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 27 participants, all of whom received the study drug maralixibat (also known as LUM001). The trial was looking at a condition called cholestasis — a liver problem where bile does not flow properly — and measured several things over roughly 14 weeks: how many participants experienced unwanted health events (called adverse events) while taking the drug, changes in bile acid levels in the blood, changes in liver enzyme levels, changes in bilirubin levels (a substance linked to liver function), and changes in itching severity. Participants were given different doses of the drug. Twenty-three of the 27 participants completed the study, and four did not finish. The reported data shows that when it came to unwanted health events during the study, the numbers varied by dose group: 1 participant at the 1 mg dose, 2 at the 2.5 mg dose, 2 at the 5 mg dose, 1 at the 7.5 mg dose, and 19 at the 10 mg dose experienced such events. For bile acids in the blood, the reported starting (baseline) level was about 38.9 micromoles per litre, and by week 14 the reported change was a decrease of about 14.8 micromoles per litre. For liver enzymes, the reported data shows starting levels and changes across three enzymes (ALT, AST, and ALP), with baseline figures of approximately 108.5, 88.3, and 471.6 units per litre respectively, and reported changes of 10.5, 11.7, and 36.7 units per litre respectively — though the direction of those changes (up or down) was not clearly labelled in the submitted data. For bilirubin, starting levels were reported as approximately 1.22 mg/dL (total) and 0.60 mg/dL (direct), with reported changes of 0.24 and 0.19 mg/dL respectively. For itching, participants started with a weekly score of around 15 out of a possible 70 (scored on a daily 0–10 scale), and the reported change by the end of the study was a decrease of approximately 7.67 points. The reported data also shows changes in cholesterol markers: total cholesterol started at around 213 mg/dL with a reported change of −21.2 mg/dL, and LDL cholesterol started at around 121.4 mg/dL with a reported change of −16.3 mg/dL. It is important to note that these are the numbers as submitted to the registry, and the data as provided does not always specify which direction certain changes went or include full context for each figure. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 37184523) ↗
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
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 are between 18 and 80 years old (inclusive)
- You have been diagnosed with Primary Sclerosing Cholangitis (PSC), a liver condition affecting the bile ducts
- If you have inflammatory bowel disease (IBD), your symptoms are currently mild to moderate at most, based on your doctor's assessment
- If you take azathioprine for IBD, you have not had an IBD flare-up for at least 6 months
- If you are a woman who could become pregnant, you have a negative pregnancy test at the start of the study
- If you are a sexually active woman who could become pregnant, you are willing to use a highly effective form of birth control throughout the trial
- You can read and understand English well enough to fill in questionnaires and use an electronic diary (eDiary) app
- You are willing and able to use a daily electronic diary for at least 20 weeks
- You agree to accept the software licensing agreement on the eDiary app at the start of the study
- You can complete at least 10 diary entries per week (morning or evening) across two back-to-back weeks during the screening period
- You have access to a phone for scheduled check-in calls from the study team
- You are willing to follow the study procedures and sign a consent form
Who may not be able to join:
- You have a specific form called "small duct PSC," where bile duct changes show up on tests but not on imaging
- You have a significant narrowing (blockage) in your bile ducts that has not been recently checked to confirm it is not cancerous (within the last 6 months)
- You have had a procedure to treat a significant bile duct blockage within the last 6 months
- You have had a serious IBD flare-up within the last 3 months
- Your bile duct condition is caused by something other than PSC (such as gallstones, past surgery, certain infections, or other specific conditions)
- Your liver enzyme levels (AST or ALT) are significantly elevated — 5 or more times above the normal upper limit — at screening
- You have another significant liver disease, as determined by the study doctor
- You have a medical condition unrelated to your liver that can cause certain blood test results (specifically ALP) to be abnormally high (confirm with trial site)
- You have a known diagnosis of HIV
- You are expected to need surgery within 20 weeks of joining the study
- You are pregnant, breastfeeding, or planning to become pregnant within 20 weeks of joining
- You have a history of cancer, except for certain common skin cancers, or if current tests suggest possible cancer
- You have a family history of a hereditary cancer syndrome
- You have had issues with alcohol or substance misuse in the past year
- You have taken part in another clinical trial or used an experimental treatment within the past 30 days (or longer, depending on the drug)
- There are concerns about your ability or willingness to reliably follow the study procedures, as judged by the study team
- You have any other health condition that the study doctor believes could affect your safety or ability to complete the study
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.
GP referral letter
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Trial details
Where this trial is recruiting
Primary endpoints
Number of Participants With Treatment-Emergent Adverse Events (TEAEs); Change From Baseline in Fasting Serum Bile Acid Level at Week 14
Can't join this trial?
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.