Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Gaucher Disease: full-pipeline trial registry
7 of 14 indexed Gaucher Disease trials are currently recruiting.
Corpus placement: #1,230 of 28,707 conditions by registered trial count.
14 US clinical trials · 7 currently recruiting · #1,230 of 28,707 conditions by registered trial count
Gaucher Disease: 14 US clinical trials tracked, 7 recruiting.
Gaucher Disease is the subject of 14 registered US clinical trials on ClinicalTrials.gov, 7 of them currently open to new participants. 3 are in Phase 3-4 (later-stage) and 4 in Phase 1-2 (earlier-stage). The most active sponsor is Shire, running 3 of these trials.
Enrollment posture brief
Registry condition gaucher-disease
OPEN 7 | RECR 50% | LATE 3 | EARLY 4 | RANK #1230 | SPON 3
Volume-matched condition peers
Nearest open-share peer: Abdominal Aortic Aneurysms (50% recruiting · 6 trials)
Gaucher Disease lists 7 open slots (50% of 14 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 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 is recruiting above its size-band peers.
7 of 14 indexed trials (50%) 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 research.
Shire accounts for 3 of 14 trials (21.4%). 10 distinct sponsors appear on at least one study for this condition label.
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Most Gaucher Disease trials plan to enrol fewer than 100 people.
Median target enrolment is 31 among the 14 trials that report a genuine target (sentinel values excluded). 11 of those (78.6%) plan for fewer than 100 participants.
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Gaucher Disease splits between interventional and observational designs.
7 interventional and 7 observational studies make up this index (50% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
14 total, page 1 of 1
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
Genzyme, a Sanofi Company
NCT00358943
Gaucher Disease Outcome Survey (GOS)
Shire
NCT03291223
A Long-term Follow-up Study of Gaucher Disease
Duke University
NCT03190837
Drug Discovery for Parkinson's With Mutations in the GBA Gene
New York Stem Cell Foundation Research Institute
NCT05536388
Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease
University of Minnesota
NCT05843552
The GBA Multimodal Study in Parkinson's Disease
Pacific Parkinson's Research Centre
NCT04101968
A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
Prevail Therapeutics
NCT05487599
Long Term Follow-Up for Safety of AVR-RD-02
John Bernat
NCT06488261
Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase
Shire
NCT00478647
A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
Pfizer
NCT00376168
Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase
Pfizer
NCT00712348
A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
Amicus Therapeutics
NCT00433147
Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease
Shire
NCT02574286
A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease
Amicus Therapeutics
NCT00446550
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 1 |
| Phase 2 | 3 |
| Phase 3 | 2 |
| Phase 4 | 1 |
Top Sponsors
Named interventions
Most-linked intervention names on Gaucher Disease records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Gaucher Disease 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 page
14 registered trials, 7 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 7 Gaucher Disease trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Shire sponsors the most Gaucher Disease trials on record, review their full trial history before evaluating a specific study. See Shire'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 condition card totals 14 registered trials; most-linked intervention Plant cell expressed recombinant glucocerebrosidase (prGCD); 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.