Condition (MeSH)
Niemann-Pick Disease, Type C
ClinicalTrials.gov lists 36 trials for Niemann-Pick Disease, Type C or a narrower condition beneath it. Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) has the most among industry and government lead sponsors, with 5.
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Trials per phase
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36 of the 36 trials are tagged with Niemann-Pick Disease, Type C itself; the rest are tagged with a narrower condition beneath it.
When did Niemann-Pick Disease, Type C trials start? (18)
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
- Phase 4
- 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 |
| 2025 | 2 |
| 2023 | 1 |
| 2022 | 2 |
| 2021 | 2 |
| 2020 | 4 |
| 2019 | 4 |
| 2018 | 3 |
| 2017 | 3 |
| 2016 | 1 |
Showing 10 of 18 earlier years.
No forecast is made for this condition: it averages 1.2 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 4 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 Niemann-Pick Disease, Type C trials in? (9)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 4 |
| Phase 1/2 | 5 |
| Phase 2 | 6 |
| Phase 2/3 | 4 |
| Phase 3 | 6 |
| Phase 4 | 1 |
| Not applicable | 1 |
| Observational | 8 |
| Expanded access | 1 |
How many Niemann-Pick Disease, Type C trials are recruiting? (8)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 6 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 1 |
| Available | 1 |
| Completed | 15 |
Showing 5 of 8 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Niemann-Pick Disease, Type C trials? (9)
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 |
|---|---|
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 5 |
| Azafaros B.V. | 4 |
| Cyclo Therapeutics, Inc. | 4 |
| Mandos LLC | 4 |
| ZevraDenmark | 3 |
Showing 5 of 9 sponsors.
Which drugs are being tested for Niemann-Pick Disease, Type C? (6)
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 |
|---|---|
| VTS-270 | 7 |
| AZ-3102 | 4 |
| Hydroxypropyl Beta Cyclodextrin | 3 |
| Miglustat | 3 |
| N-Acetyl-L-Leucine | 2 |
Showing 5 of 6 drugs.
Interventions as named in the registry (9)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Hydroxypropyl-β-cyclodextrin | 1 |
| Biopsy | 1 |
| Alemtuzumab s.c. | 1 |
| N Acetyl Cysteine | 1 |
| Lithium Carbonate Pill | 1 |
Showing 5 of 9 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 Niemann-Pick Disease, Type C 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 |
|---|---|---|
| Pneumonia | 11 | 4 |
| Seizure | 11 | 4 |
| Dysphagia | 9 | 3 |
| Death | 7 | 2 |
| Pneumonia aspiration | 7 | 3 |
Showing 5 of 10 adverse events.
Which publications cover Niemann-Pick Disease, Type C? (38)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Convergent Validity of the Fine Motor, Speech, and Cognitive Domains of the 5-Domain Niemann-Pick Disease Type C Clinical Severity Scale. | Journal of child neurology | 2026 | Yes |
| Disease-Modifying, Neuroprotective Effect of N-Acetyl-l-Leucine in Adult and Pediatric Patients With Niemann-Pick Disease Type C. | Neurology | 2025 | Yes |
| Trial of <i>N</i>-Acetyl-l-Leucine in Niemann-Pick Disease Type C. | The New England journal of medicine | 2024 | Yes |
| Swallowing characterization of adult-onset Niemann-Pick, type C1 patients. | Orphanet journal of rare diseases | 2024 | Yes |
| Intravenous 2-hydroxypropyl-β-cyclodextrin (Trappsol® Cyclo™) demonstrates biological activity and impacts cholesterol metabolism in the central nervous system and peripheral tissues in adult subjects with Niemann-Pick Disease Type C1: Results of a phase 1 trial. | Molecular genetics and metabolism | 2022 | Yes |
Showing 5 of 38 publications.
Prevalence (27)
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 |
|---|---|---|---|---|---|
| Niemann-Pick disease type C | Prevalence at birth | 1-9 / 1 000 000 | United Kingdom | GB | Exact |
| Niemann-Pick disease type C | Prevalence at birth | 1-9 / 1 000 000 | United Kingdom | GB | Unspecified |
| Niemann-Pick disease type C | Prevalence at birth | 1-9 / 1 000 000 | United Kingdom | GB | Exact |
| Niemann-Pick disease type C | Prevalence at birth | 1-9 / 1 000 000 | Sweden | Not stated | Exact |
| Niemann-Pick disease type C | Point prevalence | 1-9 / 100 000 | Europe | Not stated | Unspecified |
Showing 5 of 27 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Niemann-Pick disease type C | 646 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Niemann-Pick disease type C | 646 | 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.