Condition (MeSH)
Mucopolysaccharidosis VII
ClinicalTrials.gov lists 18 trials for Mucopolysaccharidosis VII or a narrower condition beneath it. Ultragenyx Pharmaceutical Inc has the most among industry and government lead sponsors, with 6.
Search trials, drugs, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.
Trials per phase
18 of the 18 trials are tagged with Mucopolysaccharidosis VII itself; the rest are tagged with a narrower condition beneath it.
When did Mucopolysaccharidosis VII trials start? (9)
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 3
- Observational
Top of scale: 3
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 |
|---|---|
| 2022 | 2 |
| 2021 | 2 |
| 2018 | 3 |
| 2017 | 1 |
| 2015 | 2 |
| 2014 | 2 |
| 2013 | 1 |
| 2009 | 2 |
| 2008 | 1 |
No forecast is made for this condition: it averages 0.4 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 1 trial 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 VII trials in? (6)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 1 |
| Phase 1/2 | 1 |
| Phase 2 | 4 |
| Phase 3 | 2 |
| Observational | 8 |
| Expanded access | 2 |
How many Mucopolysaccharidosis VII trials are recruiting? (8)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 4 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 2 |
| Available | 1 |
| Completed | 7 |
Showing 5 of 8 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Mucopolysaccharidosis VII trials? (3)
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 |
|---|---|
| Ultragenyx Pharmaceutical Inc | 6 |
| CENTOGENE GmbH Rostock | 1 |
| Institut National de la Santé Et de la Recherche Médicale, France | 1 |
Which drugs are being tested for Mucopolysaccharidosis VII? (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 |
|---|---|
| UX003 | 6 |
| Cytoxan | 2 |
| Busulfan | 2 |
Interventions as named in the registry (12)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Alemtuzumab s.c. | 2 |
| Stem Cell Transplantation | 2 |
| cALD HR-D (High-Risk, Regimen C) | 1 |
| Allogeneic Stem Cell Transplantation | 1 |
| Cyclosporine 0.05% | 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 VII 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 |
| Adenoidal Hypertrophy | 2 | 1 |
| Auto recovery | 2 | 1 |
| Diarrhea | 2 | 1 |
| Graft failure | 2 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Mucopolysaccharidosis VII? (5)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in pediatric subjects < 5 years with mucopolysaccharidosis VII. | Molecular genetics and metabolism | 2022 | Yes |
| The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII. | Molecular genetics and metabolism | 2020 | Yes |
| Tolerance induction and microglial engraftment after fetal therapy without conditioning in mice with Mucopolysaccharidosis type VII. | Science translational medicine | 2020 | Yes |
| Pharmacokinetic and Pharmacodynamic Modeling to Optimize the Dose of Vestronidase Alfa, an Enzyme Replacement Therapy for Treatment of Patients with Mucopolysaccharidosis Type VII: Results from Three Trials. | Clinical pharmacokinetics | 2019 | Yes |
| A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease. | Molecular genetics and metabolism | 2018 | Yes |
Prevalence (30)
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 7 | Prevalence at birth | <1 / 1 000 000 | United States | US | Unspecified |
| Mucopolysaccharidosis type 7 | Point prevalence | <1 / 1 000 000 | United States | US | Exact |
| Mucopolysaccharidosis type 7 | Prevalence at birth | <1 / 1 000 000 | United States | US | Exact |
| Mucopolysaccharidosis type 7 | Prevalence at birth | <1 / 1 000 000 | United States | US | Exact |
| Mucopolysaccharidosis type 7 | Point prevalence | <1 / 1 000 000 | United States | US | Exact |
Showing 5 of 30 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Mucopolysaccharidosis type 7 | 584 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Mucopolysaccharidosis type 7 | 584 | Exact | Orphadata Science knowledge files, Product 1 |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.