Condition (MeSH)
Mucopolysaccharidoses
ClinicalTrials.gov lists 310 trials for Mucopolysaccharidoses or a narrower condition beneath it. Shire has the most among industry and government lead sponsors, with 20.
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Trials per phase
1 more section with rows
57 of the 310 trials are tagged with Mucopolysaccharidoses itself; the rest are tagged with a narrower condition beneath it.
When did Mucopolysaccharidoses trials start? (30)
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. 2 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
Top of scale: 26
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) | 11 |
| 2025 | 10 |
| 2024 | 10 |
| 2023 | 21 |
| 2022 | 17 |
| 2021 | 17 |
| 2020 | 19 |
| 2019 | 15 |
| 2018 | 26 |
| 2017 | 15 |
Showing 10 of 30 earlier years.
Forecast of the full-year count, planned starts included, not rows of this table: about 11 trials starting in 2026 and about 11 in 2027, the average of the 24 months through September, counted as registered and carried forward. 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 28 percent and ran about 20 percent low against the count later registered; forecasts made before the year began were off by about 39 percent and ran about 37 percent low against the count later registered. 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. The 2026 forecast is below the 11 already registered with a 2026 start date: at the cutoff, 3 trials were already registered to start in October to December, more than the method's monthly average allows for, and the method does not use those planned starts.
Prediction, not a registry value: median of 36 months from start to primary completion across 34 trials still running at the 2026-10-09 snapshot; registered median of 30 months across 198 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Mucopolysaccharidoses trials in? (9)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 24 |
| Phase 1/2 | 50 |
| Phase 2 | 30 |
| Phase 2/3 | 20 |
| Phase 3 | 23 |
| Phase 4 | 15 |
| Not applicable | 30 |
| Observational | 112 |
| Expanded access | 6 |
How many Mucopolysaccharidoses 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 | 9 |
| Recruiting | 30 |
| Enrolling by invitation | 9 |
| Active, not recruiting | 27 |
| Available | 3 |
Showing 5 of 12 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Mucopolysaccharidoses trials? (14)
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 |
|---|---|
| Shire | 20 |
| BioMarin Pharmaceutical | 18 |
| JCR Pharmaceuticals Co., Ltd. | 11 |
| Genzyme, a Sanofi Company | 10 |
| Ultragenyx Pharmaceutical Inc | 10 |
Showing 5 of 14 sponsors.
Which drugs are being tested for Mucopolysaccharidoses? (8)
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 |
|---|---|
| Laronidase | 15 |
| Idursulfase | 12 |
| BMN 110 | 10 |
| JR-141 | 7 |
| N-acetylgalactosamine 4-sulfatase | 6 |
Showing 5 of 8 drugs.
Interventions as named in the registry (7)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Idursulfase IT (1 mg) | 6 |
| Alemtuzumab s.c. | 5 |
| Cyclosporine 0.05% | 4 |
| 1% Sevoflurane | 3 |
| Hematopoietic Stem Cell Transplantation | 3 |
Showing 5 of 7 interventions.
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do Mucopolysaccharidoses 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 |
|---|---|---|
| Knee deformity | 76 | 5 |
| Poor venous access | 55 | 9 |
| Device failure | 46 | 8 |
| Pneumonia | 39 | 21 |
| Pyrexia | 39 | 17 |
Showing 5 of 10 adverse events.
Which publications cover Mucopolysaccharidoses? (34)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Natural history of multiple sulfatase deficiency: Retrospective phenotyping and functional variant analysis to characterize an ultra-rare disease. | Journal of inherited metabolic disease | 2020 | Yes |
| Challenging behavior in mucopolysaccharidoses types I-III and day-to-day coping strategies: a cross sectional explorative study. | Orphanet journal of rare diseases | 2020 | Yes |
| Recent trends in mucopolysaccharidosis research. | Journal of human genetics | 2019 | Yes |
| Epidemiology of mucopolysaccharidoses. | Molecular genetics and metabolism | 2017 | Yes |
| Quantitative neuroimaging in mucopolysaccharidoses clinical trials. | Molecular genetics and metabolism | 2017 | Yes |
Showing 5 of 34 publications.
Prevalence (66)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Mucopolysaccharidosis | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Unspecified |
| Mucopolysaccharidosis | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Exact |
| Mucopolysaccharidosis | Point prevalence | 1-9 / 1 000 000 | United States | US | Exact |
| Mucopolysaccharidosis | Point prevalence | 1-9 / 1 000 000 | United States | US | Exact |
| Mucopolysaccharidosis | Point prevalence | 1-9 / 1 000 000 | United States | US | Unspecified |
Showing 5 of 66 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Mucopolysaccharidosis | 79213 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Mucopolysaccharidosis | 79213 | Exact | Orphadata Science knowledge files, Product 1 |
Sub-conditions in MeSH (6)
MeSH descendants one level down with at least one trial, most trials first.
Not on this page
Not measured: delivery record.