Clinical trial NCT05392751: 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
Overall status: TERMINATED
Phase: PHASE1
PHASE2
Conditions: Retinitis Pigmentosa
Retinitis Pigmentosa Syndrome
Keywords: RP
Retinitis Pigmentosa
EA-2353
inherited retinal disease
IRD
Lead sponsor: Endogena Therapeutics, Inc
Sponsor class: INDUSTRY
First posted: 2022-05-26
Enrollment: 14
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
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.
Primary purpose: TREATMENT

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.

Eligibility 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.

Primary outcomes:
measure: Incidence and severity of adverse events
timeFrame: through study completion at 24 months
measure: Incidence and severity of dose limiting toxicities (DLTs)
timeFrame: through study completion at 24 months
measure: Establish the maximum tolerated dose (MTD) as determined by occurrence of DLTs
timeFrame: through study completion at 24 months
Secondary outcomes:
Other outcomes:
Locations:
city: Miami
country: United States
facility: Endogena Site 005
state: Florida
city: Ann Arbor
country: United States
facility: Endogena Site 003
state: Michigan
city: Portland
country: United States
facility: Endogena Site 004
state: Oregon
city: Dallas
country: United States
facility: Endogena Site 002
state: Texas
city: McAllen
country: United States
facility: Endogena Site 001
state: Texas
Study officials:
affiliation: Endogena Therapeutics, Inc
name: Moreno Menghini, MD
role: STUDY_DIRECTOR
References: