A Twelve-Month, Open-Label, Randomized, Parallel Active Control, Single and Repeat Dose, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 Delivered Intravenously (IV) in Ambulatory and Ventilator-free Patients With Late-Onset Glycogen Storage Disease-II (GSD-II) (Pompe Disease)

Trial Profile

A Twelve-Month, Open-Label, Randomized, Parallel Active Control, Single and Repeat Dose, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 Delivered Intravenously (IV) in Ambulatory and Ventilator-free Patients With Late-Onset Glycogen Storage Disease-II (GSD-II) (Pompe Disease)

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 26 Sep 2017

At a glance

  • Drugs VAL 1221 (Primary)
  • Indications Glycogen storage disease type II
  • Focus Adverse reactions
  • Sponsors Valerion Therapeutics
  • Most Recent Events

    • 20 Sep 2017 Planned number of patients changed from 16 to 12.
    • 20 Sep 2017 Planned End Date changed from 1 Jun 2018 to 1 Dec 2018.
    • 20 Sep 2017 Planned primary completion date changed from 1 Jun 2018 to 1 Dec 2018.
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