Condition (MeSH)
Mucopolysaccharidosis II
ClinicalTrials.gov lists 77 trials for Mucopolysaccharidosis II or a narrower condition beneath it. Shire has the most among industry and government lead sponsors, with 15.
Search trials, drugs, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.
Trials per phase (log scale)
77 of the 77 trials are tagged with Mucopolysaccharidosis II itself; the rest are tagged with a narrower condition beneath it.
When did Mucopolysaccharidosis II 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.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Phase 4
- Observational
Top of scale: 9
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 |
|---|---|
| 2025 | 1 |
| 2024 | 1 |
| 2023 | 5 |
| 2022 | 9 |
| 2021 | 4 |
| 2020 | 4 |
| 2019 | 3 |
| 2018 | 7 |
| 2017 | 7 |
| 2016 | 1 |
Showing 10 of 23 earlier years.
No forecast is made for this condition: it averages 3.4 trial starts a year over the five years to September, under the 5 a year the method needs.
Prediction, not a registry value: median of 36 months from start to primary completion across 11 trials still running at the 2026-10-09 snapshot; registered median of 40 months across 50 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Mucopolysaccharidosis II trials in? (8)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 8 |
| Phase 1/2 | 12 |
| Phase 2 | 7 |
| Phase 2/3 | 12 |
| Phase 3 | 7 |
| Phase 4 | 4 |
| Observational | 26 |
| Expanded access | 1 |
How many Mucopolysaccharidosis II 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 | 3 |
| Active, not recruiting | 15 |
| Available | 1 |
| Completed | 40 |
Showing 5 of 8 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Mucopolysaccharidosis II 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 |
|---|---|
| Shire | 15 |
| JCR Pharmaceuticals Co., Ltd. | 7 |
| REGENXBIO Inc. | 6 |
| Green Cross Corporation | 5 |
| Takeda | 5 |
Showing 5 of 10 sponsors.
Which drugs are being tested for Mucopolysaccharidosis II? (5)
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 |
|---|---|
| Idursulfase | 12 |
| JR-141 | 7 |
| Hunterase | 3 |
| tividenofusp alfa | 3 |
| SB-913 | 2 |
Interventions as named in the registry (10)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Idursulfase IT (1 mg) | 6 |
| Adalimumab-US | 2 |
| Alemtuzumab s.c. | 2 |
| cALD HR-D (High-Risk, Regimen C) | 1 |
| Long Term Follow-Up | 1 |
Showing 5 of 10 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 II 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 |
|---|---|---|
| Device failure | 34 | 5 |
| Cerebrospinal fluid leakage | 24 | 4 |
| Pyrexia | 23 | 6 |
| Pneumonia | 14 | 7 |
| Device breakage | 12 | 4 |
Showing 5 of 10 adverse events.
Which publications cover Mucopolysaccharidosis II? (33)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| An Intravenous Brain-Penetrant Enzyme Therapy for Mucopolysaccharidosis II. | The New England journal of medicine | 2026 | Yes |
| Unmet needs of adults living with mucopolysaccharidosis II: data from the Hunter Outcome Survey. | Orphanet journal of rare diseases | 2025 | Yes |
| A post hoc analysis of Projected Retained Ability Scores (PRAS) for the longitudinal assessment of cognitive functioning in patients with neuronopathic mucopolysaccharidosis II receiving intrathecal idursulfase-IT. | Orphanet journal of rare diseases | 2023 | Yes |
| Neurodevelopmental status and adaptive behavior of pediatric patients with mucopolysaccharidosis II: a longitudinal observational study. | Orphanet journal of rare diseases | 2023 | Yes |
| Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II. | Molecular genetics and metabolism | 2022 | Yes |
Showing 5 of 33 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 2 | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Unspecified |
| Mucopolysaccharidosis type 2 | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Exact |
| Mucopolysaccharidosis type 2 | Point prevalence | <1 / 1 000 000 | United States | US | Unspecified |
| Mucopolysaccharidosis type 2 | Point prevalence | <1 / 1 000 000 | United States | US | Exact |
| Mucopolysaccharidosis type 2 | 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 2 | 580 | Exact | Orphadata Science knowledge files, Product 1 |
| Mucopolysaccharidosis type 2 | 580 | 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.