Clinical Trials in the USA / NCT03115086
Active, not recruiting
Observational
The REPLACE Registry for Cholbam® (Cholic Acid)
NCT03115086 · tracked via the Priya Life Science USA tracker
Condition(s) studied
Bile Acid Synthesis Disorders
Investigational drug(s) / intervention(s)
Cholbam
Cholbam: Cholbam prescribed according to the approved label.
Study summary
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.
Eligibility
Inclusion Criteria:
1. Male and female patients, of any age.
2. 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.
3. The patient has a diagnosis for which Cholbam is indicated.
4. The patient is or will be treated with Cholbam at the time of signing the informed consent form (ICF) (enrollment).
Exclusion Criteria:
1\. Patients who, by judgement of the Investigator, will not be able to comply with the requirements of the protocol will be excluded
Primary outcome measure(s)
- Number of participants with worsening cholestasis — 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. - Number of participants with new-onset cholestasis — 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 — 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. - Changes in serum levels of fat-soluble vitamins — 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 — 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 — 10 Years
Growth failure that the physician judges to be attributable to malabsorption. - Death — 10 Years
AEs and SAEs leading to death will be recorded. The relatedness of death to Cholbam or disease progression will be recorded. - Adverse effects on pregnancy, pregnancy outcomes, and infant status — 10 Years
Adverse effects on pregnancy, pregnancy outcomes, and infant status will be recorded. - All Adverse Events (AEs) and Serious AEs (SAEs) — 10 years
All other AEs and SAEs will be collected.
Trial sites (24)
| Facility | City | Region | Status |
|---|---|---|---|
| University of Alabama Birmingham | Birmingham | Alabama | |
| Stanford School of Medicine | Palo Alto | California | |
| UC San Francisco | San Francisco | California | |
| Kidz Pediatric Multispecialty Group | Hollywood | Florida | |
| Nemours duPont Pediatrics Specialty Clinic | Jacksonville | Florida | |
| Nicklaus Children's Hospital | Miami | Florida | |
| Children's Healthcare of Atlanta | Atlanta | Georgia | |
| Children's Center For Digestive Health | Atlanta | Georgia | |
| The Community Health Clinic | Topeka | Indiana | |
| Ochsner Hospital for Children | New Orleans | Louisiana | |
| University of Minnesota | Minneapolis | Minnesota | |
| Mayo Clinic | Rochester | Minnesota | |
| Office of Dr. Esperanza Font-Montgomery MD | Columbia | Missouri | |
| Washington University at St Louis | St Louis | Missouri | |
| NYU Langone - NYU Grossman School of Medicine | New York | New York | |
| Icahn School Of Medicine at Mount Sinai | New York | New York | |
| Montefiore Medical Center | New York | New York | |
| Golisano Children's Hospital, URMC | Rochester | New York | |
| UNC Chapel Hill | Chapel Hill | North Carolina | |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | |
| University of Oklahoma | Oklahoma City | Oklahoma | |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | |
| Monroe-Carell Jr. Children's Hospital at Vanderbilt | Nashville | Tennessee | |
| Primary Children's Hospital | Salt Lake City | Utah |
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Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT03115086 on ClinicalTrials.gov ↗ ← All trials in the USA