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Mucopolysaccharidosis II: early-phase trial registry
3 of 8 indexed Mucopolysaccharidosis II trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #2,176 of 28,707 conditions by registered trial count.
8 US clinical trials · 3 currently recruiting · #2,176 of 28,707 conditions by registered trial count
Mucopolysaccharidosis II: 8 US clinical trials tracked, 3 recruiting.
Mucopolysaccharidosis II is the subject of 8 registered US clinical trials on ClinicalTrials.gov, 3 of them currently open to new participants. 1 are in Phase 3-4 (later-stage) and 5 in Phase 1-2 (earlier-stage). The most active sponsor is Denali Therapeutics, running 2 of these trials.
Enrollment posture brief
Registry condition mucopolysaccharidosis-ii
OPEN 3 | RECR 38% | LATE 1 | EARLY 5 | RANK #2176 | SPON 2
Volume-matched condition peers
Nearest open-share peer: Acute-graft-versus-host Disease (38% recruiting · 8 trials)
Mucopolysaccharidosis II lists 3 open slots (38% of 8 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Mucopolysaccharidosis II on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Mucopolysaccharidosis II is recruiting below its size-band peers.
3 of 8 indexed trials (37.5%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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One sponsor concentrates Mucopolysaccharidosis II research.
Denali Therapeutics accounts for 2 of 8 trials (25%). 7 distinct sponsors appear on at least one study for this condition label.
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Most Mucopolysaccharidosis II trials plan to enrol fewer than 100 people.
Median target enrolment is 47 among the 8 trials that report a genuine target (sentinel values excluded). 7 of those (87.5%) plan for fewer than 100 participants.
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Mucopolysaccharidosis II research is mostly interventional.
6 interventional and 2 observational studies make up this index (75% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
8 total, page 1 of 1
Registry of Patients Diagnosed With Lysosomal Storage Diseases
University of California, San Francisco
NCT05619900
A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)
Denali Therapeutics
NCT05371613
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Joanne Kurtzberg, MD
NCT02254863
A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
JCR Pharmaceuticals Co.
NCT04573023
A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome
Denali Therapeutics
NCT04251026
Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
NCT03153319
Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
Sangamo Therapeutics
NCT04628871
Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NCT00004454
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 4 |
| Phase 2 | 1 |
| Phase 3 | 1 |
Top Sponsors
Named interventions
Most-linked intervention names on Mucopolysaccharidosis II records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Mucopolysaccharidosis II trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Mucopolysaccharidosis II page
8 registered trials, 3 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 3 Mucopolysaccharidosis II trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Denali Therapeutics sponsors the most Mucopolysaccharidosis II trials on record, review their full trial history before evaluating a specific study. See Denali Therapeutics's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Mucopolysaccharidosis II condition card totals 8 registered trials; most-linked intervention tividenofusp alfa; top US site state California. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.