Expanded Access Program for RP in Adults

NCT ID: NCT06574997

Last Updated: 2025-07-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

AVAILABLE

Study Classification

EXPANDED_ACCESS

Brief Summary

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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.

Detailed Description

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This expanded access protocol provides access to OCU400 for a subset of patients. Subjects will undergo specific ocular testing included in the study. Subjects will follow-up with physical examination, review of concomitant medication, blood work and ocular examinations for up to 52 weeks (about 12 months) post dosing.

Conditions

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Retinitis Pigmentosa

Interventions

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OCU400

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

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

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

Exclusion Criteria

1. Subject lacks evidence of outer nuclear layer, i.e., containing the nuclei of the retinal photoreceptors as determined by SD- OCT.
2. 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.
3. Previous treatment with a gene-therapy or cell therapy product, excluding OCU400.
4. Previous treatment with any investigational drug or device within one year, excluding OCU400.
5. Any contraindications for subretinal injection.
6. Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months
Minimum Eligible Age

18 Years

Maximum Eligible Age

99 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ocugen

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Huma Qamar, MD, MPH, CMI

Role: STUDY_CHAIR

Ocugen

Locations

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Associated Retina Consultants

Phoenix, Arizona, United States

Site Status AVAILABLE

Advanced Research

Deerfield Beach, Florida, United States

Site Status AVAILABLE

Erie Retina Research

Erie, Pennsylvania, United States

Site Status AVAILABLE

Gundersen Health

La Crosse, Wisconsin, United States

Site Status AVAILABLE

Countries

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United States

Central Contacts

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Umair Qazi, MD, MPH

Role: CONTACT

484-237-3390

Mahvish Tafseer, MD, ACRP-CP

Role: CONTACT

Facility Contacts

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Mallory Mintert, MS

Role: primary

480-999-5458

Jillian Bollinger, BS

Role: backup

4809995458

Danahe Navarrete

Role: primary

954-302-3047 ext. 103

Samarth Shah, MD, Ph.D

Role: backup

Bethany Bielak, M.D

Role: primary

814-737-0203

Trinity Lee

Role: primary

608-775-9539

Chase Penzkover

Role: backup

Other Identifiers

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OCU400-EAP

Identifier Type: -

Identifier Source: org_study_id

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