical trial NCT06388200: A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Retinitis Pigmentosa
Keywords: Modified Gene therapy
Retinitis Pigmentosa
RHO
RP
NR2E3
Subretinal Injection
Lead sponsor: Ocugen
Sponsor class: INDUSTRY
First posted: 2024-04-29
Enrollment: 150
Sex: ALL
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.

Detailed 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 criteria: Inclusion Criteria:

1. Males or females ≥ 5 years of age
2. Clinical diagnosis of RP with confirmed genetic diagnosis of autosomal dominant RHO mutation
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 ≤ 75 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

Primary outcomes:
measure: Number of participants with change in functional vision in the study eye of RP subjects as assessed by LDNA
timeFrame: 52 weeks
measure: Number of participants with change in functional vision in the study eye in Gene Agnostic group as assessed by LDNA
timeFrame: 52 weeks
Secondary outcomes:
measure: Number of participants with change in functional vision in the all treated eyes of RP subjects as assessed by LDNA
timeFrame: 52 weeks
measure: Number of participants with change in functional vision in the all treated eyes of subjects in Gene Agnostic Arm as assessed by LDNA
timeFrame: 52 weeks
measure: Number of participants with change in visual function in subjects with RHO mutation as assessed by LLVA letter score
timeFrame: 52 weeks
measure: Number of participants with change in visual function in Gene Agnostic Arm as assessed by LLVA letter scores
timeFrame: 52 weeks
measure: Ocular and non-ocular Events
timeFrame: 52 weeks
Other outcomes:
measure: Patients Global Impression of Change (PGIC) score
timeFrame: 52 weeks
Locations:
city: Phoenix
country: United States
facility: Associated Retina Consultants
state: Arizona
city: La Jolla
country: United States
facility: University of Southern Califormia
state: California
city: Los Angeles
country: United States
facility: University of Southern California, Roski Eye Insitute
state: California
city: Deerfield Beach
country: United States
facility: Advanced Research, LLC.
state: Florida
city: Miami
country: United States
facility: Bascom Palmer Eye Institute, University of Miami, Miller School of Medicine
state: Florida
city: Erie
country: United States
facility: Erie Retina Research LLC
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt Eye Institute
state: Tennessee
city: Bellaire
country: United States
facility: Retina Consultants of Texas
state: Texas
city: Dallas
country: United States
facility: Retina Foundation of the Southwest
state: Texas
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: McAllen
country: United States
facility: Valley Retina Institute
state: Texas
city: La Crosse
country: United States
facility: Gundersen Health System
state: Wisconsin
city: Calgary
country: Canada
facility: Calgary Retina Consultants
state: Alberta
city: Edmonton
country: Canada
facility: University of Alberta
state: Alberta
city: Vancouver
country: Canada
facility: The University of British Columbia
state: British Columbia
city: Toronto
country: Canada
facility: Sunnybrook Health Sciences Centre
state: Ontario
Study officials:
affiliation: Ocugen
name: Huma Qamar
role: STUDY_CHAIR
References: