The REPLACE Registry for Cholbam®/Kolbam® (Cholic Acid)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03115086|
Recruitment Status : Recruiting
First Posted : April 14, 2017
Last Update Posted : July 1, 2020
|Condition or disease|
|Bile Acid Synthesis Disorders|
|Study Type :||Observational [Patient Registry]|
|Estimated Enrollment :||110 participants|
|Target Follow-Up Duration:||10 Years|
|Official Title:||A Prospective, Observational, Non-Interventional, Post-Marketing, Patient Registry to Collect Data on Routine Clinical Care in Patients Treated With Cholbam®/Kolbam® (Cholic Acid)|
|Actual Study Start Date :||July 10, 2017|
|Estimated Primary Completion Date :||July 2038|
|Estimated Study Completion Date :||July 2039|
- Number of Participants with New-Onset or Worsening Cholestasis [ Time Frame: 10 Years ]Safety and effectiveness will be measured by new-onset or worsening cholestasis. Worsening cholestasis will be identified by measuring direct total bilirubin concentration, and will be defined as a 25% increase from previous measurement if the total bilirubin is >1mg/dL. 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 [ Time Frame: 10 Years ]Safety and effectiveness will be measured by steatorrhea leading to poor growth in children, which will be defined as a decrease in percentiles from the original percentile at enrollment to the registry study.
- Number of Participants changes in Serum Levels of Fat Soluble Vitamins [ Time Frame: 10 Years ]Safety and effectiveness will be measured changes in serum levels of fat soluble vitamins measured by deficiencies 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.
- Number of Participants with Growth Failure [ Time Frame: 10 Years ]Safety and effectiveness will be measured by growth failure will be defined as a decrease in percentiles from the original percentile at entry point into the registry study.
- Number of Participants with Treated Related Adverse Events [ Time Frame: 10 years ]Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03115086
|Contact: Retrophin Medical Informationfirstname.lastname@example.org|
|Principal Investigator:||Feriandas Greblikas, MD||Senior Medical Director|