A Phase 3, Prospective, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Chenodeoxycholic Acid (CDCA), With an Open-Label Cohort, in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX)
试验速览
- 阶段
- 3 期
- 状态
- 撤回
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Change from baseline at Week 4 in average number of stools with a BSS score of 6 or 7 per day
研究概览
简要总结
This study is designed to demonstrate the beneficial effect of CDCA in the treatment of CTX-associated diarrhea in approximately 10 participants aged 2-75 years old with newly diagnosed CTX or suspected CTX who have never received treatment with CDCA.
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
- 2 Years 至 75 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent form (or assent form as appliable)
- •Aged from 2 to 75 years old
- •Has a new or suspected diagnosis of CTX as defined by an elevated plasma cholestanol concentration (>10 mg/L/>25.7 μmol/L) in conjunction with a clinical presentation consistent with the diseased as assessed by the investigator
- •Has never received treatment with CDCA
- •Has never received treatment with other bile acid products
排除标准
- •Any medical condition that, in the opinion of the investigator, precludes the participant's participation in the study
- •Presence of known hepatocyte dysfunction or bile ductal abnormalities such as intrahepatic cholestasis, primary biliary cirrhosis or sclerosing cholangitis
- •Inability to adhere to treatment and visit schedule
- •Female participants who are pregnant
- •Female participants who are breast feeding
- •Female participants who are using estrogen-containing compounds and cannot/will not discontinue them for the duration of the study
- •Female participants of childbearing potential who are not using locally approved birth control method(s) or double barrier contraception (ie, condom and diaphragm, condom or diaphragm and spermicidal gel or foam)
- •Taking any of the following medications: bile acid products; inhibitors of bile acid transporters; bile acid binding resins; aluminum-based antacids; coumarin and its derivatives; cholestyramine; ciclosporin; sirolimus; or phenobarbital
研究组 & 干预措施
Group 1 Diarrhea-Evaluable
Diarrhea-evaluable participants aged 12-75 years old who did not use continence aids that precluded assessment or evaluation of stool frequency and consistency during the screening/baseline period; randomized 1:1 to receive either placebo or CDCA.
干预措施: Chenodeoxycholic acid (Drug)
Group 1 Diarrhea-Evaluable
Diarrhea-evaluable participants aged 12-75 years old who did not use continence aids that precluded assessment or evaluation of stool frequency and consistency during the screening/baseline period; randomized 1:1 to receive either placebo or CDCA.
干预措施: Chenodeoxycholic acid Placebo (Drug)
Group 2a Non-Diarrhea Evaluable
Non-diarrhea-evaluable participants aged 12-75 years old who do not have stable, clinically burdensome diarrhea or their diarrhea cannot be fully characterized.
干预措施: Chenodeoxycholic acid (Drug)
Group 2b Pediatrics
Participants aged 2 to less than 12 years old with or without clinically burdensome diarrhea.
干预措施: Chenodeoxycholic acid (Drug)
结局指标
主要结局
Change from baseline at Week 4 in average number of stools with a BSS score of 6 or 7 per day
时间窗: Up to 4 weeks
次要结局
- Number of participants with discontinuations due to AEs(Up to 20 weeks)
- Change in urine and/or plasma bile alcohol levels from baseline to Week 12(Up to 12 weeks)
- Incidence of, severity/intensity of, and relationship to study drug of SAEs(Up to 20 weeks)
- Incidence of, severity/intensity of, and changes in physical examination(Up to 20 weeks)
- Change in plasma cholestanol levels from baseline to Week 12(Up to 12 weeks)
- Incidence of, severity/intensity of, and changes in laboratory values(Up to 20 weeks)
- Incidence of, severity/intensity of, and relationship to study drug of AEs(Up to 20 weeks)
- Incidence of, severity/intensity of, and changes in vital signs(Up to 20 weeks)
- Incidence of, severity/intensity of AESIs(Up to 20 weeks)