Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.

ClinicalTrials.gov 1 recruiting now official registry

Gaucher Disease, Type 1 clinical trials

1 of 9 indexed Gaucher Disease, Type 1 trials are currently recruiting.

9 US clinical trials · 1 currently 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.

The research picture

Gaucher Disease, Type 1 has 9 registered US clinical trials, 1 of them open to new participants right now, about 11% of the total.

1
recruiting participants now
11%
of trials open to enrollment
3
in Phase 3–4 (later-stage)
4
top sponsor: Genzyme, a Sanofi Company
Phase 12Phase 24Phase 33

Active & Recent Trials

9 total, page 1 of 1

RECRUITING Phase 1 15 participants

A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)

Prevail Therapeutics

NCT05487599

ACTIVE NOT RECRUITING Phase 1 75 participants

Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

Spur Therapeutics

NCT06545136

ACTIVE NOT RECRUITING Phase 2 12 participants

Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

Genzyme, a Sanofi Company

NCT02843035

COMPLETED Phase 3 160 participants

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

COMPLETED Phase 3 95 participants

An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease

Shire

NCT00635427

COMPLETED Phase 3 40 participants

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)

Genzyme, a Sanofi Company

NCT00891202

COMPLETED Phase 2 30 participants

A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy

Amicus Therapeutics

NCT00433147

COMPLETED Phase 2 26 participants

A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients

Genzyme, a Sanofi Company

NCT00358150

COMPLETED Phase 2 19 participants

A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease

Amicus Therapeutics

NCT00446550

Phase Distribution

PhaseTrial count
Phase 1 2
Phase 2 4
Phase 3 3

Top Sponsors

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

Reading the Gaucher Disease, Type 1 Trial Landscape

9 indexed Gaucher Disease, Type 1 studies · 1 recruiting (11%).

Phase mix: 3 late (III/IV) vs 6 early (I/II).

Top sponsor: Genzyme, a Sanofi Company (4 trials), 4 others listed. 9 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, Type 1?

PlainTrial tracks 9 US clinical trials for Gaucher Disease, Type 1, of which 1 are currently recruiting participants. Data sourced from ClinicalTrials.gov.

How do I find a recruiting trial for Gaucher Disease, Type 1?

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.

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.

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.

Verify with NIH →

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.