Condition (MeSH)
Pantothenate Kinase-Associated Neurodegeneration
ClinicalTrials.gov lists 12 trials for Pantothenate Kinase-Associated Neurodegeneration or a narrower condition beneath it. ApoPharma has the most among industry and government lead sponsors, with 2.
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Trials per phase
12 of the 12 trials are tagged with Pantothenate Kinase-Associated Neurodegeneration itself; the rest are tagged with a narrower condition beneath it.
When did Pantothenate Kinase-Associated Neurodegeneration 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 3
- Not applicable
- Observational
Top of scale: 2
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 | 1 |
| 2023 | 1 |
| 2021 | 1 |
| 2019 | 1 |
| 2018 | 1 |
| 2017 | 1 |
| 2015 | 1 |
| 2014 | 1 |
| 2013 | 1 |
| 2012 | 2 |
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 Pantothenate Kinase-Associated Neurodegeneration trials in? (4)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 3 | 3 |
| Not applicable | 2 |
| Observational | 6 |
| Expanded access | 1 |
How many Pantothenate Kinase-Associated Neurodegeneration trials are recruiting? (5)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 2 |
| Completed | 5 |
| Terminated | 1 |
| No longer available | 1 |
| Unknown status | 3 |
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Pantothenate Kinase-Associated Neurodegeneration trials? (4)
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 |
|---|---|
| ApoPharma | 2 |
| Casa di Cura San Raffaele Cassino | 1 |
| Chiesi Canada Corp | 1 |
| Travere Therapeutics, Inc. | 1 |
Interventions as named in the registry (3)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Deferiprone oral solution | 2 |
| Fosmetpantotenate | 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 Pantothenate Kinase-Associated Neurodegeneration 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 Pantothenate Kinase-Associated Neurodegeneration? (5)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
Prevalence (3)
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 |
|---|---|---|---|---|---|
| Pantothenate kinase-associated neurodegeneration | Point prevalence | 1-9 / 1 000 000 | Europe | Not stated | Exact |
| Pantothenate kinase-associated neurodegeneration | Point prevalence | 1-9 / 1 000 000 | Europe | Not stated | Unspecified |
| Pantothenate kinase-associated neurodegeneration | Point prevalence | 1-9 / 1 000 000 | Europe | Not stated | Exact |
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Pantothenate kinase-associated neurodegeneration | 157850 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Pantothenate kinase-associated neurodegeneration | 157850 | 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.