Active — Not RecruitingNot Applicable

The REPLACE Registry for Cholbam® (Cholic Acid)

United States55 participantsStarted 2017-07-10

Plain-language 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.

Who can participate

SexALL

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Inclusion criteria

✓. Male and female patients, of any age.
✓. 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.
✓. The patient has a diagnosis for which Cholbam is indicated.
✓. The patient is or will be treated with Cholbam at the time of signing the informed consent form (ICF) (enrollment).

What they're measuring

Number of participants with worsening cholestasis

Timeframe: 10 Years

Number of participants with new-onset cholestasis

Timeframe: 10 Years

Number of participants with steatorrhea leading to poor growth

Timeframe: 10 Years

Changes in serum levels of fat-soluble vitamins

Timeframe: 10 Years

Neuropathic signs or symptoms related to fat-soluble vitamin deficiencies

Timeframe: 10 Years

Number of participants with growth failure

Timeframe: 10 Years

Death

Timeframe: 10 Years

Adverse effects on pregnancy, pregnancy outcomes, and infant status

Timeframe: 10 Years

Trial details

NCT IDNCT03115086

SponsorMirum Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2038-07

View on ClinicalTrials.gov More Bile Acid Synthesis Disorders trials