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

April 24, 2024 updated by: Ocugen

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed 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.

Study Type

Interventional

Enrollment (Estimated)

150

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85020
    • Texas
      • Bellaire, Texas, United States, 77401

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Single

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: RHO Arm
Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10E10 vg/eye
Genetic: Sub-Retinal Administration of OCU400-301
Sub-Retinal Administration of OCU400-301
Experimental: Gene Agnostic Arm
Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10E10 vg/eye
Genetic: Sub-Retinal Administration of OCU400-301
Sub-Retinal Administration of OCU400-301
No Intervention: Control for RHO Arm
Will not receive any active study intervention
No Intervention: Control for Gene Agnostic Arm
Will not receive any active study intervention

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of participants with change in functional vision in the study eye of RP subjects as assessed by LDNA
Time Frame: 52 weeks
Change in the LDNA assessment from screening in the study eye of RP subjects with RHO mutation will be compared to controls
52 weeks
Number of participants with change in functional vision in the study eye in Gene Agnostic group as assessed by LDNA
Time Frame: 52 weeks
Change in the LDNA assessment from screening in the study eye in Gene Agnostic Arm subjects will be compared to controls
52 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of participants with change in functional vision in the all treated eyes of RP subjects as assessed by LDNA
Time Frame: 52 weeks
Change in the LDNA assessment from screening in all treated eyes of RP subjects with RHO mutation will be compared to controls
52 weeks
Number of participants with change in functional vision in the all treated eyes of subjects in Gene Agnostic Arm as assessed by LDNA
Time Frame: 52 weeks
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
Number of participants with change in visual function in subjects with RHO mutation as assessed by LLVA letter score
Time Frame: 52 weeks
Change in LLVA letter scores in RP subjects with mutations in RHO will be compared to controls
52 weeks
Number of participants with change in visual function in Gene Agnostic Arm as assessed by LLVA letter scores
Time Frame: 52 weeks
Change in LLVA letter scores in Gene Agnostic Arm subjects will be compared to controls
52 weeks
Ocular and non-ocular Events
Time Frame: 52 weeks
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

Other Outcome Measures

Outcome Measure
Time Frame
Patients Global Impression of Change (PGIC) score
Time Frame: 52 weeks
52 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ocugen

Investigators

  • Study Chair: Huma Qamar, Ocugen

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

April 26, 2024

Primary Completion (Estimated)

June 30, 2025

Study Completion (Estimated)

October 30, 2026

Study Registration Dates

First Submitted

April 9, 2024

First Submitted That Met QC Criteria

April 24, 2024

First Posted (Actual)

April 29, 2024

Study Record Updates

Last Update Posted (Actual)

April 29, 2024

Last Update Submitted That Met QC Criteria

April 24, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • OCU400-301

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Retinitis Pigmentosa

Clinical Trials on Sub-Retinal Administration of OCU400-301

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Rwanda

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe