Condition (MeSH)
Mucopolysaccharidosis IV
ClinicalTrials.gov lists 100 trials for Mucopolysaccharidosis IV or a narrower condition beneath it. BioMarin Pharmaceutical has the most among industry and government lead sponsors, with 11.
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Trials per phase
1 more section with rows
100 of the 100 trials are tagged with Mucopolysaccharidosis IV itself; the rest are tagged with a narrower condition beneath it.
When did Mucopolysaccharidosis IV trials start? (23)
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. 1 trial is 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: 8
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) | 5 |
| 2025 | 7 |
| 2024 | 4 |
| 2023 | 8 |
| 2022 | 6 |
| 2021 | 7 |
| 2020 | 7 |
| 2019 | 6 |
| 2018 | 6 |
| 2017 | 2 |
Showing 10 of 23 earlier years.
Forecast of the full-year count, planned starts included, not rows of this table: about 7 trials starting in 2026 and about 6 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.
Prediction, not a registry value: median of 33 months from start to primary completion across 10 trials still running at the 2026-10-09 snapshot; registered median of 17 months across 60 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Mucopolysaccharidosis IV trials in? (9)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 3 |
| Phase 1/2 | 5 |
| Phase 2 | 12 |
| Phase 2/3 | 3 |
| Phase 3 | 5 |
| Phase 4 | 5 |
| Not applicable | 22 |
| Observational | 44 |
| Expanded access | 1 |
How many Mucopolysaccharidosis IV trials are recruiting? (9)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 2 |
| Recruiting | 18 |
| Enrolling by invitation | 4 |
| Active, not recruiting | 4 |
| Completed | 50 |
Showing 5 of 9 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Mucopolysaccharidosis IV trials? (8)
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 |
|---|---|
| BioMarin Pharmaceutical | 11 |
| QED Therapeutics, a BridgeBio company | 8 |
| CENTOGENE GmbH Rostock | 2 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 2 |
| Pfizer | 2 |
Showing 5 of 8 sponsors.
Which drugs are being tested for Mucopolysaccharidosis IV? (4)
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 |
|---|---|
| BMN 110 | 10 |
| Infigratinib | 3 |
| Recifercept | 2 |
| Inulin | 2 |
Interventions as named in the registry (11)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| 1% Sevoflurane | 3 |
| BMN 110 - Every Other Week | 2 |
| Infigratinib 0.25 mg/kg/day | 2 |
| Phase II: NTC015 medium dose | 1 |
| Hematopoetic Stem Cell Transplantation | 1 |
Showing 5 of 11 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 Mucopolysaccharidosis IV 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 | 45 | 5 |
| Lower respiratory tract infection | 17 | 6 |
| Cervical cord compression | 14 | 5 |
| Otitis media | 14 | 5 |
Showing 5 of 10 adverse events.
Which publications cover Mucopolysaccharidosis IV? (24)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Abnormally increased carotid intima media-thickness and elasticity in patients with Morquio A disease. | Orphanet journal of rare diseases | 2020 | Yes |
| Effect of enzyme replacement therapy on the growth of patients with Morquio A. | Journal of human genetics | 2019 | Yes |
| Safety, immunogenicity, and clinical outcomes in patients with Morquio A syndrome participating in 2 sequential open-label studies of elosulfase alfa enzyme replacement therapy (MOR-002/MOR-100), representing 5 years of treatment. | Molecular genetics and metabolism | 2018 | Yes |
| Impact of long-term elosulfase alfa on activities of daily living in patients with Morquio A syndrome in an open-label, multi-center, phase 3 extension study. | Molecular genetics and metabolism | 2018 | Yes |
| Long-term Immunogenicity of Elosulfase Alfa in the Treatment of Morquio A Syndrome: Results From MOR-005, a Phase III Extension Study. | Clinical therapeutics | 2017 | Yes |
Showing 5 of 24 publications.
Prevalence (42)
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 type 4 | Point prevalence | <1 / 1 000 000 | United States | US | Exact |
| Mucopolysaccharidosis type 4 | Point prevalence | <1 / 1 000 000 | United States | US | Exact |
| Mucopolysaccharidosis type 4 | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Exact |
| Mucopolysaccharidosis type 4 | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Exact |
| Mucopolysaccharidosis type 4 | Point prevalence | 1-5 / 10 000 | Europe | Not stated | Exact |
Showing 5 of 42 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Mucopolysaccharidosis type 4 | 582 | Exact | Orphadata Science knowledge files, Product 1 |
| Mucopolysaccharidosis type 4 | 582 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.