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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05392751
Recruitment Status : Active, not recruiting
First Posted : May 26, 2022
Last Update Posted : April 27, 2023
Sponsor:
Information provided by (Responsible Party):
Endogena Therapeutics, Inc

Study Description
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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 25 months.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated 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.
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 : June 2, 2022
Estimated Primary Completion Date : June 30, 2025
Estimated Study Completion Date : June 30, 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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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
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Primary Outcome Measures :
  1. Incidence and severity of adverse events [ Time Frame: through study completion at 24 months ]
  2. Incidence and severity of dose limiting toxicities (DLTs) [ Time Frame: through study completion at 24 months ]
  3. Establish the maximum tolerated dose (MTD) as determined by occurrence of DLTs [ Time Frame: through study completion at 24 months ]

Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05392751


Locations
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United States, Florida
Endogena Site 005
Miami, Florida, United States, 33136
United States, Michigan
Endogena Site 003
Ann Arbor, Michigan, United States, 48109
United States, Oregon
Endogena Site 004
Portland, Oregon, United States, 97232
United States, Texas
Endogena Site 002
Dallas, Texas, United States, 75231
Endogena Site 001
McAllen, Texas, United States, 78503
Sponsors and Collaborators
Endogena Therapeutics, Inc
Investigators
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Study Director: Moreno Menghini, MD Endogena Therapeutics, Inc
More Information
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Responsible Party: Endogena Therapeutics, Inc
ClinicalTrials.gov Identifier: NCT05392751    
Other Study ID Numbers: END-AU-CS101/201
First Posted: May 26, 2022    Key Record Dates
Last Update Posted: April 27, 2023
Last Verified: April 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Endogena Therapeutics, Inc:
RP
Retinitis Pigmentosa
EA-2353
inherited retinal disease
IRD
Additional relevant MeSH terms:
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Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases
 Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn