Clinical Trial: The Replace Registry

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Observational [Patient Registry]

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)

Brief Summary: This is a prospective, observational, non-interventional patient registry designed to document long term safety and clinical outcomes for 10 years in patients treated with Cholbam/Kolbam, including those who have been using Cholbam/Kolbam and those who start Cholbam/Kolbam treatment at enrollment.

Detailed Summary: No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician.
Sponsor: Retrophin, Inc.

Current Primary Outcome:

• 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.
• Num
  
  

  Original Primary Outcome: Same as current
  
  

  Current Secondary Outcome:
  
  

  Original Secondary Outcome:
  
  Information By: Retrophin, Inc.
  

  Dates:
  Date Received: January 10, 2017
  Date Started: May 2017
  Date Completion: August 2030
  Last Updated: April 10, 2017
  Last Verified: April 2017