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 Jun 10, 2026, 5:35 AM EDT

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

Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Glycogen Storage Disease Type II (GSD-II) Clear all
Recruiting No phase listed Observational

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

NCT01665326
View directory record Official record
Conditions
Pompe Disease
Interventions
Observational
Other
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2029
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 7, 2026 · Synced Jun 10, 2026, 5:35 AM 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 Jun 10, 2026, 5:35 AM 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 Jun 10, 2026, 5:35 AM 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 Jun 10, 2026, 5:35 AM 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 Jun 10, 2026, 5:35 AM 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 Jun 10, 2026, 5:35 AM EDT
Completed Phase 2 Interventional

Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies

NCT00763932
View directory record Official record
Conditions
Pompe Disease Infantile-Onset, Glycogen Storage Disease Type II
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
5
States / cities
Oakland, California • Orange, California • Omaha, Nebraska + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2014 · Synced Jun 10, 2026, 5:35 AM EDT
Completed No phase listed Observational

Pompe Telemedicine Developmental Study

NCT02950298
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease II
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
6 Years to 18 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 27, 2021 · Synced Jun 10, 2026, 5:35 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Screening of Lysosomal Storage Disorders Diseases in Minority Groups

NCT03812042
View directory record Official record
Conditions
Lysosomal Storage Diseases
Interventions
Enzyme assay and molecular sequencing
Diagnostic Test
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
Not listed
Enrollment
100,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2019
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jan 21, 2019 · Synced Jun 10, 2026, 5:35 AM EDT
Completed No phase listed Observational

Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

NCT01451879
View directory record Official record
Conditions
Pompe Disease
Interventions
Rituximab, Miglustat
Drug
Lead sponsor
University of Florida
Other
Eligibility
Up to 65 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2017
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Dec 7, 2017 · Synced Jun 10, 2026, 5:35 AM 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 Jun 10, 2026, 5:35 AM 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 Jun 10, 2026, 5:35 AM 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 Jun 10, 2026, 5:35 AM 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 Jun 10, 2026, 5:35 AM 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 Jun 10, 2026, 5:35 AM 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 Jun 10, 2026, 5:35 AM EDT
Terminated Early Phase 1 Interventional

A Pilot Study of Pyridostigmine in Pompe Disease

NCT02357225
View directory record Official record
Conditions
Pompe Disease
Interventions
Pyridostigmine Bromide
Drug
Lead sponsor
University of Florida
Other
Eligibility
8 Years to 60 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated May 14, 2018 · Synced Jun 10, 2026, 5:35 AM 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
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced Jun 10, 2026, 5:35 AM EDT
Terminated Phase 4 Interventional Results available

A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease

NCT01526785
View directory record Official record
Conditions
Pompe Disease
Interventions
Alglucosidase alfa
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
1 Year and older
Enrollment
113 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
51
States / cities
Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 48 more
Source: ClinicalTrials.gov public record
Updated Dec 7, 2015 · Synced Jun 10, 2026, 5:35 AM EDT
Completed No phase listed Observational

Pompe Lactation Sub-Registry

NCT00566878
View directory record Official record
Conditions
Glycogen Storage Disease, Pompe Disease
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Female only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2024
U.S. locations
2
States / cities
Cambridge, Massachusetts • Grand Rapids, Michigan
Source: ClinicalTrials.gov public record
Updated Mar 18, 2024 · Synced Jun 10, 2026, 5:35 AM 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 May 26, 2026 · Synced Jun 10, 2026, 5:35 AM 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 Jun 10, 2026, 5:35 AM EDT
Completed No phase listed Observational Results available

Response to Diaphragmatic Pacing in Subjects With Pompe Disease

NCT02354651
View directory record Official record
Conditions
Pompe Disease
Interventions
Diaphragm conditioning via phrenic nerve stimulation
Other
Lead sponsor
University of Florida
Other
Eligibility
2 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jul 31, 2023 · Synced Jun 10, 2026, 5:35 AM EDT