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Glycogen Storage Disease Type II

1
Pipeline Programs
1
Companies
12
Clinical Trials
1 recruiting
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
0
0
0
1
Early DiscoveryClinical DevelopmentMarket

On Market (1)

Approved therapies currently available

Sanofi
NEXVIAZYMEApproved
avalglucosidase alfa-ngpt
Sanofi
Hydrolytic Lysosomal Glycogen-specific Enzyme [EPC]injection2021
22M Part D

Competitive Landscape

1 companies ranked by most advanced pipeline stage

Sanofi
SanofiPARIS, France
6 programs
1
Alglucosidase alfa GZ419829N/A1 trial
Pompe Disease Registry ProtocolN/A1 trial
MyozymePHASE_1_21 trial
NEXVIAZYME(Alglucosidase alfa)PHASE_21 trial
MyozymePHASE_2_35 trials
+1 more programs
Active Trials
NCT04848779Active Not Recruiting16Est. Oct 2026
NCT00231400Recruiting2,000Est. Jan 2034
NCT00053573Completed20Est. Nov 2006
+7 more trials

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
SanofiMyozyme
SanofiMyozyme
SanofiMyozyme
SanofiMyozyme
SanofiMyozyme
SanofiAlglucosidase alfa
SanofiMyozyme
SanofiMyozyme
SanofiAlglucosidase alfa GZ419829
SanofiMyozyme
SanofiPompe Disease Registry Protocol
SanofiMyozyme

Clinical Trials (12)

Total enrollment: 2,154 patients across 12 trials

Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment

Start: Dec 2018Est. completion: Dec 202010 patients
Phase 4Completed

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

Start: Oct 2009Est. completion: Mar 20134 patients
Phase 4Completed

Immune Tolerance Induction Study

Start: Dec 2008Est. completion: Feb 20204 patients
Phase 4Terminated

Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

Start: Jun 2005Est. completion: Dec 200616 patients
Phase 2/3Completed

A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease

Start: Apr 2003Est. completion: Sep 200516 patients
Phase 2/3Completed
NCT00051935SanofiAlglucosidase alfa

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

Start: Jan 2003Est. completion: Oct 20032 patients
Phase 2Completed

rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)

Start: Feb 2003Est. completion: Nov 200620 patients
Phase 1/2Completed

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

Start: Oct 2021Est. completion: Jan 202457 patients
N/ACompleted
NCT04848779SanofiAlglucosidase alfa GZ419829

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

Start: Jun 2021Est. completion: Oct 202616 patients
N/AActive Not Recruiting

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

Start: Nov 2004Est. completion: Dec 20069 patients
N/ACompleted
NCT00231400SanofiPompe Disease Registry Protocol

Pompe Disease Registry Protocol

Start: Sep 2004Est. completion: Jan 20342,000 patients
N/ARecruiting

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

Start: Dec 2003Est. completion: Feb 2007
N/AApproved For Marketing

Related Jobs

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

1 actively recruiting trials targeting 2,154 patients
1 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.