A Phase 3 Study to Evaluate the Effects of Chenodeoxycholic Acid in Adult and Pediatric Patients With Cerebrotendinous Xanthomatosis
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 19
- 试验地点
- 1
- 主要终点
- Change From Baseline in Urine 23S-Pentol During the Two Double-Blind Periods
研究概览
简要总结
The study is made up of two cohorts: a randomized double-blind crossover (placebo withdrawal with rescue) study among patients ≥ 16 years of age (adult cohort) and an open-label dose titration study among pediatric patients ≥1 month and <16 years of age (pediatric cohort)
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
- 1 Month 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female at least 1 month or older at screening.
- •Clinical diagnosis of CTX with biochemical confirmation.
- •Women of childbearing potential must agree to the use of one highly reliable method of contraception during the study, plus one additional barrier method during sexual activity.
- •Males must be surgically sterile, or males and their sexual partners must together agree to use medically accepted methods of contraception that are considered highly reliable during the course of the study.
排除标准
- •Genetic testing does not confirm CTX.
- •Malabsorption disorder or confounding inflammatory gastrointestinal condition (for example, irritable bowel syndrome).
- •Documented history of heart failure.
- •Treated with medications which impact bile acid absorption such as bile acid sequestering agents (eg, cholestyramine, colestipol, aluminum-based antacids.
- •Treated with cholic acid medication.
- •Female patient who is pregnant, plans to become pregnant during the course of the study, or is breastfeeding.
- •Positive at screening for the human immunodeficiency virus (HIV) or markers indicating acute or chronic hepatitis B infection or hepatitis C infection.
研究组 & 干预措施
Adult Cohort
Patients in the adult cohort will participate in a randomized, double-blind, placebo-controlled, 2 period × 2-treatment crossover study with rescue medication and open-label run-in to assess the efficacy and safety of CDCA.
干预措施: Blinded CDCA 250 mg TID (Drug)
Adult Cohort
Patients in the adult cohort will participate in a randomized, double-blind, placebo-controlled, 2 period × 2-treatment crossover study with rescue medication and open-label run-in to assess the efficacy and safety of CDCA.
干预措施: Placebo (Drug)
Adult Cohort
Patients in the adult cohort will participate in a randomized, double-blind, placebo-controlled, 2 period × 2-treatment crossover study with rescue medication and open-label run-in to assess the efficacy and safety of CDCA.
干预措施: Open-Label CDCA 250 mg TID (Drug)
Adult Cohort
Patients in the adult cohort will participate in a randomized, double-blind, placebo-controlled, 2 period × 2-treatment crossover study with rescue medication and open-label run-in to assess the efficacy and safety of CDCA.
干预措施: Rescue Medication CDCA 250 mg TID (Drug)
Pediatric Cohort
Pediatric cohort patients (≥1 month and <16 years) will participate in a 24-week, open-label cohort with an 8-week titration period and a 16-week treatment period at the tolerated dose.
干预措施: CDCA Weight-Based Dose TID (Drug)
结局指标
主要结局
Change From Baseline in Urine 23S-Pentol During the Two Double-Blind Periods
时间窗: Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16
Primary Analysis of Change from Baseline in Urine 23S-Pentol (Natural Log-Transformed) during the Two Double-blind Periods- Paired T-test
次要结局
- Change From Baseline Plasma Cholestanol During the Two Double-Blind Periods(Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16)
- Change From Baseline Plasma 7αC4 During the Two Double-Blind Periods(Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16)
- Proportion of Participants Who Received Rescue Treatment During Two Double-Blind Periods(Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16)