Retinitis PigmentosaPhase 1/2NCT05392751
Dose Escalation Study to Evaluate the Safety/Tolerability and Efficacy of EA-2353 in Subjects With Retinitis Pigmentosa
A Phase 1/2 study testing EA-2353 in people with Retinitis Pigmentosa and Retinitis Pigmentosa Syndrome, run by Endogena Therapeutics, Inc at 5 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Retinitis Pigmentosa, Retinitis Pigmentosa Syndrome
- What is being tested?
- EA-2353
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 5 sitesUnited States
- Study dates
- 2 Jun 2022to 24 Apr 2024
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Endogena Site 003
Ann Arbor, Michigan, 48109
Endogena Site 002
Dallas, Texas, 75231
Endogena Site 001
McAllen, Texas, 78503
Endogena Site 005
Miami, Florida, 33136
Endogena Site 004
Portland, Oregon, 97232
About this study
Official title: Open-label, Multiple Dose, Dose Escalation Study to Evaluate the Safety/Tolerability and Efficacy of EA-2353 in Subjects With Retinitis Pigmentosa
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.
Read the 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.
- Being tested
- EA-2353
- Keywords
- RP, Retinitis Pigmentosa, EA-2353, inherited retinal disease, IRD
- Medical subject headings
- Retinitis Pigmentosa, RHYNS syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EA-2353Drug
EA-2353 Ophthalmic Suspension
| Study group | Role | What they receive |
|---|---|---|
| EA-2353 | Experimental | EA-2353 Ophthalmic Suspension will be administered via unilateral intravitreal injection into the Study Eye weekly for one month (4 injections) Receives: Drug: EA-2353 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of adverse events
Measured: through study completion at 24 months
Incidence and severity of dose limiting toxicities (DLTs)
Measured: through study completion at 24 months
Establish the maximum tolerated dose (MTD) as determined by occurrence of DLTs
Measured: through study completion at 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 May 2022
- Study start2 Jun 2022
- Primary completion24 Apr 2024
- Study completion24 Apr 2024
Why it stopped: Study terminated by the Sponsor. All study participants completed at least 12 months of safety follow-up instead of 24 mo. The reason for earlier termination was due to a corporate decision; not any safety concerns of EA-2353.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis PigmentosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- 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)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Endogena Therapeutics, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Moreno Menghini, MD, Study director, Endogena Therapeutics, Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05392751
- Sponsor's protocol ID
- END-AU-CS101/201
- Status last verified
- Oct 2024
- First submitted
- 23 May 2022
- Last updated
- 15 Oct 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-label, Multiple Dose, Dose Escalation Study to Evaluate the Safety/Tolerability and Efficacy of EA-2353 in Subjects With Retinitis Pigmentosa
- ClinicalTrials.gov ID
- NCT05392751
- Acronym / sponsor ID
- END-AU-CS101/201
- Keywords
- RP, Retinitis Pigmentosa, EA-2353, inherited retinal disease, IRD
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study terminated by the Sponsor. All study participants completed at least 12 months of safety follow-up instead of 24 mo. The reason for earlier termination was due to a corporate decision; not any safety concerns of EA-2353.
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Endogena Therapeutics, IncIndustry (pharma, biotech, devices)
- Study start
- 2 Jun 2022 (actual)
- Primary completion
- 24 Apr 2024 (actual)
- Study completion
- 24 Apr 2024 (actual)
- First posted
- 26 May 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- 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.
| Arm | Type | Receives |
|---|---|---|
| EA-2353 | Experimental | Drug: EA-2353EA-2353 Ophthalmic Suspension will be administered via unilateral intravitreal injection into the Study Eye weekly for one month (4 injections) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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)
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EA-2353EA-2353 Ophthalmic Suspension | Drug | EA-2353 |
Primary outcome
Incidence and severity of adverse events
Time frame: through study completion at 24 months
Incidence and severity of dose limiting toxicities (DLTs)
Time frame: through study completion at 24 months
Establish the maximum tolerated dose (MTD) as determined by occurrence of DLTs
Time frame: through study completion at 24 months
Results and safety
The study was stopped early (reason given: Study terminated by the Sponsor. All study participants completed at least 12 months of safety follow-up instead of 24 mo. The reason for earlier termination was due to a corporate decision; not any safety concerns of EA-2353.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Moreno Menghini, MD study director
Endogena Therapeutics, Inc
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing EA-2353 in people with Retinitis Pigmentosa and Retinitis Pigmentosa Syndrome, run by Endogena Therapeutics, Inc at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 15 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Study terminated by the Sponsor. All study participants completed at least 12 months of safety follow-up instead of 24 mo. The reason for earlier termination was due to a corporate decision; not any safety concerns of EA-2353.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of adverse events (time frame: through study completion at 24 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Oct 2024.