This trial is terminated!
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Your journey
1What's a trial
2Find
3Review
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More info
You can access this
clinical trial
if you have
Pompe Disease
and you are
over 18
years old
1
2
This is a medium phase trial determining
the safety and dosage of the new treatment, as well as its efficacy.
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The purpose

This Phase I/II open-label, randomized, dose-escalation study will assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of VAL-1221 versus Myozyme®/Lumizyme® in participants with late-onset glycogen storage disease-II (GSD-II) (Pompe disease)

Provided treatments

  • Drug: VAL-1221
  • Drug: RhGAA

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: NCT02898753. The sponsor of the trial is Valerion Therapeutics, LLC and it is looking for 12 volunteers for the current phase.
Official trial title:
A Three-Month, Open-Label, Randomized, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 Versus Myozyme®/Lumizyme® in Patients With Late-Onset GSD-II (Pompe Disease) Followed by Open-Label Treatment With VAL-1221 in All Patients