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

Retinitis Pigmentosa Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05392751
• Other study ID #: END-AU-CS101/201
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: June 2, 2022
• Est. completion date: June 30, 2025

Study information

• Verified date: January 2024
• Source: Endogena Therapeutics, Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 25 months.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 14
• Est. completion date: June 30, 2025
• Est. primary completion date: June 30, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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 between 20/50 and able to count fingers (CF)

6. Have clear ocular media.

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

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.

Related Conditions & MeSH terms

• Retinitis
• Retinitis Pigmentosa
• Retinitis Pigmentosa Syndrome

Intervention

Drug:
• EA-2353
EA-2353 Ophthalmic Suspension

Locations

Country	Name	City	State
United States	Endogena Site 003	Ann Arbor	Michigan
United States	Endogena Site 002	Dallas	Texas
United States	Endogena Site 001	McAllen	Texas
United States	Endogena Site 005	Miami	Florida
United States	Endogena Site 004	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
Endogena Therapeutics, Inc

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence and severity of adverse events		through study completion at 24 months
Primary	Incidence and severity of dose limiting toxicities (DLTs)		through study completion at 24 months
Primary	Establish the maximum tolerated dose (MTD) as determined by occurrence of DLTs		through study completion at 24 months