| Status | Study |
| Completed |
Study Name: Cardiopulmonary Exercise Test to Quantify Enzyme Replacement Response in Pediatric Pompe Disease Condition: Enzyme Replacement Therapy in Pompe Disease Date: 2017-04-30 |
| Not yet recruiting |
Study Name: Search for Serum/Plasma Biomarkers in Pompe's Disease Condition: Late Onset Pompe Disease Pompe Disease Date: 2017-01-27 Interventions: Drug: Enzyme Replacement Agent Patients will be treated following the decision of their doctors. The inv |
| Not yet recruiting |
Study Name: A Study to Assess Safety and Efficacy of NeoGAA Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa Condition: Glycogen Storage Disease Type II-Pompe's Disease Date: 2016-12-20 Interventions: Drug: GZ402666 Pharmaceutical |
| Not yet recruiting |
Study Name: VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Patients With Late-Onset Pompe Disease Condition: Pompe Disease Date: 2016-09-08 Interventions: Drug: VAL-1221 1 mg/kg VAL-122 |
| Completed |
Study Name: Feasability and Interest of Screening for Infantile Pompe's Diseases at Birth Condition: Pompe's Disease Date: 2016-09-06 Interventions: Other: observational (no intervention) |
| Completed |
Study Name: Prevalence of Heterozygote Mothers for Pompe's Disease Among Mothers Having Delivered in French Guiana Condition: Pompe's Disease Date: 2016-09-06 Interventions: Other: observational |
| Recruiting |
Study Name: Pompe Telemedicine Developmental Study Condition: Pompe Disease Glycogen Storage Disease II Date: 2016-06-22 |
| Recruiting |
Study Name: Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (ATBIG-Pompe-Study) Condition: Pompe Disease Date: 2016-06-20 Interventions: Drug: glucosidase alfa Long-term use in an observational study of licenced drug |
| Recruiting |
Study Name: Respiratory Muscle Training in L-Onset Pompe Disease (LOPD) Condition: Glycogen Storage Disease Type II Date: 2016-06-13 Interventions: Device: RMT therapy using modified RMT device |
| Recruiting |
Study Name: Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease Condition: Glycogen Storage Disease Type II-Pompe's Disease Date: 2016-05-23 Interventions: Drug: GZ402666 |