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ClinicalTrials.gov public records Last synced Oct 9, 2026, 9:29 PM EDT

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Showing 49–72 of 98 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Glycogen Storage Disease Type II Pompe Disease Clear all
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 4, 2026 · Synced Oct 9, 2026, 9:29 PM EDT
Completed Not applicable Interventional

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

NCT03893240
View directory record Official record
Conditions
Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
Interventions
Neutralizing Antibody to SPK-3006 capsid
Diagnostic Test
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
6
States / cities
Phoenix, Arizona • Orange, California • Kansas City, Kansas + 3 more
Source: ClinicalTrials.gov public record
Updated Mar 24, 2023 · Synced Oct 9, 2026, 9:29 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 5, 2015 · Synced Oct 9, 2026, 9:29 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 28, 2021 · Synced Oct 9, 2026, 9:29 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 Mar 1, 2024 · Synced Oct 9, 2026, 9:29 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

NCT02000310
View directory record Official record
Conditions
Lysosomal Storage Disorders
Interventions
Not listed
Lead sponsor
O & O Alpan LLC
Other
Eligibility
1 Day to 100 Years
Enrollment
80 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2022
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Feb 23, 2021 · Synced Oct 9, 2026, 9:29 PM EDT
Completed Not applicable Interventional

Pompe Disease QMUS and EIM

NCT02742298
View directory record Official record
Conditions
Pompe Disease
Interventions
Muscle ultrasound, Electrical Impedance Myography
Device · Other
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 25, 2020 · Synced Oct 9, 2026, 9:29 PM EDT
Terminated Phase 4 Interventional Results available

A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

NCT01597596
View directory record Official record
Conditions
Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenosis 2, Acid Maltase Deficiency
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Up to 12 Months
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
14
States / cities
Little Rock, Arkansas • Oakland, California • Gainsville, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Jan 18, 2016 · Synced Oct 9, 2026, 9:29 PM EDT
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 25, 2022 · Synced Oct 9, 2026, 9:29 PM 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 9, 2026, 9:29 PM 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 9, 2026, 9:29 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 7, 2026 · Synced Oct 9, 2026, 9:29 PM EDT
Completed Phase 1Phase 2 Interventional Results available

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

NCT02675465
View directory record Official record
Conditions
Pompe Disease
Interventions
ATB200, AT2221
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
12
States / cities
Phoenix, Arizona • Orange, California • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Oct 23, 2025 · Synced Oct 9, 2026, 9:29 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 13, 2026 · Synced Oct 9, 2026, 9:29 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 11, 2025 · Synced Oct 9, 2026, 9:29 PM EDT
Recruiting No phase listed Observational

Avalglucosidase Alfa Pregnancy Study

NCT05734521
View directory record Official record
Conditions
Pompe Disease, Pregnancy
Interventions
avalglucosidase alfa-NGPT (GZ402666) IV, avalglucosidase alfa-NGPT (GZ402666)
Biological
Lead sponsor
Sanofi
Industry
Eligibility
Female only
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2032
U.S. locations
1
States / cities
Bridgewater, New Jersey
Source: ClinicalTrials.gov public record
Updated Jan 16, 2026 · Synced Oct 9, 2026, 9:29 PM EDT
Active, not recruiting Phase 3 Interventional

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

NCT03911505
View directory record Official record
Conditions
Pompe Disease (Late-onset)
Interventions
Cipaglucosidase Alfa, Miglustat
Biological · Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
0 Years to 17 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
9
States / cities
Gainesville, Florida • Jacksonville, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 17, 2026 · Synced Oct 9, 2026, 9:29 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 3, 2026 · Synced Oct 9, 2026, 9:29 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 17, 2022 · Synced Oct 9, 2026, 9:29 PM 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 9, 2026, 9:29 PM EDT
Completed Not applicable Interventional

Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease

NCT00074932
View directory record Official record
Conditions
Glycogen Storage Disease Type II, Glycogenosis 2
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2006
U.S. locations
8
States / cities
Aurora, Colorado • Wichita, Kansas • Cambridge, Massachusetts + 5 more
Source: ClinicalTrials.gov public record
Updated Feb 6, 2014 · Synced Oct 9, 2026, 9:29 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 4, 2024 · Synced Oct 9, 2026, 9:29 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 16, 2026 · Synced Oct 9, 2026, 9:29 PM 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 2, 2019 · Synced Oct 9, 2026, 9:29 PM EDT