The REPLACE Registry for CholbamĀ® (Cholic Acid)
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
- Bile Acid Synthesis Disorders
Eligibility
- Eligible Ages
- All ages
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- 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
- 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
Arm | Description | Assigned Intervention |
---|---|---|
Existing User | Patients who have been using Cholbam for at least 30 days |
|
New User | First-time initiators of Cholbam |
|
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.