Recruitment

Recruitment Status
Active, not recruiting
Estimated Enrollment
18

Summary

Conditions
Glycogen Storage Disease Type II Pompe Disease
Type
Interventional
Phase
Phase 2 & Phase 3
Design
  • Allocation: N/A
  • Intervention Model: Single Group Assignment
  • Masking: None (Open Label)
  • Primary Purpose: Treatment

Participation Requirements

Age
Younger than 125 years
Gender
Both males and females

Description

The duration of the study will be 6 years from the date the first patient enters the study. Each patient will continue with the study until the patient withdraws, the Investigator withdraws the patient, or the Sponsor terminates the study. An additional follow-up phase will begin after the patient's...

The duration of the study will be 6 years from the date the first patient enters the study. Each patient will continue with the study until the patient withdraws, the Investigator withdraws the patient, or the Sponsor terminates the study. An additional follow-up phase will begin after the patient's last planned visit in the 6-year study and last until avalglucosidase alfa is approved in the patient's country for all patients.

Locations

København Ø, 2100
Leuven, 3000
Mainz, 55131
München, 80336
Newcastle Upon Tyne, NE1 4LP
...
København Ø, 2100
Leuven, 3000
Mainz, 55131
München, 80336
Newcastle Upon Tyne, NE1 4LP
Dallas, Texas, 75390
Fairfax, Virginia, 22030
Rotterdam, 3015 GJ
Saint Louis, Missouri, 63110
Durham, North Carolina, 27710
Paris, 75013
Münster, 48149
Nice, 06000
Jacksonville, Florida, 32209
Cincinnati, Ohio, 45219
Kansas City, Kansas, 66160-7321
Phoenix, Arizona, 85013

Tracking Information

NCT #
NCT02032524
Collaborators
Not Provided
Investigators
Study Director: Clinical Sciences & Operations Sanofi