Status | Study |
Completed |
Study Name: Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease Receiving Enzyme Replacement Therapy Condition: Pompe Disease Date: 2013-06-21 Interventions: Drug: Albuterol Initially 4 mg |
Completed |
Study Name: Albuterol in Individuals With Late Onset Pompe Disease (LOPD) Condition: Pompe Disease Date: 2013-05-08 Interventions: Drug: Albuterol Albuterol 4 mg |
Recruiting |
Study Name: Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease Condition: Pompe Disease Date: 2012-08-13 Interventions: Other: Observational This is a longitudinal study focused on the emerging natural history of Infantile P |
Withdrawn |
Study Name: High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease Condition: Glycogen Storage Disease Type II Date: 2012-08-01 Interventions: Other: High Protein and Exercise Therapy along-with Nocturnal Enteral Feeding |
Recruiting |
Study Name: Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies Condition: Pompe Disease Date: 2011-09-15 Interventions: Drug: Rituximab Clinically pre |
Completed |
Study Name: Screening for Early Detection and Prevention of Pompe Disease in Israel Using Tandem Mass Spectrometry Condition: Pompe Disease Date: 2011-07-07 Interventions: Other: Drawing blood spots from Newborns Dry blood spots would be taken for determination of Alpha Gluco |
Completed |
Study Name: Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease Condition: Pompe Disease Date: 2009-07-13 Interventions: Drug: rAAV1-CMV-GAA (study agent) Administration |
Completed |
Study Name: Newborn Screening Assay of Pompe's Disease Condition: Pompe Disease Date: 2008-07-09 Interventions: Other: Pompe disease newborn screening DBS will be tested for acid alpha-glucosidase (GAA)activity. Newb |
Recruiting |
Study Name: A Long Term Follow up Study in Late-onset Pompe Disease Condition: Pompe Disease Date: 2008-07-09 |
Recruiting |
Study Name: Muscle Response to Enzyme Replacement Therapy in Pompe Disease Condition: Pompe Disease Date: 2008-07-09 |