Gene Therapy

OCU400 High Dose for Retinitis Pigmentosa

Ocugen Site 8 - Mid Atlantic Retina - Wills Eye Hospital, Philadelphia, PA
Targeting 2 different conditionsOCU400 High Dose +2 morePhase 1 & 2RecruitingResearch Sponsored by Ocugen

Study 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.

Eligible Conditions
  • Retinitis Pigmentosa
  • Leber Congenital Amaurosis


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

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

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)

Find a site

Who is running the clinical trial?

OcugenLead Sponsor
8 Previous Clinical Trials
880 Total Patients Enrolled
Huma Qamar, MD, MPH, CMIStudy Director
2 Previous Clinical Trials
442 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 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
How old are they?
18 - 65
What site did they apply to?
Ocugen Site 6 - Emory University
Ocugen Site 5 - University of California, San Diego (UCSD) - Shiley Eye Institute
Ocugen Site 1 - Retina Foundation of the Southwest
What portion of applicants met pre-screening 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.
I have usher syndrome. I am hoping this clinical trial will help me with my vision loss.
What motivates me is that I can me a part of this trail, that can change my life my mother and 1,000 of people.

What questions have other patients asked about this trial?

How long is the trail? What dates are involved? Is there a trial recruiting now or in the near future that I can apply for?
How long does the screening take? How often would I have to visit for subsequent testing or procedures over the course of the trial?

How responsive is this trial?

Average response time
  • < 1 Day
Typically responds via
Phone Call
Most responsive sites:
  1. Ocugen Site 6 - Emory University: < 24 hours
  2. Ocugen Site 1 - Retina Foundation of the Southwest: < 24 hours
  3. Ocugen Site 5 - University of California, San Diego (UCSD) - Shiley Eye Institute: < 48 hours
~21 spots leftby Feb 2024