Condition (MeSH)
Peroxisomal Disorders
ClinicalTrials.gov lists 85 trials for Peroxisomal Disorders or a narrower condition beneath it. Genetix Biotherapeutics Inc. has the most among industry and government lead sponsors, with 5.
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Trials per phase (log scale)
1 more section with rows
17 of the 85 trials are tagged with Peroxisomal Disorders itself; the rest are tagged with a narrower condition beneath it.
When did Peroxisomal Disorders trials start? (24)
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. 1 trial is registered to start in 2027 or later, and not listed here.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Phase 4
- Not applicable
- Observational
Top of scale: 8
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 |
|---|---|
| 2025 | 2 |
| 2024 | 2 |
| 2023 | 8 |
| 2022 | 4 |
| 2021 | 4 |
| 2020 | 2 |
| 2019 | 6 |
| 2018 | 8 |
| 2017 | 5 |
| 2016 | 5 |
Showing 10 of 24 earlier years.
No forecast is made for this condition: it averages 3.2 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 2 trials 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 Peroxisomal Disorders trials in? (9)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 6 |
| Phase 1/2 | 5 |
| Phase 2 | 12 |
| Phase 2/3 | 5 |
| Phase 3 | 6 |
| Phase 4 | 1 |
| Not applicable | 13 |
| Observational | 35 |
| Expanded access | 2 |
How many Peroxisomal Disorders trials are recruiting? (9)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 2 |
| Recruiting | 19 |
| Active, not recruiting | 5 |
| Available | 1 |
| Completed | 31 |
Showing 5 of 9 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Peroxisomal Disorders trials? (6)
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 |
|---|---|
| Genetix Biotherapeutics Inc. | 5 |
| Novartis Pharmaceuticals | 3 |
| MED-LIFE DISCOVERIES LP | 2 |
| Minoryx Therapeutics, S.L. | 2 |
| Mirum Pharmaceuticals, Inc. | 2 |
Showing 5 of 6 sponsors.
Which drugs are being tested for Peroxisomal Disorders? (6)
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 |
|---|---|
| Melphalan | 3 |
| CLOFARABINE | 2 |
| Cytoxan | 2 |
| Cholic Acid | 2 |
| Busulfan | 2 |
Showing 5 of 6 drugs.
Interventions as named in the registry (9)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Alemtuzumab s.c. | 5 |
| Canakinumab, ACZ885 | 3 |
| Cyclosporine 0.05% | 3 |
| Mofetil Mycophenolate | 3 |
| Hematopoietic Stem Cell Transplantation | 2 |
Showing 5 of 9 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 Peroxisomal Disorders 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 |
|---|---|---|
| Death | 61 | 3 |
| Febrile neutropenia | 12 | 2 |
| Auto recovery | 11 | 2 |
| Pyrexia | 11 | 2 |
| Disease progression | 10 | 3 |
Showing 5 of 10 adverse events.
Which publications cover Peroxisomal Disorders? (8)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| A Retrospective Study of Hearing Loss in Patients Diagnosed with Peroxisome Biogenesis Disorders in the Zellweger Spectrum. | Ear and hearing | 2022 | 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 |
| Peroxisome biogenesis disorders in the Zellweger spectrum: An overview of current diagnosis, clinical manifestations, and treatment guidelines. | Molecular genetics and metabolism | 2016 | Yes |
| Functions of plasmalogen lipids in health and disease. | Biochimica et biophysica acta | 2012 | Yes |
| Peroxisome biogenesis disorders with prolonged survival: phenotypic expression in a cohort of 31 patients. | American journal of medical genetics. Part A | 2004 | Yes |
Showing 5 of 8 publications.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Peroxisomal disease | 68373 | Exact | Orphadata Science knowledge files, Product 1 |
| Peroxisomal disease | 68373 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Sub-conditions in MeSH (6)
MeSH descendants one level down with at least one trial, most trials first.
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: prevalence.