Independent directory Public ClinicalTrials.gov records United States
Clinical Trials Finder
Objective public record navigation
Search Results

Search by objective public record fields.

Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.

Clear filters
ClinicalTrials.gov public records Last synced Jul 23, 2026, 10:12 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 98 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Glycogen Storage Disease Type II Pompe Disease Clear all
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 4, 2014 · Synced Jul 23, 2026, 10:12 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 Jul 23, 2026, 10:12 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 Jul 23, 2026, 10:12 PM EDT
Completed Phase 4 Interventional Results available

High Dose or High Dose Frequency Study of Alglucosidase Alfa

NCT00483379
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
6 Months and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
9
States / cities
Birmingham, Alabama • Stanford, California • Washington D.C., District of Columbia + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 6, 2014 · Synced Jul 23, 2026, 10:12 PM EDT
Completed Phase 4 Interventional Results available

Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

NCT01410890
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD II)
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
4
States / cities
Valhalla, New York • Cincinnati, Ohio • Salt Lake City, Utah + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 27, 2022 · Synced Jul 23, 2026, 10:12 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 6, 2026 · Synced Jul 23, 2026, 10:12 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 24, 2020 · Synced Jul 23, 2026, 10:12 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 Jul 23, 2026, 10:12 PM EDT
Completed No phase listed Observational

The Effects of Respiratory Muscle Strength Training (RMST) on Inspiratory and Expiratory Muscle Strength in Adults and Children With Pompe Disease

NCT01701154
View directory record Official record
Conditions
Pompe Disease
Interventions
respiratory muscle strength training
Other
Lead sponsor
Duke University
Other
Eligibility
3 Years to 99 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 29, 2023 · Synced Jul 23, 2026, 10:12 PM EDT
Completed Phase 4 Interventional Results available

Growth and Development Study of Alglucosidase Alfa

NCT00486889
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Up to 24 Months
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2021
U.S. locations
3
States / cities
Gainesville, Florida • Decatur, Georgia • Detroit, Michigan
Source: ClinicalTrials.gov public record
Updated Aug 25, 2022 · Synced Jul 23, 2026, 10:12 PM EDT
Terminated Phase 4 Interventional Results available

Immune Tolerance Induction Study

NCT00701701
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
Interventions
Myozyme® (alglucosidase alfa)
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
1 Month and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2020
U.S. locations
4
States / cities
Louisville, Kentucky • Durham, North Carolina • Salt Lake City, Utah + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 6, 2022 · Synced Jul 23, 2026, 10:12 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 12, 2026 · Synced Jul 23, 2026, 10:12 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 Jul 16, 2026 · Synced Jul 23, 2026, 10:12 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 Jul 23, 2026, 10:12 PM EDT
Completed Phase 2 Interventional Results available

Drug-drug Interaction Study

NCT01380743
View directory record Official record
Conditions
Pompe Disease
Interventions
duvoglustat, rhGAA
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
11
States / cities
Phoenix, Arizona • Scottsdale, Arizona • Orange, California + 8 more
Source: ClinicalTrials.gov public record
Updated Jun 25, 2025 · Synced Jul 23, 2026, 10:12 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease

NCT00976352
View directory record Official record
Conditions
Pompe Disease
Interventions
rAAV1-CMV-GAA (study agent) Administration, RMST
Drug · Other
Lead sponsor
University of Florida
Other
Eligibility
2 Years to 18 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Sep 13, 2018 · Synced Jul 23, 2026, 10:12 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 Jul 23, 2026, 10:12 PM EDT
Completed Phase 2Phase 3 Interventional

A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease

NCT00059280
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Up to 26 Weeks
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
4
States / cities
Gainesville, Florida • Durham, North Carolina • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2014 · Synced Jul 23, 2026, 10:12 PM EDT
Not listed No phase listed Observational

Clinical Specimen Collection From Pompe Disease Patients

NCT04476550
View directory record Official record
Conditions
Pompe Disease
Interventions
Filgrastim
Drug
Lead sponsor
Serhat Gumrukcu, MD PhD
Other
Eligibility
3 Years to 30 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Toluca Lake, California
Source: ClinicalTrials.gov public record
Updated May 18, 2022 · Synced Jul 23, 2026, 10:12 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 Jul 23, 2026, 10:12 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 10, 2025 · Synced Jul 23, 2026, 10:12 PM EDT
Terminated Phase 2 Interventional Results available

Extension Study for Patients Who Have Participated in a BMN 701 Study

NCT01435772
View directory record Official record
Conditions
Pompe Disease
Interventions
BMN 701
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
13 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
4
States / cities
San Diego, California • Gainesville, Florida • Tampa, Florida + 1 more
Source: ClinicalTrials.gov public record
Updated May 21, 2018 · Synced Jul 23, 2026, 10:12 PM EDT
Recruiting No phase listed Observational

A Global Prospective Observational Registry of Patients With Pompe Disease

NCT06121011
View directory record Official record
Conditions
Pompe Disease
Interventions
Cipaglucosidase alfa, Miglustat, Alglucosidase alfa or Avalglucosidase alfa, Untreated
Biological · Drug · Other
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2034
U.S. locations
15
States / cities
Little Rock, Arkansas • Irvine, California • Jacksonville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2026 · Synced Jul 23, 2026, 10:12 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 22, 2026 · Synced Jul 23, 2026, 10:12 PM EDT