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ClinicalTrials.gov public records Last synced May 22, 2026, 3:53 AM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Mucopolysaccharidosis (MPS) Clear all
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
MPS IV A
Interventions
BMN 110 Weekly, Placebo, BMN 110 Every Other Week
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
5 Years and older
Enrollment
177 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
6
States / cities
Oakland, California • Wilmington, Delaware • Washington D.C., District of Columbia + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2014 · Synced May 22, 2026, 3:53 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Sanfilippo Syndrome Type B
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
1 Year to 10 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
2
States / cities
Oakland, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 16, 2021 · Synced May 22, 2026, 3:53 AM EDT
Recruiting Phase 2Phase 3 Interventional
View directory record Official record
Conditions
Sanfilippo Syndrome, MPS3
Interventions
Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day
Drug
Lead sponsor
Ozlem Goker-Alpan
Other
Eligibility
18 Years to 99 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 19, 2025 · Synced May 22, 2026, 3:53 AM EDT
Active, not recruiting Phase 3 Interventional
View directory record Official record
Conditions
MPS-IH (Hurler Syndrome)
Interventions
Experimental: OTL-203, Active Comparator: Allo-HSCT
Genetic
Lead sponsor
Orchard Therapeutics
Industry
Eligibility
28 Days to 30 Months
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 21, 2025 · Synced May 22, 2026, 3:53 AM EDT
Recruiting Phase 2Phase 3 Interventional
View directory record Official record
Conditions
MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
Interventions
UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy, Adjuvant IM Therapy
Biological · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2027
U.S. locations
2
States / cities
Columbus, Ohio • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 23, 2026 · Synced May 22, 2026, 3:53 AM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Mucopolysaccharidosis Type I
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
6 Years and older
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 31, 2019 · Synced May 22, 2026, 3:53 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Pompe Disease Infantile-Onset, Neuronopathic Gaucher Disease, Wolman Disease
Interventions
There is no intervention
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 64 Years
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2050
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Apr 7, 2026 · Synced May 22, 2026, 3:53 AM EDT
Withdrawn Phase 1 Interventional
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
Genetic HMI-203
Biological
Lead sponsor
Homology Medicines, Inc
Industry
Eligibility
18 Years to 45 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
5
States / cities
Oakland, California • New Haven, Connecticut • Hackensack, New Jersey + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2023 · Synced May 22, 2026, 3:53 AM EDT
Terminated Phase 1 Interventional
View directory record Official record
Conditions
Mucopolysaccharidosis I, Lysosomal Storage Diseases, Spinal Cord Compression
Interventions
laronidase
Drug
Lead sponsor
Patricia I. Dickson, M.D.
Individual
Eligibility
8 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2011
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Feb 20, 2013 · Synced May 22, 2026, 3:53 AM EDT
Approved For Marketing No phase listed Expanded access
View directory record Official record
Conditions
Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
Interventions
BMN 110
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
U.S. locations
24
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Oakland, California + 21 more
Source: ClinicalTrials.gov public record
Updated Apr 1, 2014 · Synced May 22, 2026, 3:53 AM EDT
Terminated Not applicable Interventional Results available
View directory record Official record
Conditions
Maroteaux-Lamy Syndrome
Interventions
Naglazyme®
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
3 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 22, 2020 · Synced May 22, 2026, 3:53 AM EDT
Not listed No phase listed Observational
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
Long-term Follow-Up
Other
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
28 Months and older · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 9, 2021 · Synced May 22, 2026, 3:53 AM EDT
Not listed Phase 2Phase 3 Interventional
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIA
Interventions
LYS-SAF302
Drug
Lead sponsor
LYSOGENE
Industry
Eligibility
6 Months and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
4
States / cities
Orange, California • Minneapolis, Minnesota • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 30, 2021 · Synced May 22, 2026, 3:53 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
Interventions
BMN 110
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 5 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
2
States / cities
Oakland, California • Manhasset, New York
Source: ClinicalTrials.gov public record
Updated Aug 9, 2017 · Synced May 22, 2026, 3:53 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
MPS 7, Sly Syndrome, Mucopolysaccharidosis, MPS VII
Interventions
UX003, Placebo
Drug · Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
5 Years to 35 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
4
States / cities
Oakland, California • Orange, California • Miami, Florida + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2020 · Synced May 22, 2026, 3:53 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Mucopolysaccharidosis (MPS), Hunter Syndrome
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
2 Years to 18 Years · Male only
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 16, 2021 · Synced May 22, 2026, 3:53 AM EDT
Terminated Phase 2 Interventional Results available
View directory record Official record
Conditions
Sanfilippo Syndrome, Mucopolysaccharidosis (MPS)
Interventions
HGT-1410
Drug
Lead sponsor
Shire
Industry
Eligibility
12 Months to 48 Months
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
2
States / cities
Minneapolis, Minnesota • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 10, 2021 · Synced May 22, 2026, 3:53 AM EDT
Completed Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Mucopolysaccharidosis III
Interventions
anakinra
Biological
Lead sponsor
Lynda E Polgreen
Other
Eligibility
4 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Oct 21, 2024 · Synced May 22, 2026, 3:53 AM EDT
Terminated Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
MPS I
Interventions
SB-318
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
5 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Jan 25, 2023 · Synced May 22, 2026, 3:53 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
No Intervention
Other
Lead sponsor
Denali Therapeutics Inc.
Industry
Eligibility
Up to 30 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
3
States / cities
Oakland, California • Chapel Hill, North Carolina • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 9, 2024 · Synced May 22, 2026, 3:53 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Mucopolysaccharidoses
Interventions
Not listed
Lead sponsor
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Other
Eligibility
5 Years to 35 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2019
U.S. locations
3
States / cities
Oakland, California • Torrance, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 21, 2024 · Synced May 22, 2026, 3:53 AM EDT
Completed Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Mucopolysaccharidosis I
Interventions
JR-171
Drug
Lead sponsor
JCR Pharmaceuticals Co., Ltd.
Industry
Eligibility
Not listed
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Aug 27, 2025 · Synced May 22, 2026, 3:53 AM EDT
Active, not recruiting No phase listed Observational
View directory record Official record
Conditions
Hemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II
Interventions
SB-318, SB-913, SB-FIX
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
5
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated May 2, 2024 · Synced May 22, 2026, 3:53 AM EDT
Terminated Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Mucopolysaccharidosis Type 3 B
Interventions
rAAV9.CMV.hNAGLU
Biological
Lead sponsor
Abeona Therapeutics, Inc
Industry
Eligibility
Not listed
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated May 4, 2022 · Synced May 22, 2026, 3:53 AM EDT