Sanofi: Glycogen Storage Disease Type II trials

The registry lists 41 Glycogen Storage Disease Type II studies with Sanofi as lead sponsor. 4 are open to participants now and 13 have results posted.

Phase 1 2Phase 2 10Phase 3 6Phase 4 13No phase 13(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPDRecruitingNCT06666413Phase 4
• Avalglucosidase Alfa Pregnancy StudyRecruitingNCT05734521
• Pompe Pregnancy Sub-RegistryRecruitingNCT00567073
• Pompe Disease Registry ProtocolRecruitingNCT00231400
• A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase AlfaActive, not recruitingNCT03019406Phase 2Results posted
• Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseCompletedNCT02782741Phase 3Results posted
• Avalglucosidase Alfa Extension StudyCompletedNCT02032524Phase 2Results posted
• A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe DiseaseTerminatedNCT01597596Phase 4Results posted
• A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe DiseaseTerminatedNCT01526785Phase 4Results posted
• Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe DiseaseCompletedNCT01410890Phase 4Results posted
• Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase AlfaCompletedNCT01288027Phase 4Results posted
• Immune Tolerance Induction StudyTerminatedNCT00701701Phase 4Results posted
• 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 DiseaseCompletedNCT00701129Phase 4Results posted
• Growth and Development Study of Alglucosidase AlfaCompletedNCT00486889Phase 4Results posted
• High Dose or High Dose Frequency Study of Alglucosidase AlfaCompletedNCT00483379Phase 4Results posted
• Late-Onset Treatment Study Extension ProtocolCompletedNCT00455195Phase 4Results posted
• A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe DiseaseCompletedNCT00158600Phase 3Results posted
• Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)Active, not recruitingNCT05164055Phase 4
• 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 SettingCompletedNCT05073783
• Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase AlfaActive, not recruitingNCT04910776Phase 3
• A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)CompletedNCT04848779
• Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa TreatmenCompletedNCT04676373Phase 4
• Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa TreatmentCompletedNCT03687333Phase 4
• Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.CompletedNCT01898364Phase 1
• Alglucosidase Alfa Pompe Safety Sub-RegistryCompletedNCT01710813
• 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.CompletedNCT00765414Phase 2
• 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) StudiesCompletedNCT00763932Phase 2
• Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®CompletedNCT00731081
• Pompe Lactation Sub-RegistryCompletedNCT00566878
• Alglucosidase Alfa Temporary Access ProgramApproved for marketingNCT00520143
• Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory SupportCompletedNCT00268944Phase 3
• A Study of rhGAA in Patients With Late-Onset Pompe DiseaseCompletedNCT00250939Phase 2
• Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602CompletedNCT00125879Phase 2/3
• Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe DiseaseCompletedNCT00113035
• A Prospective, Observational Study in Patients With Late-Onset Pompe DiseaseCompletedNCT00077662
• Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe DiseaseCompletedNCT00074932Not applicable
• Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe DiseaseApproved for marketingNCT00074919
• A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe DiseaseCompletedNCT00059280Phase 2/3
• rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)CompletedNCT00053573Phase 1/2
• A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type IICompletedNCT00051935Phase 2
• Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe DiseaseCompletedNCT00025896Phase 2