POC Study to Evaluate the Efficacy and Safety of ESK-001 in Patients With Active Intermediate, Posterior, or Pan NIU (OPTYK-1)

August 27, 2024 updated by: Alumis Inc

A Proof-of-Concept, Randomized, Double-Masked Study to Evaluate the Efficacy and Safety of ESK-001 in Patients With Active Noninfectious Intermediate, Posterior, or Panuveitis (OPTYK-1)

This is a multi-center, randomized, double-masked, proof-of-concept study in patients with Active Noninfectious Intermediate, Posterior, or Panuveitis.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

This study will consist of a 2-week screening period (+7 days), 24-week treatment period, followed by a 24-week extension period, and a 4-week follow up period for a total of up to 55 weeks. Each participant will be randomized to receive one of two doses of ESK-001 daily for 24 weeks. Participants that do not meet the treatment failure criteria at Week 24 will continue into the extension period.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
36

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • United States
    • California
      • Beverly Hills, California, United States, 90211
        • Investigator Site#1065
      • Los Angeles, California, United States, 90033
        • Investigator Site #1073
      • Los Angeles, California, United States, 90095
        • Investigator Site #1072
      • Palo Alto, California, United States, 94303
        • Investigator Site #1080
      • Sacramento, California, United States, 95825
        • Investigator Site #1079
    • Colorado
      • Lakewood, Colorado, United States, 80228
        • Investigator Site#1068
    • Illinois
      • Oak Park, Illinois, United States, 60304
        • Investigator Site #1086
    • Maryland
      • Hagerstown, Maryland, United States, 21740
        • Investigator Site #1085
    • Massachusetts
      • Waltham, Massachusetts, United States, 02451
        • Investigator Site #1069
    • New Jersey
      • Palisades Park, New Jersey, United States, 07650
        • Investigator Site #1076
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Investigator Site #1083
    • Pennsylvania
      • Erie, Pennsylvania, United States, 16507
        • Investigator Site #1064
    • Tennessee
      • Nashville, Tennessee, United States, 37203
        • Investigator Site#1066
      • Nashville, Tennessee, United States, 37232
        • Investigator Site #1084
    • Texas
      • Bellaire, Texas, United States, 77401
        • Investigator Site#1075
      • Katy, Texas, United States, 77494
        • Investigator Site #1074
      • Plano, Texas, United States, 75075
        • Investigator Site #1078

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  • Able and willing to provide consent
  • Male and females, age 18 to 70 years
  • Diagnosis of active noninfectious intermediate, posterior or panuveitis

  • Must have active uveitis at Screening in at least one eye as defined by:

    1. Active inflammatory chorioretinal and/or inflammatory retinal vascular lesion or lesions, or
    2. ≥ 2+ VH in accordance with the NEI/SUN criteria
  • Males and females must use highly effective methods of contraception for the entirety of the study

Key Exclusion Criteria:

  • Diagnosis of infectious uveitis
  • Has elevated intraocular pressures or severe glaucoma
  • Positive for HIV, Hepatitis B or C or active or inadequately latent tuberculosis at screening
  • Positive for syphilis at screening
  • Patients with QTcF >450 msec (both males and females) at screening
  • Known active malignancy or history of malignancy within the past 5 years
  • History of chronic drug or alcohol abuse
  • Live vaccines
  • No planned ocular or any other surgery during the course of the study

Other protocol-defined inclusion/exclusion criteria apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: ESK-001 Dose Level 1
ESK-001 administered as an oral tablet
Drug: ESK-001
Oral tablet
Experimental: ESK-001 Dose Level 2
ESK-001 administered as an oral tablet
Drug: ESK-001
Oral tablet

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
To compare the efficacy between 2 ESK-001 treatment groups for the treatment of active NIU as measured by the proportion of patients failing treatment by Week 24
Time Frame: 24 Weeks
The proportion of patients failing treatment for active NIU by Week 24 between 2 ESK-001 treatment groups. Treatment failure is defined as a patient meeting at least 1 of the treatment failure criteria in at least 1 eye at Week 24.
24 Weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
To compare the efficacy between 2 ESK-001 treatment groups for the treatment of active NIU as measured by the time to treatment failure on or after Week 24
Time Frame: 24 Weeks
Time to treatment failure on or after Week 24 between 2 ESK-001 treatment groups. Time to treatment failure is defined as time to the visit of the first treatment failure as defined per protocol. Median time to treatment failure in each treatment group will be reported and compared.
24 Weeks
To assess the safety and tolerability of ESK-001 in patients with active NIU by comparing the incidence and proportion of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs).
Time Frame: Through study completion, up to 52 weeks
Clinical and laboratory assessments including blood tests, ECGs, hematological measures and physical examinations throughout the study. The number and proportion of TEAEs, SAEs, AEs leading to study discontinuation, and AEs of greater than grade 3 severity will recorded per treatment group and across all groups.
Through study completion, up to 52 weeks
To characterize the pharmacokinetics (PK) of ESK-001 (Cmax)
Time Frame: Through study completion, up to 48 weeks
Plasma concentrations and PK parameters including steady-state maximum observed concentration (Cmax) for ESK-001 at Day 1, Week 24, and Week 48.
Through study completion, up to 48 weeks
To characterize the pharmacokinetics (PK) of ESK-001 (Ctrough)
Time Frame: Through study completion, up to 48 weeks
Plasma concentrations and PK parameters including steady-state trough observed plasma concentration (Ctrough) for ESK-001 at Week 24, and Week 48.
Through study completion, up to 48 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Alumis Inc

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 14, 2023

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 13, 2024

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 13, 2024

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 29, 2023

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 18, 2023

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 20, 2023

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 28, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 27, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • ESK-001-014

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings