Condition (MeSH)
Neuroaxonal Dystrophies
ClinicalTrials.gov lists 19 trials for Neuroaxonal Dystrophies or a narrower condition beneath it. Biojiva LLC has the most among industry and government lead sponsors, with 3.
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Trials per phase
9 of the 19 trials are tagged with Neuroaxonal Dystrophies itself; the rest are tagged with a narrower condition beneath it.
When did Neuroaxonal Dystrophies trials start? (10)
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 2/3
- Phase 3
- Phase 4
- Not applicable
- Observational
Top of scale: 3
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 |
|---|---|
| 2024 | 2 |
| 2023 | 1 |
| 2021 | 2 |
| 2019 | 3 |
| 2018 | 3 |
| 2017 | 1 |
| 2015 | 2 |
| 2014 | 1 |
| 2013 | 1 |
| 2012 | 2 |
No forecast is made for this condition: it averages 0.8 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 Neuroaxonal Dystrophies trials in? (6)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 2/3 | 1 |
| Phase 3 | 3 |
| Phase 4 | 1 |
| Not applicable | 5 |
| Observational | 8 |
| Expanded access | 1 |
How many Neuroaxonal Dystrophies trials are recruiting? (6)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 3 |
| Active, not recruiting | 1 |
| Completed | 9 |
| Terminated | 2 |
| No longer available | 1 |
Showing 5 of 6 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Neuroaxonal Dystrophies trials? (5)
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 |
|---|---|
| Biojiva LLC | 3 |
| ApoPharma | 2 |
| Casa di Cura San Raffaele Cassino | 1 |
| Chiesi Canada Corp | 1 |
| Travere Therapeutics, Inc. | 1 |
Which drugs are being tested for Neuroaxonal Dystrophies? (1)
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 |
|---|---|
| Desipramine | 1 |
Interventions as named in the registry (4)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Deferiprone oral solution | 2 |
| Fosmetpantotenate | 1 |
| type A botulinum toxin | 1 |
| Deferiprone pill | 1 |
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 Neuroaxonal Dystrophies 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 |
|---|---|---|
| Dystonia | 30 | 3 |
| Hospitalization | 27 | 1 |
| Neutropenia | 11 | 2 |
| Vomiting | 9 | 3 |
| Pneumonia | 8 | 3 |
Showing 5 of 10 adverse events.
Which publications cover Neuroaxonal Dystrophies? (15)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Genome sequencing reveals novel noncoding variants in PLA2G6 and LMNB1 causing progressive neurologic disease. | Molecular genetics & genomic medicine | 2022 | Yes |
| C19orf12 mutation causing mitochondrial membrane-protein Associated Neurodegeneration masquerading as spastic paraplegia. | Parkinsonism & related disorders | 2021 | Yes |
| PLA2G6 mutations associated with a continuous clinical spectrum from neuroaxonal dystrophy to hereditary spastic paraplegia. | Clinical genetics | 2017 | Yes |
| Disruption of Golgi morphology and altered protein glycosylation in PLA2G6-associated neurodegeneration. | Journal of medical genetics | 2016 | Yes |
| Infantile and childhood onset PLA2G6-associated neurodegeneration in a large North African cohort. | European journal of neurology | 2015 | Yes |
Showing 5 of 15 publications.
Sub-conditions in MeSH (1)
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 and rare-disease identity.