Clinical trial NCT05953688: POC Study to Evaluate the Efficacy and Safety of ESK-001 in Patients With Active Intermediate, Posterior, or Pan NIU
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)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Uveitis Posterior Non-Infectious
Uveitis, Intermediate
Noninfectious Panuveitis
Lead sponsor: Alumis Inc
Sponsor class: INDUSTRY
First posted: 2023-07-20
Enrollment: 36
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: 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
timeFrame: 24 Weeks
Secondary outcomes:
measure: 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
timeFrame: 24 Weeks
measure: 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).
timeFrame: Through study completion, up to 52 weeks
measure: To characterize the pharmacokinetics (PK) of ESK-001 (Cmax)
timeFrame: Through study completion, up to 48 weeks
measure: To characterize the pharmacokinetics (PK) of ESK-001 (Ctrough)
timeFrame: Through study completion, up to 48 weeks
Other outcomes:
Locations:
city: Beverly Hills
country: United States
facility: Investigator Site#1065
state: California
city: Los Angeles
country: United States
facility: Investigator Site #1073
state: California
city: Los Angeles
country: United States
facility: Investigator Site #1072
state: California
city: Palo Alto
country: United States
facility: Investigator Site #1080
state: California
city: Sacramento
country: United States
facility: Investigator Site #1079
state: California
city: Lakewood
country: United States
facility: Investigator Site#1068
state: Colorado
city: Oak Park
country: United States
facility: Investigator Site #1086
state: Illinois
city: Hagerstown
country: United States
facility: Investigator Site #1085
state: Maryland
city: Waltham
country: United States
facility: Investigator Site #1069
state: Massachusetts
city: Palisades Park
country: United States
facility: Investigator Site #1076
state: New Jersey
city: Durham
country: United States
facility: Investigator Site #1083
state: North Carolina
city: Erie
country: United States
facility: Investigator Site #1064
state: Pennsylvania
city: Nashville
country: United States
facility: Investigator Site#1066
state: Tennessee
city: Nashville
country: United States
facility: Investigator Site #1084
state: Tennessee
city: Bellaire
country: United States
facility: Investigator Site#1075
state: Texas
city: Katy
country: United States
facility: Investigator Site #1074
state: Texas
city: Plano
country: United States
facility: Investigator Site #1078
state: Texas
Study officials:
References: