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ClinicalTrials.gov public records Last synced May 21, 2026, 8:12 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 189 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Mucopolysaccharidosis Clear all
Not listed No phase listed Observational
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIB
Interventions
Not listed
Lead sponsor
Allievex Corporation
Industry
Eligibility
Up to 18 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Oct 25, 2022 · Synced May 21, 2026, 8:12 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
MPS IVA, Morquio Syndrome
Interventions
Not listed
Lead sponsor
Oregon Health and Science University
Other
Eligibility
1 Year and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 16, 2019 · Synced May 21, 2026, 8:12 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
Interventions
Aldurazyme, placebo
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2005
U.S. locations
8
States / cities
Mobile, Alabama • Atlanta, Georgia • Rochester, New York + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2015 · Synced May 21, 2026, 8:12 PM EDT
Completed Phase 3 Interventional
View directory record Official record
Conditions
Achondroplasia
Interventions
Infigratinib 0.25 mg/kg/day, Placebo Comparator 0.25 mg/kg/day
Drug
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
3 Years to 17 Years
Enrollment
114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
7
States / cities
San Francisco, California • Aurora, Colorado • Baltimore, Maryland + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 16, 2026 · Synced May 21, 2026, 8:12 PM EDT
Active, not recruiting No phase listed Observational
View directory record Official record
Conditions
Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I
Interventions
Laronidase therapy and a stem cell transplant
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
0 Years to 3 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 29, 2025 · Synced May 21, 2026, 8:12 PM EDT
Completed Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions
Hematopoietic stem cell transplantation
Procedure
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 22, 2016 · Synced May 21, 2026, 8:12 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
Interventions
Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc.
Diagnostic Test
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Wilmington, Delaware
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced May 21, 2026, 8:12 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Morquio Disease, Mucopolysaccharidosis IV
Interventions
Not listed
Lead sponsor
Nadia Ali, PhD
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 3, 2013 · Synced May 21, 2026, 8:12 PM EDT
Enrolling by invitation No phase listed Observational
View directory record Official record
Conditions
Mucopolysaccharidosis I
Interventions
No Intervention
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Orange, California
Source: ClinicalTrials.gov public record
Updated Apr 28, 2026 · Synced May 21, 2026, 8:12 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Enchondromatosis
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
2 Years to 100 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced May 21, 2026, 8:12 PM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Sanfilippo Syndrome Type A (MPS IIIA)
Interventions
SOBI003
Drug
Lead sponsor
Swedish Orphan Biovitrum
Industry
Eligibility
18 Months to 78 Months
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
2
States / cities
Oakland, California • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Feb 24, 2022 · Synced May 21, 2026, 8:12 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced May 21, 2026, 8:12 PM EDT
Completed Phase 2 Interventional
View directory record Official record
Conditions
Achondroplasia
Interventions
Infigratinib 0.016 mg/kg, Infigratinib 0.032 mg/kg, Infigratinib 0.064 mg/kg, Infigratinib 0.128 mg/kg, Infigratinib 0.25 mg/kg
Drug
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
3 Years to 11 Years
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
5
States / cities
Oakland, California • Wilmington, Delaware • Baltimore, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 21, 2025 · Synced May 21, 2026, 8:12 PM EDT
Enrolling by invitation Phase 4 Interventional
View directory record Official record
Conditions
MPS III B
Interventions
AX 250
Drug
Lead sponsor
Allievex Corporation
Industry
Eligibility
Not listed
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
2
States / cities
Oakland, California • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 8, 2023 · Synced May 21, 2026, 8:12 PM EDT
Completed Phase 1 Interventional Results available
View directory record Official record
Conditions
Hurler Syndrome
Interventions
Laronidase
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 14 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 19, 2020 · Synced May 21, 2026, 8:12 PM EDT
Completed Phase 4 Interventional Results available
View directory record Official record
Conditions
Mucopolysaccharidosis VI, Maroteaux-Lamy Syndrome
Interventions
Naglazyme
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 1 Year
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
2
States / cities
Los Angeles, California • Oakland, California
Source: ClinicalTrials.gov public record
Updated Jul 21, 2011 · Synced May 21, 2026, 8:12 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIA
Interventions
DNL126
Drug
Lead sponsor
Denali Therapeutics Inc.
Industry
Eligibility
0 Years to 18 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
4
States / cities
Oakland, California • Iowa City, Iowa • Chapel Hill, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 4, 2025 · Synced May 21, 2026, 8:12 PM EDT
Not listed Phase 2 Interventional
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIB, MPS III B
Interventions
AX 250
Drug
Lead sponsor
Allievex Corporation
Industry
Eligibility
0 Years to 18 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
2
States / cities
Oakland, California • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 17, 2023 · Synced May 21, 2026, 8:12 PM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
Interventions
Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)
Biological · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
149 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2029
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 6, 2026 · Synced May 21, 2026, 8:12 PM EDT
Completed Phase 4 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Irritable Bowel Syndrome
Interventions
curcumin, placebo
Dietary Supplement
Lead sponsor
Kaiser Permanente
Other
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Mar 10, 2015 · Synced May 21, 2026, 8:12 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Mucopolysaccharidosis I, Hurlers Syndrome, Hurler-Scheie Syndrome
Interventions
rhIDU (recombinant human-Alpha-L-Iduronidase), Placebo
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
5 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2001
U.S. locations
2
States / cities
New York, New York • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 6, 2015 · Synced May 21, 2026, 8:12 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
MPS VI
Interventions
Not listed
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Apr 1, 2014 · Synced May 21, 2026, 8:12 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Sanfilippo Syndrome Type C
Interventions
Not listed
Lead sponsor
Phoenix Nest
Industry
Eligibility
12 Months and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced May 21, 2026, 8:12 PM EDT
Terminated No phase listed Observational
View directory record Official record
Conditions
Inherited Metabolic Disorders (IMD)
Interventions
Safety and efficacy assessments
Other
Lead sponsor
Magenta Therapeutics, Inc.
Industry
Eligibility
Up to 16 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 14, 2021 · Synced May 21, 2026, 8:12 PM EDT