Condition (MeSH)
Lysosomal Storage Diseases, Nervous System
ClinicalTrials.gov lists 705 trials for Lysosomal Storage Diseases, Nervous System or a narrower condition beneath it. Genzyme, a Sanofi Company has the most among industry and government lead sponsors, with 67.
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Trials per phase (log scale)
1 more section with rows
2 of the 705 trials are tagged with Lysosomal Storage Diseases, Nervous System itself; the rest are tagged with a narrower condition beneath it.
When did Lysosomal Storage Diseases, Nervous System trials start? (34)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year. 4 trials are registered to start in 2027 or later, and not listed here.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Phase 4
- Not applicable
- Observational
- Other (Expanded access)
Top of scale: 49
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 26 |
| 2025 | 25 |
| 2024 | 30 |
| 2023 | 49 |
| 2022 | 38 |
| 2021 | 41 |
| 2020 | 34 |
| 2019 | 37 |
| 2018 | 35 |
| 2017 | 25 |
Showing 10 of 34 earlier years.
Forecast of the full-year count, planned starts included, not rows of this table: about 32 trials starting in 2026 and about 28 in 2027, the average of the 24 months through September, counted as registered and carried forward, with about 0.7 times the starts already registered for the rest of 2026 added. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 15 percent and ran about 1 percent high against the count later registered; forecasts made before the year began were off by about 26 percent and ran about 20 percent low against the count later registered. The part-way figures are from forecasts that used a multiple fitted only on earlier years. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.
Prediction, not a registry value: median of 36 months from start to primary completion across 46 trials still running at the 2026-10-09 snapshot; registered median of 32 months across 384 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Lysosomal Storage Diseases, Nervous System trials in? (9)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 58 |
| Phase 1/2 | 59 |
| Phase 2 | 73 |
| Phase 2/3 | 13 |
| Phase 3 | 70 |
| Phase 4 | 45 |
| Not applicable | 67 |
| Observational | 305 |
| Expanded access | 15 |
How many Lysosomal Storage Diseases, Nervous System trials are recruiting? (12)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 25 |
| Recruiting | 101 |
| Enrolling by invitation | 8 |
| Active, not recruiting | 43 |
| Available | 5 |
Showing 5 of 12 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Lysosomal Storage Diseases, Nervous System trials? (10)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| Genzyme, a Sanofi Company | 67 |
| Amicus Therapeutics | 38 |
| Shire | 34 |
| Sanofi | 26 |
| National Institute of Neurological Disorders and Stroke (NINDS) | 19 |
Showing 5 of 10 sponsors.
Which drugs are being tested for Lysosomal Storage Diseases, Nervous System? (15)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| ALGLUCOSIDASE ALFA | 28 |
| REPLAGAL | 20 |
| Migalastat | 20 |
| AGALSIDASE BETA (GZ419828) | 20 |
| Miglustat | 14 |
Showing 5 of 15 drugs.
Which serious adverse events do Lysosomal Storage Diseases, Nervous System trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Pneumonia | 100 | 32 |
| Pyrexia | 72 | 26 |
| Death | 64 | 6 |
| Dehydration | 34 | 20 |
| Vomiting | 33 | 16 |
Showing 5 of 10 adverse events.
Sub-conditions in MeSH (5)
MeSH descendants one level down with at least one trial, most trials first.
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: publications, prevalence, and rare-disease identity.