Condition (MeSH)
Zellweger Syndrome
ClinicalTrials.gov lists 11 trials for Zellweger Syndrome or a narrower condition beneath it. Mirum Pharmaceuticals, Inc. has the most among industry and government lead sponsors, with 2.
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Trials per phase
11 of the 11 trials are tagged with Zellweger Syndrome itself; the rest are tagged with a narrower condition beneath it.
When did Zellweger Syndrome trials start? (9)
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 2
- Phase 3
- Observational
Top of scale: 2
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 |
|---|---|
| 2023 | 1 |
| 2019 | 1 |
| 2018 | 1 |
| 2017 | 2 |
| 2016 | 1 |
| 2014 | 1 |
| 2013 | 1 |
| 2012 | 1 |
| 1992 | 1 |
No forecast is made for this condition: it averages 0.2 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: none of the 11 trials evaluated at the 2026-10-09 snapshot is predicted and still running. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Zellweger Syndrome trials in? (4)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 2 | 2 |
| Phase 3 | 2 |
| Not applicable | 1 |
| Observational | 6 |
How many Zellweger Syndrome trials are recruiting? (4)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 3 |
| Active, not recruiting | 2 |
| Completed | 5 |
| Terminated | 1 |
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Zellweger Syndrome trials? (1)
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. | 2 |
Which drugs are being tested for Zellweger Syndrome? (3)
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 |
|---|---|
| Cholic Acid | 2 |
| Betaine | 1 |
| Ursodeoxycholic Acid | 1 |
Interventions as named in the registry (10)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| cALD HR-D (High-Risk, Regimen C) | 1 |
| Cholic Acids | 1 |
| Osteopetrosis Haploidentical Only Preparative Regimen | 1 |
| Hydroxychloroquin | 1 |
| cALD SR-B (Standard-Risk, Regimen B) | 1 |
Showing 5 of 10 interventions.
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 Zellweger 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 |
|---|---|---|
| Disease progression | 7 | 1 |
| Dehydration | 2 | 1 |
| Diarrhoea | 2 | 1 |
| Urinary tract infection | 2 | 1 |
| Coagulopathy | 1 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Zellweger Syndrome? (12)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Zellweger Spectrum Disorder: Ophthalmic Findings from a New Natural History Study Cohort and Scoping Literature Review. | Ophthalmology | 2023 | Yes |
| A Retrospective Study of Hearing Loss in Patients Diagnosed with Peroxisome Biogenesis Disorders in the Zellweger Spectrum. | Ear and hearing | 2022 | Yes |
| Clinical, neuroradiological, and molecular characterization of patients with atypical Zellweger spectrum disorder caused by PEX16 mutations: a case series. | Neurogenetics | 2022 | Yes |
| Open-label Phase 3 Continuation Study of Cholic Acid in Patients With Inborn Errors of Bile Acid Synthesis. | Journal of pediatric gastroenterology and nutrition | 2020 | Yes |
| A metabolomic map of Zellweger spectrum disorders reveals novel disease biomarkers. | Genetics in medicine : official journal of the American College of Medical Genetics | 2018 | Yes |
Showing 5 of 12 publications.
Prevalence (13)
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 |
|---|---|---|---|---|---|
| Infantile Refsum disease | Point prevalence | Unknown | Worldwide | Not stated | Unspecified |
| Zellweger syndrome | Point prevalence | Unknown | Worldwide | Not stated | Exact |
| Zellweger syndrome | Prevalence at birth | 1-9 / 100 000 | Israel | Not stated | Exact |
| Zellweger syndrome | Prevalence at birth | 1-9 / 100 000 | Israel | Not stated | Unspecified |
| Zellweger syndrome | Prevalence at birth | 1-9 / 100 000 | Israel | Not stated | Exact |
Showing 5 of 13 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Zellweger syndrome | 912 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Zellweger syndrome | 912 | 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.