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ClinicalTrials.gov public records Last synced Oct 6, 2026, 11:34 AM EDT

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Showing 49–72 of 97 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Glycogen Storage Disease Type II GSD II Clear all
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 19, 2022 · Synced Oct 6, 2026, 11:34 AM EDT
Recruiting No phase listed Observational

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

NCT01665326
View directory record Official record
Conditions
Pompe Disease
Interventions
Observational
Other
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2029
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Oct 6, 2026, 11:34 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 2, 2018 · Synced Oct 6, 2026, 11:34 AM EDT
Active, not recruiting Phase 2 Interventional Accepts healthy volunteers Results available

A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa

NCT03019406
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
6 Months to 17 Years
Enrollment
22 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2027
U.S. locations
3
States / cities
Valhalla, New York • Durham, North Carolina • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jan 30, 2026 · Synced Oct 6, 2026, 11:34 AM EDT
Terminated Phase 3 Interventional Results available

BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

NCT01924845
View directory record Official record
Conditions
Late-onset Pompe Disease
Interventions
BMN 701
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
8
States / cities
Phoenix, Arizona • Orange, California • Gainesville, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 14, 2018 · Synced Oct 6, 2026, 11:34 AM 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 26, 2025 · Synced Oct 6, 2026, 11:34 AM EDT
Recruiting Not applicable Interventional

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease

NCT07478172
View directory record Official record
Conditions
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome, Primary Lateral Sclerosis, Spinal Muscular Atrophy, Charcot Marie Tooth Disease (CMT), Fascioscapulohumeral Muscular Dystrophy, Inclusion Body Myositis, Mitochondrial Myopathy, Nemaline Myopathy, Centronuclear Myopathy, Postpolio Syndrome, Pompe Disease (Late-onset), Chronic Inflammatory Demyelinating Polyneuropathy, Hereditary Spastic Paraplegia, Postural Orthostatic Tachycardia Syndrome (POTS), Progressive Muscular Atrophy
Interventions
Whole-body Electrical Muscle Stimulation Exercise
Device
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced Oct 6, 2026, 11:34 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 5, 2015 · Synced Oct 6, 2026, 11:34 AM EDT
Completed Phase 4 Interventional Results available

Late-Onset Treatment Study Extension Protocol

NCT00455195
View directory record Official record
Conditions
Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD-II), Glycogenesis Type II, Acid Maltase Deficiency (AMD)
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
8 Years and older
Enrollment
81 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
26
States / cities
Anchorage, Alaska • Phoenix, Arizona • Beverly Hills, California + 23 more
Source: ClinicalTrials.gov public record
Updated Mar 7, 2014 · Synced Oct 6, 2026, 11:34 AM 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 28, 2021 · Synced Oct 6, 2026, 11:34 AM EDT
Completed Phase 2Phase 3 Interventional

Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

NCT00125879
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
6
States / cities
Birmingham, Alabama • Gainesville, Florida • Miami, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 6, 2014 · Synced Oct 6, 2026, 11:34 AM 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 2, 2019 · Synced Oct 6, 2026, 11:34 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 Sep 2, 2026 · Synced Oct 6, 2026, 11:34 AM 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 11, 2025 · Synced Oct 6, 2026, 11:34 AM 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 5, 2014 · Synced Oct 6, 2026, 11:34 AM EDT
Completed Phase 2 Interventional

Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies

NCT00763932
View directory record Official record
Conditions
Pompe Disease Infantile-Onset, Glycogen Storage Disease Type II
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
5
States / cities
Oakland, California • Orange, California • Omaha, Nebraska + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 5, 2014 · Synced Oct 6, 2026, 11:34 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 23, 2026 · Synced Oct 6, 2026, 11:34 AM 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 15, 2018 · Synced Oct 6, 2026, 11:34 AM 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 24, 2026 · Synced Oct 6, 2026, 11:34 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 28, 2022 · Synced Oct 6, 2026, 11:34 AM EDT
Completed Phase 2Phase 3 Interventional

A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease

NCT00059280
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Up to 26 Weeks
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
4
States / cities
Gainesville, Florida • Durham, North Carolina • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 5, 2014 · Synced Oct 6, 2026, 11:34 AM EDT
Active, not recruiting Phase 3 Interventional

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

NCT04910776
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
avalglucosidase alfa
Drug
Lead sponsor
Sanofi
Industry
Eligibility
0 Days to 12 Months
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
6
States / cities
Stanford, California • Minneapolis, Minnesota • Hawthorne, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Oct 6, 2026, 11:34 AM EDT
Completed Phase 2 Interventional

A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II

NCT00051935
View directory record Official record
Conditions
Glycogen Storage Disease Type II, Pompe Disease, Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions
Alglucosidase alfa
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003
U.S. locations
1
States / cities
New Brunswick, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 5, 2014 · Synced Oct 6, 2026, 11:34 AM EDT