Mirum Pharmaceuticals
Mirum Pharmaceuticals sponsors 28 registered US clinical trials on ClinicalTrials.gov, 5 of them currently recruiting, and 18 completed. 8 of these trials are in Phase 3-4 (later-stage) and 17 in Phase 1-2 (earlier-stage). The most-studied condition in this pipeline is Progressive Familial Intrahepatic Cholestasis (PFIC), with 2 trials.
Trial Pipeline
28 total, page 1 of 1
A Trial Evaluating Brelovitug vs Delayed Treatment for the Treatment of Chronic Hepatitis Delta Infection (AZURE-4)
NCT07298330
Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)
NCT07209462
Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND)
NCT06553768
Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
NCT06193928
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis
NCT05050136
A Trial Evaluating BJT-778 vs Delayed Treatment for the Treatment of Chronic Hepatitis Delta Infection
NCT06907290
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)
NCT04663308
The REPLACE Registry for Cholbam® (Cholic Acid)
NCT03115086
A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).
NCT04729751
Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai
NCT04524390
A Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (OHANA)
NCT04718961
Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)
NCT04270682
MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study
NCT04168385
An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)
NCT04185363
A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)
NCT03905330
Volixibat (SHP626) in the Treatment of Adults With Nonalcoholic Steatohepatitis (NASH)
NCT02787304
A Study to Investigate How the Study Drug SHP626 is Eliminated From the Body After One Dose
NCT02571192
Study to Assess the Relative Potency of Multiple Oral Doses of LUM001 and SHP626 in Overweight and Obese Adults as Assessed by Fecal Bile Acid Excretion
NCT02475317
An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome
NCT02117713
Safety and Tolerability Study of SHP626 in Overweight and Obese Adults
NCT02287779
Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome
NCT02057692
Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease
NCT02131623
Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 (Maralixibat) in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic Cholestasis
NCT02057718
Open Label Study to Evaluate Safety and Efficacy of LUM001 in Patients With Primary Sclerosing Cholangitis
NCT02061540
Phase 2 Study to Evaluate LUM001 in Combination With Ursodeoxycholic Acid in Patients With Primary Biliary Cirrhosis
NCT01904058
Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis
NCT01115582
Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis
NCT01438411
Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
NCT00007020
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 3 |
| Phase 2 | 14 |
| Phase 3 | 8 |
What the Pipeline for Mirum Pharmaceuticals Shows
According to the ClinicalTrials.gov registry, Mirum Pharmaceuticals is linked to 28 US clinical trials across every stage of research activity. Of those, 5 studies are currently recruiting, about 18% of the sponsor's indexed portfolio, and 18 are already marked complete, representing roughly 64% of the total.
The phase mix for Mirum Pharmaceuticals reports 8 late-stage studies (Phase 3 and Phase 4 combined) and 17 earlier-phase studies (Phase 1 and Phase 2).
The top therapeutic focus area indexed for Mirum Pharmaceuticals is Progressive Familial Intrahepatic Cholestasis (PFIC) with 2 linked trials, and 9 other condition areas appear in the top list above.
How to read these pipeline numbers
Recruiting share is one of the more practical signals here: it reflects how much of a sponsor's research is presently open to new participants, while the completed share indicates the depth of finished work that has already contributed registry results. Both counts come directly from the public ClinicalTrials.gov dataset and are refreshed on the registry side; this page mirrors the latest data pull without altering it.
A portfolio weighted toward Phase 3 usually reflects an organization advancing candidates toward regulatory review, where the research centers on comparative efficacy and broader safety across larger populations. A heavier Phase 1 and Phase 2 tilt generally indicates exploratory work, safety, dosing, and early signal detection, and is common among research-forward sponsors that seed many early programs. Phase 4 entries, when present, track interventions already in real-world use and typically focus on long-term safety, effectiveness across subgroups, or formulation comparisons.
That distribution is a quick read of where the organization concentrates its research attention; it does not imply product availability, market share, or any clinical endorsement. All numbers on this page come from ClinicalTrials.gov maintained by the National Library of Medicine, and counts can shift as new studies are registered or existing ones update their status. This information is provided for reference and educational purposes only, not as medical, investment, or regulatory advice, verify current details directly with ClinicalTrials.gov before relying on any figure here.
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial from this sponsor, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.