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Clinical trials
catalyst calendar across sponsors →Studies where MIRM is the lead sponsor, as filed with ClinicalTrials.gov. Completion dates are the sponsor's own projected windows and are revised as a study runs. Active studies first, then completed and stopped — newest readout first within each.
34 interventional · 3 observational · 1 expanded access · 20 with posted results
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| Study | Phase | Status | Interventions | Conditions | Enrollment | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|---|
| The REPLACE Registry for Cholbam® (Cholic Acid)NCT03115086observationalNumber of participants with worsening cholestasis | — | Active, not recruiting | Drug: Cholbam | Bile Acid Synthesis Disorders | 55 | Jul 2038 | ≤ 4,362 d | 2026-07-31 |
| Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the European Union (LEAP-EU)NCT07290257Adverse Events (AEs)Open-label · Other | Phase 4 | Recruiting | Drug: Livmarli Oral Product | Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis | 230 | 2030-09-22 | in 1,493 d | 2026-06-23 |
| A Trial Evaluating BJT-778 vs Delayed Treatment for the Treatment of Chronic Hepatitis Delta InfectionNCT06907290Percentage of participants with a composite endpointRandomized · Open-label · Treatment | Phase 2/3 | Active, not recruiting | Drug: Brelovitug 300 mg, Brelovitug 900 mg, Delayed Treatment with Brelovitug 300mg | Chronic Hepatitis D Infection | 150 | May 2029 | ≤ 1,014 d | 2026-03-02 |
| Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)NCT06193928observationalIncidence of Long-Term Clinical Outcomes | — | Recruiting | Drug: Livmarli, Livmarli | Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis | 70 | 2028-09-20 | in 761 d | 2025-05-25 |
| Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)NCT07209462Incidence and severity of treatment-emergent adverse events and Withdrawal due to adverse events and withdrawal due to adverse eventsRandomized · Double-masked · Treatment | Phase 2 | Recruiting | Drug: Low dose of MRM-3379, Middle Dose of MRM-3379, High dose of MRM-3379, Placebo, Low dose of MRM-3379 Open-Label | Fragile X Syndrome | 60 | Oct 2027 | ≤ 436 d | 2026-08-07 |
| Study to Evaluate Switching to Brelovitug for the Treatment of CHD in Participants Receiving BulevirtideNCT07454837Proportion of participants with undetectable HDV RNA (<LLOQ Target not detected [TND])Randomized · Open-label · Treatment | Phase 2/3 | Recruiting | Drug: Brelovitug (BJT-778), Bulevirtide | Chronic Hepatitis D | 120 | 2027-08-31 | in 375 d | 2026-06-29 |
| A Trial Evaluating Brelovitug (BJT-778) vs Bulevirtide for the Treatment of Chronic Hepatitis Delta Infection (AZURE-2)NCT07200908Percentage of participants with a composite endpoint of virologic response and ALT normalizationRandomized · Open-label · Treatment | Phase 3 | Recruiting | Drug: Brelovitug 300 mg, Bulevirtide 2 mg and Brelovitug - 300 mg | Chronic Hepatitis D Infection | 172 | 2027-06-30 | in 313 d | 2026-06-15 |
| A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary CholangitisNCT05050136Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaireRandomized · Double-masked · Treatment | Phase 2 | Recruiting | Drug: Volixibat, Placebo | Primary Biliary Cholangitis, PBC | 260 | Apr 2027 | ≤ 252 d | 2026-05-14 |
| Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND)NCT06553768Mean change in the ItchRO(Obs) severity scoreRandomized · Quadruple-masked · Treatment | Phase 3 | Active, not recruiting | Drug: Maralixibat · Other: Placebo | Cholestatic Liver Disease (Except ALGS, PFIC, PBC and PSC) | 90 | Oct 2026 | ≤ 71 d | 2026-08-11 |
| A Trial Evaluating Brelovitug vs Delayed Treatment for the Treatment of Chronic Hepatitis Delta Infection (AZURE-4)NCT07298330Percentage of participants with a composite endpoint of virologic response and ALT normalization at Week 24 in brelovitug arms compared to response at Week 12 of delayed-treatment armRandomized · Open-label · Treatment | Phase 3 | Active, not recruiting | Drug: Brelovitug 300 mg, Brelovitug 900 mg, Delayed Treatment with Brelovitug 300mg | Chronic Hepatitis D Infection | 80 | Aug 2026 | ≤ 10 d | 2026-06-29 |
| A Study to Evaluate the Absorption, Distribution, Metabolism, and Excretion of [14C]-MRM-3379NCT07712718Total radioactivity recovery of MRM-3379 in urine and fecesOpen-label · Basic science | Phase 1 | Recruiting | Drug: [14C]-MRM-3379 | Healthy Volunteers | 8 | Jul 2026 | — | 2026-07-17 |
| A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)NCT04663308Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaireRandomized · Double-masked · Treatment | Phase 2 | Active, not recruiting | Drug: Volixibat, Placebo | Primary Sclerosing Cholangitis | 182 | Jun 2026 | — | 2026-02-04 |
| An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)NCT04185363results2026-06-23Mean Change From Baseline Over Time in the Average Morning ItchRO(Obs) Severity ScoreOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Maralixibat | Progressive Familial Intrahepatic Cholestasis (PFIC) | 84 | 2025-04-23actual | — | 2026-06-23 |
| 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).NCT04729751results2026-06-02Frequency of Treatment-emergent Adverse Events [TEAEs]Open-label · TreatmentStudy Protocol and Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Maralixibat | Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome, Cholestatic Liver Disease | 27 | 2024-12-17actual | — | 2026-06-02 |
| MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat StudyNCT04168385results2025-12-08Incidence of Treatment-Emergent Adverse EventsOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Maralixibat | Cholestatic Liver Disease | 52 | 2024-09-04actual | — | 2025-12-08 |
| Evaluation of Maralixibat in Biliary Atresia Response Post-KasaiNCT04524390results2025-02-17Mean Change in Total Serum Bilirubin LevelsRandomized · Triple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Maralixibat · Other: Placebo | Biliary Atresia | 75 | 2023-11-07actual | — | 2025-03-19 |
| Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)NCT04270682results2024-10-28Change From Baseline in Urine 23S-Pentol During the Two Double-Blind PeriodsRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Blinded CDCA 250 mg TID, Placebo, Open-Label CDCA 250 mg TID, Rescue Medication CDCA 250 mg TID, CDCA Weight-Based Dose TID | CTX | 19 | 2023-07-13actual | — | 2024-10-28 |
| A Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (OHANA)NCT04718961results2024-08-06Assess the Safety and Tolerability of Volixibat in Participants With ICPRandomized · Double-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Terminatedsponsor's stated reason: Due to company's assessment of enrollment feasibility. | Drug: Volixibat, Placebo | Intrahepatic Cholestasis of Pregnancy | 4 | 2022-12-07actual | — | 2024-08-06 |
| A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)NCT03905330results2023-12-11Mean Change in the Average Morning ItchRO(Obs) Severity Score in the Primary CohortRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Maralixibat · Other: Placebo | Progressive Familial Intrahepatic Cholestasis (PFIC) | 93 | 2022-09-01actual | — | 2023-12-11 |
| An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS)NCT02047318results2021-11-19Change From MRX Baseline to Week 48 in Fasting sBA LevelsOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: LUM001 (Maralixibat) | Alagille Syndrome | 19 | 2020-06-17actual | — | 2021-11-19 |
| A Placebo-controlled Study of Maralixibat (SHP625) in Pediatric Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)NCT03353454Treatment Response as Measured by the Observer Itch Reported Outcome (ItchRO[Obs])Randomized · Double-masked · Treatment | Phase 3 | Withdrawnsponsor's stated reason: The study was withdrawn due to change of ownership of the study drug maralixibat. Future studies of maralixibat will be posted by Mirum Pharmaceuticals. | Drug: Maralixibat, Placebo | Progressive Familial Intrahepatic Cholestasis (PFIC) | 0 | 2020-06-15 | — | 2019-03-18 |
| 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 SyndromeNCT02117713results2021-06-22Change From MRX Baseline to Week 48 in Fasting Serum Bile Acid (sBA)Double-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: LUM001 (Maralixibat) | Alagille Syndrome | 34 | 2020-06-01actual | — | 2021-07-01 |
| Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS)NCT02160782results2021-07-14Change From Week 18 to Week 22 in Fasting sBA Levels in Participants Who Had a Reduction in sBA ≥50% From Baseline to Week 12 or Week 18Randomized · Triple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: LUM001 (Maralixibat), Placebo | Alagille Syndrome | 31 | 2020-05-28actual | — | 2021-07-14 |
| 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 CholestasisNCT02057718results2021-07-12Change From Baseline to Endpoint (Week 13) in Fasting sBA LevelOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: LUM001 (Maralixibat) | Progressive Familial Intrahepatic Cholestasis (PFIC) | 33 | 2020-05-08actual | — | 2023-10-23 |
| Volixibat (SHP626) in the Treatment of Adults With Nonalcoholic Steatohepatitis (NASH)NCT02787304results2019-11-25Number of Subjects Achieving Binary Response on Liver Histology Between Volixibat (SHP626) and Placebo at Week 48Randomized · Double-masked · TreatmentStatistical Analysis Plan ↗ · Study Protocol ↗ | Phase 2 | Terminated | Drug: SHP626, Placebo | Non-Alcoholic Steatohepatitis | 197 | 2018-07-27actual | — | 2019-11-25 |
| An Open-label Study to Evaluate the Long-term Safety and Tolerability of LUM001 in Patients With Primary Biliary CirrhosisNCT02321306Number of Participants with Adverse Events as a Measure of SafetyOpen-label · Treatment | Phase 2 | Withdrawnsponsor's stated reason: The study stopped early, before enrolling its first participant. | Drug: LUM001 | Primary Biliary Cirrhosis | 0 | Dec 2017 | — | 2019-03-19 |
| Evaluation of LUM001 in the Reduction of Pruritus in Alagille SyndromeNCT02057692results2018-01-23Change From Baseline to Endpoint (Week 13/Early Termination) in PruritusRandomized · Triple-masked · Treatment | Phase 2 | Completed | Drug: LUM001, Placebo | Alagille Syndrome | 37 | 2016-11-16actual | — | 2019-03-26 |
| Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid SynthesisNCT01438411results2020-08-21Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)Open-label · Treatment | Phase 3 | Completed | Drug: Cholic Acid | Bile Acid Synthesis Defect | 53 | Jul 2016actual | — | 2023-10-03 |
| Open Label Study to Evaluate Safety and Efficacy of LUM001 in Patients With Primary Sclerosing CholangitisNCT02061540results2017-04-12Number of Participants With Treatment-Emergent Adverse Events (TEAEs)Open-label · Treatment | Phase 2 | Completed | Drug: LUM001 | Primary Sclerosing Cholangitis (PSC) | 27 | Feb 2016actual | — | 2019-03-29 |
| 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 ExcretionNCT02475317Change From Baseline of Fecal Bile Acid Excretion After Dosing (Day 6 and Day 7)Randomized · Double-masked · Other | Phase 1 | Completed | Drug: Placebo, Maralixibat, Volixibat | Healthy Volunteers | 84 | 2015-12-01actual | — | 2019-04-04 |
| A Study to Investigate How the Study Drug SHP626 is Eliminated From the Body After One DoseNCT02571192Pharmacokinetic parameters will be determined from the plasma and blood concentration time data of total radioactivity and from the plasma concentration-time data for SHP626 by non-compartmental analysis.Open-label · Basic science | Phase 1 | Completed | Drug: SHP626 · Radiation: 5.95 μCi RAD | Non-Alcoholic Steatohepatitis | 8 | 2015-10-01actual | — | 2019-05-01 |
| Safety and Tolerability Study of SHP626 in Overweight and Obese AdultsNCT02287779results2016-12-07Number of Participants With Treatment-emergent Adverse Events (TEAEs) Related to Standard HematologyRandomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: SHP626, Placebo | Non-Alcoholic Steatohepatitis | 84 | 2015-06-19actual | — | 2019-03-26 |
| Phase 2 Study to Evaluate LUM001 in Combination With Ursodeoxycholic Acid in Patients With Primary Biliary CirrhosisNCT01904058results2016-07-06Change From Baseline in Pruritus Using Adult Itch Reported Outcome (ItchRO) Weekly Sum Score at Week 13/ Early Termination (ET)Randomized · Double-masked · Treatment | Phase 2 | Completed | Drug: LUM001, Placebo, Ursodeoxycholic Acid | PBC, Primary Biliary Cirrhosis | 66 | Apr 2015actual | — | 2019-03-27 |
| Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver DiseaseNCT02131623observationalValidation of ItchRO measure | — | Completed | — | Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis | 23 | Mar 2015actual | — | 2019-03-18 |
| Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille SyndromeNCT01903460results2015-12-07Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid LevelRandomized · Triple-masked · Treatment | Phase 2 | Completed | Drug: LUM001, Placebo | Alagille Syndrome | 20 | Feb 2015actual | — | 2019-03-28 |
| Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid SynthesisNCT01115582results2016-12-09Serum TransaminasesOpen-label · Treatment | Phase 3 | Completed | Drug: Cholic acid | Inborn Errors of Bile Acid Synthesis | 16 | Aug 2010actual | — | 2023-10-03 |
| Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic AcidNCT00007020results2020-07-15Number of Participants With Excretion of Atypical Bile Acids in Urine by CategoryOpen-label · Treatment | Phase 3 | Completed | Drug: Cholic Acids | Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders, Cholestasis | 85 | Dec 2009actual | — | 2023-10-03 |
| A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)NCT04530994expanded access | — | Approved for marketing | Drug: maralixibat | Alagille Syndrome | — | — | — | 2021-10-20 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
Source: ClinicalTrials.gov, retrieved 2026-08-19