A Prospective, Observational, Non-Interventional, Post-Marketing, Patient Registry to Collect Data on Routine Clinical Care in Patients Treated With Cholbam® (Cholic Acid)
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 55
- 试验地点
- 38
- 主要终点
- Number of participants with new-onset cholestasis
研究概览
简要总结
This is a prospective, observational, non-interventional patient registry study designed to document product safety and effectiveness outcomes for 10 years in patients treated with Cholbam, including those who have been using Cholbam for at least 30 days (existing users) and those who are first-time initiators of Cholbam.
详细描述
No experimental intervention is involved. Patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female patients, of any age.
- •The patient and/or the patient's parent/legal guardian is willing and able to provide signed informed consent, and the patient, if less than 18 years of age, is willing to provide assent as appropriate and in accordance with local regulatory, IRB, and EC requirements.
- •The patient has a diagnosis for which Cholbam is indicated.
- •The patient is or will be treated with Cholbam at the time of signing the informed consent form (ICF) (enrollment).
排除标准
- •1. Patients who, by judgement of the Investigator, will not be able to comply with the requirements of the protocol will be excluded
研究组 & 干预措施
Existing User
Patients who have been using Cholbam for at least 30 days
干预措施: Cholbam (Drug)
New User
First-time initiators of Cholbam
干预措施: Cholbam (Drug)
结局指标
主要结局
Number of participants with new-onset cholestasis
时间窗: 10 Years
Patients with new-onset cholestasis will be identified by detecting abnormal direct bilirubin concentration \>1mg/dL or direct bilirubin greater than 20% of the total bilirubin if total bilirubin is \>5mg/dL.
Number of participants with steatorrhea leading to poor growth
时间窗: 10 Years
Steatorrhea leading to poor growth in children, which will be defined as a decrease in growth percentiles from the original percentile at enrollment to the registry study.
Death
时间窗: 10 Years
AEs and SAEs leading to death will be recorded. The relatedness of death to Cholbam or disease progression will be recorded.
Number of participants with worsening cholestasis
时间窗: 10 Years
Worsening cholestasis will be identified by measuring direct total bilirubin concentration in blood and will be defined as a 25% increase from previous measurement if the total bilirubin is \>1mg/dL.
Changes in serum levels of fat-soluble vitamins
时间窗: 10 Years
Fat-soluble vitamin (A, D, E, K) deficiencies will be identified by comparing results from serum assays (Vitamin A: Retinol, Vitamin D: 250HD2 + 250HD3, Vitamin K: Serum Vitamin K, Vitamin E: Serum Vitamin E) for each vitamin with standard ranges.
Neuropathic signs or symptoms related to fat-soluble vitamin deficiencies
时间窗: 10 Years
Neuropathic signs or symptoms related to fat-soluble vitamin deficiencies (peripheral neuropathy, cerebellar ataxia) will be assessed by the Investigator.
Number of participants with growth failure
时间窗: 10 Years
Growth failure that the physician judges to be attributable to malabsorption.
Adverse effects on pregnancy, pregnancy outcomes, and infant status
时间窗: 10 Years
Adverse effects on pregnancy, pregnancy outcomes, and infant status will be recorded.
All Adverse Events (AEs) and Serious AEs (SAEs)
时间窗: 10 years
All other AEs and SAEs will be collected.
次要结局
- Changes in Cholbam dosing regimens and reasons for any dose modifications or treatment discontinuations(10 years)
- Changes from baseline in weight(10 years)
- Changes from baseline in length/height(10 years)
- Age-appropriate developmental milestones in infants(10 years)
- Changes in international normalized ratio (INR)(10 years)
- Changes in albumin(10 years)
- Changes in direct bilirubin(10 years)
- Changes in alanine aminotransferase (ALT)(10 years)
- All indications for which Cholbam has been prescribed(10 years)
- Changes from baseline in head circumference in infants(10 years)
- Changes in prothrombin time (PT)(10 years)
- Presence or absence of urinary bile acids and levels of bile acid intermediaries and urinary bile alcohol(10 years)
- Changes in bilirubin(10 years)
- Changes in aspartate aminotransferase (AST)(10 years)
- Changes in alkaline phosphatase(10 years)
- Changes in gamma-glutamyl transferase (GGT)(10 years)