17 completed, 10 recruiting, 1 active not recruiting, 1 approved for marketing. 6 Phase 2, 3 Phase 3, 2 Not applicable, 2 Phase 1, 2 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 6 | Completed 6 |
| Phase 3 | 3 | Completed 2, Active not recruiting 1 |
| Not applicable | 2 | Completed 1, Unknown 1 |
| Phase 1 | 2 | Completed 2 |
| Phase 4 | 2 | Completed 1, Recruiting 1 |
Follow Alagille Syndrome: new trials by email Literature Review of Alagille Syndrome trials Research Report on Alagille Syndrome
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Determine the Prevalence of Exocrine Pancreatic Insufficiency (EPI) in Pediatric and Adult Participants With Alagille Syndrome After Liver Transplantation Fecal Fat - Exocrine Pancreatic Insuficiency | Recruiting | Digestive Care, Inc. | 30 | 2026-08-03 | 2026-10-13 | |
| A Study to Observe the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment Alagille Syndrome | Recruiting | Ipsen | 30 | 2026-07-10 | 2026-10-23 | |
| Pediatric Evaluation and Registry for Liver Cholestasis in Canada PFIC - Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome (ALGS) | Recruiting | Children's Hospital of Eastern Ontario | 220 | 2026-04-21 | 2026-10-08 | |
| Dental Dyschromia and Quality of Life in Early Prolonged Hyperbilirubinemia Questionnaires - Dental Diseases | Recruiting | University Hospital, Toulouse | 120 | 2026-04-07 | 2026-05-12 | |
| A Database Study of Maralixibat (TAK-625) in Participants With Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) Maralixibat - Alagille Syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC) | Recruiting | Takeda | 50 | 2026-01-13 | 2026-02-20 | |
| 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) Livmarli Oral Product - Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis | Phase 4 | Recruiting | Mirum Pharmaceuticals, Inc. | 230 | 2025-09-25 | 2026-06-23 |
| A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment Alagille Syndrome | Recruiting | Ipsen | 30 | 2025-04-22 | 2026-10-23 | |
| Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US) Livmarli - Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis | Recruiting | Mirum Pharmaceuticals, Inc. | 70 | 2023-09-21 | 2026-10-08 | |
| Decreasing Hemorrhage Risk in Children With Alagille Syndrome Assessment for bleeding disorder with tailored post-operative care - Alagille Syndrome, Pulmonary Artery Stenoses | Not applicable | Unknown | Stanford University | 40 | 2023-04-18 | 2023-05-06 |
| A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS) TAK-625 - Alagille Syndrome (ALGS) | Phase 3 | Completed | Takeda | 7 | 2023-01-16 | 2026-01-29 |
| Atorvastatin Therapy on Xanthoma in Alagille Syndrome atorvastatin - Alagille Syndrome, Atorvastatin | Phase 4 | Completed | Children's Hospital of Fudan University | 15 | 2022-03-22 | 2026-03-27 |
| 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). Maralixibat - Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome | Phase 2 | Completed | Mirum Pharmaceuticals, Inc. | 27 | 2021-09-09 | 2026-06-02 |
| Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome Odevixibat - Alagille Syndrome | Phase 3 | Active not recruiting | Albireo, an Ipsen Company | 62 | 2021-09-03 | 2026-10-23 |
| Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome Odevixibat, Placebo - Alagille Syndrome | Phase 3 | Completed | Albireo | 52 | 2021-03-19 | 2023-11-02 |
| Diagnostic Accuracy of CAD4TB and C-reactive Protein Assay as Triage Tests for Pulmonary Tuberculosis Accuracy of CAD4TB and Afinion CRP assay for pulmonary TB, CAD4COVID+WBC and COVID-19 RDT for SARS-CoV-2 infection - Pulmonary Tuberculosis, Tuberculosis Diagnosis | Completed | Klaus Reither | 1400 | 2021-02-15 | 2025-09-22 | |
| An Open Label, Single-dose, Single Period ADME Study of A4250 in Healthy Subjects 3 mg [14C]-A4250 capsule - Orphan Cholestatic Liver Diseases, Progressive Familial Intrahepatic Cholestasis | Phase 1 | Completed | Albireo | 6 | 2017-01-31 | 2024-03-07 |
| FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE) Liver Stiffness Measurement (LSM) - Biliary Atresia, Alagille Syndrome | Completed | Arbor Research Collaborative for Health | 552 | 2016-11-16 | 2024-08-28 | |
| Spanish Registry of Autoimmune and Cholestatic Liver Diseases (ColHai) Hepatitis Autoimmune Disease, Primary Biliary Cholangitis (PBC) | Recruiting | Asociación Española para el Estudio del Hígado | 5000 | 2016-01-01 | 2025-01-10 | |
| 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 LUM001 (Maralixibat) - Alagille Syndrome | Phase 2 | Completed | Mirum Pharmaceuticals, Inc. | 34 | 2015-03-16 | 2021-07-01 |
| Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome LUM001, Placebo - Alagille Syndrome | Phase 2 | Completed | Mirum Pharmaceuticals, Inc. | 37 | 2014-11-24 | 2019-03-26 |
Showing 20 of 31. Sign in to see all of them.
Cholestasis, progressive familial intrahepatic 1 (10) · Cholestasis (6) · Liver Diseases (5) · Biliary Atresia (4) · Liver Cirrhosis, Biliary (4) · Bile Duct Diseases (2) · Biliary Tract Diseases (2) · Genetic Diseases, Inborn (2) · Pruritus (2) · Rare Diseases (2) · alpha 1-Antitrypsin Deficiency (2) · 3-Methylglutaconic Aciduria, Type V (1) · Achalasia Addisonianism Alacrimia syndrome (1) · Acrodysostosis (1)
Abnormalities, Multiple (31) · Cardiovascular Abnormalities (31) · Cardiovascular Diseases (31) · Congenital Abnormalities (31) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (31) · Heart Defects, Congenital (31) · Cholestasis, Intrahepatic (30) · Digestive System Diseases (30) · Bile Duct Diseases (29) · Biliary Tract Diseases (29) · Genetic Diseases, Inborn (29) · Liver Diseases (26) · Cholestasis (25) · Pathological Conditions, Signs and Symptoms (11) · Pathologic Processes (9)
maralixibat (8) · odevixibat (2) · Atorvastatin (1) · Cholestyramine Resin (1) · Magnesium Sulfate (1) · Moire Topography (1) · Restraint, Physical (1) · Surveys and Questionnaires (1) · gluconic acid (1)
Mirum Pharmaceuticals, Inc. (10) · Albireo (3) · Arbor Research Collaborative for Health (2) · Ipsen (2) · Stanford University (2) · Takeda (2) · Albireo, an Ipsen Company (1) · Asociación Española para el Estudio del Hígado (1) · Children's Hospital of Eastern Ontario (1) · Children's Hospital of Fudan University (1) · Digestive Care, Inc. (1) · Klaus Reither (1) · National Center for Research Resources (NCRR) (1) · National Human Genome Research Institute (NHGRI) (1) · Sanford Health (1)
United States (17) · Canada (7) · France (7) · United Kingdom (7) · Belgium (5) · Italy (4) · Poland (4) · Germany (3) · Netherlands (3) · Spain (3) · Australia (2) · Japan (2) · Malaysia (2) · Turkey (Türkiye) (2) · Brazil (1)
Ed Doo, MD (2) · John Magee, MD (2) · Lisa Henn, PhD (2) · Averell Sherker, MD (1) · Benjamin Shneider, MD (1) · James Heubi (1) · Jeffrey Feinstein, MD, MPH (1) · Kathleen Loomes, MD (1) · Katrina Loh, MD (1) · Klaus Reither, MD, PhD (1) · Noelle Ebel, MD (1)
PMID 42002216, PMID 12183720, PMID 25651489, PMID 34694263, PMID 34755627, PMID 35131835, PMID 36096175, PMID 37620921, PMID 38055647, PMID 38670135, PMID 39190813
U01DK062436 (2), U01DK062445 (2), U01DK062452 (2), U01DK062453 (2), U01DK062456 (2), U01DK062466 (2), U01DK062470 (2), U01DK062481 (2), U01DK062497 (2), U01DK062500 (2), U01DK062503 (2), U01DK084536 (2), U01DK084538 (2), U01DK084575 (2), U01DK103135 (2)