Clinical Trials Logo

Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT06388200
Other study ID # OCU400-301
Secondary ID
Status Recruiting
Phase Phase 3
First received
Last updated
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
Health authority
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

Study Design


Related Conditions & MeSH terms


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
See also
  Status Clinical Trial Phase
Recruiting NCT01432847 - Cell Collection to Study Eye Diseases
Completed NCT04983914 - Retrospective NIS to Evaluate the Patient Benefit of TES
Recruiting NCT03845218 - Retinitis Pigmentosa Clinical Measures and Repeatability Testing of Potential Outcome Measures
Completed NCT00231010 - Molecular Genetics of Retinal Degenerations
Active, not recruiting NCT04611503 - PDE6A Gene Therapy for Retinitis Pigmentosa Phase 1/Phase 2
Completed NCT02909985 - Visual Activity Evoked by Infrared in Humans After Dark Adaptation N/A
Recruiting NCT01914913 - Clinical Study to Evaluate Safety and Efficacy of BMMNC in Retinitis Pigmentosa Phase 1/Phase 2
Completed NCT01949623 - Biomarkers In Retinitis Pigmentosa (BIRP) N/A
Completed NCT01835002 - Transcorneal Electrical Stimulation - Multicenter Safety Study N/A
Completed NCT00407602 - Argus® II Retinal Stimulation System Feasibility Protocol N/A
Completed NCT00515814 - Retina Implant Pilot Trial to Evaluate Safety & Efficacy in Blind Patients Having Degenerated Photo-receptors N/A
Completed NCT00100230 - DHA and X-Linked Retinitis Pigmentosa Phase 2
Active, not recruiting NCT00378742 - Repository for Inherited Eye Diseases
Terminated NCT05085964 - An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa Phase 2
Recruiting NCT05805007 - Safety and Tolerability Study of Gene Editing Drug ZVS203e in Participants With Retinitis Pigmentosa Early Phase 1
Recruiting NCT05909488 - Role of UC-MSC and CM to Inhibit Vision Loss in Retinitis Pigmentosa Phase I/II Phase 2/Phase 3
Recruiting NCT06291935 - Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene Phase 1
Recruiting NCT03078309 - The Effects of Cannabis on Visual Functions in Healthy and Retinitis Pigmentosa Patients Early Phase 1
Completed NCT04238858 - Effects of Subtenon-injected Autologous Platelet-rich Plasma on Visual Functions in Eyes With Retinitis Pigmentosa N/A
Active, not recruiting NCT01680510 - The Effect of Oral Administration of 9-cis β Carotene Rich Powder of the Alga Dunaliella Bardawil Phase 1/Phase 2