NCT00007020Phase 3Completed

Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

Study sponsor: Mirum Pharmaceuticals, Inc. · MIRM

Compare the available registry edits below. Primary completion is the date data collection ends for the main study measures; it does not tell you when results will be published.

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Primary completion2009-12Actual

Enrollment · participants85Actual

Source updated2023-10-03Last update listed in the registry record

Version history

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Study details

Conditions studied: Infantile Refsum's Disease; Zellweger Syndrome; Adrenoleukodystrophy; Peroxisomal Disorders; Cholestasis
Treatments or interventions studied: type: DRUG; name: Cholic Acids; otherNames: Cholic; Cholic Acid; Cholic Acid Capsules
Primary outcomes · main study measures: measure: Number of Participants With Excretion of Atypical Bile Acids in Urine by Category; description: Patients with excretion of atypical bile acids in urine by category, from worst status before treatment (baseline, BL) to best status on treatment (OT); timeFrame: Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
History coverage: Research sample: selected versions from May 2023 onward plus a preceding baseline where available. Only selected fields were retained; endpoint measures were truncated to 300 characters. This is not complete trial or sponsor history. Daily snapshots begin when collection starts.

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