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)
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- Alumis Inc
- 入组人数
- 36
- 试验地点
- 17
- 主要终点
- 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
研究概览
简要总结
This is a multi-center, randomized, double-masked, proof-of-concept study in patients with Active Noninfectious Intermediate, Posterior, or Panuveitis.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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.
研究组 & 干预措施
ESK-001 Dose Level 1
ESK-001 administered as an oral tablet
干预措施: ESK-001 (Drug)
ESK-001 Dose Level 2
ESK-001 administered as an oral tablet
干预措施: ESK-001 (Drug)
结局指标
主要结局
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
时间窗: 24 Weeks
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.
次要结局
- 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(24 Weeks)
- To characterize the pharmacokinetics (PK) of ESK-001 (Cmax)(Through study completion, up to 48 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).(Through study completion, up to 52 weeks)
- To characterize the pharmacokinetics (PK) of ESK-001 (Ctrough)(Through study completion, up to 48 weeks)