Completed Glycogen Storage Disease Type II trials: Trial Insights

Filters: Condition: Glycogen Storage Disease Type II Status: Completed Clear all

69 trials match Source: ClinicalTrials.gov registrations

Trials started per year
Category	Trials
2001	1
2003	4
2004	2
2005	6
2007	5
2008	2
2009	2
2010	2
2011	3
2012	3
2013	6
2014	5
2015	6
2016	4
2017	4
2018	3
2019	3
2020	1
2021	4
2022	2

Trials by status
Category	Trials
Completed	69

Top lead sponsors
Category	Trials
Genzyme, a Sanofi Company	24
Duke University	8
University of Florida	6
Amicus Therapeutics	5
Centre Hospitalier Universitaire de Nice	2
Centre Hospitalier de Cayenne	2
LMU Klinikum	2
National Taiwan University Hospital	2
University of California, Irvine	2
AskBio Inc	1

Top conditions
Category	Trials
Glycogen Storage Disease Type II	69
Glycogen Storage Disease	3
Macular Degeneration	3
Lysosomal Storage Diseases	2
Mucopolysaccharidosis I	2
Muscular Diseases	2
Adrenal Hyperplasia, Congenital	1
Adrenoleukodystrophy	1
Anaphylaxis	1
Andersen Syndrome	1

Trials

Showing 51 to 69 of 69. Each trial links to its page on Pharma Observatory.

A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease NCT00515398ObservationalCompletedAmicus TherapeuticsStarted 2007
High Dose or High Dose Frequency Study of Alglucosidase Alfa NCT00483379Phase 4CompletedGenzyme, a Sanofi CompanyStarted 2007
Late-Onset Treatment Study Extension Protocol NCT00455195Phase 4CompletedGenzyme, a Sanofi CompanyStarted 2007
Scotoma Reduction in AMD Patients Treated With Ranibizumab NCT00467935Phase 1/2CompletedRetina Research FoundationStarted 2007
Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme® NCT00731081ObservationalCompletedGenzyme, a Sanofi CompanyStarted 2007
Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support NCT00268944Phase 3CompletedGenzyme, a Sanofi CompanyStarted 2005
A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease NCT00158600Phase 3CompletedGenzyme, a Sanofi CompanyStarted 2005
Newborn Screening Assay of Pompe's Disease NCT01758354Not applicableCompletedNational Taiwan University HospitalStarted 2005
Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602 NCT00125879Phase 2/3CompletedGenzyme, a Sanofi CompanyStarted 2005
Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease NCT00113035ObservationalCompletedGenzyme, a Sanofi CompanyStarted 2005
A Study of rhGAA in Patients With Late-Onset Pompe Disease NCT00250939Phase 2CompletedGenzyme, a Sanofi CompanyStarted 2005
Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease NCT00074932Not applicableCompletedGenzyme, a Sanofi CompanyStarted 2004
A Prospective, Observational Study in Patients With Late-Onset Pompe Disease NCT00077662ObservationalCompletedGenzyme, a Sanofi CompanyStarted 2004
A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease NCT00059280Phase 2/3CompletedGenzyme, a Sanofi CompanyStarted 2003
Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies NCT00763932Phase 2CompletedGenzyme, a Sanofi CompanyStarted 2003
rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease) NCT00053573Phase 1/2CompletedGenzyme, a Sanofi CompanyStarted 2003
A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II NCT00051935Phase 2CompletedGenzyme, a Sanofi CompanyStarted 2003
Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease NCT00025896Phase 2CompletedGenzyme, a Sanofi CompanyStarted 2001
Genetic and Family Studies of Inherited Muscle Diseases NCT00001331ObservationalCompletedNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)Started 1993

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