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Showing 1–24 of 124 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Glycogen Storage Disorders Clear all
Completed No phase listed Observational

Identification and Characterization of Novel Non-Coding Variants That Contribute to Genetic Disorders

NCT04399694
View directory record Official record
Conditions
Genetic Disease, Inborn Errors of Metabolism, Glycogen Storage Disease, Lysosomal Storage Diseases, Storage Disease
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 14, 2026 · Synced Sep 5, 2026, 2:30 PM EDT
Recruiting No phase listed Observational

Accuracy of Home Lactate Meter and Accu-chek Glucometer in Patients With Glycogen Storage Disease

NCT07459582
View directory record Official record
Conditions
Glycogen Storage Disease Type IA, Glycogen Storage Disease Type I, Glycogen Storage Disease Type IB, Glycogen Storage Disease Xi
Interventions
Lactate Meter, Accu Chek
Other · Device
Lead sponsor
Connecticut Children's Medical Center
Other
Eligibility
Up to 60 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Hartford, Connecticut
Source: ClinicalTrials.gov public record
Updated May 31, 2026 · Synced Sep 5, 2026, 2:30 PM 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 12, 2014 · Synced Sep 5, 2026, 2:30 PM EDT
Completed Phase 3 Interventional Results available

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

NCT00158600
View directory record Official record
Conditions
Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions
alglucosidase alfa, Placebo
Biological · Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
8 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
5
States / cities
Beverly Hills, California • Washington D.C., District of Columbia • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 27, 2015 · Synced Sep 5, 2026, 2:30 PM EDT
Completed Phase 3 Interventional Results available

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

NCT04138277
View directory record Official record
Conditions
Pompe Disease (Late-onset)
Interventions
AT2221, ATB200
Drug · Biological
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
119 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
18
States / cities
Little Rock, Arkansas • Irvine, California • Gainesville, Florida + 14 more
Source: ClinicalTrials.gov public record
Updated Mar 4, 2026 · Synced Sep 5, 2026, 2:30 PM EDT
Recruiting No phase listed Observational

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

NCT06636383
View directory record Official record
Conditions
Glycogen Storage Disease Type Ia
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
2 Years and older
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2036
U.S. locations
9
States / cities
Orange, California • Denver, Colorado • Hartford, Connecticut + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Sep 5, 2026, 2:30 PM EDT
Completed No phase listed Observational

Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

NCT01451879
View directory record Official record
Conditions
Pompe Disease
Interventions
Rituximab, Miglustat
Drug
Lead sponsor
University of Florida
Other
Eligibility
Up to 65 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2017
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Dec 7, 2017 · Synced Sep 5, 2026, 2:30 PM EDT
Recruiting No phase listed Observational

A Global Prospective Observational Registry of Patients With Pompe Disease

NCT06121011
View directory record Official record
Conditions
Pompe Disease
Interventions
Cipaglucosidase alfa, Miglustat, Alglucosidase alfa or Avalglucosidase alfa, Untreated
Biological · Drug · Other
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2034
U.S. locations
15
States / cities
Little Rock, Arkansas • Irvine, California • Jacksonville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2026 · Synced Sep 5, 2026, 2:30 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa)

NCT06735755
View directory record Official record
Conditions
Glycogen Storage Disease Type Ia
Interventions
BEAM-301: Single dose of BEAM-301 administered by IV
Drug
Lead sponsor
Beam Therapeutics Inc.
Industry
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
3
States / cities
Orange, California • Farmington, Connecticut • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Dec 14, 2025 · Synced Sep 5, 2026, 2:30 PM EDT
Terminated Early Phase 1 Interventional

A Pilot Study of Pyridostigmine in Pompe Disease

NCT02357225
View directory record Official record
Conditions
Pompe Disease
Interventions
Pyridostigmine Bromide
Drug
Lead sponsor
University of Florida
Other
Eligibility
8 Years to 60 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated May 14, 2018 · Synced Sep 5, 2026, 2:30 PM EDT
Terminated No phase listed Observational

Prospective Collection of Biospecimen in Pediatric Patients and Adult Guardians Diagnosed With Glycogen Storage Disease Type 1B (GSD1b)

NCT05915910
View directory record Official record
Conditions
Glycogen Storage Disease Type IB
Interventions
Specimen Donation
Diagnostic Test
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
4 Years to 85 Years
Enrollment
6 participants
Timeline
2023 – 2024
U.S. locations
1
States / cities
Woburn, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 18, 2024 · Synced Sep 5, 2026, 2:30 PM EDT
Active, not recruiting No phase listed Observational

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

NCT04848779
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
Alglucosidase alfa GZ419829
Drug
Lead sponsor
Sanofi
Industry
Eligibility
0 Days to 6 Months
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
5
States / cities
Hawthorne, New York • Durham, North Carolina • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2026 · Synced Sep 5, 2026, 2:30 PM EDT
Completed No phase listed Observational

Natural History and Functional Status Study of Patients With Lafora Disease

NCT03876522
View directory record Official record
Conditions
Lafora Disease
Interventions
Not listed
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
5 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
2
States / cities
Los Angeles, California • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Sep 27, 2022 · Synced Sep 5, 2026, 2:30 PM EDT
Completed Phase 3 Interventional Results available

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

NCT03729362
View directory record Official record
Conditions
Pompe Disease (Late-onset)
Interventions
Cipaglucosidase Alfa, Miglustat, Alglucosidase Alfa, Placebo
Biological · Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
125 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
25
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Irvine, California + 21 more
Source: ClinicalTrials.gov public record
Updated Sep 10, 2025 · Synced Sep 5, 2026, 2:30 PM EDT
Recruiting No phase listed Observational

Pompe Pregnancy Sub-Registry

NCT00567073
View directory record Official record
Conditions
Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-onset), Glycogenesis 2 Acid Maltase Deficiency
Interventions
Not listed
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2034
U.S. locations
15
States / cities
Phoenix, Arizona • Jacksonville, Florida • Atlanta, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced Sep 5, 2026, 2:30 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Transfer Study in Patients With Late Onset Pompe Disease

NCT04174105
View directory record Official record
Conditions
Pompe Disease (Late-onset)
Interventions
zocaglusagene nuzaparvovec
Genetic
Lead sponsor
Astellas Gene Therapies
Industry
Eligibility
18 Years to 80 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2035
U.S. locations
3
States / cities
Orange, California • Palo Alto, California • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Sep 5, 2026, 2:30 PM EDT
Active, not recruiting No phase listed Observational

Developing a Management Approach for Patients With "Late-Onset" Pompe Disease

NCT03694561
View directory record Official record
Conditions
Pompe Disease, Pompe Disease (Late-onset), GAA Deficiency
Interventions
Observational
Other
Lead sponsor
Duke University
Other
Eligibility
3 Months to 54 Months
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Sep 5, 2026, 2:30 PM EDT
Completed Not applicable Interventional

Diet and Exercise in Pompe Disease

NCT02363153
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II, Lysosomal Storage Diseases
Interventions
Diet and Exercise
Other
Lead sponsor
University of Florida
Other
Eligibility
15 Years to 55 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated May 13, 2021 · Synced Sep 5, 2026, 2:30 PM EDT
Completed No phase listed Observational

Pompe Telemedicine Developmental Study

NCT02950298
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease II
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
6 Years to 18 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 27, 2021 · Synced Sep 5, 2026, 2:30 PM EDT
Completed Phase 2 Interventional Results available

Drug-drug Interaction Study

NCT01380743
View directory record Official record
Conditions
Pompe Disease
Interventions
duvoglustat, rhGAA
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
11
States / cities
Phoenix, Arizona • Scottsdale, Arizona • Orange, California + 8 more
Source: ClinicalTrials.gov public record
Updated Jun 25, 2025 · Synced Sep 5, 2026, 2:30 PM EDT
Completed No phase listed Observational

Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease

NCT00113035
View directory record Official record
Conditions
Pompe Disease
Interventions
Not listed
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
8 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005
U.S. locations
5
States / cities
Beverly Hills, California • Washington D.C., District of Columbia • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated May 4, 2015 · Synced Sep 5, 2026, 2:30 PM EDT
Completed No phase listed Observational

Study of Glycogen Storage Disease Expression in Carriers

NCT02057731
View directory record Official record
Conditions
Glycogen Storage Disease
Interventions
Glycogen Storage Disease markers
Genetic
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 100 Years
Enrollment
114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Dec 1, 2016 · Synced Sep 5, 2026, 2:30 PM EDT
Recruiting No phase listed Observational

GBE Deficiency (GSD IV and APBD) Natural History Study

NCT02683512
View directory record Official record
Conditions
Glycogen Storage Disease Type IV, Adult Polyglucosan Body Disease, GSD4, GSD IV, APBD
Interventions
No intervention
Other
Lead sponsor
Duke University
Other
Eligibility
0 Years to 90 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2035
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 4, 2026 · Synced Sep 5, 2026, 2:30 PM EDT
Not yet recruiting Phase 2 Interventional

Evaluating the Efficacy and Safety of D-galactose in PGM1-CDG (AVTX-801)

NCT05402332
View directory record Official record
Conditions
PGM1-CDG - Phosphoglucomutase 1-Related Congenital Disorder of Glycosylation
Interventions
AVTX-801, Placebo
Drug
Lead sponsor
Eva Morava-Kozicz
Other
Eligibility
18 Years to 60 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 15, 2026 · Synced Sep 5, 2026, 2:30 PM EDT