Condition (MeSH)
Alagille Syndrome
ClinicalTrials.gov lists 31 trials for Alagille Syndrome or a narrower condition beneath it. Mirum Pharmaceuticals, Inc. has the most among industry and government lead sponsors, with 10.
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Trials per phase
31 of the 31 trials are tagged with Alagille Syndrome itself; the rest are tagged with a narrower condition beneath it.
When did Alagille Syndrome trials start? (14)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Trials over time, by phase
- Phase 1
- Phase 2
- Phase 3
- Phase 4
- Not applicable
- Observational
Top of scale: 4
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 5 |
| 2025 | 2 |
| 2023 | 3 |
| 2022 | 1 |
| 2021 | 4 |
| 2017 | 1 |
| 2016 | 2 |
| 2015 | 1 |
| 2014 | 3 |
| 2013 | 3 |
Showing 10 of 14 earlier years.
No forecast is made for this condition: it averages 2.2 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 1 trial still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Alagille Syndrome trials in? (7)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 2 |
| Phase 2 | 6 |
| Phase 3 | 3 |
| Phase 4 | 2 |
| Not applicable | 2 |
| Observational | 15 |
| Expanded access | 1 |
How many Alagille Syndrome trials are recruiting? (6)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 10 |
| Active, not recruiting | 1 |
| Completed | 17 |
| Approved for marketing | 1 |
| Suspended | 1 |
Showing 5 of 6 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Alagille Syndrome trials? (8)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| Mirum Pharmaceuticals, Inc. | 10 |
| Albireo | 3 |
| Ipsen | 2 |
| Takeda | 2 |
| Albireo, an Ipsen Company | 1 |
Showing 5 of 8 sponsors.
Which drugs are being tested for Alagille Syndrome? (4)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| LUM001 | 10 |
| A4250 | 3 |
| CHOLESTYRAMINE | 1 |
| ATORVASTATIN | 1 |
Interventions as named in the registry (4)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Calcium Chloride/Calcium Gluconate | 1 |
| 3 mg [14C]-A4250 capsule | 1 |
| Livmarli Oral Product | 1 |
| CRC (A3384) | 1 |
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do Alagille Syndrome trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Pyrexia | 8 | 4 |
| Vomiting | 5 | 3 |
| Forearm fracture | 4 | 2 |
| Abdominal pain | 3 | 2 |
| Diarrhoea | 3 | 2 |
Showing 5 of 10 adverse events.
Which publications cover Alagille Syndrome? (11)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT): a phase 3, double-blind, randomised, placebo-controlled trial. | The lancet. Gastroenterology & hepatology | 2024 | Yes |
| Maralixibat Treatment Response in Alagille Syndrome is Associated with Improved Health-Related Quality of Life. | The Journal of pediatrics | 2023 | Yes |
| Neurodevelopmental Outcomes in Children With Inherited Liver Disease and Native Liver. | Journal of pediatric gastroenterology and nutrition | 2022 | Yes |
| Efficacy and safety of maralixibat treatment in patients with Alagille syndrome and cholestatic pruritus (ICONIC): a randomised phase 2 study. | Lancet (London, England) | 2021 | Yes |
| Alagille syndrome mutation update: Comprehensive overview of JAG1 and NOTCH2 mutation frequencies and insight into missense variant classification. | Human mutation | 2019 | Yes |
Showing 5 of 11 publications.
Prevalence (12)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Alagille syndrome | Point prevalence | Unknown | Worldwide | Not stated | Exact |
| Alagille syndrome | Point prevalence | 1-9 / 100 000 | Australia | Not stated | Unspecified |
| Alagille syndrome | Prevalence at birth | 1-9 / 100 000 | Europe | Not stated | Unspecified |
| Alagille syndrome | Prevalence at birth | 1-9 / 100 000 | Australia | Not stated | Exact |
| Alagille syndrome | Point prevalence | 1-9 / 100 000 | Australia | Not stated | Exact |
Showing 5 of 12 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Alagille syndrome | 52 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Alagille syndrome | 52 | Exact | Orphadata Science knowledge files, Product 1 |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.