CompletedPhase 3

Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis

United States53 participantsStarted 2010-01

Plain-language summary

The primary purpose of the study is to evaluate the therapeutic efficacy and safety of cholic acid in subjects with identified inborn errors of bile acid synthesis.

Who can participate

SexALL

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Inclusion criteria

✓. The subject and/or parent/legal guardian must have provided informed consent prior to study start.
✓. The subject must have a diagnosis of an inborn error of bile acid synthesis.
✓. The subject must be willing and able to comply with all study assessments and procedures.
✓. Subjects with other organ dysfunction will not be excluded

What they're measuring

Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)

Timeframe: At baseline, then every 12 months for an average of 3.5 years

Trial details

NCT IDNCT01438411

SponsorMirum Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2016-07

Results submitted2020-06-29

View on ClinicalTrials.gov More Bile Acid Synthesis Defect trials