An Open Label, Single Center, Nonrandomized Continuation Study of Cholic Acid Capsules to Treat Subjects With Inborn Errors of Bile Acid Synthesis
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 53
- 试验地点
- 1
- 主要终点
- Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)
研究概览
简要总结
The primary purpose of the study is to evaluate the therapeutic efficacy and safety of cholic acid in subjects with identified inborn errors of bile acid synthesis.
详细描述
This is a Phase 3, open-label, single center, nonrandomized study. This continuation protocol will consist of eligible subjects who have previously received cholic acid through the Cincinnati Children's Hospital Medical Center (CCHMC) Compassionate Use (91-10-10), CAC-001-01 study protocols and newly diagnosed subjects.
New subjects will be infants, children, adolescents identified from urine samples obtained from the clinical services of programs across the U.S., Canada, South America, Europe, and Asia. Subject or their legal representative will receive information regarding the study, and the principle investigator (PI) or designee will obtain informed consent. Serum and urine samples will be collected and sent to CCHMC to measure complete bile acid profile analysis. Clinical records including medical history, physical exams, vital signs, and laboratory assessments performed as standard of care will be reviewed to ensure subject eligibility and determine baseline values.
Subjects who have participated in Protocols conducted under IND 45,470 will be consented to continue to receive cholic acid capsules under this continuation protocol. Subjects will serve as their own controls and no placebo will be utilized.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects who received cholic acid through CCHMC protocols 91-10-10 or CAC-002-01 and meet the following criteria are eligible for study participation.
- •The subject and/or parent/legal guardian must have provided informed consent prior to study start.
- •The subject must have a diagnosis of an inborn error of bile acid synthesis.
- •The subject must be willing and able to comply with all study assessments and procedures.
- •Subjects with other organ dysfunction will not be excluded
排除标准
- 未提供
研究组 & 干预措施
Cholic Acid
Active drug
干预措施: Cholic Acid (Drug)
结局指标
主要结局
Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)
时间窗: At baseline, then every 12 months for an average of 3.5 years
The level of atypical urinary bile acid secretion was scored using a scale of: 0, normal; 1, slight; 2, significant; or 3, marked. A Cochran-Mantel-Haenszel (CMH) test with modified ridit scoring was used to compare the difference between the score at baseline and the worst post-baseline score during treatment with cholic acid in this single-arm trial.
次要结局
- Clinical Laboratory Results: Alkaline Phosphatase(At baseline, then every 12 months for an average of 3.5 years)
- Clinical Laboratory Results: Prothrombin Time(At baseline, then every 12 months for an average of 3.5 years)
- Clinical Laboratory Results: Bilirubin(At baseline, then every 12 months for an average of 3.5 years)
- Evaluation of Serum Transaminases: AST(At baseline, then every 12 months for an average of 3.5 years)
- Physical Examinations: Body Weight(At baseline, then every 12 months for an average of 3.5 years)
- Incidence of Adverse Events(At baseline, then every 12 months for an average of 3.5 years)
- Evaluation of Serum Transaminases: ALT(At baseline, then every 12 months for an average of 3.5 years)
- Clinical Laboratory Results: Gamma Glutamyl Transferase (GGT)(At baseline, then every 12 months for an average of 3.5 years)
- Physical Examinations: Height(At baseline, then every 12 months for an average of 3.5 years)