The REPLACE Registry for Cholbam® (Cholic Acid)

Mirum Pharmaceuticals

Status: Active, not recruiting

Conditions: Bile Acid Synthesis Disorders

Treatments: Drug: Cholbam

Study type: Observational

Funder types: Industry

Identifiers

NCT03115086

031CHO15001

Details and patient eligibility

About

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.

Full description

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

Enrollment: 55 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

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).

Exclusion criteria

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

Trial design

55 participants in 2 patient groups

Existing User

Description:

Patients who have been using Cholbam for at least 30 days

Treatment:

Drug: Cholbam

New User

Description:

First-time initiators of Cholbam

Treatment:

Drug: Cholbam