Safety of Tofacitinib, an Oral Janus Kinase Inhibitor, in Primary Sjogren's Syndrome; a Phase Ib-IIa Placebo-controlled Clinical Trial and Associated Mechanistic Studies
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 23
- 试验地点
- 1
- 主要终点
- Number of Adverse Events by Grade/Category
研究概览
简要总结
Background:
An autoimmune disease is one in which the immune system attacks a person's own body. Sjogren's syndrome (SS) is an autoimmune disease. It often involves multiple systems and organs of the body. Researchers are trying to find new, more effective and safe treatments for SS.
Objective:
To evaluate the safety and tolerance of tofacitinib in people with SS.
Eligibility:
Adults ages 18-75 with SS.
Design:
Participants will be screened on a separate protocol. They will undergo:
- Medical and dental history
- Physical exam
- Medicine review
- Electrocardiogram to test the heart s electrical activity (Participants will lay on a table. Sticky pads will be placed on their body.)
- Eye exam and test for dry eyes
- Oral, head, and neck exams
- Plaque collection (Dental plaques and tongue and mucosal scrapings will be collected using a small tongue depressor.)
- Salivary gland ultrasound
- Blood and urine tests
- Minor salivary gland biopsy (The lower lip will be numbed. Several tiny salivary glands will be removed through a small incision.)
- Saliva collection
- Disease assessment.
Participants will repeat some of the screening tests during the study.
Participants will take capsules of the study drug or a placebo by mouth for 168 days.
Participants will have tests to measure blood pressure and the speed of blood flow through the organs. They will also have a test that examines the function and reaction of the blood vessels. For these tests, they will wear blood pressure cuffs and other sensors.
Participants will complete questionnaires about their health.
Participants will have 9 study visits over 28 weeks. They may be contacted by phone between study visits.
详细描述
Study Description:
As a primary objective, this study represents an innovative investigative measure of the safety and tolerability of JAK inhibition in participants with primary Sjogren's syndrome. Secondary objectives will include investigating the effects of Tofacitinib on target tissues (e.g., salivary glands), systemic inflammation, and on vascular function in SS participants. We also aim to identify biomarkers of response that may be useful as endpoints in future studies.
Objectives:
Primary Objective:
-To determine the safety and tolerability of Tofacitinib in participants with SS and mild to moderate disease activity.
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Drug: Tofacitinib
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive tofacitinib 5 mg orally twice daily for 168 days.
干预措施: tofacitinib (Drug)
Placebo
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive placebo orally twice daily for 168 days.
干预措施: Placebo (Other)
结局指标
主要结局
Number of Adverse Events by Grade/Category
时间窗: Up to day 196
Count of adverse events by grade was assessed using the National Cancer Institute (NCI), Common Terminology Criteria for Adverse Events (CTCAE), Version 5.0. Grade 1: Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2: Moderate; minimal, local or noninvasive intervention indicated; limiting age appropriate instrumental ADL. Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care ADL. Grade 4: Life-threatening consequences; urgent intervention indicated. Grade 5: Death related to adverse event. Serious is defined as any grade 3 or higher adverse event. Toxicity is defined as any study drug-related Grade 3 or higher adverse event.
Participants With Adverse Events
时间窗: Up to day 196
Number participants with any adverse events by grade and severity was assessed using the National Cancer Institute (NCI), Common Terminology Criteria for Adverse Events (CTCAE), Version 5.0. Grade 1 Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2 Moderate; minimal, local or noninvasive intervention indicated; limiting age appropriate instrumental activity of daily living (ADL). Grade 3 Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care ADL. Grade 4 Life-threatening consequences; urgent intervention indicated. Grade 5 Death related to adverse event. Serious is defined as any grade 3 or higher adverse event. Toxicity is defined as any study drug-related Grade 3 or higher adverse event.
次要结局
- Change in Physicians Global Assessment (PGA) Score(Day 168 minus day 1)
- Change in EULAR Sjögren's Syndrome (SS) Disease Activity Index (ESSDAI) Score(Day 168 minus day 1)
- Change in Whole Unstimulated Saliva Flow(Day 168 minus day 1)
- Change in Whole Stimulated Saliva Flow(Day 168 minus day 1)
- Change in EULAR Sjögren's Syndrome (SS) Patient Reported Index (ESSPRI)(Day 168 minus day 1)