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Gaucher Disease clinical trials
7 of 14 indexed Gaucher Disease trials are currently recruiting.
14 US clinical trials · 7 currently 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.
The research picture
Gaucher Disease has 14 registered US clinical trials, 7 of them open to new participants right now, about 50% of the total.
- 7
- recruiting participants now
- 50%
- of trials open to enrollment
- 3
- in Phase 3–4 (later-stage)
- 3
- top sponsor: Shire
Active & 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
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Gaucher Disease Trial Landscape
14 indexed Gaucher Disease studies · 7 recruiting (50%).
Phase mix: 3 late (III/IV) vs 4 early (I/II).
Top sponsor: Shire (3 trials), 9 others listed. 14 shown below, recruiting first.
How to read this condition's trial landscape
That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.
Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.
All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation, eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.
Frequently Asked Questions
How many clinical trials are there for Gaucher Disease?
PlainTrial tracks 14 US clinical trials for Gaucher Disease, of which 7 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for Gaucher Disease?
Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.
Is this data current?
Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.
Related
Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.
Read our methodology - how this data is sourced, computed, and verified.
Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial for this condition, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.