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

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

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.

Study Overview

• Status: Terminated
• Conditions: Retinitis Pigmentosa; Retinitis Pigmentosa Syndrome
• Intervention / Treatment: Drug: EA-2353

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 25 months.

• Study Type: Interventional
• Enrollment (Actual): 14
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • Florida
    • Miami, Florida, United States, 33136
      • Endogena Site 005
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • Endogena Site 003
  • Oregon
    • Portland, Oregon, United States, 97232
      • Endogena Site 004
  • Texas
    • Dallas, Texas, United States, 75231
      • Endogena Site 002
    • McAllen, Texas, United States, 78503
      • Endogena Site 001

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence and severity of adverse events	through study completion at 24 months
Incidence and severity of dose limiting toxicities (DLTs)	through study completion at 24 months
Establish the maximum tolerated dose (MTD) as determined by occurrence of DLTs	through study completion at 24 months

Collaborators and Investigators

• Sponsor: Endogena Therapeutics, Inc
• Investigators: Study Director: Moreno Menghini, MD, Endogena Therapeutics, Inc

Study record dates

Study Major Dates

• Study Start (Actual): June 2, 2022
• Primary Completion (Actual): April 24, 2024
• Study Completion (Actual): April 24, 2024

Study Registration Dates

• First Submitted: May 23, 2022
• First Submitted That Met QC Criteria: May 23, 2022
• First Posted (Actual): May 26, 2022

Study Record Updates

• Last Update Posted (Actual): October 15, 2024
• Last Update Submitted That Met QC Criteria: October 10, 2024
• Last Verified: October 1, 2024

More Information

Terms related to this study

• Keywords: RP; Retinitis Pigmentosa; IRD; EA-2353; inherited retinal disease
• Additional Relevant MeSH Terms: Eye Diseases; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Eye Diseases, Hereditary; Retinal Dystrophies; Retinitis; Retinitis Pigmentosa
• Other Study ID Numbers: END-AU-CS101/201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No