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Condition (MeSH)

Sphingolipidoses

ClinicalTrials.gov lists 558 trials for Sphingolipidoses or a narrower condition beneath it.

MeSH D013106 Nervous System Diseases C10 C16 C18 Listed by Orphanet as a rare disease
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Phase 1 Phase 1 48 Phase 1/2 Phase 1/2 46 Phase 2 Phase 2 60 Phase 2/3 Phase 2/3 11 Phase 3 Phase 3 64 Phase 4 Phase 4 26 Not applicable Not applicable 48 Observational Observational 243 Phase 1 Phase 1 48 Phase 1/2 Phase 1/2 46 Phase 2 Phase 2 60 Phase 2/3 Phase 2/3 11 Phase 3 Phase 3 64 Phase 4 Phase 4 26 Not applicable Not applicable 48 Observational Observational 243

Trials per phase (log scale)

1 more section with rows

23 of the 558 trials are tagged with Sphingolipidoses itself; the rest are tagged with a narrower condition beneath it.

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The PDF carries up to 50 rows of each section, where the page shows the first few, with the sources listed. Enter a work email and confirm it from the message we send, and the report follows by email.

When did Sphingolipidoses trials start? (34)

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. 4 trials are registered to start in 2027 or later, and not listed here.

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: 41

1991: 2 trials (Observational 2) 1993: 2 trials (Phase 2 1, Phase 1 1) 1994: 1 trials (Observational 1) 1995: 4 trials (Observational 2, Not applicable 1, Phase 2/3 1) 1997: 2 trials (Observational 1, Phase 4 1) 1998: 3 trials (Not applicable 1, Phase 2 2) 1999: 5 trials (Observational 2, Phase 3 1, Phase 2 1, Phase 1 1) 2000: 1 trials (Observational 1) 2001: 7 trials (Observational 4, Phase 4 3) 2002: 9 trials (Observational 3, Phase 2 5, Phase 1 1) 2003: 5 trials (Observational 1, Phase 2 3, Phase 1 1) 2004: 9 trials (Observational 2, Phase 4 2, Phase 3 1, Phase 2 3, Phase 1/2 1) 2005: 5 trials (Observational 2, Phase 4 2, Phase 1 1) 2006: 13 trials (Observational 3, Phase 4 2, Phase 3 1, Phase 2 6, Phase 1 1) 2007: 13 trials (Observational 4, Not applicable 2, Phase 3 2, Phase 2/3 1, Phase 2 2, Phase 1/2 1, Phase 1 1) 2008: 19 trials (Observational 5, Phase 3 8, Phase 2 4, Phase 1/2 1, Phase 1 1) 2009: 18 trials (Observational 6, Not applicable 2, Phase 3 3, Phase 2 2, Phase 1/2 2, Phase 1 3) 2010: 23 trials (Observational 16, Not applicable 2, Phase 4 1, Phase 3 2, Phase 1/2 1, Phase 1 1) 2011: 29 trials (Observational 11, Not applicable 1, Phase 3 7, Phase 2/3 1, Phase 2 3, Phase 1/2 1, Phase 1 5) 2012: 16 trials (Observational 4, Not applicable 3, Phase 4 1, Phase 3 1, Phase 2 2, Phase 1/2 4, Phase 1 1) 2013: 11 trials (Observational 1, Not applicable 3, Phase 3 1, Phase 2 1, Phase 1/2 1, Phase 1 4) 2014: 22 trials (Observational 9, Not applicable 2, Phase 2 5, Phase 1/2 3, Phase 1 3) 2015: 28 trials (Observational 10, Not applicable 2, Phase 4 2, Phase 3 2, Phase 2/3 3, Phase 2 2, Phase 1/2 2, Phase 1 5) 2016: 17 trials (Observational 9, Not applicable 2, Phase 4 2, Phase 3 1, Phase 2/3 1, Phase 1 2) 2017: 19 trials (Observational 9, Not applicable 2, Phase 3 3, Phase 2 1, Phase 1/2 2, Phase 1 2) 2018: 30 trials (Observational 16, Not applicable 1, Phase 4 1, Phase 3 7, Phase 2 3, Phase 1/2 1, Phase 1 1) 2019: 32 trials (Observational 13, Not applicable 4, Phase 4 1, Phase 3 2, Phase 2/3 1, Phase 2 4, Phase 1/2 5, Phase 1 2) 2020: 30 trials (Observational 16, Not applicable 3, Phase 4 3, Phase 3 2, Phase 2 1, Phase 1/2 4, Phase 1 1) 2021: 31 trials (Observational 14, Not applicable 4, Phase 4 3, Phase 3 2, Phase 2 1, Phase 1/2 4, Phase 1 3) 2022: 31 trials (Observational 13, Not applicable 2, Phase 4 1, Phase 3 8, Phase 2 1, Phase 1/2 5, Phase 1 1) 2023: 41 trials (Observational 26, Not applicable 3, Phase 4 1, Phase 3 3, Phase 2/3 2, Phase 2 3, Phase 1/2 2, Phase 1 1) 2024: 23 trials (Observational 14, Not applicable 2, Phase 2 1, Phase 1/2 3, Phase 1 3) 2025: 21 trials (Observational 13, Not applicable 3, Phase 3 3, Phase 2/3 1, Phase 1/2 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
2026 (partial year) 18
2025 21
2024 23
2023 41
2022 31
2021 31
2020 30
2019 32
2018 30
2017 19

Showing 10 of 34 earlier years.

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Forecast of the full-year count, planned starts included, not rows of this table: about 23 trials starting in 2026 and about 21 in 2027, the average of the 24 months through September, counted as registered and carried forward, with about 0.7 times the starts already registered for the rest of 2026 added. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 15 percent and ran about 1 percent high against the count later registered; forecasts made before the year began were off by about 26 percent and ran about 20 percent low against the count later registered. The part-way figures are from forecasts that used a multiple fitted only on earlier years. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.

Prediction, not a registry value: median of 36 months from start to primary completion across 35 trials still running at the 2026-10-09 snapshot; registered median of 32 months across 303 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Sphingolipidoses trials in? (9)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 48
Phase 1/2 46
Phase 2 60
Phase 2/3 11
Phase 3 64
Phase 4 26
Not applicable 48
Observational 243
Expanded access 12

How many Sphingolipidoses trials are recruiting? (12)

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

Trials by recruitment status
Status Trials
Not yet recruiting 20
Recruiting 80
Enrolling by invitation 5
Active, not recruiting 31
Available 4

Showing 5 of 12 statuses.

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

Which lead sponsors run the most Sphingolipidoses trials? (12)

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.

Lead sponsors
Sponsor Trials
Genzyme, a Sanofi Company 34
Shire 34
Amicus Therapeutics 29
Sanofi 22
National Institute of Neurological Disorders and Stroke (NINDS) 18

Showing 5 of 12 sponsors.

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Which drugs are being tested for Sphingolipidoses? (14)

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.

Drugs tested
Drug Trials
REPLAGAL 20
Migalastat 20
AGALSIDASE BETA (GZ419828) 20
Velaglucerase Alfa 13
Pegunigalsidase Alfa 12

Showing 5 of 14 drugs.

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Interventions as named in the registry (1)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
Alemtuzumab s.c. 5

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 Sphingolipidoses trials report? (10)

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 64 6
Pyrexia 54 20
Pneumonia 51 21
Atrial fibrillation 32 12
Vomiting 29 13

Showing 5 of 10 adverse events.

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Which publications cover Sphingolipidoses? (4)

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

Publications
Publication Journal Year Major topic
Cancer and sphingolipid storage disease therapy using novel synthetic analogs of sphingolipids. Chemistry and physics of lipids 2012 Yes
Cerebral glycolipidoses: clinical characteristics of 41 pediatric patients. Journal of child neurology 2004 Yes
Substrate reduction therapy in mouse models of the glycosphingolipidoses. Philosophical transactions of the Royal Society of London. Series B, Biological sciences 2003 Yes
Enzymatic abnormalities in diseases of sphingolipid metabolism. Clinical chemistry 1967 No

Rare-disease identity (2)

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

Rare-disease identity
Orphanet disease ORPHA code Relation Source
Sphingolipidosis 79225 Exact Orphanet Rare Disease Ontology (ORDO)
Sphingolipidosis 79225 Exact Orphadata Science knowledge files, Product 1

Sub-conditions in MeSH (7)

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.

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