Condition (MeSH)
Adrenoleukodystrophy
ClinicalTrials.gov lists 62 trials for Adrenoleukodystrophy 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
62 of the 62 trials are tagged with Adrenoleukodystrophy itself; the rest are tagged with a narrower condition beneath it.
When did Adrenoleukodystrophy 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: 6
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 | 1 |
| 2024 | 2 |
| 2023 | 6 |
| 2022 | 3 |
| 2021 | 4 |
| 2020 | 2 |
| 2019 | 3 |
| 2018 | 5 |
| 2017 | 4 |
| 2016 | 5 |
Showing 10 of 24 earlier years.
No forecast is made for this condition: it averages 2.4 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 Adrenoleukodystrophy trials in? (9)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 5 |
| Phase 1/2 | 5 |
| Phase 2 | 8 |
| Phase 2/3 | 5 |
| Phase 3 | 4 |
| Phase 4 | 1 |
| Not applicable | 12 |
| Observational | 20 |
| Expanded access | 2 |
How many Adrenoleukodystrophy 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 | 13 |
| Active, not recruiting | 2 |
| Available | 1 |
| Completed | 24 |
Showing 5 of 9 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Adrenoleukodystrophy 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 |
| Minoryx Therapeutics, S.L. | 2 |
| NeuroVia, Inc. | 2 |
| Poxel SA | 2 |
| SwanBio Therapeutics, Inc. | 2 |
Showing 5 of 6 sponsors.
Which drugs are being tested for Adrenoleukodystrophy? (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 |
|---|---|
| Melphalan | 3 |
| CLOFARABINE | 2 |
| Sobetirome | 2 |
Interventions as named in the registry (12)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Alemtuzumab s.c. | 4 |
| Cyclosporine 0.05% | 3 |
| Mofetil Mycophenolate | 3 |
| Hematopoietic Stem Cell Transplantation | 2 |
| HU - Hydroxyurea | 2 |
Showing 5 of 12 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 Adrenoleukodystrophy 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 | 55 | 2 |
| Febrile neutropenia | 12 | 2 |
| Pyrexia | 11 | 2 |
| Auto recovery | 9 | 1 |
| Disease progression | 9 | 2 |
Showing 5 of 10 adverse events.
Which publications cover Adrenoleukodystrophy? (29)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy. | The New England journal of medicine | 2024 | Yes |
| Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy. | The New England journal of medicine | 2024 | Yes |
| Male Carrier of X-Linked Adrenal Leukodystrophy Due to 47, XXY Karyotype. | JAMA neurology | 2024 | Yes |
| Safety and efficacy of leriglitazone for preventing disease progression in men with adrenomyeloneuropathy (ADVANCE): a randomised, double-blind, multi-centre, placebo-controlled phase 2-3 trial. | The Lancet. Neurology | 2023 | Yes |
| Interval between contrast administration and T1-weighted MRI for cerebral adrenoleukodystrophy: a single-case observation. | European radiology experimental | 2023 | Yes |
Showing 5 of 29 publications.
Prevalence (9)
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 |
|---|---|---|---|---|---|
| X-linked adrenoleukodystrophy | Prevalence at birth | 1-5 / 10 000 | United States | US | Exact |
| X-linked adrenoleukodystrophy | Prevalence at birth | 1-5 / 10 000 | United States | US | Unspecified |
| X-linked adrenoleukodystrophy | Prevalence at birth | 1-5 / 10 000 | United States | US | Exact |
| X-linked adrenoleukodystrophy | Point prevalence | 1-9 / 1 000 000 | Norway | Not stated | Unspecified |
| X-linked adrenoleukodystrophy | Prevalence at birth | 1-9 / 1 000 000 | Israel | Not stated | Exact |
Showing 5 of 9 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| X-linked adrenoleukodystrophy | 43 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| X-linked adrenoleukodystrophy | 43 | 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.