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Showing 1–24 of 46 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Late-Onset Pompe Disease Clear all
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 5, 2014 · Synced Sep 25, 2026, 7:39 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 25, 2026, 7:39 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 6, 2014 · Synced Sep 25, 2026, 7:39 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 25, 2026, 7:39 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 1, 2020 · Synced Sep 25, 2026, 7:39 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 Sep 25, 2026, 7:39 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 22, 2023 · Synced Sep 25, 2026, 7:39 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 22, 2026 · Synced Sep 25, 2026, 7:39 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 Sep 25, 2026, 7:39 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 25, 2026, 7:39 PM EDT
Completed Not applicable Interventional

High Dose Inspiratory Muscle Training in LOPD

NCT05431127
View directory record Official record
Conditions
Late-Onset Pompe Disease, Lysosomal Disease
Interventions
IMT therapy using the Pr02 mobile device
Device
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 30, 2025 · Synced Sep 25, 2026, 7:39 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 Sep 25, 2026, 7:39 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 4, 2026 · Synced Sep 25, 2026, 7:39 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 25, 2026, 7:39 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 25, 2026, 7:39 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 Sep 25, 2026, 7:39 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 16, 2026 · Synced Sep 25, 2026, 7:39 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 8, 2023 · Synced Sep 25, 2026, 7:39 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 4, 2019 · Synced Sep 25, 2026, 7:39 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 25, 2026, 7:39 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 Sep 25, 2026, 7:39 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 25, 2026, 7:39 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 29, 2019 · Synced Sep 25, 2026, 7:39 PM EDT
Completed Not applicable Interventional

Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.

NCT02654886
View directory record Official record
Conditions
Pompe Disease, Muscle Weakness
Interventions
Resistance Exercise Training
Other
Lead sponsor
University of California, Irvine
Other
Eligibility
16 Years to 75 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated Sep 25, 2019 · Synced Sep 25, 2026, 7:39 PM EDT