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ClinicalTrials.gov public records Last synced May 21, 2026, 11:25 PM EDT

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Showing 1–24 of 46 matching trials from the live ClinicalTrials.gov search.

Local D1 index available.

Filters: Condition: Pompe Disease (Late-onset) Clear all

Completed No phase listed Observational Accepts healthy volunteers Results available

Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease

NCT02354664

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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 May 21, 2026, 11:25 PM EDT

Recruiting No phase listed Observational

Pompe Pregnancy Sub-Registry

NCT00567073

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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 May 21, 2026, 11:25 PM EDT

Terminated No phase listed Observational

STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT

NCT03347253

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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 May 21, 2026, 11:25 PM EDT

Completed Phase 1 Interventional

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

NCT02240407

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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 May 21, 2026, 11:25 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

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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 Apr 7, 2026 · Synced May 21, 2026, 11:25 PM EDT

Active, not recruiting Phase 1Phase 2 Interventional

Gene Transfer Study in Patients With Late Onset Pompe Disease

NCT04174105

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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 Feb 1, 2026 · Synced May 21, 2026, 11:25 PM EDT

Terminated Phase 3 Interventional Results available

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

NCT01924845

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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 May 21, 2026, 11:25 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

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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 May 21, 2026, 11:25 PM EDT

Active, not recruiting Phase 1Phase 2 Interventional

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

NCT04093349

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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 May 21, 2026, 11:25 PM EDT

Completed Phase 4 Interventional Results available

Late-Onset Treatment Study Extension Protocol

NCT00455195

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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 May 21, 2026, 11:25 PM EDT

Completed Phase 2Phase 3 Interventional

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

NCT00125879

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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 May 21, 2026, 11:25 PM EDT

Withdrawn Phase 2 Interventional

Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease

NCT04094948

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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 May 21, 2026, 11:25 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

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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 May 21, 2026, 11:25 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

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Conditions: Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
Interventions: AB-1009 (GAA Gene); Genetic
Lead sponsor: AskBio Inc; Industry
Eligibility: 18 Years and older

Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2032
U.S. locations: 10
States / cities: Phoenix, Arizona • Irvine, California • Palo Alto, California + 7 more

Source: ClinicalTrials.gov public record

Updated May 5, 2026 · Synced May 21, 2026, 11:25 PM EDT

Completed No phase listed Observational

Pompe Gene Therapy- Screening for Eligibility

NCT03285126

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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 May 21, 2026, 11:25 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

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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 Apr 29, 2026 · Synced May 21, 2026, 11:25 PM EDT

Completed Not applicable Interventional

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

NCT03893240

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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 May 21, 2026, 11:25 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

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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 May 21, 2026, 11:25 PM EDT

Completed Phase 1 Interventional

AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease

NCT03533673

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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 May 21, 2026, 11:25 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

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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 May 21, 2026, 11:25 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

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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 10, 2018 · Synced May 21, 2026, 11:25 PM EDT

Completed No phase listed Observational

Identification of Tongue Involvement in Late-Onset Pompe Disease

NCT02765828

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Conditions: Myopathy, Neuropathy, Glycogen Storage Disease Type II (Late-onset Pompe Disease)
Interventions: Observational study; Other
Lead sponsor: Duke University; Other
Eligibility: 12 Years and older

Enrollment: 73 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2019
U.S. locations: 1
States / cities: Durham, North Carolina

Source: ClinicalTrials.gov public record

Updated Jul 5, 2021 · Synced May 21, 2026, 11:25 PM EDT

Completed Not applicable Interventional

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

NCT02801539

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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 May 21, 2026, 11:25 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

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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 May 21, 2026, 11:25 PM EDT