Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Lysosomal Storage Disease: early-phase trial registry
A small indexed set (3 trials) with no open enrollment listed right now.
Corpus placement: #5,209 of 28,707 conditions by registered trial count.
3 US clinical trials · none currently recruiting · #5,209 of 28,707 conditions by registered trial count
Lysosomal Storage Disease: 3 US clinical trials tracked, none currently recruiting.
Lysosomal Storage Disease is the subject of 3 registered US clinical trials on ClinicalTrials.gov, none currently recruiting. 0 are in Phase 3-4 (later-stage) and 1 in Phase 1-2 (earlier-stage). The most active sponsor is Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), running 1 of these trials.
Enrollment posture brief
Registry condition lysosomal-storage-disease
OPEN 0 | RECR 0% | LATE 0 | EARLY 1 | RANK #5209 | SPON 1
Volume-matched condition peers
Nearest open-share peer: Accelerated Phase Chronic Myelogenous Leukemia (0% recruiting · 12 trials)
Lysosomal Storage Disease has no open enrollment in this registry pull (3 archived studies). Peers show how other volume-matched labels compare on open share. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Lysosomal Storage 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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Lysosomal Storage Disease is recruiting below its size-band peers.
0 of 3 indexed trials (0%) 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.
Indexed trials (enrollment closed)
3 total, page 1 of 1
The Classification and Cause of Leukodystrophies of Unknown Cause
National Institute of Neurological Disorders and Stroke (NINDS)
NCT00001671
Study of Inborn Errors of Cholesterol Synthesis and Related Disorders
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NCT00046202
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
Duke University
NCT00744692
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 1 |
Top Sponsors
Named interventions
Most-linked intervention names on Lysosomal Storage Disease records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Lysosomal Storage 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 Lysosomal Storage Disease page
3 registered trials, 0 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- No Lysosomal Storage Disease trials are currently recruiting in this registry; that can change as new trials post, or check the broader research index. Browse research indexes
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) sponsors the most Lysosomal Storage Disease trials on record, review their full trial history before evaluating a specific study. See Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)'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 Lysosomal Storage Disease condition card totals 3 registered trials; most-linked intervention Unrelated Umbilical Cord Blood Transplant; top US site state North Carolina. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.