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

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: Active, not recruiting
• Conditions: Uveitis, Intermediate; Noninfectious Panuveitis; Uveitis Posterior Non-Infectious
• Intervention / Treatment: Drug: ESK-001

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: Interventional
• Enrollment (Actual): 36
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Alumis; Phone Number: 650-538-2502; Email: clinicaltrials@alumis.com

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

Eligibility Criteria

• 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

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
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

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	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

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 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).	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)	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)	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

• Sponsor: Alumis Inc

Study record dates

Study Major Dates

• Study Start (Actual): June 14, 2023
• Primary Completion (Estimated): September 30, 2024
• Study Completion (Estimated): April 30, 2025

Study Registration Dates

• First Submitted: June 29, 2023
• First Submitted That Met QC Criteria: July 18, 2023
• First Posted (Actual): July 20, 2023

Study Record Updates

• Last Update Posted (Actual): April 8, 2024
• Last Update Submitted That Met QC Criteria: April 5, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Uveal Diseases; Choroid Diseases; Choroiditis; Uveitis, Posterior; Uveitis; Panuveitis; Uveitis, Intermediate; Pars Planitis
• Other Study ID 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