Back to resultsPOC Study to Evaluate the Efficacy and Safety of ESK-001 in Patients With Active Intermediate, Posterior, or Pan NIU (OPTYK-1)
Primary Purpose
Uveitis Posterior Non-Infectious, Uveitis, Intermediate, Noninfectious Panuveitis
Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
ESK-001
Sponsored by
About this trial
This is an interventional treatment trial for Uveitis Posterior Non-Infectious
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: Active inflammatory chorioretinal and/or inflammatory retinal vascular lesion or lesions, or ≥ 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.
Sites / Locations
- Investigator Site#1065Recruiting
- Investigator Site#1068Recruiting
- Investigator Site #1069Recruiting
- Investigator Site #1076Recruiting
- Investigator Site #1064Recruiting
- Investigator Site#1066Recruiting
- Investigator Site#1075Recruiting
- Investigator Site #1074Recruiting
- Investigator Site # 1078
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
ESK-001 Dose Level 1
ESK-001 Dose Level 2
Arm Description
ESK-001 administered as an oral tablet
ESK-001 administered as an oral tablet
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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.
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.
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.
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05953688
Brief Title
POC Study to Evaluate the Efficacy and Safety of ESK-001 in Patients With Active Intermediate, Posterior, or Pan NIU
Acronym
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)
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 14, 2023 (Actual)
Primary Completion Date
September 30, 2024 (Anticipated)
Study Completion Date
April 30, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alumis Inc
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Uveitis Posterior Non-Infectious, Uveitis, Intermediate, Noninfectious Panuveitis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
30 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
ESK-001 Dose Level 1
Arm Type
Experimental
Arm Description
ESK-001 administered as an oral tablet
Arm Title
ESK-001 Dose Level 2
Arm Type
Experimental
Arm Description
ESK-001 administered as an oral tablet
Intervention Type
Drug
Intervention Name(s)
ESK-001
Intervention Description
Oral tablet
Primary Outcome Measure Information:
Title
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
Description
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.
Time Frame
24 Weeks
Secondary Outcome Measure Information:
Title
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
Description
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.
Time Frame
24 Weeks
Title
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).
Description
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.
Time Frame
Through study completion, up to 52 weeks
Title
To characterize the pharmacokinetics (PK) of ESK-001 (Cmax)
Description
Plasma concentrations and PK parameters including steady-state maximum observed concentration (Cmax) for ESK-001 at Day 1, Week 24, and Week 48.
Time Frame
Through study completion, up to 48 weeks
Title
To characterize the pharmacokinetics (PK) of ESK-001 (Ctrough)
Description
Plasma concentrations and PK parameters including steady-state trough observed plasma concentration (Ctrough) for ESK-001 at Week 24, and Week 48.
Time Frame
Through study completion, up to 48 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
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:
Active inflammatory chorioretinal and/or inflammatory retinal vascular lesion or lesions, or
≥ 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Alumis
Phone
650-538-2502
Email
clinicaltrials@alumis.com
Facility Information:
Facility Name
Investigator Site#1065
City
Beverly Hills
State/Province
California
ZIP/Postal Code
90211
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Invite Only
Facility Name
Investigator Site#1068
City
Lakewood
State/Province
Colorado
ZIP/Postal Code
80228
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Invite Only
Facility Name
Investigator Site #1069
City
Waltham
State/Province
Massachusetts
ZIP/Postal Code
02451
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Invite Only
Facility Name
Investigator Site #1076
City
Palisades Park
State/Province
New Jersey
ZIP/Postal Code
07650
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Invite Only
Facility Name
Investigator Site #1064
City
Erie
State/Province
Pennsylvania
ZIP/Postal Code
16507
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Invite Only
Facility Name
Investigator Site#1066
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37203
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Invite Only
Facility Name
Investigator Site#1075
City
Bellaire
State/Province
Texas
ZIP/Postal Code
77401
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Invite Only
Facility Name
Investigator Site #1074
City
Katy
State/Province
Texas
ZIP/Postal Code
77494
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Invite Only
Facility Name
Investigator Site # 1078
City
Plano
State/Province
Texas
ZIP/Postal Code
75075
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Invite Only
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
POC Study to Evaluate the Efficacy and Safety of ESK-001 in Patients With Active Intermediate, Posterior, or Pan NIU
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