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Your journey
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More info
You can access this
clinical trial
if you have
Glycogen Storage Disease Type II-Pompe's Disease
and you are
between 6 and 17
years old
2
This is a second phase trial assessing
efficacy and side effects of the new treatment.
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The purpose

Primary Objective: To evaluate the safety profile of avalglucosidase alfa in patients with infantile-onset Pompe disease (IOPD) previously treated with alglucosidase alfa. Secondary Objective: To characterize the pharmacokinetic profile of avalglucosidase alfa and to evaluate the preliminary efficacy of avalglucosidase alfa in comparison to alglucosidase alfa.

Provided treatments

  • Drug: avalglucosidase alfa GZ402666
  • Drug: alglucosidase alfa GZ419829

Locations near you

Unfortunately, there are no recruiting locations near you. Please check the list with all locations below.
Tris trial is registered with FDA with number: NCT03019406. The sponsor of the trial is Genzyme, a Sanofi Company and it is looking for 22 volunteers for the current phase.
Official trial 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