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Showing 25–46 of 22 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Late-Onset Pompe Disease Clear all
Recruiting No phase listed Observational

Pompe Disease Registry Protocol

NCT00231400
View directory record Official record
Conditions
Glycogen Storage Disease Type II, Pompe Disease
Interventions
Not listed
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2034
U.S. locations
92
States / cities
Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 70 more
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Jul 23, 2026, 2:21 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 21, 2025 · Synced Jul 23, 2026, 2:21 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 Jul 23, 2026, 2:21 PM 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 13, 2018 · Synced Jul 23, 2026, 2:21 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 Jul 23, 2026, 2:21 PM EDT
Recruiting No phase listed Observational

Long-Term Development of Muscular Dystrophy Outcome Assessments

NCT05989620
View directory record Official record
Conditions
LGMD1B, LGMD1C, LGMD1D, LGMD1E, LGMD1F, LGMD1G, LGMD1H, LGMD2A, LGMD2B, LGMD2C, LGMD2D, LGMD2E, LGMD2F, LGMD2G, LGMD2I, LGMD2J, LGMD2K, LGMD2L, LGMD2M, LGMD2N, LGMD2O, LGMD2P, LGMD2Q, LGMD2S, LGMD2T, LGMD2U, LGMD2W, LGMD2X, LGMD2Y
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
6 Years to 50 Years
Enrollment
1,000 participants
Timeline
2023 – 2029
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Nov 17, 2025 · Synced Jul 23, 2026, 2:21 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 16, 2022 · Synced Jul 23, 2026, 2:21 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 Jul 23, 2026, 2:21 PM EDT
Completed Phase 1 Interventional

Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease

NCT02240407
View directory record Official record
Conditions
Pompe Disease
Interventions
Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase, Rapamycin, saline, Rituxan, Diphenhydramine, Acetaminophen, Lidocaine, LMX 4 Topical Cream
Genetic · Drug · Other
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 50 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 Apr 4, 2022 · Synced Jul 23, 2026, 2:21 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 1, 2019 · Synced Jul 23, 2026, 2:21 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 23, 2023 · Synced Jul 23, 2026, 2:21 PM 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, 2:21 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
3
States / cities
Irvine, California • Durham, North Carolina • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jun 28, 2026 · Synced Jul 23, 2026, 2:21 PM EDT
Completed No phase listed Observational

A Prospective, Observational Study in Patients With Late-Onset Pompe Disease

NCT00077662
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
Not listed
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
8 Years and older
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2006
U.S. locations
3
States / cities
Washington D.C., District of Columbia • St Louis, Missouri • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated May 4, 2015 · Synced Jul 23, 2026, 2:21 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 Jul 23, 2026, 2:21 PM EDT
Completed Phase 4 Interventional Results available

Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

NCT01288027
View directory record Official record
Conditions
Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenesis 2 Acid Maltase Deficiency
Interventions
Alglucosidase Alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
9
States / cities
Orange, California • Gainesville, Florida • Kansas City, Kansas + 6 more
Source: ClinicalTrials.gov public record
Updated Dec 18, 2014 · Synced Jul 23, 2026, 2:21 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 Jul 23, 2026, 2:21 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 5, 2014 · Synced Jul 23, 2026, 2:21 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 16, 2026 · Synced Jul 23, 2026, 2:21 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 Jul 23, 2026, 2:21 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 Jul 23, 2026, 2:21 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 – 2026
U.S. locations
9
States / cities
Gainesville, Florida • Jacksonville, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 26, 2025 · Synced Jul 23, 2026, 2:21 PM EDT