Condition (MeSH)
Mucopolysaccharidosis VI
ClinicalTrials.gov lists 36 trials for Mucopolysaccharidosis VI or a narrower condition beneath it. BioMarin Pharmaceutical has the most among industry and government lead sponsors, with 7.
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Trials per phase
36 of the 36 trials are tagged with Mucopolysaccharidosis VI itself; the rest are tagged with a narrower condition beneath it.
When did Mucopolysaccharidosis VI trials start? (20)
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.
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: 4
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 |
|---|---|
| 2023 | 1 |
| 2022 | 2 |
| 2021 | 2 |
| 2018 | 3 |
| 2017 | 4 |
| 2016 | 1 |
| 2015 | 1 |
| 2014 | 2 |
| 2013 | 2 |
| 2012 | 2 |
Showing 10 of 20 earlier years.
No forecast is made for this condition: it averages 0.6 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 2 trials still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Mucopolysaccharidosis VI trials in? (8)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 3 |
| Phase 1/2 | 4 |
| Phase 2 | 7 |
| Phase 2/3 | 1 |
| Phase 3 | 3 |
| Phase 4 | 1 |
| Not applicable | 1 |
| Observational | 16 |
How many Mucopolysaccharidosis VI trials are recruiting? (7)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 3 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 4 |
| Completed | 19 |
| Terminated | 6 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Mucopolysaccharidosis VI trials? (4)
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 | 7 |
| Aldagen | 1 |
| CENTOGENE GmbH Rostock | 1 |
| Institut National de la Santé Et de la Recherche Médicale, France | 1 |
Which drugs are being tested for Mucopolysaccharidosis VI? (3)
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 |
|---|---|
| N-acetylgalactosamine 4-sulfatase | 6 |
| Cytoxan | 2 |
| Busulfan | 2 |
Interventions as named in the registry (12)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Adalimumab-US | 2 |
| Alemtuzumab s.c. | 2 |
| Stem Cell Transplantation | 2 |
| cALD HR-D (High-Risk, Regimen C) | 1 |
| Busulfan, Cyclophosphamide, ATG | 1 |
Showing 5 of 12 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 VI 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 |
|---|---|---|
| Death | 6 | 1 |
| Pneumonia | 6 | 3 |
| Broncopneumopathy | 5 | 1 |
| Convulsion | 3 | 2 |
| Respiratory distress | 3 | 2 |
Showing 5 of 10 adverse events.
Which publications cover Mucopolysaccharidosis VI? (14)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Multi-year enzyme expression in patients with mucopolysaccharidosis type VI after liver-directed gene therapy. | Med (New York, N.Y.) | 2025 | Yes |
| Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI. | NEJM evidence | 2022 | Yes |
| Long-term outcomes of patients with mucopolysaccharidosis VI treated with galsulfase enzyme replacement therapy since infancy. | Molecular genetics and metabolism | 2021 | Yes |
| Enzyme replacement therapy outcomes across the disease spectrum: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program. | Journal of inherited metabolic disease | 2019 | Yes |
| Cervical cord compression in mucopolysaccharidosis VI (MPS VI): Findings from the MPS VI Clinical Surveillance Program (CSP). | Molecular genetics and metabolism | 2016 | Yes |
Showing 5 of 14 publications.
Prevalence (81)
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 6 | Prevalence at birth | <1 / 1 000 000 | United States | US | Exact |
| Mucopolysaccharidosis type 6 | Prevalence at birth | <1 / 1 000 000 | United States | US | Exact |
| Mucopolysaccharidosis type 6 | Point prevalence | <1 / 1 000 000 | United States | US | Unspecified |
| Mucopolysaccharidosis type 6 | Point prevalence | <1 / 1 000 000 | United States | US | Exact |
| Mucopolysaccharidosis type 6 | Point prevalence | <1 / 1 000 000 | United States | US | Exact |
Showing 5 of 81 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Mucopolysaccharidosis type 6 | 583 | Exact | Orphadata Science knowledge files, Product 1 |
| Mucopolysaccharidosis type 6 | 583 | 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.