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Clinical trial

An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients With Infantile-onset Pompe Disease Treated With Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response

NCT03019406 is registered in ClinicalTrials.gov with Genzyme, a Sanofi Company as lead sponsor.

NCT NCT03019406
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Registry record

As filed in ClinicalTrials.gov.

Title
An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients With Infantile-onset Pompe Disease Treated With Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response
Lead sponsor
Genzyme, a Sanofi Company
Phase
Phase 2
Overall status
Active, not recruiting
Start date
October 12, 2017
Completion date
August 10, 2027
Enrollment
22 (actual)
Sites
12

Conditions

As the registry lists them; a condition with its own page is linked.

glycogen storage disease type ii-pompe's disease