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 Sep 15, 2026, 2:38 AM EDT

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

Showing 25–40 of 16 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Intervention: rhGAA Clear all
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 Sep 15, 2026, 2:38 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 Sep 15, 2026, 2:38 AM EDT
Completed Phase 1Phase 2 Interventional Results available

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

NCT02675465
View directory record Official record
Conditions
Pompe Disease
Interventions
ATB200, AT2221
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
12
States / cities
Phoenix, Arizona • Orange, California • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Oct 22, 2025 · Synced Sep 15, 2026, 2:38 AM 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 Sep 15, 2026, 2:38 AM 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 Sep 15, 2026, 2:38 AM EDT
Approved For Marketing No phase listed Expanded access

Alglucosidase Alfa Temporary Access Program

NCT00520143
View directory record Official record
Conditions
Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-Onset), Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions
alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
U.S. locations
74
States / cities
Sheffield, Alabama • Anchorage, Alaska • Phoenix, Arizona + 71 more
Source: ClinicalTrials.gov public record
Updated Feb 5, 2014 · Synced Sep 15, 2026, 2:38 AM EDT
Completed Phase 4 Interventional Results available

An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease

NCT00701129
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II
Interventions
Alglucosidase Alfa, Methotrexate, Rituximab
Biological · Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
2
States / cities
Louisville, Kentucky • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 12, 2014 · Synced Sep 15, 2026, 2:38 AM EDT
Completed Phase 2 Interventional

A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II

NCT00051935
View directory record Official record
Conditions
Glycogen Storage Disease Type II, Pompe Disease, Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions
Alglucosidase alfa
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003
U.S. locations
1
States / cities
New Brunswick, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 4, 2014 · Synced Sep 15, 2026, 2:38 AM 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
View directory record Official record
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 Sep 15, 2026, 2:38 AM 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 Sep 15, 2026, 2:38 AM 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 4, 2015 · Synced Sep 15, 2026, 2:38 AM 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 15, 2026, 2:38 AM 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 15, 2026, 2:38 AM 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 15, 2026, 2:38 AM 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 Sep 15, 2026, 2:38 AM EDT
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 15, 2026, 2:38 AM EDT