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Condition (MeSH)

Niemann-Pick Disease, Type B

ClinicalTrials.gov lists 3 trials for Niemann-Pick Disease, Type B or a narrower condition beneath it.

MeSH D052537 Nervous System Diseases C10 C15 C16 C18 Listed by Orphanet as a rare disease
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Phase 2 Phase 2 1 Not applicable Not applicable 2 Phase 2 Phase 2 1 Not applicable Not applicable 2

Trials per phase

3 of the 3 trials are tagged with Niemann-Pick Disease, Type B itself; the rest are tagged with a narrower condition beneath it.

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When did Niemann-Pick Disease, Type B trials start? (3)

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
  • Not applicable

Top of scale: 1

2008: 1 trials (Phase 2 1) 2022: 1 trials (Not applicable 1) 2023: 1 trials (Not applicable 1)

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.

Trials started per year
Year Trials started
2023 1
2022 1
2008 1

No forecast is made for this condition: it averages 0.4 trial starts a year over the five years to September, under the 5 a year the method needs.

Recruitment duration, a model prediction: none of the 3 trials evaluated at the 2026-10-09 snapshot is predicted and still running. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Niemann-Pick Disease, Type B trials in? (2)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 2 1
Not applicable 2

How many Niemann-Pick Disease, Type B trials are recruiting? (2)

Shown in this order: the order a trial moves through, from not yet recruiting to unknown.

Trials by recruitment status
Status Trials
Terminated 1
Unknown status 2

Status as registered on ClinicalTrials.gov. Each trial is counted once.

Which serious adverse events do Niemann-Pick Disease, Type B trials report? (3)

Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.

Serious adverse events in this condition's trials
Adverse event Subjects affected Trials
Death 6 1
Auto recovery 2 1
Graft failure 2 1

Which publications cover Niemann-Pick Disease, Type B? (1)

Shown in this order: major-topic papers first, then newest, then most linked to trials.

Publications
Publication Journal Year Major topic
Clearance of Hepatic Sphingomyelin by Olipudase Alfa Is Associated With Improvement in Lipid Profiles in Acid Sphingomyelinase Deficiency. The American journal of surgical pathology 2016 Yes

Prevalence (6)

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.

Prevalence
Orphanet disease Type Class Geography Jurisdiction Mapping relation
Chronic visceral acid sphingomyelinase deficiency Prevalence at birth 1-9 / 1 000 000 Europe Not stated Unspecified
Chronic visceral acid sphingomyelinase deficiency Prevalence at birth 1-9 / 1 000 000 Europe Not stated Exact
Chronic visceral acid sphingomyelinase deficiency Point prevalence Unknown Europe Not stated Exact
Chronic visceral acid sphingomyelinase deficiency Point prevalence Unknown Europe Not stated Exact
Chronic visceral acid sphingomyelinase deficiency Point prevalence Unknown Europe Not stated Unspecified

Rare-disease identity (2)

Shown in this order: exact, broader, narrower, other.

Rare-disease identity
Orphanet disease ORPHA code Relation Source
Chronic visceral acid sphingomyelinase deficiency 77293 Exact Orphanet Rare Disease Ontology (ORDO)
Chronic visceral acid sphingomyelinase deficiency 77293 Exact Orphadata Science knowledge files, Product 1

Not on this page

Not measured: delivery record.

Checked, with nothing recorded for this condition: lead sponsors and sub-conditions in mesh.

Not computed for conditions with fewer than 10 trials: drugs tested.

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