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

Uveitis, Intermediate Clinical Trial

— OPTYK-1  

Official title:

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)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05953688
• Other study ID #: ESK-001-014
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: June 14, 2023
• Est. completion date: April 30, 2025

Study information

• Verified date: April 2024
• Source: Alumis Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 36
• Est. completion date: April 30, 2025
• Est. primary completion date: September 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 70 Years

Eligibility

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.

Related Conditions & MeSH terms

• Noninfectious Panuveitis
• Panuveitis
• Uveitis
• Uveitis, Intermediate

Intervention

Drug:
• ESK-001
Oral tablet

Locations

Country	Name	City	State
United States	Investigator Site#1075	Bellaire	Texas
United States	Investigator Site#1065	Beverly Hills	California
United States	Investigator Site #1083	Durham	North Carolina
United States	Investigator Site #1064	Erie	Pennsylvania
United States	Investigator Site #1085	Hagerstown	Maryland
United States	Investigator Site #1074	Katy	Texas
United States	Investigator Site#1068	Lakewood	Colorado
United States	Investigator Site #1072	Los Angeles	California
United States	Investigator Site #1073	Los Angeles	California
United States	Investigator Site #1084	Nashville	Tennessee
United States	Investigator Site#1066	Nashville	Tennessee
United States	Investigator Site #1086	Oak Park	Illinois
United States	Investigator Site #1076	Palisades Park	New Jersey
United States	Investigator Site #1080	Palo Alto	California
United States	Investigator Site #1078	Plano	Texas
United States	Investigator Site #1079	Sacramento	California
United States	Investigator Site #1069	Waltham	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Alumis Inc

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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
Secondary	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