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

Pompe Disease (Late-onset) Clinical Trial

Official title:
Prospective, Open-label, Single-arm, Exploratory Study of the Effect and Safety of rhGAA in Patients With Advanced Late-onset Pompe Disease Who Are Receiving Respiratory Support

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 is to evaluate the safety and efficacy of rhGAA in patients with advanced Late-onset Pompe disease.

Clinical Trial Description

n/a

Study Design

Allocation: Non-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 NCT00268944
Study type Interventional
Source Sanofi
Contact
Status Completed
Phase Phase 3
Start date December 2005
Completion date June 2007
View Details
See also
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Recruiting NCT05951790 - Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease N/A
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Withdrawn NCT04094948 - Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease Phase 2
Completed NCT00250939 - A Study of rhGAA in Patients With Late-Onset Pompe Disease Phase 2
Recruiting NCT03911505 - ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD Phase 3
Recruiting NCT06150820 - A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease N/A
Completed NCT03729362 - A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease Phase 3
Not yet recruiting NCT06178432 - Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD) Early Phase 1
Active, not recruiting NCT04138277 - A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD Phase 3