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

Glycogen Storage Disease Type II Clinical Trial

Official title:
A Long-Term Continuation Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

Clinical Trial Summary

Pompe disease (also known as glycogen storage disease type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The overall objective of this study is to evaluate the long-term safety and efficacy of Myozyme treatment in patients with infantile-onset Pompe disease.

Clinical Trial Description

n/a

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT00125879
Study type Interventional
Source Sanofi
Contact
Status Completed
Phase Phase 2/Phase 3
Start date June 2005
Completion date December 2006
View Details
See also
  Status Clinical Trial Phase
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Recruiting NCT04910776 - Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa Phase 3
Not yet recruiting NCT05017402 - Higher Dose of Alglucosidase Alpha for Pompe Disease
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Completed NCT00701129 - 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 Phase 4
Active, not recruiting NCT04093349 - A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) Phase 1/Phase 2
Completed NCT02363153 - Diet and Exercise in Pompe Disease N/A
Completed NCT00074932 - Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease N/A
Completed NCT00025896 - Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease Phase 2
Completed NCT00001331 - Genetic and Family Studies of Inherited Muscle Diseases N/A
Completed NCT00077662 - A Prospective, Observational Study in Patients With Late-Onset Pompe Disease N/A
Recruiting NCT05951790 - Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease N/A
Recruiting NCT04848779 - A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)
Active, not recruiting NCT05164055 - Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase) Phase 4
Recruiting NCT02761421 - Effect of Motor Development, Motor Function and Electrophysiologic Findings of IOPD Under ERT N/A
Active, not recruiting NCT02635269 - Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy N/A
Completed NCT00051935 - A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II Phase 2
Completed NCT00053573 - rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease) Phase 1/Phase 2
Completed NCT02801539 - Respiratory Muscle Training in L-Onset Pompe Disease (LOPD) N/A
Completed NCT03687333 - Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment Phase 4