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

NCT05392751 | Terminated Phase 1 FDA Drug

• 1 other identifier: END-AU-CS101/201
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 5

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.

Conditions

Retinitis Pigmentosa; Retinitis Pigmentosa Syndrome

Keywords

RP; Retinitis Pigmentosa; EA-2353; inherited retinal disease; IRD

Outcome Measures

Primary Outcomes (3)

• 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

Study Arms (1)

EA-2353

EXPERIMENTAL

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

Drug: EA-2353

Interventions

EA-2353 DRUG

EA-2353 Ophthalmic Suspension

EA-2353

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Endogena Therapeutics, Inc: lead

Study Sites (5)

Endogena Site 005

Miami, Florida, 33136, United States

Location

Endogena Site 003

Ann Arbor, Michigan, 48109, United States

Location

Endogena Site 004

Portland, Oregon, 97232, United States

Location

Endogena Site 002

Dallas, Texas, 75231, United States

Location

Endogena Site 001

McAllen, Texas, 78503, United States

Location

MeSH Terms

Conditions

Retinitis Pigmentosa; RHYNS syndrome

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Moreno Menghini, MD

  Endogena Therapeutics, Inc

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

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

Study Record Dates

• First Submitted: May 23, 2022
• First Posted: May 26, 2022
• Study Start: June 2, 2022
• Primary Completion: April 24, 2024
• Study Completion: April 24, 2024
• Last Updated: October 15, 2024

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)