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

Alumis

Status and phase: Terminated; Phase 2

Conditions: Noninfectious Panuveitis; Uveitis Posterior Non-Infectious; Uveitis, Intermediate

Treatments: Drug: ESK-001

Study type: Interventional

Funder types: Industry

Identifiers

NCT05953688

ESK-001-014

Details and patient eligibility

About

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

Full 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.

Enrollment: 36 patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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36 participants in 2 patient groups

ESK-001 Dose Level 1

Experimental group

Description:

ESK-001 administered as an oral tablet

Treatment:

Drug: ESK-001

ESK-001 Dose Level 2

Experimental group

Description:

ESK-001 administered as an oral tablet

Treatment:

Drug: ESK-001