|Principal Investigator: Linda Book|
|Keywords: Cholbam , Kolbam , Cholic Acid||Department: Pediatric Administration|
|IRB Number: 00115154|
|Specialty: Pediatric Gastroenterology|
|Recruitment Status: Not yet recruiting|
The purpose of the study is to assess primarily the long term safety of treatment with Cholbam/Kolbam (cholic acid) capsules with respect to incidence rates of worsening cholestasis, steatorrhea leading to poor growth, fat-soluble vitamin deficiency, or neuropathic process related to a fat-soluble vitamin deficiency, and the incidence of death and adverse effects on pregnancy, pregnancy outcomes and infant status. Additional evaluations will include dosing regimens and reasons for any dose modifications, weight gain, length/height and developmental outcomes.
The primary objective of this registry study is to describe the long-term safety of treatment with Cholbam/Kolbam, with particular attention to the following:
1. New-onset cholestasis
2. Worsening cholestasis
3. Steatorrhea leading to poor growth.
4. Changes in serum levels of fat- soluble vitamins
5. Signs of fat-soluble vitamin deficiencies that the physician judges to be attributable to malabsorption, including neuropathic process related to fat-soluble vitamin deficiency (peripheral neuropathy, cerebellar ataxia) and other neurological abnormalities
6. Growth failure that the physician judges to be attributable to malabsorption
8. Adverse effects on pregnancy, pregnancy outcomes and infant status
9. All other adverse events (AEs) and serious adverse events (SAEs)
The secondary objectives are to describe:
1. Changes in Cholbam/Kolbam dosing regimens and reasons for any dose modifications or treatment discontinuations.
2. Changes in weight.
3. Changes in length/height and in infants, head circumference.
4. Age-appropriate developmental milestones in infants (as measured by World Health Organization [WHO] Motor Development Milestones).
5. Changes prothrombin time (PT), international normalized ratio (INR) and albumin, and monitoring alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma-glutamyl transferase (GGT), and alkaline phosphatase.
6. Presence or absence of bile acid intermediates.
7. All indications for which Cholbam/Kolbam has been prescribed.
The objective of the aRMM sub-study is to evaluate the utility of the risk minimization measures (aRMM), comprising the summary of product characteristics (SmPC) and additional educational materials, implemented in Europe for Kolbam.
A patient must meet all of the following criteria to be eligible for enrollment in the registry:
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, institutional review board (IRB), and ethics committee (EC) requirements.
3. The patient has a diagnosis for which Cholbam/Kolbam is indicated.
4. The patient is treated with Cholbam/Kolbam at enrollment.
1. Patients who will not be able to comply with the requirements of the protocol will be excluded.