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

Ocugen

Status and phase: Enrolling; Phase 3

Conditions: Retinitis Pigmentosa

Treatments: Genetic: Sub-Retinal Administration of OCU400-301

Study type: Interventional

Funder types: Industry

Identifiers

NCT06388200

OCU400-301

Details and patient eligibility

About

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.

Full 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.

Enrollment: 150 estimated patients

Sex: All

Ages: 8+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

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

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 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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150 participants in 4 patient groups

RHO Arm

Experimental group

Description:

Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10E10 vg/eye

Treatment:

Genetic: Sub-Retinal Administration of OCU400-301

Gene Agnostic Arm

Experimental group

Description:

Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10E10 vg/eye

Treatment:

Genetic: Sub-Retinal Administration of OCU400-301

Control for RHO Arm

No Intervention group

Description:

Will not receive any active study intervention

Control for Gene Agnostic Arm

No Intervention group

Description:

Will not receive any active study intervention