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

Sponsor

Endogena Therapeutics, Inc (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05392751

Collaborator

(none)

Enrollment

Locations

Arm

Anticipated Duration (Months)

2.3

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease	Intervention/Treatment	Phase
Retinitis Pigmentosa Retinitis Pigmentosa Syndrome	Drug: EA-2353	Phase 1/Phase 2

Detailed Description

This is an open-label, multiple ascending dose study to evaluate the safety, tolerability, and efficacy of EA-2353 in subjects with RP. There will be up to 4 cohorts. Each of the first three cohorts will treat 3 subjects with 4 weekly injections of EA-2353.

Unilateral intravitreal injections (IVT) will be administered into the subject's Study Eye, which is determined as the eye with worse vision based on the BCVA. Eligible subjects will be enrolled into one of the following three cohorts in an ascending sequential fashion:

Cohort 1 (low dose)
Cohort 2 (mid dose)
Cohort 3 (high dose)
Cohort 4 (maximum tolerated dose)

Patient participation will last for approximately 7 months.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

14 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Intervention Model Description:

EA-2353 will be administered to all subjects during this study. It will be administered as 4 weekly intravitreal injections in the Study Eye.EA-2353 will be administered to all subjects during this study. It will be administered as 4 weekly intravitreal injections in the Study Eye.

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Actual Study Start Date :

Jun 2, 2022

Anticipated Primary Completion Date :

Dec 31, 2023

Anticipated Study Completion Date :

Dec 31, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: EA-2353 EA-2353 Ophthalmic Suspension will be administered via unilateral intravitreal injection into the Study Eye weekly for one month (4 injections)	Drug: EA-2353 EA-2353 Ophthalmic Suspension

Arm

Intervention/Treatment

Experimental: EA-2353

EA-2353 Ophthalmic Suspension will be administered via unilateral intravitreal injection into the Study Eye weekly for one month (4 injections)

Drug: EA-2353

EA-2353 Ophthalmic Suspension

Outcome Measures

Primary Outcome Measures

Incidence and severity of adverse events [through study completion at 6 months]
Incidence and severity of dose limiting toxicities (DLTs) [through study completion at 6 months]
Establish the maximum tolerated dose (MTD) as determined by occurrence of DLTs [through study completion at 6 months]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Male or female, ≥ 18 years of age.
Have a clinical and molecular diagnosis of RP.
Understand the language of the informed consent and are willing and able to provide written informed consent prior to any study procedures.
Are willing to comply with the protocol and follow the instructions including attendance at all scheduled study visits.
BCVA in the worse eye between 20/50 and able to count fingers (CF)
Have clear ocular media.
Have pupillary dilation sufficient to allow for quality images.
Have a central retinal thickness (center point) >100 microns on SD-OCT.

Exclusion Criteria:

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.
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).
Presence of a significant ocular disease or disorder in the Study Eye.
Have a history of any vitreoretinal surgery ever in the Study Eye.
Have received any prior cell or gene therapy for RP.
Have history or current abuse of alcohol and/or drugs.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Endogena Site 005	Miami	Florida	United States	33136
2	Endogena Site 003	Ann Arbor	Michigan	United States	48109
3	Endogena Site 006	Durham	North Carolina	United States	27710
4	Endogena Site 004	Portland	Oregon	United States	97232
5	Endogena Site 002	Dallas	Texas	United States	75231
6	Endogena Site 001	McAllen	Texas	United States	78503