List Results
Refine Search
Results by Topic
Results on Map
Search Details
Found 33 studies with search of: Pompe Disease
Hide studies that are not seeking new volunteers.
  Display Options
Rank Status Study
1 Completed rhGAA in Patients With Infantile-Onset Glycogen Storage Disease-II (Pompe Disease)
Conditions: Glycogen Storage Disease Type II;   Pompe Disease;   Acid Maltase Deficiency Disease;   Glycogenosis 2
Intervention: Biological: Myozyme
2 Completed Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
Conditions: Glycogen Storage Disease Type II;   Glycogenosis 2
Intervention: Biological: Myozyme
3 Recruiting Pompe Pregnancy Sub-Registry
Conditions: Glycogen Storage Disease Type II (GSD II);;   Pompe Disease (Late-Onset);;   Glycogenesis 2 Acid Maltase Deficiency
Intervention:
4 Completed A Placebo-Controlled Study of Safety and Effectiveness of Myozyme in Patients With Late-Onset Pompe Disease
Conditions: Pompe Disease (Late-Onset);   Glycogen Storage Disease Type II (GSD-II);   Acid Maltase Deficiency Disease;   Glycogenosis 2
Interventions: Drug: Myozyme (alglucosidase alfa);   Drug: Placebo
5 Completed A Study of rhGAA in Patients With Late-Onset Pompe Disease
Conditions: Pompe Disease (Late-Onset);   Glycogen Storage Disease Type II (GSD-II);   Acid Maltase Deficiency Disease;   Glycogenosis 2
Intervention: Biological: Myozyme
6 Not yet recruiting Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
Condition: Pompe Disease
Intervention: Drug: rAAV1-CMV-GAA
7 Completed Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support
Conditions: Pompe Disease (Late-Onset);   Glycogen Storage Disease Type II (GSD-II);   Acid Maltase Deficiency Disease;   Glycogenosis 2
Intervention: Biological: Myozyme
8 Completed A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II
Conditions: Glycogen Storage Disease Type II;   Pompe Disease;   Acid Maltase Deficiency Disease;   Glycogenosis 2
Intervention: Drug: Alglucosidase alfa
9 Recruiting Pompe Disease Registry
Conditions: Glycogen Storage Disease Type II;   Pompe Disease
Intervention:
10 Completed A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease
Conditions: Pompe Disease;   Glycogen Storage Disease Type II
Intervention: Other: Observation
11 Approved for marketing Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease
Conditions: Glycogen Storage Disease Type II;   Glycogenosis 2
Intervention: Biological: alglucosidase alfa
12 Completed Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease
Condition: Pompe Disease
Intervention:
13 Completed Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®
Conditions: Pompe Disease (Late-Onset);   Glycogen Storage Disease Type II (GSD II);   Glycogenesis 2 Acid Maltase Deficiency
Intervention:
14 Completed Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease
Conditions: Pompe Disease;   Glycogen Storage Disease Type II;   Acid Maltase Deficiency Disease;   Glycogenosis 2
Intervention: Drug: recombinant human acid alpha-glucosidase (rhGAA)
15 Recruiting Pompe Lactation Sub-Registry
Conditions: Glycogen Storage Disease;   Pompe Disease
Intervention:
16 Completed A Prospective, Observational Study in Patients With Late-Onset Pompe Disease
Condition: Glycogen Storage Disease Type II
Intervention:
17 Completed A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-Onset Pompe Disease
Condition: Glycogen Storage Disease Type II
Intervention: Biological: Myozyme
18 Completed Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602
Condition: Glycogen Storage Disease Type II
Intervention: Biological: Myozyme
19 Completed 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.
Conditions: Pompe Disease Late-Onset;   Glycogen Storage Disease Type II GSD II
Intervention: Biological: Myozyme
20 Terminated Study to Evaluate the Safety of AT2220 in Pompe Disease
Condition: Pompe Disease
Intervention: Drug: AT2220

Previous Page Studies Shown (1-20) Next Page (21-33)
Save this search by bookmarking this page.
When you use your bookmark, the search will be performed again on the most recent collection of studies.
RSS Feeds for studies found by your search that were:
  First received in the last 14 days,  
  Last updated (includes received) in the last 30 days
  Download Options




Contact Help Desk
Lister Hill National Center for Biomedical Communications,  U.S. National Library of Medicine,
U.S. National Institutes of Health,  U.S. Department of Health & Human Services,
USA.gov,  Copyright,  Privacy,  Accessibility,  Freedom of Information Act

U.S. National Institutes of Health U.S. National Library of Medicine U.S. Department of Health & Human Services