NCT06574997

Brief Summary

This intermediate-size patient population expanded access program is to provide access to investigational product OCU400 for up to 75 patients with Retinitis Pigmentosa (RP) outside of the ongoing clinical trial Phase 3 program for patients who do not have access to alternative Food and Drug Administration (FDA)-approved products for treatment of the disease.

Trial Health

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Trial Health Score

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Geographic Reach
1 country

4 active sites

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 23, 2024

Completed
5 days until next milestone

First Posted

Study publicly available on registry

August 28, 2024

Completed
Last Updated

April 13, 2026

Status Verified

April 1, 2026

First QC Date

August 23, 2024

Last Update Submit

April 7, 2026

Conditions

Retinitis Pigmentosa

Keywords

Retinitis PigmentosaModifier Gene TherapyModified Gene TherapyRHORPNR2E3Subretinal Injection

Interventions

OCU400DRUG

Participants will receive a subretinal injection of OCU400 modifier gene therapy product.

Eligibility Criteria

Age18 Years - 99 Years
Sexall
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Males or females ≥18 years of age at the time of informed consent.
  • Confirmed clinical and CLIA certified genetic diagnosis of RP (except subjects with AD-NR2E3 mutation)
  • Presence of photoreceptors as determined by spectral-domain optical coherence tomography (SD-OCT).

You may not qualify if:

  • Subject lacks evidence of outer nuclear layer, i.e., containing the nuclei of the retinal photoreceptors as determined by SD- OCT.
  • Considered unsuitable for any reason that may either place the subject at increased risk during participation or interfere with the interpretation of the study outcomes by the Investigator, or the Sponsor after reviewing the subject medical history and condition.
  • Previous treatment with a gene-therapy or cell therapy product, excluding OCU400.
  • Previous treatment with any investigational drug or device within one year, excluding OCU400.
  • Any contraindications for subretinal injection.
  • Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Associated Retina Consultants

Phoenix, Arizona, 85020, United States

Location

Advanced Research

Deerfield Beach, Florida, 33064, United States

Location

Erie Retina Research

Erie, Pennsylvania, 16507, United States

Location

Gundersen Health

La Crosse, Wisconsin, 54601, United States

Location

MeSH Terms

Conditions

Retinitis Pigmentosa

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesRetinal DystrophiesRetinal DegenerationRetinal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Huma Qamar, MD, MPH, CMI

    Ocugen

    STUDY CHAIR

Study Design

Study Type
expanded access
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 23, 2024

First Posted

August 28, 2024

Last Updated

April 13, 2026

Record last verified: 2026-04

Locations

US(4)