Dose Escalation Study to Evaluate the Safety/Tolerability and Efficacy of EA-2353 in Subjects With Retinitis Pigmentosa

• Status: Not yet recruiting
• Trial end date: 2023-12-31
• Target enrollment: 0
• Participant gender: All

Summary

This is an open-label, multiple ascending dose study to evaluate the safety, tolerability, and efficacy of EA-2353 in subjects with RP. Unilateral intravitreal injections (IVT) will be administered into the subject's Study Eye. There will be up to 4 cohorts.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Endogena Therapeutics, Inc

Criteria

Inclusion Criteria:

1. Male or female, ≥ 18 years of age.

2. Have a clinical and molecular diagnosis of RP.

3. Understand the language of the informed consent and are willing and able to provide written informed consent prior to any study procedures.

4. Are willing to comply with the protocol and follow the instructions including attendance at all scheduled study visits.

5. BCVA in the worse eye better than or equal to count fingers (CF)

6. Have clear ocular media.

7. Have pupillary dilation sufficient to allow for quality images.

8. Have a central retinal thickness (center point) >100 microns on SD-OCT.

Exclusion Criteria:

1. Subjects who are pregnant or suspected to be pregnant and subjects who are lactating or intend to breastfeed. Female subjects of childbearing potential are required to have a negative urine pregnancy test at screening and prior to each injection, and must agree to use an acceptable method of contraception from the time of signing informed consent until the end of the study.

2. Have any uncontrolled systemic disease or non-ocular disorder which would put the subject at risk due to study treatment or procedures, influence the results of the study, or impact the subject's ability to participate in the study (e.g., infection, uncontrolled elevated blood pressure, cardiovascular disease, and inability to maintain glycemic control).

3. Presence of a significant ocular disease or disorder in the Study Eye.

4. Have a history of any vitreoretinal surgery ever in the Study Eye.

5. Have received any prior cell or gene therapy for RP.

6. Have history or current abuse of alcohol and/or drugs.