Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Gaucher Disease, Type 2: early-phase trial registry
1 of 3 indexed Gaucher Disease, Type 2 trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #5,209 of 28,707 conditions by registered trial count.
3 US clinical trials · 1 currently recruiting · #5,209 of 28,707 conditions by registered trial count
Gaucher Disease, Type 2: 3 US clinical trials tracked, 1 recruiting.
Gaucher Disease, Type 2 is the subject of 3 registered US clinical trials on ClinicalTrials.gov, 1 of them currently open to new participants. 0 are in Phase 3-4 (later-stage) and 2 in Phase 1-2 (earlier-stage). The most active sponsor is Prevail Therapeutics, running 1 of these trials.
Enrollment posture brief
Registry condition gaucher-disease-type-2
OPEN 1 | RECR 33% | LATE 0 | EARLY 2 | RANK #5209 | SPON 1
Volume-matched condition peers
Nearest open-share peer: Adherence, Treatment (33% recruiting · 6 trials)
Gaucher Disease, Type 2 lists 1 open slots (33% of 3 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 2 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 2 is recruiting below its size-band peers.
1 of 3 indexed trials (33.3%) 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.
Open and recent trials
3 total, page 1 of 1
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
University of California, San Francisco
NCT04532047
Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
Prevail Therapeutics
NCT04411654
Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
Idorsia Pharmaceuticals
NCT04470713
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 2 |
Top Sponsors
Named interventions
Most-linked intervention names on Gaucher Disease, Type 2 records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Gaucher Disease, Type 2 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 2 page
3 registered trials, 1 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 1 Gaucher Disease, Type 2 trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Prevail Therapeutics sponsors the most Gaucher Disease, Type 2 trials on record, review their full trial history before evaluating a specific study. See Prevail Therapeutics'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 2 condition card totals 3 registered trials; most-linked intervention LY3884961; top US site state Virginia. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.