Condition (MeSH)
Leukodystrophy, Globoid Cell
ClinicalTrials.gov lists 31 trials for Leukodystrophy, Globoid Cell or a narrower condition beneath it.
Search trials, drugs, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.
Trials per phase
31 of the 31 trials are tagged with Leukodystrophy, Globoid Cell itself; the rest are tagged with a narrower condition beneath it.
When did Leukodystrophy, Globoid Cell 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.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- 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) | 1 |
| 2024 | 1 |
| 2023 | 1 |
| 2022 | 1 |
| 2021 | 1 |
| 2020 | 1 |
| 2019 | 1 |
| 2018 | 3 |
| 2017 | 1 |
| 2016 | 1 |
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: 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 Leukodystrophy, Globoid Cell trials in? (7)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 2 |
| Phase 1/2 | 3 |
| Phase 2 | 7 |
| Phase 2/3 | 1 |
| Phase 3 | 1 |
| Not applicable | 1 |
| Observational | 16 |
How many Leukodystrophy, Globoid Cell trials are recruiting? (8)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 1 |
| Recruiting | 7 |
| Active, not recruiting | 4 |
| Completed | 10 |
| Suspended | 1 |
Showing 5 of 8 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Leukodystrophy, Globoid Cell trials? (7)
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 |
|---|---|
| Forge Biologics, Inc | 2 |
| Magenta Therapeutics, Inc. | 2 |
| Aldagen | 1 |
| CENTOGENE GmbH Rostock | 1 |
| Gemma Biotherapeutics | 1 |
Showing 5 of 7 sponsors.
Which drugs are being tested for Leukodystrophy, Globoid Cell? (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 |
|---|---|
| Melphalan | 3 |
| CLOFARABINE | 2 |
| Cytoxan | 2 |
| Busulfan | 2 |
Interventions as named in the registry (11)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Alemtuzumab s.c. | 5 |
| Cyclosporine 0.05% | 3 |
| Mofetil Mycophenolate | 3 |
| Hematopoietic Stem Cell Transplantation | 2 |
| HU - Hydroxyurea | 2 |
Showing 5 of 11 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, Globoid Cell 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 |
| Auto recovery | 11 | 2 |
| Disease Progression | 4 | 1 |
| Graft Failure | 4 | 1 |
| Autoimmune hemolytic anemia | 3 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Leukodystrophy, Globoid Cell? (21)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy. | Molecular therapy : the journal of the American Society of Gene Therapy | 2024 | Yes |
| Infantile Krabbe disease (0-12 months), progression, and recommended endpoints for clinical trials. | Annals of clinical and translational neurology | 2024 | Yes |
| Preclinical studies in Krabbe disease: A model for the investigation of novel combination therapies for lysosomal storage diseases. | Molecular therapy : the journal of the American Society of Gene Therapy | 2023 | Yes |
| Efficacy and Safety of a Krabbe Disease Gene Therapy. | Human gene therapy | 2022 | Yes |
| Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease. | Blood | 2021 | Yes |
Showing 5 of 21 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 |
|---|---|---|---|---|---|
| Krabbe disease | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Unspecified |
| Krabbe disease | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Exact |
| Krabbe disease | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Exact |
| Krabbe disease | Prevalence at birth | 1-9 / 1 000 000 | Worldwide | Not stated | Exact |
| Krabbe disease | Prevalence at birth | 1-9 / 100 000 | Portugal | Not stated | Unspecified |
Showing 5 of 36 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Krabbe disease | 487 | Exact | Orphadata Science knowledge files, Product 1 |
| Krabbe disease | 487 | 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.