Alglucosidase Alfa Pompe Safety Sub-Registry

Late-Onset Treatment Study Extension Protocol

NCT00455195 ·Status: COMPLETED ·Phase: PHASE4

Treatment Frequency Reduction in Pompe Disease

NCT06575829 ·Status: NOT_YET_RECRUITING ·Phase: PHASE4

Pompe Disease Registry Protocol

NCT00231400 ·Status: RECRUITING

Avalglucosidase Alfa Extension Study

NCT02032524 ·Status: COMPLETED ·Phase: PHASE2

Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

NCT05164055 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE4

Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (STIG-Pompe-Study)

NCT02824068 ·Status: COMPLETED

Alglucosidase Alfa Temporary Access Program

NCT00520143 ·Status: APPROVED_FOR_MARKETING

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

NCT00158600 ·Status: COMPLETED ·Phase: PHASE3

Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease

NCT00074932 ·Status: COMPLETED ·Phase: NA

Growth and Development Study of Alglucosidase Alfa

NCT00486889 ·Status: COMPLETED ·Phase: PHASE4

China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD

NCT06666413 ·Status: RECRUITING ·Phase: PHASE4

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 ·Status: COMPLETED

BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

NCT01924845 ·Status: TERMINATED ·Phase: PHASE3

Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

NCT01230801 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease

NCT01526785 ·Status: TERMINATED ·Phase: PHASE4

Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support

NCT00268944 ·Status: COMPLETED ·Phase: PHASE3

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

NCT01665326 ·Status: RECRUITING

A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

NCT01597596 ·Status: TERMINATED ·Phase: PHASE4

A Study of rhGAA in Patients With Late-Onset Pompe Disease

NCT00250939 ·Status: COMPLETED ·Phase: PHASE2

Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease

NCT00025896 ·Status: COMPLETED ·Phase: PHASE2

A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II

NCT00051935 ·Status: COMPLETED ·Phase: PHASE2

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

NCT00763932 ·Status: COMPLETED ·Phase: PHASE2

Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease

NCT00113035 ·Status: COMPLETED

Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.

NCT00765414 ·Status: COMPLETED ·Phase: PHASE2

A Prospective, Observational Study in Patients With Late-Onset Pompe Disease

NCT00077662 ·Status: COMPLETED