Mirum Pharmaceuticals, Inc.: Alagille Syndrome trials

The registry lists 10 Alagille Syndrome studies with Mirum Pharmaceuticals, Inc. as lead sponsor. 2 are open to participants now and 6 have results posted.

Phase 2 6Phase 4 1No phase 3(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• 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)RecruitingNCT07290257Phase 4
• Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)RecruitingNCT06193928
• 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).CompletedNCT04729751Phase 2Results posted
• Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS)CompletedNCT02160782Phase 2Results posted
• 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 SyndromeCompletedNCT02117713Phase 2Results posted
• Evaluation of LUM001 in the Reduction of Pruritus in Alagille SyndromeCompletedNCT02057692Phase 2Results posted
• 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)CompletedNCT02047318Phase 2Results posted
• Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille SyndromeCompletedNCT01903460Phase 2Results posted
• A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)Approved for marketingNCT04530994
• Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver DiseaseCompletedNCT02131623