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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Lysosomal Storage Disorder Clear all
Terminated No phase listed Observational

Natural History Study of Children With Metachromatic Leukodystrophy

NCT01963650
View directory record Official record
Conditions
Lipid Metabolism Disorders, Metachromatic Leukodystrophy (MLD), Nervous System Diseases, Brain Diseases, Central Nervous System Diseases, Demyelinating Diseases, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Sphingolipidoses, Hereditary Central Nervous System Demyelinating Diseases, Metabolic Inborn Brain Diseases, Lysosomal Storage Diseases, Metabolic Diseases, Sulfatidosis
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
Up to 12 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
4
States / cities
Torrance, California • Washington D.C., District of Columbia • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 16, 2021 · Synced Sep 23, 2026, 5:02 PM EDT
Completed No phase listed Observational

Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion

NCT05186324
View directory record Official record
Conditions
Fabry Disease
Interventions
Interview
Other
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
Not listed
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
7
States / cities
Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2023 · Synced Sep 23, 2026, 5:02 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

NCT06149403
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 Sep 23, 2026, 5:02 PM EDT
Recruiting Phase 2 Interventional

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

NCT07123155
View directory record Official record
Conditions
Pompe Disease
Interventions
S-606001, Placebo
Drug
Lead sponsor
Shionogi
Industry
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
9
States / cities
Irvine, California • Gainesville, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Sep 23, 2026, 5:02 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

NCT07282847
View directory record Official record
Conditions
Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
Interventions
AB-1009 (GAA Gene)
Genetic
Lead sponsor
AskBio Inc
Industry
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
9
States / cities
Phoenix, Arizona • Irvine, California • Palo Alto, California + 6 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2026 · Synced Sep 23, 2026, 5:02 PM EDT
Completed Not applicable Interventional

A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I

NCT00852358
View directory record Official record
Conditions
Cognitive Decline, Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Scheie Syndrome, Lysosomal Storage Disease
Interventions
laronidase
Drug
Lead sponsor
Patricia I. Dickson, M.D.
Individual
Eligibility
6 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
3
States / cities
Oakland, California • Torrance, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 25, 2016 · Synced Sep 23, 2026, 5:02 PM EDT
Completed No phase listed Observational

Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices

NCT02838368
View directory record Official record
Conditions
Pompe Disease
Interventions
Not listed
Lead sponsor
University of California, Irvine
Other
Eligibility
8 Years and older
Enrollment
921 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated Feb 24, 2019 · Synced Sep 23, 2026, 5:02 PM EDT
Completed Phase 1Phase 2 Interventional

Scotoma Reduction in AMD Patients Treated With Ranibizumab

NCT00467935
View directory record Official record
Conditions
Macular Degeneration
Interventions
intravitreal injection Lucentis (ranibizumab)
Drug
Lead sponsor
Retina Research Foundation
Other
Eligibility
50 Years to 90 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007
U.S. locations
1
States / cities
Slingerlands, New York
Source: ClinicalTrials.gov public record
Updated Jul 28, 2011 · Synced Sep 23, 2026, 5:02 PM EDT
Withdrawn Phase 2 Interventional

Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease

NCT04094948
View directory record Official record
Conditions
Pompe Disease (Late-onset)
Interventions
Clenbuterol, Placebos
Drug
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Aug 16, 2022 · Synced Sep 23, 2026, 5:02 PM EDT
Completed Phase 2 Interventional Results available

Stem Cell Transplantation for Hurler

NCT00176917
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Mannosidosis, Mucolipidosis Type II (I-cell Disease)
Interventions
Stem Cell Transplant, Busulfan, Cyclophosphamide, ATG
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 27, 2017 · Synced Sep 23, 2026, 5:02 PM EDT
Completed No phase listed Observational

Establishment of Biomarkers for Fabry Disease

NCT01165697
View directory record Official record
Conditions
Fabry Disease
Interventions
Fluorescein angiography
Other
Lead sponsor
Ohio State University
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 12, 2017 · Synced Sep 23, 2026, 5:02 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

NCT00069641
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
iduronate-2-sulfatase enzyme replacement therapy, Placebo
Biological
Lead sponsor
Shire
Industry
Eligibility
5 Years to 25 Years · Male only
Enrollment
96 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
4
States / cities
Oakland, California • St Louis, Missouri • Chapel Hill, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2021 · Synced Sep 23, 2026, 5:02 PM EDT
Terminated No phase listed Observational

Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB

NCT02293408
View directory record Official record
Conditions
MPS IIIB (Sanfilippo B Syndrome)
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
1 Year and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
2
States / cities
Minneapolis, Minnesota • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 12, 2018 · Synced Sep 23, 2026, 5:02 PM EDT
Completed No phase listed Observational

Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up

NCT01884220
View directory record Official record
Conditions
Wolman Disease, Cholesterol Ester Storage Disease, Acid Cholesteryl Ester Hydrolase Deficiency, Type 2
Interventions
There are no interventions in this study.
Other
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 27, 2015 · Synced Sep 23, 2026, 5:02 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

NCT06545136
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
FLT201
Genetic
Lead sponsor
Spur Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Nov 3, 2025 · Synced Sep 23, 2026, 5:02 PM EDT
Available No phase listed Expanded access

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

NCT05795361
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Idursulfase-IT
Drug
Lead sponsor
Takeda
Industry
Eligibility
Male only
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
17
States / cities
Phoenix, Arizona • Oakland, California • Wilmington, Delaware + 14 more
Source: ClinicalTrials.gov public record
Updated Apr 12, 2026 · Synced Sep 23, 2026, 5:02 PM EDT
Terminated No phase listed Observational

The Natural History of Mucolipidosis Type IV

NCT01067742
View directory record Official record
Conditions
Mucolipidosis Type IV
Interventions
Not listed
Lead sponsor
Baylor Research Institute
Other
Eligibility
1 Year to 64 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2021
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 25, 2026 · Synced Sep 23, 2026, 5:02 PM EDT
Completed No phase listed Observational

Clinical Biomarkers in Alpha-mannosidosis

NCT02141503
View directory record Official record
Conditions
Alpha-Mannosidosis
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
5 Years to 60 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 2, 2019 · Synced Sep 23, 2026, 5:02 PM EDT
Completed No phase listed Observational

A Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP)

NCT06064656
View directory record Official record
Conditions
Fibrodysplasia Ossificans Progressiva (FOP)
Interventions
Non-Interventional
Other
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
131 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
1
States / cities
Tarrytown, New York
Source: ClinicalTrials.gov public record
Updated Jul 2, 2025 · Synced Sep 23, 2026, 5:02 PM EDT
Active, not recruiting No phase listed Observational

Long Term Follow-Up for Safety of AVR-RD-02

NCT06488261
View directory record Official record
Conditions
Gaucher Disease
Interventions
AVR-RD-02
Biological
Lead sponsor
John Bernat
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2037
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Jul 21, 2025 · Synced Sep 23, 2026, 5:02 PM EDT
Not listed No phase listed Observational

A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

NCT03227042
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 Sep 23, 2026, 5:02 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients

NCT01678898
View directory record Official record
Conditions
Fabry Disease
Interventions
PRX-102
Drug
Lead sponsor
Protalix
Industry
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
9
States / cities
Sacramento, California • Atlanta, Georgia • Iowa City, Iowa + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 12, 2023 · Synced Sep 23, 2026, 5:02 PM EDT
Completed No phase listed Observational

Registry of Fabry Disease - A Multicenter Observational Study

NCT00055016
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2008
U.S. locations
2
States / cities
Bethesda, Maryland • Cambridge, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Sep 23, 2026, 5:02 PM EDT
Completed No phase listed Observational

Identification of Tongue Involvement in Late-Onset Pompe Disease

NCT02765828
View directory record Official record
Conditions
Myopathy, Neuropathy, Glycogen Storage Disease Type II (Late-onset Pompe Disease)
Interventions
Observational study
Other
Lead sponsor
Duke University
Other
Eligibility
12 Years and older
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 5, 2021 · Synced Sep 23, 2026, 5:02 PM EDT