Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Gaucher Disease, Type 1: early-phase trial registry
1 of 9 indexed Gaucher Disease, Type 1 trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #1,916 of 28,707 conditions by registered trial count.
9 US clinical trials · 1 currently recruiting · #1,916 of 28,707 conditions by registered trial count
Gaucher Disease, Type 1: 9 US clinical trials tracked, 1 recruiting.
Gaucher Disease, Type 1 is the subject of 9 registered US clinical trials on ClinicalTrials.gov, 1 of them currently open to new participants. 3 are in Phase 3-4 (later-stage) and 6 in Phase 1-2 (earlier-stage). The most active sponsor is Genzyme, a Sanofi Company, running 4 of these trials.
Enrollment posture brief
Registry condition gaucher-disease-type-1
OPEN 1 | RECR 11% | LATE 3 | EARLY 6 | RANK #1916 | SPON 4
Volume-matched condition peers
Nearest open-share peer: Alcohol Use Disorders (11% recruiting · 9 trials)
Gaucher Disease, Type 1 lists 1 open slots (11% of 9 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Gaucher Disease, Type 1 on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Gaucher Disease, Type 1 is recruiting below its size-band peers.
1 of 9 indexed trials (11.1%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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One sponsor concentrates Gaucher Disease, Type 1 research.
Genzyme, a Sanofi Company accounts for 4 of 9 trials (44.4%). 5 distinct sponsors appear on at least one study for this condition label.
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Most Gaucher Disease, Type 1 trials plan to enrol fewer than 100 people.
Median target enrolment is 30 among the 9 trials that report a genuine target (sentinel values excluded). 8 of those (88.9%) plan for fewer than 100 participants.
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Gaucher Disease, Type 1 research is mostly interventional.
9 interventional and 0 observational studies make up this index (100% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
9 total, page 1 of 1
A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
Prevail Therapeutics
NCT05487599
Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)
Spur Therapeutics
NCT06545136
Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension
Genzyme, a Sanofi Company
NCT02843035
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)
Genzyme, a Sanofi Company
NCT00943111
An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease
Shire
NCT00635427
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)
Genzyme, a Sanofi Company
NCT00891202
A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
Amicus Therapeutics
NCT00433147
A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients
Genzyme, a Sanofi Company
NCT00358150
A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease
Amicus Therapeutics
NCT00446550
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 2 |
| Phase 2 | 4 |
| Phase 3 | 3 |
Top Sponsors
Named interventions
Most-linked intervention names on Gaucher Disease, Type 1 records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Gaucher Disease, Type 1 trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Gaucher Disease, Type 1 page
9 registered trials, 1 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 1 Gaucher Disease, Type 1 trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Genzyme, a Sanofi Company sponsors the most Gaucher Disease, Type 1 trials on record, review their full trial history before evaluating a specific study. See Genzyme, a Sanofi Company's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Gaucher Disease, Type 1 condition card totals 9 registered trials; most-linked intervention Eliglustat tartrate; top US site state New York. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.