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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

Active, not recruiting · Phase 2 · NCT03019406

Conditions: Glycogen Storage Disease Type II-Pompe's Disease

At a glance

Research payments reported, 2025
$664,175
U.S. investigators named
3
Sites paid
5
Planned or actual enrollment
22

About the trial

Sponsor
Genzyme, a Sanofi Company
Tested
Avalglucosidase alfa (GZ402666) (drug), Alglucosidase alfa (GZ419829) (drug)
Dates
Started 2017-10-12; completion 2027-08-10
Results posted
Yes

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.

From ClinicalTrials.gov, last updated 2026-01-30. Full record, eligibility and contacts (opens in a new tab)

Industry funding

Genzyme Corporation reported $664,175 in research payments for this study in 2025 (27 payments).

Products named: Nexviazyme

Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.

U.S. investigators named on this study

Clinicians the company named as principal investigators in its Open Payments reports.

Sites paid to run it

Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.