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

Launched by OCUGEN · Apr 24, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new gene therapy called OCU400 to see how well it works, how safe it is, and how well patients can tolerate it. The focus is on people with retinitis pigmentosa (RP), a genetic eye disease that affects vision. This trial is specifically looking for patients with certain genetic mutations, including the RHO mutation, which is commonly associated with RP. The study will take place at multiple locations and aims to enroll 150 participants.

To be eligible for the trial, participants need to be at least 8 years old and have a confirmed diagnosis of RP with specific genetic mutations. They should have a certain level of vision and still have some functioning light-sensitive cells in their eyes. During the trial, participants will receive the OCU400 treatment and will have regular check-ups to monitor their vision and overall health. It's important to note that individuals who have had certain recent eye surgeries, active infections, or specific medical conditions may not qualify for this study. If you or someone you know is affected by RP and meets the criteria, this trial could be an opportunity to explore a potential new treatment.

Gender

ALL

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