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

Retinitis Pigmentosa Clinical Trial

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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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06388200
• Other study ID #: OCU400-301
• Status: Recruiting
• Phase: Phase 3
• Start date: April 26, 2024
• Est. completion date: October 30, 2026

Study information

• Verified date: April 2024
• Source: Ocugen
• Contact: Umair Qazi, MD, MPH
• Phone: 484-237-3390
• Email: umair.qazi[@]ocugen.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: October 30, 2026
• Est. primary completion date: June 30, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 8 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Retinitis
• Retinitis Pigmentosa

Intervention

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

Locations

Country	Name	City	State
United States	Retina Consultants of Texas	Bellaire	Texas
United States	Associated Retina Consultants	Phoenix	Arizona

Sponsors (1)

Lead Sponsor	Collaborator
Ocugen

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Patients Global Impression of Change (PGIC) score		52 weeks
Primary	Number of participants with change in functional vision in the study eye of RP subjects as assessed by LDNA	Change in the LDNA assessment from screening in the study eye of RP subjects with RHO mutation will be compared to controls	52 weeks
Primary	Number of participants with change in functional vision in the study eye in Gene Agnostic group as assessed by LDNA	Change in the LDNA assessment from screening in the study eye in Gene Agnostic Arm subjects will be compared to controls	52 weeks
Secondary	Number of participants with change in functional vision in the all treated eyes of RP subjects as assessed by LDNA	Change in the LDNA assessment from screening in all treated eyes of RP subjects with RHO mutation will be compared to controls	52 weeks
Secondary	Number of participants with change in functional vision in the all treated eyes of subjects in Gene Agnostic Arm as assessed by LDNA	Change in the LDNA assessment from screening in all treated eyes of subjects in Gene Agnostic Arm subjects will be compared to controls	52 weeks
Secondary	Number of participants with change in visual function in subjects with RHO mutation as assessed by LLVA letter score	Change in LLVA letter scores in RP subjects with mutations in RHO will be compared to controls	52 weeks
Secondary	Number of participants with change in visual function in Gene Agnostic Arm as assessed by LLVA letter scores	Change in LLVA letter scores in Gene Agnostic Arm subjects will be compared to controls	52 weeks
Secondary	Ocular and non-ocular Events	Incidence and severity of Study Drug-related adverse events (SDAE), Treatment-Emergent adverse events (TEAEs), Serious adverse events (SAEs) and Adverse Events of Special Interest (AESI).	52 weeks