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ClinicalTrials.gov public records Last synced Jul 23, 2026, 10:54 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: Glycogen Metabolism Clear all
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 Jul 23, 2026, 10:54 AM EDT
Completed Phase 1 Interventional

Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.

NCT01898364
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD II), Acid Maltase Deficiency
Interventions
GZ402666
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
7
States / cities
Phoenix, Arizona • Jacksonville, Florida • Kansas City, Kansas + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 22, 2023 · Synced Jul 23, 2026, 10:54 AM EDT
Completed No phase listed Observational

Pompe Lactation Sub-Registry

NCT00566878
View directory record Official record
Conditions
Glycogen Storage Disease, Pompe Disease
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Female only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2024
U.S. locations
2
States / cities
Cambridge, Massachusetts • Grand Rapids, Michigan
Source: ClinicalTrials.gov public record
Updated Mar 18, 2024 · Synced Jul 23, 2026, 10:54 AM EDT
Completed Phase 4 Interventional Results available

Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

NCT01410890
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD II)
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
4
States / cities
Valhalla, New York • Cincinnati, Ohio • Salt Lake City, Utah + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 27, 2022 · Synced Jul 23, 2026, 10:54 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 1, 2019 · Synced Jul 23, 2026, 10:54 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease

NCT00976352
View directory record Official record
Conditions
Pompe Disease
Interventions
rAAV1-CMV-GAA (study agent) Administration, RMST
Drug · Other
Lead sponsor
University of Florida
Other
Eligibility
2 Years to 18 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Sep 13, 2018 · Synced Jul 23, 2026, 10:54 AM 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 Jul 23, 2026, 10:54 AM EDT
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 Jul 23, 2026, 10:54 AM EDT
Approved For Marketing No phase listed Expanded access

Alglucosidase Alfa Temporary Access Program

NCT00520143
View directory record Official record
Conditions
Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-Onset), Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions
alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
U.S. locations
74
States / cities
Sheffield, Alabama • Anchorage, Alaska • Phoenix, Arizona + 71 more
Source: ClinicalTrials.gov public record
Updated Feb 5, 2014 · Synced Jul 23, 2026, 10:54 AM EDT
Completed Not applicable Interventional Results available

Repeated Eye Injections of Aflibercept for Treatment of Wet Age Related Macular Degeneration

NCT01773954
View directory record Official record
Conditions
Age-Related Macular Degeneration, Macular Degeneration
Interventions
Intravitreal Aflibercept Injection
Drug
Lead sponsor
MidAtlantic Retina
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
3
States / cities
Cherry Hill, New Jersey • Huntingdon Valley, Pennsylvania • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 1, 2018 · Synced Jul 23, 2026, 10:54 AM EDT
Completed Early Phase 1 Interventional

Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I

NCT03665636
View directory record Official record
Conditions
Glycogen Storage Disease Type I
Interventions
Triheptanoin
Drug
Lead sponsor
Areeg El-Gharbawy
Other
Eligibility
1 Month to 65 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Dec 14, 2021 · Synced Jul 23, 2026, 10:54 AM EDT
Recruiting Phase 2 Interventional

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

NCT06092034
View directory record Official record
Conditions
Danon Disease
Interventions
RP-A501
Genetic
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years and older · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2032
U.S. locations
3
States / cities
La Jolla, California • Boston, Massachusetts • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 3, 2026 · Synced Jul 23, 2026, 10:54 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)

NCT03517085
View directory record Official record
Conditions
GSD1
Interventions
DTX401, steroid regimen
Genetic · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
3
States / cities
Farmington, Connecticut • Ann Arbor, Michigan • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 17, 2022 · Synced Jul 23, 2026, 10:54 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 12, 2014 · Synced Jul 23, 2026, 10:54 AM EDT
Completed No phase listed Observational

Pregnancy and Birth Outcome in Women With Pompe Disease

NCT01556516
View directory record Official record
Conditions
Pompe Disease
Interventions
Not listed
Lead sponsor
O & O Alpan LLC
Other
Eligibility
18 Years and older · Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Sep 27, 2016 · Synced Jul 23, 2026, 10:54 AM EDT
Terminated Phase 4 Interventional Results available

Immune Tolerance Induction Study

NCT00701701
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
Interventions
Myozyme® (alglucosidase alfa)
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
1 Month and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2020
U.S. locations
4
States / cities
Louisville, Kentucky • Durham, North Carolina • Salt Lake City, Utah + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 6, 2022 · Synced Jul 23, 2026, 10:54 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

NCT04093349
View directory record Official record
Conditions
Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
Interventions
SPK-3006
Genetic
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2032
U.S. locations
10
States / cities
Phoenix, Arizona • Orange, California • Atlanta, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 26, 2024 · Synced Jul 23, 2026, 10:54 AM EDT
Completed Phase 1 Interventional

AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease

NCT03533673
View directory record Official record
Conditions
Pompe Disease
Interventions
ACTUS-101
Genetic
Lead sponsor
AskBio Inc
Industry
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 12, 2026 · Synced Jul 23, 2026, 10:54 AM EDT
Recruiting Not applicable Interventional

Very Low Carbohydrate Diets and Glucagon Response in T1DM

NCT04200391
View directory record Official record
Conditions
Type1diabetes
Interventions
Very low carbohydrate diet
Other
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Years to 40 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 17, 2024 · Synced Jul 23, 2026, 10:54 AM 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 Jun 9, 2026 · Synced Jul 23, 2026, 10:54 AM 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 Jul 23, 2026, 10:54 AM EDT
Not listed No phase listed Observational

Clinical Specimen Collection From Pompe Disease Patients

NCT04476550
View directory record Official record
Conditions
Pompe Disease
Interventions
Filgrastim
Drug
Lead sponsor
Serhat Gumrukcu, MD PhD
Other
Eligibility
3 Years to 30 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Toluca Lake, California
Source: ClinicalTrials.gov public record
Updated May 18, 2022 · Synced Jul 23, 2026, 10:54 AM EDT
Completed No phase listed Observational Results available

Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

NCT03970278
View directory record Official record
Conditions
Glycogen Storage Disease Type IA, Von Gierke's Disease (GSD Type Ia)
Interventions
No intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
3
States / cities
Farmington, Connecticut • Ann Arbor, Michigan • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced Jul 23, 2026, 10:54 AM 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 Jul 23, 2026, 10:54 AM EDT