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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Pediatric Evaluation and Registry for Liver Cholestasis in CanadaAbnormalities, Multiple, Alagille syndrome, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, bile duct disorder, biliary tract disorder, cardiovascular disorder, cholestasis, congenital heart disease, hereditary disease, intrahepatic cholestasis, liver disorder, progressive familial intrahepatic cholestasis type 1RecruitingNot stated2026-10-08NCT07411716
A Trial Evaluating Brelovitug (BJT-778) vs Bulevirtide for the Treatment of Chronic Hepatitis Delta Infection (AZURE-2)Digestive System Diseases, Infections, RNA Virus Infections, hepatitis, hepatitis D virus infection, liver disorder, viral hepatitis, viral infectious diseaseRecruitingPhase 32026-10-08NCT07200908
Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)Abnormalities, Multiple, Alagille syndrome, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, bile duct disorder, biliary tract disorder, cardiovascular disorder, cholestasis, congenital heart disease, hereditary disease, intrahepatic cholestasis, liver disorder, progressive familial intrahepatic cholestasis type 1RecruitingNot stated2026-10-08NCT06193928
Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND)Cholangitis, Sclerosing, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Abnormalities, Digestive System Diseases, Pathological Conditions, Signs and Symptoms, Pruritus, Signs and Symptoms, Skin Manifestations, Skin and Connective Tissue Diseases, bile duct disorder, biliary atresia, biliary tract disorder, cholangitis, skin disorderActive, not recruitingPhase 32026-10-06NCT06553768
Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Sex Chromosome Disorders, X-linked disease, chromosomal disorder, fragile X syndrome, hereditary disease, inherited neurodegenerative disorder, intellectual disability, nervous system disorderRecruitingPhase 22026-09-29NCT07209462
MRX-901: An Open-label Study to Assess Safety and Tolerability of Maralixibat in Participants With Intrahepatic Cholestasis of Pregnancy (ICP)intrahepatic cholestasis of pregnancyNot yet recruitingPhase 22026-09-16NCT07823127
Study to Evaluate Switching to Brelovitug for the Treatment of CHD in Participants Receiving BulevirtideChronic Disease, Digestive System Diseases, Disease Attributes, Hepatitis D, Chronic, Hepatitis, Chronic, Infections, Pathologic Processes, Pathological Conditions, Signs and Symptoms, RNA Virus Infections, hepatitis, hepatitis D virus infection, liver disorder, viral hepatitis, viral infectious diseaseRecruitingPhase 2 / Phase 32026-09-10NCT07454837
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)Cholangitis, Sclerosing, Digestive System Diseases, Pathological Conditions, Signs and Symptoms, Pruritus, Signs and Symptoms, Skin Manifestations, Skin and Connective Tissue Diseases, bile duct disorder, biliary tract disorder, cholangitis, cholestasis, primary sclerosing cholangitis, skin disorderActive, not recruitingPhase 22026-08-26NCT04663308
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary CholangitisDigestive System Diseases, Fibrosis, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Pruritus, Signs and Symptoms, Skin Manifestations, Skin and Connective Tissue Diseases, bile duct disorder, biliary tract disorder, cholangitis, cholestasis, cirrhosis of liver, intrahepatic cholestasis, liver disorder, primary biliary cholangitis, skin disorderActive, not recruitingPhase 22026-08-25NCT05050136
The REPLACE Registry for Cholbam® (Cholic Acid)Abnormalities, Multiple, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Peroxisomal Disorders, Urogenital Diseases, Zellweger spectrum disorders, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of bile acid synthesis, inborn errors of metabolism, kidney disorder, liver disorder, metabolic disease, nervous system disorder, urinary system disorderActive, not recruitingNot stated2026-07-31NCT03115086
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening ProgramBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Ceroid Lipofuscinosis, Neuronal, 2, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Fabry disease, GM1 gangliosidosis, Gangliosidoses, Gaucher disease, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Infant, Newborn, Diseases, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Mucopolysaccharidosis III, Neurobehavioral Manifestations, Neurologic Manifestations, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, Skin and Connective Tissue Diseases, Sulfatidosis, Wolman disease, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrotendinous xanthomatosis, cerebrovascular disorder, cholesteryl ester storage disease, connective tissue disorder, cutaneous mucinosis, demyelinating disease, hereditary disease, histiocytosis, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mucopolysaccharidosis, mucopolysaccharidosis type 2, mucopolysaccharidosis type 4, mucopolysaccharidosis type 6, mucopolysaccharidosis type 7, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosis, vascular disorder, xanthomatosisEnrolling by invitationNot stated2026-07-28NCT05368038
A Trial Evaluating Brelovitug vs Delayed Treatment for the Treatment of Chronic Hepatitis Delta Infection (AZURE-4)Digestive System Diseases, Infections, RNA Virus Infections, hepatitis, hepatitis D virus infection, liver disorder, viral hepatitis, viral infectious diseaseActive, not recruitingPhase 32026-06-29NCT07298330
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)Abnormalities, Multiple, Alagille syndrome, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathological Conditions, Signs and Symptoms, Pruritus, Signs and Symptoms, Skin Manifestations, Skin and Connective Tissue Diseases, bile duct disorder, biliary tract disorder, cardiovascular disorder, cholestasis, congenital heart disease, hereditary disease, intrahepatic cholestasis, liver disorder, progressive familial intrahepatic cholestasis type 1, skin disorderRecruitingPhase 42026-06-23NCT07290257
A Trial Evaluating BJT-778 vs Delayed Treatment for the Treatment of Chronic Hepatitis Delta InfectionDigestive System Diseases, Infections, RNA Virus Infections, hepatitis, hepatitis D virus infection, liver disorder, viral hepatitis, viral infectious diseaseActive, not recruitingPhase 2 / Phase 32026-03-02NCT06907290
Maralixibat for Intrahepatic Cholestasis of PregnancyDigestive System Diseases, Pathological Conditions, Signs and Symptoms, Pruritus, Signs and Symptoms, Skin Manifestations, Skin and Connective Tissue Diseases, bile duct disorder, biliary tract disorder, cholestasis, intrahepatic cholestasis of pregnancy, skin disorderNot yet recruitingPhase 22026-02-05NCT07389031