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ClinicalTrials.gov public records Last synced Sep 30, 2026, 7:25 PM EDT

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Showing 1–24 of 46 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Pompe Disease (Late-Onset) Clear all
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 8, 2025 · Synced Sep 30, 2026, 7:25 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 Sep 30, 2026, 7:25 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 Sep 30, 2026, 7:25 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 Sep 30, 2026, 7:25 PM 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 Sep 30, 2026, 7:25 PM EDT
Active, not recruiting Not applicable Interventional

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

NCT06150820
View directory record Official record
Conditions
Pompe Disease (Late-onset)
Interventions
No Intervention
Other
Lead sponsor
Astellas Gene Therapies
Industry
Eligibility
16 Years to 69 Years
Enrollment
119 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
10
States / cities
Atlanta, Georgia • Kansas City, Kansas • Ann Arbor, Michigan + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 18, 2026 · Synced Sep 30, 2026, 7:25 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 15, 2019 · Synced Sep 30, 2026, 7:25 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 Sep 30, 2026, 7:25 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 5, 2015 · Synced Sep 30, 2026, 7:25 PM EDT
Completed Phase 2 Interventional

Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.

NCT00765414
View directory record Official record
Conditions
Pompe Disease Late-Onset, Glycogen Storage Disease Type II GSD II
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older · Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Feb 5, 2014 · Synced Sep 30, 2026, 7:25 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 11, 2025 · Synced Sep 30, 2026, 7:25 PM EDT
Completed Phase 1 Interventional

Albuterol in Individuals With Late Onset Pompe Disease (LOPD)

NCT01859624
View directory record Official record
Conditions
Pompe Disease
Interventions
Albuterol
Drug
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 5, 2019 · Synced Sep 30, 2026, 7:25 PM 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 23, 2023 · Synced Sep 30, 2026, 7:25 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 Sep 30, 2026, 7:25 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

NCT01230801
View directory record Official record
Conditions
Pompe Disease
Interventions
BMN 701
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
13 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
3
States / cities
La Jolla, California • Gainesville, Florida • Kansas City, Kansas
Source: ClinicalTrials.gov public record
Updated Jun 11, 2018 · Synced Sep 30, 2026, 7:25 PM EDT
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 23, 2026 · Synced Sep 30, 2026, 7:25 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 24, 2024 · Synced Sep 30, 2026, 7:25 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

NCT07282847
View directory record Official record
Conditions
Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
Interventions
AB-1009 (GAA Gene)
Genetic
Lead sponsor
AskBio Inc
Industry
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
9
States / cities
Phoenix, Arizona • Irvine, California • Palo Alto, California + 6 more
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Sep 30, 2026, 7:25 PM EDT
Completed Not applicable Interventional

Respiratory Muscle Training in L-Onset Pompe Disease (LOPD)

NCT02801539
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
RMT therapy using modified RMT device, Sham-RMT therapy using modified RMT device
Device
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
22 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 30, 2019 · Synced Sep 30, 2026, 7:25 PM EDT
Terminated Phase 1Phase 2 Interventional

VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease

NCT02898753
View directory record Official record
Conditions
Pompe Disease
Interventions
VAL-1221, RhGAA
Drug
Lead sponsor
Valerion Therapeutics, LLC
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
2
States / cities
Orange, California • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 2, 2020 · Synced Sep 30, 2026, 7:25 PM EDT
Completed Phase 3 Interventional Results available

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

NCT04138277
View directory record Official record
Conditions
Pompe Disease (Late-onset)
Interventions
AT2221, ATB200
Drug · Biological
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
119 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
18
States / cities
Little Rock, Arkansas • Irvine, California • Gainesville, Florida + 14 more
Source: ClinicalTrials.gov public record
Updated Mar 5, 2026 · Synced Sep 30, 2026, 7:25 PM 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 Sep 30, 2026, 7:25 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 Sep 30, 2026, 7:25 PM EDT
Terminated No phase listed Observational Results available

A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)

NCT02191917
View directory record Official record
Conditions
Late-onset Pompe Disease
Interventions
Respiratory muscle strength measurements by different techniques.
Procedure
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jan 9, 2023 · Synced Sep 30, 2026, 7:25 PM EDT