This intermediate-size patient population expanded access program is to provide access toinvestigational 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 accessto alternative Food and Drug Administration (FDA)-approved products for treatment of thedisease.
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.
Drug: OCU400
Participants will receive a subretinal injection of OCU400 modifier gene therapy product.
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
Associated Retina Consultants
Phoenix, Arizona, United States
Advanced Research
Deerfield Beach, Florida, United States
Erie Retina Research
Erie, Pennsylvania, United States
Gundersen Health
La Crosse, Wisconsin, United States
Umair Qazi, MD, MPH
484-237-3390
umair.qazi@ocugen.com
Mahvish Tafseer, MD, ACRP-CP
mahvish.tafseer@ocugen.com
Huma Qamar, MD, MPH, CMI, Study Chair
Ocugen