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Gene Therapy

OCU400 for Retinitis Pigmentosa (OCU400 Trial)

Phase 1 & 2

Recruiting

Research Sponsored by Ocugen

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 2 days

Treatment Varies

Follow Up 1 year

Awards & highlights

OCU400 Trial Summary

This trial is testing a new treatment for retinitis pigmentosa, a degenerative eye disease. The treatment involves injecting a drug into the retina, and the trial will assess the safety of the treatment.

Who is the study for?

This trial is for adults over 18 with certain genetic mutations causing retinitis pigmentosa or Leber Congenital Amaurosis. Participants must have a specific level of visual impairment and be able to perform a mobility test in low light. They can't join if they're pregnant, breastfeeding, have had recent eye surgery, previous gene therapy, or any condition that might affect the study results.Check my eligibility

What is being tested?

The trial tests three doses (low, medium, high) of OCU400 on patients with retinitis pigmentosa and Leber Congenital Amaurosis to evaluate its safety and effectiveness. It includes an initial phase followed by a natural history study across multiple centers involving up to 124 subjects.See study design

What are the potential side effects?

Potential side effects are not explicitly listed but may include typical risks associated with subretinal injections such as eye irritation, infection risk post-surgery, inflammation inside the eye or vision changes due to the procedure.

OCU400 Trial Timeline

Screening ~ 2 days0 visits

Treatment ~ Varies

Follow Up ~ 1 year

Screening ~ 2 days

Treatment ~ Varies

Follow Up ~1 year

This trial's timeline: 2 days for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Serious adverse events (SAEs)

Study Drug-related adverse events (SDAE)

Treatment-Emergent adverse events (TEAEs)

Secondary outcome measures

Best-corrected visual acuity (BCVA)

Indirect ophthalmoscopy

Intraocular pressure (IOP)

+5 more

Other outcome measures

Changes in ellipsoid zone width/length on wide-field 45° SD-OCT

Contrast sensitivity

Full Field Electroretinogram

+5 more

OCU400 Trial Design

6Treatment groups

Experimental Treatment

Active Control

Group I: Pediatric ArmExperimental Treatment1 Intervention

Pediatric subjects will receive the medium dose concentration and will have subjects with RP and LCA

Group II: Cohort 3 (High Dose)Experimental Treatment1 Intervention

Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation, RHO mutations subgroup and LCA patients with CEP290

Group III: Cohort 2 (Mid Dose)Experimental Treatment1 Intervention

Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation or RHO mutations subgroup

Group IV: Cohort 1 (Low Dose)Experimental Treatment1 Intervention

Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation or RHO mutations subgroup

Group V: Adult ArmExperimental Treatment1 Intervention

Following DSMB confirmation, adult LCA subjects with CEP290 mutation will receive a medium dose concentration of OCU400.

Group VI: Natural History Study (OCU400-104)Active Control1 Intervention

A Prospective and Retrospective Natural History Study of RP and LCA: This is an observatory study for the prospective natural history of RP and LCA in adult and pediatric subjects. The study will also collect and review retrospective data and ophthalmology examination of natural history and progression of disease for all subjects starting with earliest timepoint on or after the date of their diagnosis of RP or LCA. Subjects will be seen up to a total of four times during the 12 months of the Observational Period, at baseline, 3 months, 6 months and 12 months. A total of up to 100 subjects will be enrolled in the study, including: Approximately 76 newly enrolled subjects consisting of 50 adult RP subjects 6 adult LCA subjects 20 pediatric RP/LCA subjects. Up to 24 subjects that reconsent from the OCU400-101 study (subjects from OCU400-101 will provide data on their untreated eye)

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

OcugenLead Sponsor

9 Previous Clinical Trials

943 Total Patients Enrolled

Huma Qamar, MD, MPH, CMIStudy DirectorOcugen

3 Previous Clinical Trials

505 Total Patients Enrolled

Media Library

OCU400 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05203939 — Phase 1 & 2

Retinitis Pigmentosa Research Study Groups: Natural History Study (OCU400-104), Adult Arm, Cohort 1 (Low Dose), Cohort 2 (Mid Dose), Cohort 3 (High Dose), Pediatric Arm

Retinitis Pigmentosa Clinical Trial 2023: OCU400 Highlights & Side Effects. Trial Name: NCT05203939 — Phase 1 & 2

OCU400 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05203939 — Phase 1 & 2

Retinitis Pigmentosa Patient Testimony for trial: Trial Name: NCT05203939 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many people are being treated as part of this experiment?

"In order for this clinical trial to move forward, 18 individuals who meet the pre-specified inclusion criteria must enroll. These patients can be based out of Ocugen Site 6 - Emory University in Atlanta, Georgia or Ocugen Site 8 - Mid Atlantic Retina - Wills Eye Hospital in Philadelphia, Pennsylvania."

Answered by AI

Is this trial taking place in numerous stateside facilities?

"There are 8 clinical trial sites presently operational. They are situated in Atlanta, Chicago, Philadelphia and other cities. If you want to enroll in this study, it may be helpful to choose a location that is nearest to you so as to reduce travelling demands."

Answered by AI

Can patients currently sign up for this clinical trial?

"The clinicaltrials.gov website contains information suggesting that this trial is still open to patients. This study was first posted on January 24th, 2022 and updated October 19th, 2022. There are 8 locations where a total of 18 people will be accepted into the trial."

Answered by AI

What goals does this clinical trial hope to accomplish?

"The objective of this one year long clinical trial is to document Serious adverse events (SAEs). Other metrics that will be used to assess the efficacy of the intervention include changes in anti-hNR2E3 antibodies (gene), Intraocular pressure (IOP), and Indirect ophthalmoscopy."

Answered by AI

Who else is applying?

What state do they live in?

Puerto Rico

Florida

Texas

Other

How old are they?

18 - 65

What site did they apply to?

Ocugen Site 3 - Bascom Palmer Eye Institute

Ocugen Site 1 - Retina Foundation of the Southwest

Ocugen Site 5 - University of California, San Diego (UCSD) - Shiley Eye Institute

Other

What portion of applicants met pre-screening criteria?

Did not meet criteria

Met criteria

How many prior treatments have patients received?

Why did patients apply to this trial?

Im trying this first time. In hopes of improved vision for myself or others.

PatientReceived 2+ prior treatments

Living with RP is not easy..i hope You calle me for this trial. I have usher syndrome. I am hoping this clinical trial will help me with my vision loss.

PatientReceived no prior treatments