A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa (NCT06388200) | Clinical Trial Compass
Active — Not RecruitingPhase 3
A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa
United States140 participantsStarted 2024-06-18
Plain-language 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 140 subjects. Study has completed enrollment of all 140 subjects.
Who can participate
Age range3 Years
SexALL
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Inclusion criteria
✓. Males or females ≥ 3 years of age
✓. Confirmed genetic diagnosis of autosomal dominant RHO mutation with clinical diagnosis of RP
✓. Clinical Diagnosis of Syndromic or Non-Syndromic RP with/without confirmed genetic diagnosis of any other RP associated mutation (except AD-NR2E3)
✓. BCVA ≤ 80 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.
What they're measuring
1
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