Phase 2 Primary Biliary Cholangitis Trial, Completed NCT01904058 Sponsor: Mirum Pharmaceuticals, Inc. Condition: Primary Biliary Cholangitis
Back to Primary Biliary Cholangitis

Trial results

AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

According to the results reported on ClinicalTrials.gov, this trial enrolled 66 adults in total across four groups. Participants were already taking a standard medication called UDCA (ursodeoxycholic acid) and were then given either a low dose (10 mg) or higher dose (20 mg) of an investigational drug called LUM001, or a placebo (a dummy treatment with no active ingredient), for 13 weeks. The trial was primarily measuring changes in itching — a common and troublesome symptom in liver conditions — using a diary-based scoring tool called the ItchRO, where participants rated their itch twice daily on a scale of 0 (no itch) to 10 (very severe itch). Of the 66 who started, 61 completed the trial. The reported data shows that at the start of the trial, all four groups had similar weekly itch sum scores (ranging roughly from 48 to 55 points out of a possible 70). By week 13, the reported changes in that weekly sum score were: a reduction of about 24.6 points in the LUM001 10 mg group, a reduction of about 27.7 points in the LUM001 20 mg group, a reduction of about 26.2 points in the placebo group for Cohort A, and a reduction of about 22.8 points in the placebo group for Cohort B. The reported data also shows changes were tracked at weeks 4 and 8 across secondary measures, including a separate five-dimension itch scale, average daily itch scores, and blood test results for alkaline phosphatase (a liver-related enzyme) and bile acid levels (substances that can build up in liver conditions). All four groups showed reductions in itch scores across most time points and measures, with figures for blood markers varying across the groups and time points. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.

Phase 2 Primary Biliary Cholangitis Trial, Completed

NCT01904058
Completed Phase 2

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

  • You have been diagnosed with Primary Biliary Cirrhosis (a liver condition)
  • You experience moderate to severe itching related to your condition
  • You have been taking a medication called ursodeoxycholic acid (UDCA) for at least 6 months, or you are unable to take this medication due to side effects
  • You are able to understand the study information and are willing to sign a consent form before any study procedures begin

Who may not be able to join:

  • You have been diagnosed with, or currently have, another significant liver disease alongside your existing condition
  • You have previously had or are currently awaiting a liver transplant
  • You have a known HIV infection
  • You are currently pregnant or breastfeeding

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 28 July 2026
Phase 2: approximately ~30% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.
Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

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Trial details

Status
Completed
Phase
Phase 2
Sponsor
Mirum Pharmaceuticals, Inc.
Registry
ClinicalTrials.gov
Start date
1 August 2013
Est. completion
1 April 2015

Where this trial is recruiting

🇨🇦 Canada 🇬🇧 United Kingdom 🇺🇸 United States

Primary endpoints

Change From Baseline in Pruritus Using Adult Itch Reported Outcome (ItchRO) Weekly Sum Score at Week 13/ Early Termination (ET)

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov