Recruitment

Recruitment Status
Active, not recruiting
Estimated Enrollment
18

Summary

Conditions
Glycogen Storage Disease Type II Pompe Disease
Type
Interventional
Phase
Phase 2Phase 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.

Tracking Information

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