A Double Blind, Randomized Placebo Controlled Crossover Multiple Dose Study of LJN452 to Assess Safety, Tolerability and Efficacy in Patients With Primary Bile Acid Diarrhea (pBAD).
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Number of Patients Reported With Adverse Events , Serious Adverse Events and Death.
研究概览
简要总结
The purpose of this study is to determine whether LJN452 improves the symptoms of bile acid diarrhea and to assess its safety and tolerability profile in patients with primary bile acid diarrhea (pBAD) to guide decision-making regarding further clinical development in this indication.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A history of diarrheal symptoms for at least 3 months prior to dosing - Average stool frequency of at least 2 per day when off therapy AND Average stool form of >5 on Bristol Stool Chart.
- •Previous laboratory or radiological confirmation of bile acid malabsorption with either fecal bile acid loss OR 7 day 75Selenium homocholic acid taurine (75SeHCAT) retention.
- •Age ≥ 18 years.
排除标准
- •Patients with other diagnoses leading to diarrhea, including colorectal neoplasia, ulcerative colitis, Crohn's disease, celiac disease, chronic pancreatitis, drug-induced diarrhea or active infection AND Patients who have not been investigated by standard clinical assessments to exclude these disorders.
- •Treatment with bile acid sequestrants (colestyramine, colestipol, colesevelam) for 2 weeks before the first dose of LJN
- •A washout of 14 days for these agents will be allowed before first dosing.
- •Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant.
- •A positive Hepatitis B surface antigen or Hepatitis C test result.
- •History of immunodeficiency diseases, including a positive HIV (ELISA and Western blot) test result.
研究组 & 干预措施
LJN452 followed by placebo
Randomized patients in this arm will receive single oral dose of LJN452 daily for 14 days. There will be a washout period between 7 to 28 days followed by single oral dose of placebo daily for 14 days.
干预措施: LJN452 (Drug)
LJN452 followed by placebo
Randomized patients in this arm will receive single oral dose of LJN452 daily for 14 days. There will be a washout period between 7 to 28 days followed by single oral dose of placebo daily for 14 days.
干预措施: Placebo to LJN452 (Drug)
Placebo followed by LJN452
Randomized patients in this arm will receive single oral dose of placebo daily for 14 days. There will be a washout period between 7 to 28 days followed by single oral dose of LJN452 daily for 14 days.
干预措施: LJN452 (Drug)
Placebo followed by LJN452
Randomized patients in this arm will receive single oral dose of placebo daily for 14 days. There will be a washout period between 7 to 28 days followed by single oral dose of LJN452 daily for 14 days.
干预措施: Placebo to LJN452 (Drug)
结局指标
主要结局
Number of Patients Reported With Adverse Events , Serious Adverse Events and Death.
时间窗: up to Day 79
Number of patients reported with adverse events , serious adverse events and death.
Stool Frequency at Baseline, Week 1 (Period 1 & Period 2), Week 2 (Period 1 & Period 2), and Week 1 & 2 Combined
时间窗: Baseline, Week 1 (Period 1 & Period 2), Week 2 (Period 1 & Period 2), and Week 1 & 2 combined
Stool frequency at Baseline, Week 1 (Period 1 \& Period 2), Week 2 (Period 1 \& Period 2), and Week 1 \& 2 combined
Stool Form at Baseline, Week 1 (Period 1 & Period 2), Week 2 (Period 1 & Period 2), and Week 1 & 2 Combined
时间窗: Baseline, Week 1 (Period 1 & Period 2), Week 2 (Period 1 & Period 2), and Week 1 & 2 combined
Stool Form at Baseline, Week 1 (Period 1 \& Period 2), Week 2 (Period 1 \& Period 2), and Week 1 \& 2 combined Clinical Symptoms will be measured as change from baseline in stool types per Bristol Stool Scale. The Bristol Stool Scale is a medical aid designed to classify feces on a scale from 1 to 7 according to increasing wateriness.
次要结局
- Area Under the Plasma Concentration-time Profile (AUCtau) of LJN452(Day 1 (Period 1 & 2) and Day 12 (Period 1 & 2))
- Time to Reach Maximum Concentration After Drug Administration (Tmax)(Day 1 (Period 1 & 2) and Day 12 (Period 1 & 2))
- Total Dose of Rescue Medication Used at Baseline, Week 1 (Period 1 & Period 2), Week 2 (Period 1 & Period 2), and Week 1 & 2 Combined(Baseline, Week 1 (Period 1 & 2), Week 2 (Period 1 & 2), Week 1 & 2 combined)
- (Cmax) of LJN452(Day 1 (Period 1 & 2) and Day 12 (Period 1 & 2))