| MSOT in Pompe Disease (NCT05083806) | University of Erlangen-Nürnberg Medical School | 2022-05-17 |
| Frequency of Pompe Disease in Patients With Myalgia With or Without Hyper Ckemia - Data From the Reference Center (CERCA) (NCT05092230) | University Hospital Center of Martinique | 2021-11-01 |
| A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting (NCT05073783) | Sanofi | 2021-10-14 |
| High Risk Screen of Childhood Late-onset Pompe Disease in Pediatric Outpatient Clinics (NCT04981210) | National Taiwan University Hospital | 2021-08-01 |
| Development of an Assessment Tool for Health-related Quality of Life in Children and Adolescents With Pompe Disease (NCT04724213) | University Children's Hospital, Zurich | 2021-07-01 |
| A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) (NCT04093349) | Spark Therapeutics, Inc. | 2020-10-01 |
| Getting Global Rare Disease Insights Through Technology Study (NCT04758130) | M.A.G.I.C. Clinic LTD | 2020-08-07 |
| Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease (NCT03893240) | Spark Therapeutics, Inc. | 2019-06-12 |
| Developing a Management Approach for Patients With "Late-Onset" Pompe Disease (NCT03694561) | Duke University | 2019-03-25 |
| AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease (NCT03533673) | AskBio Inc | 2018-12-17 |
| Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease (NCT02240407) | University of Florida | 2017-10-17 |
| VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease (NCT02898753) | Valerion Therapeutics, LLC | 2017-06-21 |
| Search for Serum/Plasma Biomarkers in Pompe's Disease (NCT03045042) | Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau | 2017-04-01 |
| Pompe Disease QMUS and EIM (NCT02742298) | Duke University | 2016-10-24 |
| Cognitive and Neurological Pathologies in Pompe Disease (NCT04639336) | Duke University | 2016-08-03 |
| Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (STIG-Pompe-Study) (NCT02824068) | LMU Klinikum | 2016-06-01 |
| First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221 (NCT02675465) | Amicus Therapeutics | 2016-04-01 |
| Frequency of Pompe's Disease and Neuromuscular Etiologies in Patients With Restrictive Respiratory Failure Associated With Signs of Muscle Weakness (NCT02746718) | Centre Hospitalier Universitaire de Nice | 2015-12-11 |
| Pompe Telemedicine Developmental Study (NCT02950298) | Duke University | 2015-12-01 |
| Immune Modulation Therapy for Pompe Disease (NCT02525172) | National Taiwan University Hospital | 2015-08-01 |
| A Pilot Study of Pyridostigmine in Pompe Disease (NCT02357225) | University of Florida | 2015-08-01 |
| Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease (NCT02354664) | University of Florida | 2015-04-01 |
| Alglucosidase Alfa Pompe Safety Sub-Registry (NCT01710813) | Genzyme, a Sanofi Company | 2015-03-20 |
| Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease (NCT01410890) | Genzyme, a Sanofi Company | 2014-11-03 |
| CPAP for Infantile Pompe Disease (NCT02405624) | National Taiwan University Hospital | 2014-06-01 |
| Investigating Lysosomal Storage Diseases in Minority Groups (NCT02120235) | O & O Alpan LLC | 2014-02-01 |
| Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease (NCT02405598) | National Taiwan University Hospital | 2013-10-01 |
| Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy (NCT01942590) | Dwight Koeberl, M.D., Ph.D. | 2013-09-01 |
| Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients. (NCT01898364) | Genzyme, a Sanofi Company | 2013-08-19 |
| Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease (NCT01885936) | Duke University | 2013-06-01 |
| Response to Diaphragmatic Pacing in Subjects With Pompe Disease (NCT02354651) | University of Florida | 2013-05-01 |
| Albuterol in Individuals With Late Onset Pompe Disease (LOPD) (NCT01859624) | Duke University | 2012-06-01 |
| Pompe Lactation Sub-Registry (NCT00566878) | Genzyme, a Sanofi Company | 2012-03-31 |
| A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease (NCT01526785) | Genzyme, a Sanofi Company | 2012-03-01 |
| Pregnancy and Birth Outcome in Women With Pompe Disease (NCT01556516) | O & O Alpan LLC | 2012-02-01 |
| Detection of Pompe Disease in Adult Patients With Myopathies of Uncertain Origin or With Asymptomatic Hyper-CK-emia (NCT01482494) | Jordi Perez Lopez | 2011-12-01 |
| Drug-drug Interaction Study (NCT01380743) | Amicus Therapeutics | 2011-10-31 |
| Screening for Early Detection and Prevention of Pompe Disease in Israel Using Tandem Mass Spectrometry (NCT01409486) | Rambam Health Care Campus | 2011-09-01 |
| Extension Study for Patients Who Have Participated in a BMN 701 Study (NCT01435772) | BioMarin Pharmaceutical | 2011-08-15 |
| Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease (NCT01230801) | BioMarin Pharmaceutical | 2011-01-17 |
| The Effects of Respiratory Muscle Strength Training (RMST) on Inspiratory and Expiratory Muscle Strength in Adults and Children With Pompe Disease (NCT01701154) | Duke University | 2010-12-01 |
| Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease (NCT00976352) | University of Florida | 2010-09-01 |
| 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 (NCT00701129) | Genzyme, a Sanofi Company | 2009-10-01 |
| Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease (NCT01665326) | Duke University | 2009-09-01 |
| Immune Tolerance Induction Study (NCT00701701) | Genzyme, a Sanofi Company | 2008-12-14 |
| Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease (NCT00688597) | Amicus Therapeutics | 2008-12-08 |
| Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies (NCT01451879) | University of Florida | 2008-10-01 |
| Growth and Development Study of Alglucosidase Alfa (NCT00486889) | Genzyme, a Sanofi Company | 2008-08-26 |
| A Long Term Follow up Study in Late-onset Pompe Disease (NCT00713245) | National Taiwan University Hospital | 2008-05-01 |
| A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease (NCT00515398) | Amicus Therapeutics | 2007-08-01 |
| A Long-term Study for the Outcome of Pompe Disease (NCT02399748) | National Taiwan University Hospital | 2007-04-01 |
| Muscle Response to Enzyme Replacement Therapy in Pompe Disease (NCT00713453) | National Taiwan University Hospital | 2007-01-01 |
| Newborn Screening Assay of Pompe's Disease (NCT01758354) | National Taiwan University Hospital | 2005-09-01 |
| Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease (NCT00113035) | Genzyme, a Sanofi Company | 2005-05-01 |
| Pompe Disease Registry Protocol (NCT00231400) | Genzyme, a Sanofi Company | 2004-09-15 |
| rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease) (NCT00053573) | Genzyme, a Sanofi Company | 2003-02-01 |
| A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II (NCT00051935) | Genzyme, a Sanofi Company | 2003-01-01 |
| Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease (NCT00025896) | Genzyme, a Sanofi Company | 2001-05-01 |
| Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease (NCT03865836) | Amicus Therapeutics | |