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87 studies found for:    "Glycogen Storage Disease Type II" OR "Pompe disease"
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Rank Status Study
21 Completed A Prospective, Observational Study in Patients With Late-Onset Pompe Disease
Condition: Glycogen Storage Disease Type II
Intervention:
22 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
23 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
24 Withdrawn High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease
Condition: Glycogen Storage Disease Type II
Intervention: Other: High Protein and Exercise Therapy along-with Nocturnal Enteral Feeding
25 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:
26 Recruiting Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Condition: Pompe Disease
Intervention: Other: Observational
27 Active, not recruiting Growth and Development Study of Alglucosidase Alfa.
Conditions: Pompe Disease;   Glycogen Storage Disease Type II (GSD-II);   Acid Maltase Deficiency Disease
Intervention: Biological: alglucosidase alfa
28 Recruiting Immune Tolerance Induction Study
Conditions: Pompe Disease;   Glycogen Storage Disease Type II (GSD-II);   Glycogenesis 2 Acid Maltase Deficiency
Intervention: Biological: Myozyme (alglucosidase alfa)
29 Completed
Has Results
High Dose or High Dose Frequency Study of Alglucosidase Alfa
Conditions: Pompe Disease;   Glycogen Storage Disease Type II (GSD-II);   Glycogenesis 2 Acid Maltase Deficiency
Intervention: Biological: alglucosidase alfa
30 Completed
Has Results
Late-Onset Treatment Study Extension Protocol
Conditions: Pompe Disease (Late-Onset);   Glycogen Storage Disease Type II (GSD-II);   Glycogenesis Type II;   Acid Maltase Deficiency (AMD)
Intervention: Biological: alglucosidase alfa
31 Completed Newborn Screening Assay of Pompe's Disease
Condition: Pompe Disease
Intervention: Other: Pompe disease newborn screening
32 Recruiting Biomarker for Pompe Disease
Conditions: Lysosomal Storage Diseases;   Pompe Disease
Intervention:
33 Not yet recruiting Identification of Tongue Involvement in Late-Onset Pompe Disease
Conditions: Myopathy;   Neuropathy;   Glycogen Storage Disease Type II (Late-onset Pompe Disease)
Intervention: Other: Observational study
34 Recruiting A Natural History Study of Adult Onset Pompe Disease Using Muscle MRI
Condition: Pompe Disease
Intervention:
35 Not yet recruiting VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Patients With Late-Onset Pompe Disease
Condition: Pompe Disease
Interventions: Drug: VAL-1221 1 mg/kg;   Drug: VAL-1221 3 mg/kg;   Drug: VAL-1221 10 mg/kg
36 Recruiting A Long Term Follow up Study in Late-onset Pompe Disease
Condition: Pompe Disease
Intervention:
37 Unknown  Detection of Pompe Disease in Adult Patients With Myopathies of Uncertain Origin or With Asymptomatic Hyper-CK-emia
Condition: Pompe Disease
Intervention:
38 Recruiting Muscle Response to Enzyme Replacement Therapy in Pompe Disease
Condition: Pompe Disease
Intervention:
39 Recruiting Response to Diaphragmatic Pacing in Subjects With Pompe Disease
Condition: Pompe Disease
Interventions: Other: Pulmonary function testing;   Other: Resting breathing pattern;   Other: Respiratory muscle endurance test;   Other: Phrenic nerve stimulation;   Other: Maximal inspiratory pressure;   Other: Forced expiratory testing;   Other: EMG
40 Recruiting A Pilot Study of Pyridostigmine in Pompe Disease
Condition: Pompe Disease
Intervention: Drug: Pyridostigmine Bromide

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