Glycogen Storage Disease Type II

5
Pipeline Programs
1
Companies
29
Clinical Trials
3 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
1
1
2
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

Competitive Landscape

1 companies ranked by most advanced pipeline stage

Sanofi
SanofiPARIS, France
6 programs
1
1
2
1
MyozymePhase 2/31 trial
MyozymePhase 2/31 trial
NEXVIAZYME(Alglucosidase alfa)Phase 25 trials
MyozymePhase 1/21 trial
Alglucosidase alfa GZ419829N/A1 trial
+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
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2031
SanofiAlglucosidase alfa
SanofiAlglucosidase alfa
SanofiAlglucosidase alfa
SanofiAlglucosidase alfa
SanofiAlglucosidase alfa
SanofiAlglucosidase alfa
SanofiAlglucosidase alfa
SanofiAlglucosidase alfa
SanofiAlglucosidase alfa
SanofiAlglucosidase alfa
SanofiAlglucosidase alfa
SanofiAlglucosidase alfa
SanofiAlglucosidase alfa
SanofiAlglucosidase alfa
SanofiAlglucosidase alfa

Showing 15 of 28 trials with date data

Clinical Trials (29)

Total enrollment: 2,873 patients across 29 trials

NCT06666413SanofiAlglucosidase alfa

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

Start: May 2025Est. completion: May 202813 patients
Phase 4Recruiting
NCT05164055SanofiAlglucosidase alfa

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

Start: Jul 2022Est. completion: Sep 202617 patients
Phase 4Active Not Recruiting
NCT04676373SanofiAlglucosidase alfa

Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen

Start: Mar 2021Est. completion: Jul 202441 patients
Phase 4Completed
NCT03687333SanofiAlglucosidase alfa

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
NCT01410890SanofiAlglucosidase alfa

Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

Start: Nov 2014Est. completion: Nov 202021 patients
Phase 4Completed
NCT01597596SanofiAlglucosidase alfa

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

Start: Aug 2012Est. completion: Dec 20144 patients
Phase 4Terminated
NCT01526785SanofiAlglucosidase alfa

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

Start: Mar 2012Est. completion: Dec 2014113 patients
Phase 4Terminated
NCT00701129SanofiAlglucosidase alfa

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
NCT00701701SanofiAlglucosidase alfa

Immune Tolerance Induction Study

Start: Dec 2008Est. completion: Feb 20204 patients
Phase 4Terminated
NCT00486889SanofiAlglucosidase alfa

Growth and Development Study of Alglucosidase Alfa

Start: Aug 2008Est. completion: Nov 202112 patients
Phase 4Completed
NCT00483379SanofiAlglucosidase alfa

High Dose or High Dose Frequency Study of Alglucosidase Alfa

Start: May 2007Est. completion: Jul 201013 patients
Phase 4Completed
NCT00455195SanofiAlglucosidase alfa

Late-Onset Treatment Study Extension Protocol

Start: Mar 2007Est. completion: Nov 200881 patients
Phase 4Completed
NCT04910776SanofiAlglucosidase alfa

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

Start: Sep 2021Est. completion: Aug 202717 patients
Phase 3Active Not Recruiting
NCT02782741SanofiAlglucosidase alfa

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 Disease

Start: Nov 2016Est. completion: May 2023101 patients
Phase 3Completed
NCT00158600SanofiAlglucosidase alfa

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

Start: Sep 2005Est. completion: Sep 200790 patients
Phase 3Completed

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
NCT03019406SanofiAlglucosidase alfa

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 Alfa

Start: Oct 2017Est. completion: Aug 202722 patients
Phase 2Active Not Recruiting
NCT02032524SanofiAlglucosidase alfa

Avalglucosidase Alfa Extension Study

Start: Feb 2014Est. completion: Dec 202219 patients
Phase 2Completed
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
NCT00520143SanofiAlglucosidase alfa

Alglucosidase Alfa Temporary Access Program

N/AApproved For Marketing
NCT05734521SanofiAlglucosidase alfa

Avalglucosidase Alfa Pregnancy Study

Start: Oct 2022Est. completion: Oct 2032100 patients
N/ARecruiting
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
NCT01710813SanofiAlglucosidase alfa

Alglucosidase Alfa Pompe Safety Sub-Registry

Start: Mar 2015Est. completion: Apr 2021110 patients
N/ACompleted
NCT00566878SanofiAlglucosidase alfa

Pompe Lactation Sub-Registry

Start: Mar 2012Est. completion: Feb 20242 patients
N/ACompleted
NCT00074932SanofiAlglucosidase alfa

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
NCT00074919SanofiAlglucosidase alfa

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

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

Phase Legend

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

Key Insights

3 actively recruiting trials targeting 2,873 patients
1 companies competing in this space