Summary

at San Francisco, California and other locations
study started
estimated completion:

Description

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.

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)

Details

No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician.

Keywords

Bile Acid Synthesis Disorders Bile Acid Synthesis Disorder Zellweger Spectrum Disorder Peroxisomal Disorder Cholic Acid Cholbam Kolbam The Replace Registry Cholic Acids

Eligibility

You can join if…

  1. Male and female patients, of any age.
  2. The patient has a diagnosis for which Cholbam/Kolbam is indicated.
  3. The patient is treated with Cholbam/Kolbam at enrollment.

You CAN'T join if...

  1. Patients who will not be able to comply with the requirements of the protocol will be excluded.

Locations

  • UC San Francisco accepting new patients
    San Francisco California 94158 United States
  • Stanford School of Medicine accepting new patients
    Palo Alto California 94305 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Retrophin, Inc.
ID
NCT03115086
Study Type
Observational [Patient Registry]
Last Updated
July 9, 2018