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Alglucosidase Alfa 160L vs 4000L in Infantile-Onset Pompe Disease
NCT01597596Phase 4
Key facts
- Status
- Terminated
- Start
- Aug 2012
- Primary completion
- Dec 2014
- Countries
- 3
- Sponsor
- Sanofi
- Modality
- Replacement Enzymes
- Drugs
- alglucosidase alfa
- Conditions
- Pompe disease
01
Overview
Phase 4, open-label trial of alglucosidase alfa in Pompe disease (2 arms), sponsored by Sanofi. Primary endpoint: Change From Baseline in Cardiac Function at 52 weeks. Started Aug 2012; status: terminated.
02
Design
2 arms- Study design
- Non-randomizedOpen-label
4000 L Scale (Non-US)
Alglucosidase alfa manufactured at 4000 L scale
160 L Scale (US)
Alglucosidase alfa manufactured at 160 L scale
03
Who it enrols
Infants <= 12 months with documented GAA deficiency, treatment-naïve, CRIM-positive, without invasive ventilator support or heart failure.
04
Endpoints
Primary
Change From Baseline in Cardiac Function
Secondary
Percentage of Participants With Estimated Probability of Survival
Number of Participants With Invasive Ventilator-Free Survival
Change From Baseline in Motor Development Status
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