Condition (MeSH)
Mucopolysaccharidosis III
ClinicalTrials.gov lists 58 trials for Mucopolysaccharidosis III or a narrower condition beneath it. Shire 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 (log scale)
1 more section with rows
58 of the 58 trials are tagged with Mucopolysaccharidosis III itself; the rest are tagged with a narrower condition beneath it.
When did Mucopolysaccharidosis III trials start? (19)
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: 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 |
|---|---|
| 2026 (partial year) | 4 |
| 2024 | 4 |
| 2023 | 4 |
| 2022 | 2 |
| 2021 | 2 |
| 2020 | 4 |
| 2019 | 2 |
| 2018 | 4 |
| 2017 | 4 |
| 2016 | 2 |
Showing 10 of 19 earlier years.
No forecast is made for this condition: it averages 2.2 trial starts a year over the five years to September, under the 5 a year the method needs.
Prediction, not a registry value: median 36 months or more (the model's horizon) from start to primary completion across 9 trials still running at the 2026-10-09 snapshot; 7 of 9 predicted trials reach the 36-month horizon; registered median of 31 months across 34 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Mucopolysaccharidosis III trials in? (9)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 1 |
| Phase 1/2 | 19 |
| Phase 2 | 4 |
| Phase 2/3 | 4 |
| Phase 3 | 3 |
| Phase 4 | 1 |
| Not applicable | 1 |
| Observational | 24 |
| Expanded access | 1 |
How many Mucopolysaccharidosis III trials are recruiting? (8)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 4 |
| Recruiting | 6 |
| Enrolling by invitation | 3 |
| Active, not recruiting | 6 |
| Available | 1 |
Showing 5 of 8 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Mucopolysaccharidosis III trials? (12)
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 | 6 |
| Alexion Pharmaceuticals, Inc. | 5 |
| Allievex Corporation | 5 |
| Ultragenyx Pharmaceutical Inc | 4 |
| LYSOGENE | 3 |
Showing 5 of 12 sponsors.
Which drugs are being tested for Mucopolysaccharidosis III? (7)
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 |
|---|---|
| AX 250 | 5 |
| SBC-103 | 2 |
| CLOFARABINE | 2 |
| Melphalan | 2 |
| ABO-102 | 2 |
Showing 5 of 7 drugs.
Interventions as named in the registry (8)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Hematopoietic Stem Cell Transplantation | 2 |
| Alemtuzumab s.c. | 2 |
| Cyclosporine 0.05% | 2 |
| Mofetil Mycophenolate | 2 |
| rAAV9-CMV-hNAGLUop | 1 |
Showing 5 of 8 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.
How do Mucopolysaccharidosis III trials deliver against plan? (1)
Shown in this order: one row, this condition's own record. Enrollment hit target: the share of trials mapped to this condition whose actual enrollment reached the planned enrollment. On time: the share whose actual primary completion date was no later than the planned date. Each share counts only trials where we observed an estimated figure before the actual one, and n is that number of trials. Below 5 trials the count is shown as a fraction, not a percentage.
| Enrollment | Timeline |
|---|---|
| 37% hit target (n=19); 39 of 58 trials not graded | 72% on time (n=18); 40 of 58 trials not graded |
Delivery grades from ClinicalTrials.gov / AACT (CTTI) planned-vs-actual history, public domain.
Which serious adverse events do Mucopolysaccharidosis III 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 | 14 | 4 |
| Medical device change | 10 | 2 |
| Cerebrospinal fluid leakage | 9 | 2 |
| Pyrexia | 6 | 5 |
| Central nervous system infection | 5 | 3 |
Showing 5 of 10 adverse events.
Which publications cover Mucopolysaccharidosis III? (36)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Anakinra in Sanfilippo syndrome: a phase 1/2 trial. | Nature medicine | 2024 | Yes |
| A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B. | The Journal of clinical investigation | 2023 | Yes |
| Longitudinal Natural History of Pediatric Subjects Affected with Mucopolysaccharidosis IIIB. | The Journal of pediatrics | 2022 | Yes |
| Chemically modified recombinant human sulfamidase (SOBI003) in mucopolysaccharidosis IIIA patients: Results from an open, non-controlled, multicenter study. | Molecular genetics and metabolism | 2022 | Yes |
| Adults with lysosomal storage diseases in the undiagnosed diseases network. | Molecular genetics & genomic medicine | 2022 | Yes |
Showing 5 of 36 publications.
Prevalence (58)
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 3 | Point prevalence | <1 / 1 000 000 | United States | US | Exact |
| Mucopolysaccharidosis type 3 | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Exact |
| Mucopolysaccharidosis type 3 | Point prevalence | <1 / 1 000 000 | United States | US | Exact |
| Mucopolysaccharidosis type 3 | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Exact |
| Mucopolysaccharidosis type 3 | Prevalence at birth | 1-9 / 1 000 000 | Japan | Not stated | Exact |
Showing 5 of 58 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Mucopolysaccharidosis type 3 | 581 | Exact | Orphadata Science knowledge files, Product 1 |
| Mucopolysaccharidosis type 3 | 581 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Checked, with nothing recorded for this condition: sub-conditions in mesh.