drugset
drugset / Trial / NCT02898753

VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease

NCT02898753 ↗

Phase 1/2 Terminated 12 enrolled Valerion Therapeutics, LLC
RandomizedParallel-groupOpen-labelTreatment

Summary

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)

Timeline

Start
2017-06-21
Primary completion
2020-03-25
Completion
2020-03-25

Drugs

EvaluationDrugModalityDoseRoute
Subject CLERVONAFUSP ALFA Protein / enzyme biologic 3 mg/kg Intravenous
Subject CLERVONAFUSP ALFA Protein / enzyme biologic 10 mg/kg Intravenous
Subject CLERVONAFUSP ALFA Protein / enzyme biologic 30 mg/kg Intravenous
Comparator alglucosidase alfa Protein / enzyme biologic — —

Indications

glycogen storage disease due to acid maltase deficiency, late-onset