Try the modernized ClinicalTrials.gov beta website. Learn more about the modernization effort.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05392751
Recruitment Status : Recruiting
First Posted : May 26, 2022
Last Update Posted : June 28, 2022
Sponsor:
Information provided by (Responsible Party):
Endogena Therapeutics, Inc

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.

Layout table for study information
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 : December 31, 2023
Estimated Study Completion Date : December 31, 2023

Resource links provided by the National Library of Medicine


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




Primary Outcome Measures :
  1. Incidence and severity of adverse events [ Time Frame: through study completion at 6 months ]
  2. Incidence and severity of dose limiting toxicities (DLTs) [ Time Frame: through study completion at 6 months ]
  3. Establish the maximum tolerated dose (MTD) as determined by occurrence of DLTs [ Time Frame: through study completion at 6 months ]


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.


Layout table for eligibility information
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.
  8. Have a central retinal thickness (center point) >100 microns on SD-OCT.

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.

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


Contacts
Layout table for location contacts
Contact: InFocus Clinical Research 615-312-8295 clinical.studies@endogena.com
Contact: Michelle Widmann clinical.studies@endogena.com

Locations
Layout table for location information
United States, Florida
Endogena Site 005 Not yet recruiting
Miami, Florida, United States, 33136
Contact: Endogena       clinical.studies@endogena.com   
United States, Michigan
Endogena Site 003 Not yet recruiting
Ann Arbor, Michigan, United States, 48109
Contact: Endogena       clinical.studies@endogena.com   
United States, North Carolina
Endogena Site 006 Not yet recruiting
Durham, North Carolina, United States, 27710
Contact: Endogena       clinical.studies@endogena.com   
United States, Oregon
Endogena Site 004 Not yet recruiting
Portland, Oregon, United States, 97232
Contact: Endogena       clinical.studies@endogena.com   
United States, Texas
Endogena Site 002 Recruiting
Dallas, Texas, United States, 75231
Contact: Endogena       clinical.studies@endogena.com   
Endogena Site 001 Recruiting
McAllen, Texas, United States, 78503
Contact: Endogena       clinical.studies@endogena.com   
Sponsors and Collaborators
Endogena Therapeutics, Inc
Investigators
Layout table for investigator information
Study Director: Moreno Menghini, MD Endogena Therapeutics, Inc
Layout table for additonal information
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: June 28, 2022
Last Verified: June 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
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:
Layout table for MeSH terms
Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn