Condition (MeSH)
Gaucher Disease
ClinicalTrials.gov lists 173 trials for Gaucher Disease or a narrower condition beneath it. Shire has the most among industry and government lead sponsors, with 14.
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Trials per phase (log scale)
1 more section with rows
173 of the 173 trials are tagged with Gaucher Disease itself; the rest are tagged with a narrower condition beneath it.
When did Gaucher Disease trials start? (31)
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. 1 trial is 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: 12
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) | 7 |
| 2025 | 1 |
| 2024 | 11 |
| 2023 | 12 |
| 2022 | 10 |
| 2021 | 9 |
| 2020 | 7 |
| 2019 | 10 |
| 2018 | 9 |
| 2017 | 5 |
Showing 10 of 31 earlier years.
Forecast of the full-year count, planned starts included, not rows of this table: about 7 trials starting in 2026 and about 4 in 2027, the average of the 24 months through September, counted as registered and carried forward. 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 28 percent and ran about 20 percent low against the count later registered; forecasts made before the year began were off by about 39 percent and ran about 37 percent low against the count later registered. 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. The 2026 forecast is below the 7 already registered with a 2026 start date: at the cutoff, 1 trials were already registered to start in October to December, more than the method's monthly average allows for, and the method does not use those planned starts.
Prediction, not a registry value: median of 36 months from start to primary completion across 11 trials still running at the 2026-10-09 snapshot; registered median of 27 months across 89 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Gaucher Disease trials in? (9)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 22 |
| Phase 1/2 | 12 |
| Phase 2 | 18 |
| Phase 2/3 | 3 |
| Phase 3 | 23 |
| Phase 4 | 11 |
| Not applicable | 12 |
| Observational | 69 |
| Expanded access | 3 |
How many Gaucher Disease trials are recruiting? (12)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 7 |
| Recruiting | 23 |
| Enrolling by invitation | 3 |
| Active, not recruiting | 10 |
| Available | 1 |
Showing 5 of 12 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Gaucher Disease trials? (11)
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 |
|---|---|
| Shire | 14 |
| Genzyme, a Sanofi Company | 12 |
| Sanofi | 11 |
| Pfizer | 8 |
| National Institute of Neurological Disorders and Stroke (NINDS) | 6 |
Showing 5 of 11 sponsors.
Which drugs are being tested for Gaucher Disease? (8)
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 |
|---|---|
| Velaglucerase Alfa | 13 |
| Eliglustat | 12 |
| Taliglucerase alfa | 9 |
| Imiglucerase | 9 |
| Afegostat tartrate | 4 |
Showing 5 of 8 drugs.
Interventions as named in the registry (7)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| LY3884961 | 3 |
| ISU302 | 3 |
| Eliglustat GZ385660 | 2 |
| N Acetylcysteine | 2 |
| VGN-R08b | 2 |
Showing 5 of 7 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 Gaucher Disease 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 |
|---|---|---|
| Death | 55 | 2 |
| Auto recovery | 9 | 1 |
| Arthralgia | 8 | 5 |
| Syncope | 7 | 3 |
| Osteonecrosis | 6 | 3 |
Showing 5 of 10 adverse events.
Which publications cover Gaucher Disease? (134)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
Showing 5 of 134 publications.
Prevalence (48)
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 |
|---|---|---|---|---|---|
| Gaucher disease | Prevalence at birth | 1-9 / 100 000 | Australia | Not stated | Exact |
| Gaucher disease | Prevalence at birth | 1-9 / 100 000 | Czech Republic | Not stated | Unspecified |
| Gaucher disease | Point prevalence | 1-9 / 1 000 000 | France | Not stated | Exact |
| Gaucher disease | Prevalence at birth | 1-9 / 100 000 | Netherlands | Not stated | Exact |
| Gaucher disease | Point prevalence | 1-9 / 1 000 000 | Spain | Not stated | Exact |
Showing 5 of 48 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Gaucher disease | 355 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Gaucher disease | 355 | 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.