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Gene Therapy
Gene Therapy for Retinitis Pigmentosa (liMeliGhT Trial)
Phase 3
Recruiting
Research Sponsored by Ocugen
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Clinical diagnosis of RP with confirmed genetic diagnosis of autosomal dominant RHO mutation or any other mutation associated with RP
Must not have
Previous treatment with a gene-therapy or cell therapy product or treatment with any investigational drug or ocular device within one year
Active ocular/intraocular infection, any history of rhegmatogenous retinal detachment or Current retinal detachment or retinal implant
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 52 weeks
Awards & highlights
No Placebo-Only Group
Pivotal Trial
Summary
"This trial is testing a new drug called OCU400 in 150 patients with retinitis pigmentosa (RP) caused by RHO mutations or other RP-associated mutations. The study will evaluate how
Who is the study for?
This trial is for individuals with Retinitis Pigmentosa, specifically those with RHO mutations or other RP-associated mutations. It's a large study involving multiple centers and will include 150 participants.
What is being tested?
The trial is testing the efficacy, safety, and tolerability of OCU400 gene therapy administered under the retina. The study design involves randomly assigning patients to receive this treatment while keeping assessors blind to who receives what.
What are the potential side effects?
While specific side effects are not listed here, gene therapies like OCU400 may cause immune reactions, eye discomfort or damage at the injection site, changes in vision, and potential unknown long-term effects.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have RP with a confirmed genetic mutation.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I haven't had gene or cell therapy, or any experimental treatments in the last year.
Select...
I do not have an active eye infection, history of retinal detachment, or a retinal implant.
Select...
I have had eye pressure spikes or uncontrolled glaucoma due to steroids.
Select...
I have not had cataract surgery in the last 3 months, YAG capsulotomy in the last month, or any other eye surgery in the last 6 months.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 52 weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~52 weeks
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Number of participants with change in functional vision in the study eye in Gene Agnostic group as assessed by LDNA
Number of participants with change in functional vision in the study eye of RP subjects as assessed by LDNA
Secondary study objectives
Number of participants with change in functional vision in the all treated eyes of RP subjects as assessed by LDNA
Number of participants with change in functional vision in the all treated eyes of subjects in Gene Agnostic Arm as assessed by LDNA
Number of participants with change in visual function in Gene Agnostic Arm as assessed by LLVA letter scores
+2 moreAwards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Trial Design
4Treatment groups
Experimental Treatment
Active Control
Group I: RHO ArmExperimental Treatment1 Intervention
Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10E10 vg/eye
Group II: Gene Agnostic ArmExperimental Treatment1 Intervention
Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10E10 vg/eye
Group III: Control for Gene Agnostic ArmActive Control1 Intervention
Will not receive any active study intervention
Group IV: Control for RHO ArmActive Control1 Intervention
Will not receive any active study intervention
Find a Location
Who is running the clinical trial?
OcugenLead Sponsor
11 Previous Clinical Trials
965 Total Patients Enrolled
2 Trials studying Retinitis Pigmentosa
22 Patients Enrolled for Retinitis Pigmentosa
Huma QamarStudy ChairOcugen