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

Primary Purpose

Retinitis Pigmentosa, Retinitis Pigmentosa Syndrome

Status

Active

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

EA-2353

About this trial

This is an interventional treatment trial for Retinitis Pigmentosa focused on measuring RP, Retinitis Pigmentosa, EA-2353, inherited retinal disease, IRD

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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.

Exclusion Criteria:

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.

Sites / Locations

Endogena Site 005
Endogena Site 003
Endogena Site 004
Endogena Site 002
Endogena Site 001

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

EA-2353

Arm Description

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

Outcomes

Primary Outcome Measures

Incidence and severity of adverse events

Incidence and severity of dose limiting toxicities (DLTs)

Establish the maximum tolerated dose (MTD) as determined by occurrence of DLTs

Secondary Outcome Measures

Full Information

NCT ID

NCT05392751

First Posted

May 23, 2022

Last Updated

April 26, 2023

Sponsor

Endogena Therapeutics, Inc

1. Study Identification

Unique Protocol Identification Number

NCT05392751

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

June 2, 2022 (Actual)

Primary Completion Date

June 30, 2025 (Anticipated)

Study Completion Date

June 30, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Endogena Therapeutics, Inc

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Retinitis Pigmentosa, Retinitis Pigmentosa Syndrome

Keywords

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

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)

Allocation

N/A

Enrollment

14 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

EA-2353

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

EA-2353

Intervention Description

EA-2353 Ophthalmic Suspension

Primary Outcome Measure Information:

Title

Incidence and severity of adverse events

Time Frame

through study completion at 24 months

Title

Incidence and severity of dose limiting toxicities (DLTs)

Time Frame

through study completion at 24 months

Title

Establish the maximum tolerated dose (MTD) as determined by occurrence of DLTs

Time Frame

through study completion at 24 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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. Exclusion Criteria: 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Moreno Menghini, MD

Organizational Affiliation

Endogena Therapeutics, Inc

Official's Role

Study Director

Facility Information:

Facility Name

Endogena Site 005

City

Miami

State/Province

Florida

ZIP/Postal Code

33136

Country

United States

Facility Name

Endogena Site 003

City

Ann Arbor

State/Province

Michigan

ZIP/Postal Code

48109

Country

United States

Facility Name

Endogena Site 004

City

Portland

State/Province

Oregon

ZIP/Postal Code

97232

Country

United States

Facility Name

Endogena Site 002

City

Dallas

State/Province

Texas

ZIP/Postal Code

75231

Country

United States

Facility Name

Endogena Site 001

City

McAllen

State/Province

Texas

ZIP/Postal Code

78503

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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