Mirum Pharmaceuticals

28 total trials 5 currently recruiting 18 completed

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

RECRUITING Phase 3

A Trial Evaluating Brelovitug vs Delayed Treatment for the Treatment of Chronic Hepatitis Delta Infection (AZURE-4)

NCT07298330

RECRUITING Phase 2

Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)

NCT07209462

RECRUITING Phase 3

Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND)

NCT06553768

RECRUITING

Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)

NCT06193928

RECRUITING Phase 2

A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis

NCT05050136

ACTIVE NOT RECRUITING Phase 2

A Trial Evaluating BJT-778 vs Delayed Treatment for the Treatment of Chronic Hepatitis Delta Infection

NCT06907290

ACTIVE NOT RECRUITING Phase 2

A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)

NCT04663308

ACTIVE NOT RECRUITING

The REPLACE Registry for Cholbam® (Cholic Acid)

NCT03115086

COMPLETED Phase 2

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

COMPLETED Phase 2

Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai

NCT04524390

TERMINATED Phase 2

A Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (OHANA)

NCT04718961

COMPLETED Phase 3

Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)

NCT04270682

COMPLETED Phase 2

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

COMPLETED Phase 3

An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)

NCT04185363

COMPLETED Phase 3

A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)

NCT03905330

TERMINATED Phase 2

Volixibat (SHP626) in the Treatment of Adults With Nonalcoholic Steatohepatitis (NASH)

NCT02787304

COMPLETED Phase 1

A Study to Investigate How the Study Drug SHP626 is Eliminated From the Body After One Dose

NCT02571192

COMPLETED Phase 1

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

COMPLETED Phase 2

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

COMPLETED Phase 1

Safety and Tolerability Study of SHP626 in Overweight and Obese Adults

NCT02287779

COMPLETED Phase 2

Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome

NCT02057692

COMPLETED

Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease

NCT02131623

COMPLETED Phase 2

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

COMPLETED Phase 2

Open Label Study to Evaluate Safety and Efficacy of LUM001 in Patients With Primary Sclerosing Cholangitis

NCT02061540

COMPLETED Phase 2

Phase 2 Study to Evaluate LUM001 in Combination With Ursodeoxycholic Acid in Patients With Primary Biliary Cirrhosis

NCT01904058

COMPLETED Phase 3

Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis

NCT01115582

COMPLETED Phase 3

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

NCT01438411

COMPLETED Phase 3

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

NCT00007020

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.

Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

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.