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Mucopolysaccharidosis I: early-phase trial registry
1 of 6 indexed Mucopolysaccharidosis I trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #2,866 of 28,707 conditions by registered trial count.
6 US clinical trials · 1 currently recruiting · #2,866 of 28,707 conditions by registered trial count
Mucopolysaccharidosis I: 6 US clinical trials tracked, 1 recruiting.
Mucopolysaccharidosis I is the subject of 6 registered US clinical trials on ClinicalTrials.gov, 1 of them currently open to new participants. 1 are in Phase 3-4 (later-stage) and 3 in Phase 1-2 (earlier-stage). The most active sponsor is Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, running 1 of these trials.
Enrollment posture brief
Registry condition mucopolysaccharidosis-i
OPEN 1 | RECR 17% | LATE 1 | EARLY 3 | RANK #2866 | SPON 1
Volume-matched condition peers
Nearest open-share peer: Acute Renal Failure (17% recruiting · 6 trials)
Mucopolysaccharidosis I lists 1 open slots (17% of 6 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 I 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 I is recruiting below its size-band peers.
1 of 6 indexed trials (16.7%) 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 I research.
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center accounts for 1 of 6 trials (16.7%). 6 distinct sponsors appear on at least one study for this condition label.
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Most Mucopolysaccharidosis I trials plan to enrol fewer than 100 people.
Median target enrolment is 41 among the 6 trials that report a genuine target (sentinel values excluded). 5 of those (83.3%) plan for fewer than 100 participants.
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Mucopolysaccharidosis I splits between interventional and observational designs.
4 interventional and 2 observational studies make up this index (66.7% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
6 total, page 1 of 1
Registry of Patients Diagnosed With Lysosomal Storage Diseases
University of California, San Francisco
NCT05619900
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
Human Placental-Derived Stem Cell Transplantation
New York Medical College
NCT01586455
Stem Cell Transplantation for Hurler
Masonic Cancer Center, University of Minnesota
NCT00176917
A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients
Genzyme, a Sanofi Company
NCT00144768
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 2 |
| Phase 2 | 1 |
| Phase 4 | 1 |
Top Sponsors
Named interventions
Most-linked intervention names on Mucopolysaccharidosis I records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Mucopolysaccharidosis I 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 I page
6 registered trials, 1 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 1 Mucopolysaccharidosis I trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center sponsors the most Mucopolysaccharidosis I trials on record, review their full trial history before evaluating a specific study. See Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center'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 I condition card totals 6 registered trials; most-linked intervention laronidase; top US site state California. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.