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

Phase 2

Terminated

• Conditions: Noninfectious Panuveitis; Uveitis Posterior Non-Infectious; Uveitis, Intermediate
• Interventions: Drug: ESK-001

• Registration Number: NCT05953688
• Lead Sponsor: Alumis Inc

Brief Summary

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

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 Timeline

• Study Start: 2023-06-14
• Study First Submitted: 2023-06-29
• Study First Submitted QC: 2023-07-18
• Study First Posted: 2023-07-20
• Status Verified: 2024-08
• Primary Completion: 2024-08-13
• Study Completion: 2024-08-13
• Last Update Submitted: 2024-08-27
• Last Update Posted: 2024-08-28

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 36

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
ESK-001 Dose Level 2	ESK-001	ESK-001 administered as an oral tablet
ESK-001 Dose Level 1	ESK-001	ESK-001 administered as an oral tablet

Primary Outcome Measures

Name	Time	Method
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	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.

Secondary Outcome Measures

Name	Time	Method
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	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.
To characterize the pharmacokinetics (PK) of ESK-001 (Cmax)	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.
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).	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.
To characterize the pharmacokinetics (PK) of ESK-001 (Ctrough)	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.

Trial Locations

Locations (17)

Investigator Site #1076; 🇺🇸 Palisades Park, New Jersey, United States

Investigator Site #1064; 🇺🇸 Erie, Pennsylvania, United States

Investigator Site #1073; 🇺🇸 Los Angeles, California, United States

Investigator Site #1072; 🇺🇸 Los Angeles, California, United States

Investigator Site #1083; 🇺🇸 Durham, North Carolina, United States

Investigator Site#1068; 🇺🇸 Lakewood, Colorado, United States

Investigator Site #1084; 🇺🇸 Nashville, Tennessee, United States

Investigator Site#1065; 🇺🇸 Beverly Hills, California, United States

Investigator Site #1079; 🇺🇸 Sacramento, California, United States

Investigator Site #1080; 🇺🇸 Palo Alto, California, United States

Investigator Site #1086; 🇺🇸 Oak Park, Illinois, United States

Investigator Site #1085; 🇺🇸 Hagerstown, Maryland, United States

Investigator Site#1075; 🇺🇸 Bellaire, Texas, United States

Investigator Site #1078; 🇺🇸 Plano, Texas, United States

Investigator Site #1074; 🇺🇸 Katy, Texas, United States

Investigator Site#1066; 🇺🇸 Nashville, Tennessee, United States

Investigator Site #1069; 🇺🇸 Waltham, Massachusetts, United States