Cholestasis, progressive familial intrahepatic 1 clinical trials
The registry lists 33 studies for Cholestasis, progressive familial intrahepatic 1 across more than 10 countries. 13 are recruiting or about to start. 24 of them are mainly about Cholestasis, progressive familial intrahepatic 1 (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-13.
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Who runs them
Mirum Pharmaceuticals, Inc. (8) · Ipsen (4) · Albireo (3) · Takeda (2) · IRCCS Azienda Ospedaliero-Universitaria di Bologna (2) · University Medical Center Groningen (2) · University of Pittsburgh (2) · Centre Hospitalier Universitaire de Liege (1)
Who develops what
Every programme with a company-led study here in the last two years, whatever its size: 1 programme from 1 company, 1 with this as their studies’ main aim; 0 with a live phase 3 study; 0 primary completions in the next 12 months.
Comparator & background therapies
What other companies’ phase 2+ studies give as the comparator or the background here (its leading trial sponsor and partners left out): a sign of what new drugs are measured against, not by itself the standard of care, its prescriptions or its market.
None given by two companies or more yet.
Programmes
“Meets, by arms” reads each study’s arms: vs its arm against the therapy’s; + X vs a combination against it; added to it plus the therapy against the therapy; studied with given together only. The next primary completion is the next phase-3 study’s when there is one.
The 1 programme — sign in to see the whole list, free: filter by phase and aim, sort by late development, direct comparisons or the next primary completion.
Primary completion in the next 12 months (0)
Live phase 2 and 3 studies of these programmes. Primary completion is when the main measure’s data collection ends, as the registry states it (most are estimates); results come later, if they are announced.
None in the next 12 months.
Read from ClinicalTrials.gov, industry-led studies. Main aim: this is the study’s judged main condition; named, not the main aim: the sponsor names it, the study is about something else; registry mapping only: only the registry’s mapping put it here. Leading trial sponsor is the company leading the most industry studies of a drug, a measure of trial activity, not of rights. Phase 4 trial recorded: a phase 4 study exists somewhere; approval here is not checked. Not read yet: approvals and labels, exclusivity, licences, line of therapy, academic comparators, sales.
What is being tested here
Drugs under test in Cholestasis, progressive familial intrahepatic 1 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 | 4 | 7 |
| Odevixibat | 0 | 1 | 2 |
| Cholestyramine Resin | 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
- Nov 2026Odevixibat Outcomes in Patients With PFIC Versus an External Control Cohort (OvEC-PFIC) · Ipsen
- Oct 2027Familial Intrahepatic Cholestasis-related Genes Associated with Disease Susceptibility in Hepato-biliary Cancers · IRCCS Azienda Ospedaliero-Universitaria di Bologna
- Oct 2027Dental Dyschromia and Quality of Life in Early Prolonged Hyperbilirubinemia · University Hospital, Toulouse
Who these studies take
Of the 12 Cholestasis, progressive familial intrahepatic 1 studies recruiting now, 8 (67%) set no upper age limit and 4 (33%) stop at 75 or younger. 10 (83%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (8%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 9 (75%) | 9 (75%) |
| 18 | 12 (100%) | 12 (100%) |
| 30 | 9 (75%) | 9 (75%) |
| 40 | 9 (75%) | 9 (75%) |
| 50 | 9 (75%) | 9 (75%) |
| 65 | 9 (75%) | 9 (75%) |
| 75 | 8 (67%) | 8 (67%) |
| 85 | 8 (67%) | 8 (67%) |
Across all 33 studies, recruiting or not: 52% stop at 75 or younger and 52% at 65; 82% take children; 0% are for women only and 3% for men only; 15% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 33 of 33. search them.
- Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)RecruitingNCT061939281 country
- A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in ChinaRecruitingNCT075888801 country
- A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in South KoreaRecruitingNCT071859191 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
- DEFINING THE GENETIC DRIVERS OF ADULT-ONSET CHOLESTATIC LIVER DISEASERecruitingNCT07317193Not applicable1 country
- A Database Study of Maralixibat (TAK-625) in Participants With Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC)RecruitingNCT072938971 country
- Prospective Analysis of the Treatment of Progressive Familial Intrahepatic Cholestasis (TreatFIC)RecruitingNCT067781741 country
- NAtural Course and Prognosis of PFIC and Effect of Biliary DiversionEnrolling by invitationNCT039308101 country
- Genotype-phenotype Relationship Between Cryptogenic Cholestasis and Familial Intrahepatic CholestasisRecruitingNCT067812421 country
- Familial Intrahepatic Cholestasis-related Genes Associated with Disease Susceptibility in Hepato-biliary CancersRecruitingNCT067779141 country
- Progressive Familial Intrahepatic Cholestasis in Indian Children - Establishing an Indian PFIC RegistryRecruitingNCT057045171 country
- Odevixibat Outcomes in Patients With PFIC Versus an External Control Cohort (OvEC-PFIC)Active, not recruitingNCT074977241 country
- Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFICCompletedNCT03659916Phase 315 countries
- A Study to Assess the Genetic Variations in Bile Flow Disorders: Linking Progressive Familial Intrahepatic Cholestasis (PFIC)-Related Genes to Symptoms in Adults With Recurrent Cholestasis in SpainCompletedNCT071917041 country
- Odevixibat for the Treatment of Progressive Familial Intrahepatic CholestasisNo longer availableNCT044835311 country
- An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)CompletedNCT04185363Phase 316 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
- A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC)CompletedNCT05543187Phase 31 country
- Baby Detect : Genomic Newborn ScreeningCompletedNCT056874741 country
- 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
- A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)CompletedNCT03905330Phase 317 countries
- 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 CholestasisCompletedNCT02057718Phase 24 countries
- Sulfation of Bile Acids as a Biomarker for Hepatobiliary DiseasesTerminatedNCT012000821 country
- Gastrostomy-Biliary Diversion: Innovative Management for Bile Canalicular Transport DisordersStatus unknownNCT04071197Not applicable1 country
- Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver DiseaseCompletedNCT021316232 countries
- A Placebo-controlled Study of Maralixibat (SHP625) in Pediatric Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)WithdrawnNCT03353454Phase 3
- Expanded Access Protocol for an Intermediate Size Population - RAVICTI for Byler DiseaseNo longer availableNCT02094222
- Transition From Buphenyl to RAVICTI for the Therapy of Byler DiseaseNo longer availableNCT01949766
- Buphenyl Therapy for Byler's DiseaseNo longer availableNCT01784718