Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Wolman Disease: early-phase trial registry
3 of 5 indexed Wolman Disease trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #3,391 of 28,707 conditions by registered trial count.
5 US clinical trials · 3 currently recruiting · #3,391 of 28,707 conditions by registered trial count
Wolman Disease: 5 US clinical trials tracked, 3 recruiting.
Wolman Disease is the subject of 5 registered US clinical trials on ClinicalTrials.gov, 3 of them currently open to new participants. 0 are in Phase 3-4 (later-stage) and 3 in Phase 1-2 (earlier-stage). The most active sponsor is University of California, San Francisco, running 2 of these trials.
Enrollment posture brief
Registry condition wolman-disease
OPEN 3 | RECR 60% | LATE 0 | EARLY 3 | RANK #3391 | SPON 2
Volume-matched condition peers
Nearest open-share peer: Acute Kidney Injury (AKI) (60% recruiting · 5 trials)
Wolman Disease lists 3 open slots (60% of 5 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 Wolman 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.
-
Wolman Disease is recruiting above its size-band peers.
3 of 5 indexed trials (60%) 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.
-
One sponsor concentrates Wolman Disease research.
University of California, San Francisco accounts for 2 of 5 trials (40%). 4 distinct sponsors appear on at least one study for this condition label.
-
Typical Wolman Disease enrolment sits around 135 participants.
Median target enrolment is 135 among the 5 trials that report a genuine target (sentinel values excluded). 2 of those (40%) plan for fewer than 100 participants.
-
Wolman Disease splits between interventional and observational designs.
3 interventional and 2 observational studies make up this index (60% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
5 total, page 1 of 1
Lysosomal Acid Lipase (LAL) Deficiency Registry
Alexion Pharmaceuticals
NCT01633489
Registry of Patients Diagnosed With Lysosomal Storage Diseases
University of California, San Francisco
NCT05619900
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
University of California, San Francisco
NCT04532047
Stem Cell Transplant for Inborn Errors of Metabolism
Masonic Cancer Center, University of Minnesota
NCT00176904
Human Placental-Derived Stem Cell Transplantation
New York Medical College
NCT01586455
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 2 |
| Phase 2 | 1 |
Top Sponsors
Named interventions
Most-linked intervention names on Wolman Disease records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Wolman 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 Wolman Disease page
5 registered trials, 3 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 3 Wolman Disease trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- University of California, San Francisco sponsors the most Wolman Disease trials on record, review their full trial history before evaluating a specific study. See University of California, San Francisco'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 Wolman Disease condition card totals 5 registered trials; most-linked intervention Busulfan, Cyclophosphamide, Antithymocyte Globulin; top US site state California. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.