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.
No experimental intervention is involved. Patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician.
Study Type
OBSERVATIONAL
Enrollment
55
Cholbam prescribed according to the approved label.
University of Alabama Birmingham
Birmingham, Alabama, United States
Number of participants with worsening cholestasis
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.
Time frame: 10 Years
Number of participants with new-onset cholestasis
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.
Time frame: 10 Years
Number of participants with steatorrhea leading to poor growth
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.
Time frame: 10 Years
Changes in serum levels of fat-soluble vitamins
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.
Time frame: 10 Years
Neuropathic signs or symptoms related to fat-soluble vitamin deficiencies
Neuropathic signs or symptoms related to fat-soluble vitamin deficiencies (peripheral neuropathy, cerebellar ataxia) will be assessed by the Investigator.
Time frame: 10 Years
Number of participants with growth failure
Growth failure that the physician judges to be attributable to malabsorption.
Time frame: 10 Years
Death
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Stanford School of Medicine
Palo Alto, California, United States
UC San Francisco
San Francisco, California, United States
Kidz Pediatric Multispecialty Group
Hollywood, Florida, United States
Nemours duPont Pediatrics Specialty Clinic
Jacksonville, Florida, United States
Nicklaus Children's Hospital
Miami, Florida, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Children's Center For Digestive Health
Atlanta, Georgia, United States
The Community Health Clinic
Topeka, Indiana, United States
Ochsner Hospital for Children
New Orleans, Louisiana, United States
...and 13 more locations
AEs and SAEs leading to death will be recorded. The relatedness of death to Cholbam or disease progression will be recorded.
Time frame: 10 Years
Adverse effects on pregnancy, pregnancy outcomes, and infant status
Adverse effects on pregnancy, pregnancy outcomes, and infant status will be recorded.
Time frame: 10 Years
All Adverse Events (AEs) and Serious AEs (SAEs)
All other AEs and SAEs will be collected.
Time frame: 10 years
Changes in Cholbam dosing regimens and reasons for any dose modifications or treatment discontinuations
Time frame: 10 years
Changes from baseline in weight
For patients aged 18 years of or less, these data will be converted into Z-scores based on the WHO growth standards for infants aged 0-2 years and the Centers for Disease Control and Prevention growth standards for children 2-20 years. Growth failure will be defined as a decrease in percentiles from the original percentile at entry point into the registry study.
Time frame: 10 years
Changes from baseline in length/height
For patients aged 18 years of or less, these data will be converted into Z-scores based on the WHO growth standards for infants aged 0-2 years and the Centers for Disease Control and Prevention growth standards for children 2-20 years. Some patients will be adult or will have achieved final height. Growth failure will be defined as a decrease in percentiles from the original percentile at entry point into the registry study.
Time frame: 10 years
Changes from baseline in head circumference in infants
For patients aged 18 years of or less, these data will be converted into Z-scores based on the WHO growth standards for infants aged 0-2 years and the Centers for Disease Control and Prevention growth standards for children 2-20 years. Growth failure will be defined as a decrease in percentiles from the original percentile at entry point into the registry study.
Time frame: 10 years
Age-appropriate developmental milestones in infants
Measured by WHO Motor Development Milestones
Time frame: 10 years
Changes in prothrombin time (PT)
Time frame: 10 years
Changes in international normalized ratio (INR)
Time frame: 10 years
Changes in albumin
Time frame: 10 years
Changes in bilirubin
Time frame: 10 years
Changes in direct bilirubin
Time frame: 10 years
Changes in alanine aminotransferase (ALT)
Time frame: 10 years
Changes in aspartate aminotransferase (AST)
Time frame: 10 years
Changes in gamma-glutamyl transferase (GGT)
Time frame: 10 years
Changes in alkaline phosphatase
Time frame: 10 years
Presence or absence of urinary bile acids and levels of bile acid intermediaries and urinary bile alcohol
Time frame: 10 years
All indications for which Cholbam has been prescribed
Description of all indications for which Cholbam has been prescribed
Time frame: 10 years