A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
Completed
Genzyme, a Sanofi Company
Phase 3
2005-09-01
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, efficacy, and
pharmacokinetics (PK) of alglucosidase alfa treatment in patients with late-onset Pompe
disease as compared to placebo.
This study evaluates drug-drug interactions between AT2220 (duvoglustat) and recombinant
human alpha-glucosidase (rhGAA, also known as alglucosidase alfa) in participants with Pompe
Disease.
A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease
Terminated
Genzyme, a Sanofi Company
Phase 4
2012-03-01
The objective of this study was to evaluate the efficacy and safety of treatment with 4000
litre (L) alglucosidase alfa (Lumizyme®) in Pompe participants.
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
Active, not recruiting
Genzyme, a Sanofi Company
Phase 3
2016-11-02
Primary Objective:
To determine the effect of avalglucosidase alfa treatment on respiratory muscle strength
measured by percent (%) predicted forced vital capacity (FVC) in the upright position, as
compared to alglucosidase alfa.
Secondary Objective:
To determine the safety and effect of avalglucosidase alfa treatment on functional endurance
(6-minute walk test, inspiratory muscle strength (maximum inspiratory pressure), expiratory
muscle strength (maximum expiratory pressure), lower extremity muscle strength (hand-held
dynamometry), motor function (Quick Motor Function Test), and health-related quality of life
(Short Form-12).
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