Condition (MeSH)
Leukodystrophy, Metachromatic
ClinicalTrials.gov lists 49 trials for Leukodystrophy, Metachromatic or a narrower condition beneath it. Shire has the most among industry and government lead sponsors, with 7.
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Trials per phase
49 of the 49 trials are tagged with Leukodystrophy, Metachromatic itself; the rest are tagged with a narrower condition beneath it.
When did Leukodystrophy, Metachromatic trials start? (20)
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. 2 trials are 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
- Not applicable
- Observational
Top of scale: 5
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 |
| 2022 | 2 |
| 2021 | 3 |
| 2020 | 3 |
| 2019 | 2 |
| 2018 | 3 |
| 2017 | 2 |
| 2016 | 1 |
| 2015 | 2 |
| 2014 | 4 |
Showing 10 of 20 earlier years.
No forecast is made for this condition: it averages 0.6 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 Leukodystrophy, Metachromatic trials in? (8)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 3 |
| Phase 1/2 | 8 |
| Phase 2 | 10 |
| Phase 2/3 | 1 |
| Phase 3 | 2 |
| Not applicable | 6 |
| Observational | 16 |
| Expanded access | 3 |
How many Leukodystrophy, Metachromatic 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 | 7 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 3 |
| Completed | 21 |
Showing 5 of 9 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Leukodystrophy, Metachromatic 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 |
|---|---|
| Shire | 7 |
| Orchard Therapeutics | 3 |
| Talaris Therapeutics Inc. | 3 |
| Magenta Therapeutics, Inc. | 2 |
| Aldagen | 1 |
Showing 5 of 6 sponsors.
Which drugs are being tested for Leukodystrophy, Metachromatic? (5)
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 |
|---|---|
| Recombinant human Arylsulfatase A (rhASA) | 3 |
| Melphalan | 3 |
| CLOFARABINE | 2 |
| Cytoxan | 2 |
| Busulfan | 2 |
Interventions as named in the registry (10)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Alemtuzumab s.c. | 5 |
| Cyclosporine 0.05% | 3 |
| Mofetil Mycophenolate | 3 |
| Stem Cell Transplantation | 3 |
| Hematopoietic Stem Cell Transplantation | 2 |
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 Leukodystrophy, Metachromatic 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 | 60 | 2 |
| Pyrexia | 16 | 4 |
| Seizure | 13 | 1 |
| Auto recovery | 11 | 2 |
| Pneumonia | 11 | 4 |
Showing 5 of 10 adverse events.
Which publications cover Leukodystrophy, Metachromatic? (28)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Long-Term Effects of Atidarsagene Autotemcel for Metachromatic Leukodystrophy. | The New England journal of medicine | 2025 | Yes |
| Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access. | Lancet (London, England) | 2022 | Yes |
| Intravenous arylsulfatase A in metachromatic leukodystrophy: a phase 1/2 study. | Annals of clinical and translational neurology | 2021 | Yes |
| Natural history of multiple sulfatase deficiency: Retrospective phenotyping and functional variant analysis to characterize an ultra-rare disease. | Journal of inherited metabolic disease | 2020 | Yes |
| Safety of intrathecal delivery of recombinant human arylsulfatase A in children with metachromatic leukodystrophy: Results from a phase 1/2 clinical trial. | Molecular genetics and metabolism | 2020 | Yes |
Showing 5 of 28 publications.
Prevalence (36)
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 |
|---|---|---|---|---|---|
| Metachromatic leukodystrophy | Prevalence at birth | 1-9 / 100 000 | United States | US | Exact |
| Metachromatic leukodystrophy | Prevalence at birth | 1-9 / 100 000 | United States | US | Exact |
| Metachromatic leukodystrophy | Prevalence at birth | 1-9 / 100 000 | United States | US | Unspecified |
| Metachromatic leukodystrophy | Prevalence at birth | 1-9 / 100 000 | Europe | Not stated | Exact |
| Metachromatic leukodystrophy | Prevalence at birth | 1-9 / 1 000 000 | Czech Republic | Not stated | Exact |
Showing 5 of 36 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Metachromatic leukodystrophy | 512 | Exact | Orphadata Science knowledge files, Product 1 |
| Metachromatic leukodystrophy | 512 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.