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ClinicalTrials.gov public records Last synced Oct 3, 2026, 10:58 AM EDT

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Showing 97–120 of 594 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inherited Metabolic Storage Diseases Clear all
Withdrawn Phase 1 Interventional

Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

NCT05238324
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 28, 2023 · Synced Oct 3, 2026, 10:58 AM EDT
Completed No phase listed Observational

Psychological Concomitants of Morquio Syndrome (The MAP Study)

NCT01752296
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 4, 2013 · Synced Oct 3, 2026, 10:58 AM EDT
Completed Phase 4 Interventional Results available

Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

NCT01288027
View directory record Official record
Conditions
Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenesis 2 Acid Maltase Deficiency
Interventions
Alglucosidase Alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
9
States / cities
Orange, California • Gainesville, Florida • Kansas City, Kansas + 6 more
Source: ClinicalTrials.gov public record
Updated Dec 19, 2014 · Synced Oct 3, 2026, 10:58 AM EDT
Completed Phase 4 Interventional Results available

An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease

NCT00701129
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II
Interventions
Alglucosidase Alfa, Methotrexate, Rituximab
Biological · Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
2
States / cities
Louisville, Kentucky • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 13, 2014 · Synced Oct 3, 2026, 10:58 AM EDT
Withdrawn Phase 1 Interventional

A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis

NCT00679744
View directory record Official record
Conditions
G(M2) Ganglioside, Tay-Sachs Disease Ganglioside, Sandhoff Disease Ganglioside
Interventions
Pyrimethamine
Drug
Lead sponsor
Exsar Corporation
Industry
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2008
U.S. locations
2
States / cities
New York, New York • Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 25, 2013 · Synced Oct 3, 2026, 10:58 AM EDT
Completed Phase 2 Interventional Results available

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

NCT01043640
View directory record Official record
Conditions
Mucopolysaccharidosis, Hurler Syndrome, Hunter Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Adrenoleukodystrophy (ALD), Krabbe Disease, Metachromatic Leukodystrophy (MLD), Sphingolipidoses, Peroxisomal Disorders
Interventions
Campath-1H, Cyclophosphamide, Busulfan, Allogeneic stem cell transplantation, Cyclosporine A, Mycophenolate Mofetil
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 21 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2017
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 5, 2018 · Synced Oct 3, 2026, 10:58 AM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease

NCT02354664
View directory record Official record
Conditions
Pompe Disease
Interventions
Thoracic MRI, Spirometry, Inspiratory Load Compensation, Maximal Inspiratory Pressure, Resting Breathing Pattern, Respiratory Muscle Endurance Test
Other
Lead sponsor
University of Florida
Other
Eligibility
20 Years to 65 Years
Enrollment
14 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2016
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jun 24, 2024 · Synced Oct 3, 2026, 10:58 AM EDT
Completed Not applicable Interventional Results available

Evaluation of the Gastrointestinal Manifestation of Fabry's Disease

NCT02798458
View directory record Official record
Conditions
Fabry's Disease
Interventions
Smartpill, Endoscopic Mucosal Resection
Device · Procedure
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 70 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 27, 2023 · Synced Oct 3, 2026, 10:58 AM EDT
Completed No phase listed Observational

Pompe Gene Therapy- Screening for Eligibility

NCT03285126
View directory record Official record
Conditions
Pompe Disease (Late-onset)
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 2, 2019 · Synced Oct 3, 2026, 10:58 AM EDT
Completed Phase 3 Interventional Results available

A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease

NCT00376168
View directory record Official record
Conditions
Gaucher Disease
Interventions
Plant cell expressed recombinant glucocerebrosidase (prGCD)
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
3
States / cities
Coral Springs, Florida • Decatur, Georgia • New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 4, 2018 · Synced Oct 3, 2026, 10:58 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1

NCT03887533
View directory record Official record
Conditions
Niemann-Pick Disease, Type C1
Interventions
VTS-270
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
3 Years to 60 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 30, 2022 · Synced Oct 3, 2026, 10:58 AM EDT
Recruiting Phase 1 Interventional

ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I

NCT05682144
View directory record Official record
Conditions
Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
Interventions
Autologous Plasmablasts (B cells)
Biological
Lead sponsor
Immusoft of CA, Inc.
Industry
Eligibility
10 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2044
U.S. locations
2
States / cities
Oakland, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 17, 2026 · Synced Oct 3, 2026, 10:58 AM EDT
Completed Phase 1 Interventional

New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis

NCT00001736
View directory record Official record
Conditions
Cystinosis
Interventions
Cysteamine
Drug
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
Not listed
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 3, 2026, 10:58 AM EDT
Completed Phase 1Phase 2 Interventional

A Treatment Study of Mucopolysaccharidosis Type IIIB

NCT02754076
View directory record Official record
Conditions
MPS III B, Mucopolysaccharidosis Type IIIB
Interventions
AX 250
Drug
Lead sponsor
Allievex Corporation
Industry
Eligibility
1 Year to 10 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Aug 6, 2020 · Synced Oct 3, 2026, 10:58 AM EDT
Active, not recruiting Phase 3 Interventional

Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

NCT05222906
View directory record Official record
Conditions
Gaucher's Disease Type III
Interventions
Venglustat, imiglucerase
Drug
Lead sponsor
Sanofi
Industry
Eligibility
12 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
4
States / cities
New Haven, Connecticut • Iowa City, Iowa • Dallas, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Oct 3, 2026, 10:58 AM EDT
Completed No phase listed Observational

Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)

NCT01822184
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 17, 2021 · Synced Oct 3, 2026, 10:58 AM EDT
Completed No phase listed Observational

Immune Response in Subjects With Fabry Disease Who Are Switching From Agalsidase Alfa to Agalsidase Beta

NCT01745185
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
O & O Alpan LLC
Other
Eligibility
7 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 4, 2017 · Synced Oct 3, 2026, 10:58 AM EDT
Completed Phase 2 Interventional Accepts healthy volunteers

Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)

NCT02583672
View directory record Official record
Conditions
Gaucher Disease Type 1
Interventions
N-acetylcysteine
Drug
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
33 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2025
U.S. locations
2
States / cities
Minneapolis, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 18, 2026 · Synced Oct 3, 2026, 10:58 AM EDT
Completed No phase listed Observational

Neurobehavioral Phenotypes in MPS III

NCT01873911
View directory record Official record
Conditions
Sanfilippo Syndrome Type A, Sanfilippo Syndrome Type B, Hurler Syndrome
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
2 Years to 12 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 1, 2019 · Synced Oct 3, 2026, 10:58 AM EDT
Terminated Not applicable Interventional Results available

Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

NCT02156674
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 23, 2020 · Synced Oct 3, 2026, 10:58 AM EDT
Not listed No phase listed Observational

The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)

NCT05705674
View directory record Official record
Conditions
Sanfilippo Syndrome, MPS3
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
5 Years to 99 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Mar 12, 2024 · Synced Oct 3, 2026, 10:58 AM EDT
Not listed No phase listed Observational

Podocyturia - Predictor of Renal Dysfunction in Fabry Nephropathy

NCT02994303
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
University of Washington
Other
Eligibility
1 Day to 90 Years
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
3
States / cities
Decatur, Georgia • Minneapolis, Minnesota • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Aug 28, 2017 · Synced Oct 3, 2026, 10:58 AM EDT
Completed No phase listed Observational

The Natural History of Infantile Globoid Cell Leukodystrophy

NCT00983879
View directory record Official record
Conditions
Infantile Globoid Cell Leukodystrophy
Interventions
Not listed
Lead sponsor
Zymenex A/S
Industry
Eligibility
Up to 2 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 8, 2018 · Synced Oct 3, 2026, 10:58 AM EDT
Terminated No phase listed Observational

A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101

NCT04655911
View directory record Official record
Conditions
Mucopolysaccharidosis III-B
Interventions
ABO-101
Biological
Lead sponsor
Abeona Therapeutics, Inc
Industry
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated May 31, 2022 · Synced Oct 3, 2026, 10:58 AM EDT