This study is in progress, not accepting new patients

The REPLACE Registry for Cholbam® (Cholic Acid)

a study on Bile Acid Synthesis Disorders

Summary

Location
at San Francisco, California and other locations
Dates
study started
completion around

Description

Summary

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.

Official Title

A Prospective, Observational, Non-Interventional, Post-Marketing, Patient Registry to Collect Data on Routine Clinical Care in Patients Treated With Cholbam® (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, The REPLACE Registry, Existing User, New User

Eligibility

You can join if…

  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, IRB, and EC requirements.
  3. The patient has a diagnosis for which Cholbam is indicated.
  4. The patient is or will be treated with Cholbam at the time of signing the informed consent form (ICF) (enrollment).

You CAN'T join if...

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

Locations

  • UCSF
    San Francisco California 94158 United States
  • Stanford School of Medicine
    Palo Alto California 94305 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Mirum Pharmaceuticals, Inc.
ID
NCT03115086
Study Type
Observational [Patient Registry]
Participants
Expecting 55 study participants
Last Updated