Alagille Syndrome clinical trials
The registry lists 31 studies for Alagille Syndrome across more than 10 countries. 11 are recruiting or about to start. 21 of them are mainly about Alagille Syndrome (how this is decided). Per ClinicalTrials.gov, last updated 2026-10-28.
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Status
Who runs them
Mirum Pharmaceuticals, Inc. (10) · Albireo (3) · Arbor Research Collaborative for Health (2) · Ipsen (2) · Stanford University (2) · Takeda (2) · University Hospital, Toulouse (1) · Albireo, an Ipsen Company (1)
What is being tested here
Drugs under test in Alagille Syndrome studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Maralixibat | 1 | 1 | 8 |
| Odevixibat | 0 | 2 | 3 |
| Atorvastatin | 0 | 0 | 1 |
| Cholestyramine Resin | 0 | 0 | 1 |
| Magnesium Sulfate | 0 | 0 | 1 |
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 3, phase 3 first
- Feb 2027Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome · Phase 3 · Albireo, an Ipsen Company
- Aug 2027Determine the Prevalence of Exocrine Pancreatic Insufficiency (EPI) in Pediatric and Adult Participants With Alagille Syndrome After Liver Transplantation · Digestive Care, Inc.
- Oct 2027Dental Dyschromia and Quality of Life in Early Prolonged Hyperbilirubinemia · University Hospital, Toulouse
Who these studies take
Of the 10 Alagille Syndrome studies recruiting now, 7 (70%) set no upper age limit and 3 (30%) stop at 75 or younger. 9 (90%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 0 (0%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 8 (80%) | 8 (80%) |
| 18 | 10 (100%) | 10 (100%) |
| 30 | 8 (80%) | 8 (80%) |
| 40 | 8 (80%) | 8 (80%) |
| 50 | 8 (80%) | 8 (80%) |
| 65 | 7 (70%) | 7 (70%) |
| 75 | 7 (70%) | 7 (70%) |
| 85 | 7 (70%) | 7 (70%) |
Across all 31 studies, recruiting or not: 52% stop at 75 or younger and 52% at 65; 87% take children; 0% are for women only and 3% for men only; 6% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 31 of 31. search them.
- Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at SanfordRecruitingNCT017931681 country
- Determine the Prevalence of Exocrine Pancreatic Insufficiency (EPI) in Pediatric and Adult Participants With Alagille Syndrome After Liver TransplantationRecruitingNCT073355231 country
- A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing TreatmentRecruitingNCT068500381 country
- A Study to Observe the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing TreatmentRecruitingNCT075850972 countries
- Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)RecruitingNCT061939281 country
- Pediatric Evaluation and Registry for Liver Cholestasis in CanadaRecruitingNCT074117161 country
- 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 48 countries
- Dental Dyschromia and Quality of Life in Early Prolonged HyperbilirubinemiaRecruitingNCT065067341 country
- A Database Study of Maralixibat (TAK-625) in Participants With Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC)RecruitingNCT072938971 country
- Spanish Registry of Autoimmune and Cholestatic Liver Diseases (ColHai)RecruitingNCT067676051 country
- Decreasing Hemorrhage Risk in Children With Alagille SyndromeEnrolling by invitationNCT05846854Not applicable1 country
- Long-term Safety and Efficacy of Odevixibat in Patients With Alagille SyndromeActive, not recruitingNCT05035030Phase 310 countries
- Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)SuspendedNCT005712722 countries
- 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 27 countries
- Atorvastatin Therapy on Xanthoma in Alagille SyndromeCompletedNCT05488067Phase 41 country
- A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS)CompletedNCT05543174Phase 31 country
- Diagnostic Accuracy of CAD4TB and C-reactive Protein Assay as Triage Tests for Pulmonary TuberculosisCompletedNCT046663112 countries
- FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE)CompletedNCT029227512 countries
- An Open Label, Single-dose, Single Period ADME Study of A4250 in Healthy SubjectsCompletedNCT03082937Phase 11 country
- A Safety and Pharmakokinetic Study of A4250 Alone or in Combination With A3384CompletedNCT02963077Phase 1
- Efficacy and Safety of Odevixibat in Patients With Alagille SyndromeCompletedNCT04674761Phase 313 countries
- 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 21 country
- A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)Approved for marketingNCT045309941 country
- Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS)CompletedNCT02160782Phase 26 countries
- 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 22 countries
- Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille SyndromeCompletedNCT01903460Phase 21 country
- Evaluation of LUM001 in the Reduction of Pruritus in Alagille SyndromeCompletedNCT02057692Phase 22 countries
- Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver DiseaseCompletedNCT021316232 countries
- Characterization of Pulmonary Artery Stenoses in Alagille Syndrome - a Medical Record ReviewCompletedNCT015156311 country
- Study of Magnesium Sulfate in Children With Reduced Bone Density Secondary to Chronic Cholestatic Liver DiseaseCompletedNCT00007033Not applicable1 country
- Positional Cloning of the Gene(s) Responsible for Alagille SyndromeCompletedNCT000016421 country