Expanded Access Program for RP in Adults

Ocugen

Status

Conditions: Retinitis Pigmentosa

Treatments: Drug: OCU400

Study type: Expanded Access

Funder types: Industry

Identifiers

NCT06574997

OCU400-EAP

Details and patient eligibility

About

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.

Full description

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.

Sex: All

Ages: 18 to 99 years old

Volunteers: No Healthy Volunteers

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