62 studies found for:    Pompe
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Pompe (62 records)
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Rank Status Study
1 Recruiting A Natural History Study of Adult Onset Pompe Disease Using Muscle MRI
Condition: Pompe Disease
Intervention:
2 Recruiting Pompe Pregnancy Sub-Registry
Conditions: Glycogen Storage Disease Type II (GSD-II);   Pompe Disease (Late-onset);   Glycogenesis 2 Acid Maltase Deficiency
Interventions: Biological: alglucosidase alpha;   Other: No Treatment
3 Completed Newborn Screening Assay of Pompe's Disease
Condition: Pompe Disease
Intervention: Other: Pompe disease newborn screening
4 Recruiting Pompe Disease Registry
Conditions: Glycogen Storage Disease Type II;   Pompe Disease
Intervention:
5 Recruiting Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Condition: Pompe Disease
Intervention: Other: Observational
6 Recruiting Pompe Lactation Sub-Registry
Conditions: Glycogen Storage Disease;   Pompe Disease
Intervention: Biological: alglucosidase alfa
7 Completed Pompe Prevalence Study in Patients With Muscle Weakness Without Diagnosis
Condition: Pompe's Disease
Intervention: Procedure: blood test
8 Active, not recruiting Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
Condition: Pompe Disease
Interventions: Drug: Study Agent Administration;   Other: Safety Labs;   Other: Pulmonary Function Testing;   Other: RMST
9 Recruiting Pregnancy and Birth Outcome in Women With Pompe Disease
Condition: Pompe Disease
Intervention:
10 Completed The Effects of Respiratory Muscle Strength Training (RMST) on Inspiratory and Expiratory Muscle Strength in Adults and Children With Pompe Disease
Condition: Pompe Disease
Intervention: Other: respiratory muscle strength training
11 Completed
Has Results
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
Conditions: Pompe Disease;   Glycogen Storage Disease Type II
Interventions: Biological: Alglucosidase Alfa;   Drug: Methotrexate;   Drug: Rituximab
12 Recruiting Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies
Condition: Pompe Disease
Intervention:
13 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
14 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:
15 Completed Albuterol in Individuals With Late Onset Pompe Disease (LOPD)
Condition: Pompe Disease
Intervention: Drug: Albuterol
16 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
17 Completed
Has Results
A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) 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: Biological: alglucosidase alfa;   Drug: Placebo
18 Terminated Study to Evaluate the Safety of AT2220 in Pompe Disease
Condition: Pompe Disease
Intervention: Drug: AT2220
19 Not yet recruiting A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction
Conditions: Pompe Disease;   Hypersensitivity Reaction
Interventions: Procedure: Blood collection for anti-GAA antibody level;   Procedure: Punch Muscle Biopsy;   Behavioral: Survey;   Drug: Zavesca® 100 mg;   Drug: Zavesca® 300 mg
20 Recruiting Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease Receiving Enzyme Replacement Therapy
Condition: Pompe Disease
Interventions: Drug: Albuterol;   Drug: Placebo

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