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Terminated based on interim efficacy data that did not meet the Sponsor's criteria.
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This is a multi-center, randomized, double-masked, proof-of-concept study in patients with Active Noninfectious Intermediate, Posterior, or Panuveitis.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ESK-001 Dose Level 1 | Experimental | ESK-001 administered as an oral tablet |
|
| ESK-001 Dose Level 2 | Experimental | ESK-001 administered as an oral tablet |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ESK-001 | Drug | Oral tablet |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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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:
Males and females must use highly effective methods of contraception for the entirety of the study
Key Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigator Site#1065 | Beverly Hills | California | 90211 | United States | ||
| Investigator Site #1073 |
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| 24 Weeks |
| 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 |
| 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 |
| 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 |
| Los Angeles |
| California |
| 90033 |
| United States |
| Investigator Site #1072 | Los Angeles | California | 90095 | United States |
| Investigator Site #1080 | Palo Alto | California | 94303 | United States |
| Investigator Site #1079 | Sacramento | California | 95825 | United States |
| Investigator Site#1068 | Lakewood | Colorado | 80228 | United States |
| Investigator Site #1086 | Oak Park | Illinois | 60304 | United States |
| Investigator Site #1085 | Hagerstown | Maryland | 21740 | United States |
| Investigator Site #1069 | Waltham | Massachusetts | 02451 | United States |
| Investigator Site #1076 | Palisades Park | New Jersey | 07650 | United States |
| Investigator Site #1083 | Durham | North Carolina | 27705 | United States |
| Investigator Site #1064 | Erie | Pennsylvania | 16507 | United States |
| Investigator Site#1066 | Nashville | Tennessee | 37203 | United States |
| Investigator Site #1084 | Nashville | Tennessee | 37232 | United States |
| Investigator Site#1075 | Bellaire | Texas | 77401 | United States |
| Investigator Site #1074 | Katy | Texas | 77494 | United States |
| Investigator Site #1078 | Plano | Texas | 75075 | United States |
| ID | Term |
|---|---|
| D015867 | Uveitis, Intermediate |
| ID | Term |
|---|---|
| D014605 | Uveitis |
| D014603 | Uveal Diseases |
| D005128 | Eye Diseases |
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