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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Glycogen Storage Disease Type II Clear all
Completed Phase 1Phase 2 Interventional

Prophylactic Ranibizumab for Exudative Age-related Macular Degeneration

NCT02140151
View directory record Official record
Conditions
Age-related Macular Degeneration
Interventions
Ranibizumab 0.5mg
Drug
Lead sponsor
Southern California Desert Retina Consultants, MC
Other
Eligibility
50 Years and older
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
4
States / cities
Mountain View, California • Palm Desert, California • Baltimore, Maryland + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 24, 2022 · Synced Sep 28, 2026, 2:51 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy

NCT01942590
View directory record Official record
Conditions
Pompe Disease
Interventions
Clenbuterol, Placebo
Drug
Lead sponsor
Dwight Koeberl, M.D., Ph.D.
Other
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 1, 2019 · Synced Sep 28, 2026, 2:51 PM EDT
Completed Early Phase 1 Interventional

Study to Assess the Safety, Tolerability, PK and PD of ABX1100

NCT06109948
View directory record Official record
Conditions
Healthy, Late Onset Pompe Disease
Interventions
ABX1100 injection for IV infusion, Placebo injection for IV infusion
Drug
Lead sponsor
Aro Biotherapeutics
Industry
Eligibility
18 Years and older
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
3
States / cities
Orange, California • St Louis, Missouri • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced Sep 28, 2026, 2:51 PM 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 Sep 28, 2026, 2:51 PM EDT
Recruiting No phase listed Observational

Cognitive and Neurological Pathologies in Pompe Disease

NCT04639336
View directory record Official record
Conditions
Pompe Disease
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
0 Years to 18 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2029
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 3, 2026 · Synced Sep 28, 2026, 2:51 PM EDT
Terminated Phase 2 Interventional Results available

Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease

NCT00688597
View directory record Official record
Conditions
Pompe Disease
Interventions
Duvoglustat
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 74 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Sep 10, 2025 · Synced Sep 28, 2026, 2:51 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Investigating Lysosomal Storage Diseases in Minority Groups

NCT02120235
View directory record Official record
Conditions
Lysosomal Storage Disorders, Gaucher Disease, Fabry Disease, Pompe Disease, Niemann-Pick Disease
Interventions
Not listed
Lead sponsor
O & O Alpan LLC
Other
Eligibility
1 Day to 100 Years
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2018
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 3, 2017 · Synced Sep 28, 2026, 2:51 PM EDT
Not listed No phase listed Observational

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

NCT06539169
View directory record Official record
Conditions
Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
Interventions
Not listed
Lead sponsor
xCures
Industry
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Los Altos, California
Source: ClinicalTrials.gov public record
Updated Nov 13, 2024 · Synced Sep 28, 2026, 2:51 PM 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 Sep 28, 2026, 2:51 PM EDT
Withdrawn Phase 2 Interventional

High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease

NCT01656590
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
High Protein and Exercise Therapy along-with Nocturnal Enteral Feeding
Other
Lead sponsor
Columbia University
Other
Eligibility
8 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 12, 2015 · Synced Sep 28, 2026, 2:51 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 28, 2026, 2:51 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 28, 2026, 2:51 PM EDT
Completed Phase 2 Interventional Results available

Avalglucosidase Alfa Extension Study

NCT02032524
View directory record Official record
Conditions
Glycogen Storage Disease Type II Pompe Disease
Interventions
Avalglucosidase Alfa
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2022
U.S. locations
8
States / cities
Phoenix, Arizona • Jacksonville, Florida • Kansas City, Kansas + 5 more
Source: ClinicalTrials.gov public record
Updated Feb 29, 2024 · Synced Sep 28, 2026, 2:51 PM EDT
Terminated No phase listed Observational

STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT

NCT03347253
View directory record Official record
Conditions
Late-onset Pompe Disease
Interventions
Not listed
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
17
States / cities
Irvine, California • Gainesville, Florida • Decatur, Georgia + 14 more
Source: ClinicalTrials.gov public record
Updated Jul 7, 2025 · Synced Sep 28, 2026, 2:51 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease

NCT01885936
View directory record Official record
Conditions
Pompe Disease
Interventions
Albuterol, Placebo
Drug
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 14, 2019 · Synced Sep 28, 2026, 2:51 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Satiety and Glucose Indices in Adults

NCT02929849
View directory record Official record
Conditions
Obesity
Interventions
300 mg SC, 500 mg SC, Placebo
Dietary Supplement
Lead sponsor
Rutgers University
Other
Eligibility
20 Years to 59 Years
Enrollment
51 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2017
U.S. locations
1
States / cities
New Brunswick, New Jersey
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced Sep 28, 2026, 2:51 PM 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 23, 2024 · Synced Sep 28, 2026, 2:51 PM EDT
Completed No phase listed Observational

Alglucosidase Alfa Pompe Safety Sub-Registry

NCT01710813
View directory record Official record
Conditions
Pompe Disease
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
6
States / cities
Phoenix, Arizona • Durham, North Carolina • Hershey, Pennsylvania + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 12, 2021 · Synced Sep 28, 2026, 2:51 PM EDT
Completed Phase 1Phase 2 Interventional

rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)

NCT00053573
View directory record Official record
Conditions
Glycogen Storage Disease Type II, Pompe Disease, Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
6 Months to 36 Months
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
3
States / cities
Gainesville, Florida • Durham, North Carolina • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 4, 2014 · Synced Sep 28, 2026, 2:51 PM EDT
Recruiting Phase 1 Interventional

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

NCT07354724
View directory record Official record
Conditions
Late-onset Pompe Disease
Interventions
DNL952
Drug
Lead sponsor
Denali Therapeutics Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
4
States / cities
Irvine, California • St Louis, Missouri • Durham, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 16, 2026 · Synced Sep 28, 2026, 2:51 PM EDT
Completed Not applicable Interventional

Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)

NCT03255213
View directory record Official record
Conditions
Glycogen Storage Disease
Interventions
Lingual Muscle Training
Other
Lead sponsor
Duke University
Other
Eligibility
12 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 27, 2021 · Synced Sep 28, 2026, 2:51 PM EDT
Completed Phase 3 Interventional Results available

Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease

NCT02782741
View directory record Official record
Conditions
Glycogen Storage Disease Type II;Pompe's Disease
Interventions
Avalglucosidase alfa (GZ402666), Alglucosidase alfa (GZ419829)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
3 Years and older
Enrollment
101 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
21
States / cities
Phoenix, Arizona • Los Angeles, California • Orange, California + 18 more
Source: ClinicalTrials.gov public record
Updated Apr 3, 2024 · Synced Sep 28, 2026, 2:51 PM EDT
Completed Phase 4 Interventional Results available

High Dose or High Dose Frequency Study of Alglucosidase Alfa

NCT00483379
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
6 Months and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
9
States / cities
Birmingham, Alabama • Stanford, California • Washington D.C., District of Columbia + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 6, 2014 · Synced Sep 28, 2026, 2:51 PM EDT