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)
Phase of Trial: Phase I/II
Latest Information Update: 23 Oct 2017
At a glance
- Drugs VAL 1221 (Primary)
- Indications Glycogen storage disease type II
- Focus Adverse reactions
- Sponsors Valerion Therapeutics
- 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.