Purpose

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.

Condition

Eligibility

Eligible Ages
All ages
Eligible Genders
All
Accepts Healthy Volunteers
No

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

Study Design

Phase
Study Type
Observational [Patient Registry]
Observational Model
Cohort
Time Perspective
Prospective

Arm Groups

ArmDescriptionAssigned Intervention
Existing User Patients who have been using Cholbam for at least 30 days
  • Drug: Cholbam
    Cholbam prescribed according to the approved label.
New User First-time initiators of Cholbam
  • Drug: Cholbam
    Cholbam prescribed according to the approved label.

More Details

Status
Active, not recruiting
Sponsor
Mirum Pharmaceuticals, Inc.

Study Contact

Detailed Description

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

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.