NCT02061540已完成2 期
A Pilot, Open-Label Study to Evaluate the Safety, Tolerability and Efficacy of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients With Primary Sclerosing Cholangitis
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 27
- 试验地点
- 9
- 主要终点
- Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
研究概览
简要总结
The study is an open-label study in adults with primary sclerosing cholangitis to evaluate the safety, tolerability, and effect of 14-weeks of daily dosing of LUM001.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female subjects between the ages of 18-80 years, inclusive.
- •Diagnosis of PSC
- •If inflammatory bowel disease (IBD) is present, disease activity ≤ 2 (normal to moderate), using the physician assessment on the Mayo ulcerative colitis (UC) disease activity score.
- •Patients receiving azathioprine for intestinal bowel disease are eligible to participate in the study provided that they have had no IBD exacerbations for at least 6 months.
- •Females of childbearing potential must have a negative serum pregnancy test [β human chorionic gonadotropin (β-hCG)] during screening and negative urine pregnancy test at the baseline/Day 0 visit.
- •Sexually active females must be postmenopausal, surgically sterile, or if premenopausal, be prepared to use an effective method (≤ 1% failure rate) of contraception during the trial.
- •Ability to read and understand English in order to use the study-related questionnaires and the text on the eDiary screen.
- •Must be willing and able to use an eDiary daily for a minimum of 20 weeks.
- •Must digitally accept the licensing agreement in the eDiary software at the outset of the study.
- •Must complete at least 10 eDiary Adult ItchRO reports (AM or PM) during each of two consecutive weeks of the screening period prior to allocation to treatment (maximum possible reports = 14 per week).
- •Access to phone for scheduled calls from study site.
- •Must agree to comply with the study protocol procedures and provide written informed consent.
排除标准
- •Small duct PSC (clinical biochemical and histological features compatible with PSC, but having a normal cholangiogram).
- •Presence of a dominant stricture unless brushings and/or biopsies of the stricture are negative for dysplasia or malignancy within 6 months of screening.
- •Surgical or endoscopic biliary tree interventions for treatment of clinically significant strictures within 6 months of screening.
- •IBD flare (Mayo UC disease activity score > 5 including endoscopic evaluation) within 3 months prior to screening.
- •Secondary cause of sclerosing cholangitis (e.g., choledocholithiasis, post-surgical biliary stricture, intra-arterial chemotherapy, recurrent pancreatitis, IgG4 associated cholangiopathy, AIDS cholangiopathy).
- •AST or ALT ≥ 5 x ULN at screening.
- •History or presence of any other concomitant significant liver disease as assessed by the Investigator.
- •Medical conditions that may cause nonhepatic increases in ALP (e.g., Paget's disease).
- •Known history of human immunodeficiency virus (HIV) infection.
- •The anticipated need for a surgical procedure within 20 weeks from randomization.
- •Any female who is pregnant or lactating or who is planning to become pregnant within 20 weeks of randomization.
- •History of cancer, except for basal or squamous cell carcinoma of the skin, or with any laboratory or physical exam or diagnostic procedure finding suggestive of current malignancy.
- •Family history of any documented hereditary cancer syndrome.
- •History of alcohol or other substance abuse within 1 year prior to screening.
- •Receipt of an investigational drug, biologic, or medical device within 30 days prior to Screening, or 5 half-lives of the study agent, whichever is longer.
- •History of noncompliance with medical regimens, unreliability, mental instability or incompetence that could compromise the validity of informed consent or lead to noncompliance with the study protocol.
- •Any other conditions or abnormalities which, in the opinion of the Investigator or Medical Monitor, may compromise the safety of the subject, or interfere with the subject participating in or completing the study.
研究组 & 干预措施
LUM001
Experimental
LUM001 administered orally once each day
干预措施: LUM001 (Drug)
结局指标
主要结局
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
时间窗: From start of study drug administration until Week 18
An Adverse Event (AE) was defined as any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered to be related to the investigational drug product. TEAEs were AEs with a start date on or after the first dose of investigational product and started prior to the last dose of investigational product plus 14 days.
Change From Baseline in Fasting Serum Bile Acid Level at Week 14
时间窗: Baseline, Week 14
Serum bile acid levels were evaluated using blood samples collected.
次要结局
- Change From Baseline in Bilirubin Levels at Week 14(Baseline, Week 14)
- Change From Baseline in Pruritus as Measured by Adult Itch Reported Outcome (ItchRO) Weekly Sum Score(Baseline, Week 14)
- Change From Baseline in Liver Enzyme Levels in Serum(Baseline, Week 14)
研究者
研究点 (9)
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