2상모집 중단 (진행 중)
A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa
연구 개요
This is a multi-stage, phase 2, open-label, multicenter, multinational, ascending dose cohort, repeated intravenous (IV) infusion study of avalglucosidase alfa in pediatric patients with Infantile-Onset Pompe Disease (IOPD) who have been previously treated with alglucosidase alfa for a minimum of 6 months immediately prior to study entry and have demonstrated clinical decline or unsatisfactory clinical response.
- 스폰서
- 리드: 사노피
- 적응증
- Glycogen Storage Disease Type II-Pompe's Disease
- 중재
- Avalglucosidase alfa (GZ402666), Alglucosidase alfa (GZ419829)
- 목표 등록
- 22명
- 시작일
- 2017-10-12
- 완료(예정)
- 2027-08-10
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