Disease · MONDO_0015762
progressive familial intrahepatic cholestasis
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. progressive familial intrahepatic cholestasis. Diseases, record MONDO_0015762. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0015762
On this page
Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
- Highest stage of its drugs
- Phase 3, for 2 of its 3 drugs, from ClinicalTrials.gov and TTD
- Open Targets
Drugs3
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| MARALIXIBAT | CHEMBL363392 | Small molecule | Phase 3 | ClinicalTrials.gov and TTD | 10 | 9 | 9 | Drugs and targets |
| ODEVIXIBAT | CHEMBL4297588 | Small molecule | Phase 3 | ClinicalTrials.gov | 3 | 3 | 3 | Drugs and targets |
| MARALIXIBAT CHLORIDE | CHEMBL17879 | Small molecule | Phase 2 | TTD | 1 | 0 | 0 | Drugs and targets |
Targets1
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes |
|---|---|---|---|---|---|
| SLC10A2 | Ileal sodium/bile acid cotransporter | 3 | Ileal bile acid transporter inhibitor | Phase 3 | https://conductscience.org/library/genes/6555 |
Source Drugs and targets
Trials12
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT03659916 | Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC | Phase 3 | 116 | 2025-07-10 | Progressive Familial Intrahepatic Cholestasis | ODEVIXIBAT | Its record names the disease | ClinicalTrials.gov |
| NCT03905330 | A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC) | Phase 3 | 93 | 2023-12-11 | Progressive Familial Intrahepatic Cholestasis (PFIC) | MARALIXIBAT | Its record names the disease | ClinicalTrials.gov |
| NCT03566238 | This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC Types 1 or 2 | Phase 3 | 62 | 2021-09-05 | PFIC1, PFIC2 | ODEVIXIBAT | Its record names the disease | ClinicalTrials.gov |
| NCT04168385 | MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study | Phase 2 | 52 | 2025-12-08 | Cholestatic Liver Disease | MARALIXIBAT | Its record names the disease | ClinicalTrials.gov |
| NCT02057718 | 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 Cholestasis | Phase 2 | 33 | 2021-07-12 | Progressive Familial Intrahepatic Cholestasis (PFIC) | MARALIXIBAT | Its record names the disease | ClinicalTrials.gov |
| NCT05543187 | A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) | Phase 3 | 5 | 2024-09-24 | Progressive Familial Intrahepatic Cholestasis (PFIC) | MARALIXIBAT | Its record names the disease | ClinicalTrials.gov |
| NCT04185363 | An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC) | Phase 3 | 84 | Progressive Familial Intrahepatic Cholestasis (PFIC) | MARALIXIBAT | Its record names the disease | ClinicalTrials.gov | |
| NCT06193928 | Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US) | 70 | Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis | MARALIXIBAT | Its record names the disease | ClinicalTrials.gov | ||
| NCT07293897 | A Database Study of Maralixibat (TAK-625) in Participants With Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) | 50 | Alagille Syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC) | MARALIXIBAT | Its record names the disease | ClinicalTrials.gov | ||
| NCT04729751 | 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). | Phase 2 | 27 | Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome, Cholestatic Liver Disease | MARALIXIBAT | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 12
See all 12 trialsComparisons in its trials6
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial | Source |
|---|---|---|---|---|---|
| Maralixibat vs Placebo: deaths from any cause | 0 of 47 | 0 of 46 | 93 | NCT03905330 | Trial comparisons |
| Maralixibat vs Placebo: serious adverse events | 5 of 47 | 3 of 46 | 93 | NCT03905330 | Trial comparisons |
| Maralixibat: ALGS vs Maralixibat: PFIC: deaths from any cause | 1 of 40 | 0 of 12 | 52 | NCT04168385 | Trial comparisons |
| Maralixibat: ALGS vs Maralixibat: PFIC: serious adverse events | 10 of 40 | 3 of 12 | 52 | NCT04168385 | Trial comparisons |
| Primary Cohort: TAK-625 vs Supplemental Cohort: TAK-625: deaths from any cause | 0 of 3 | 0 of 2 | 5 | NCT05543187 | Trial comparisons |
| Primary Cohort: TAK-625 vs Supplemental Cohort: TAK-625: serious adverse events | 0 of 3 | 0 of 2 | 5 | NCT05543187 | Trial comparisons |
Papers from its trials5
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0015762
GET https://conductscience.org/api/diseases/v1/MONDO_0015762/drugs