NCT03019406

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

An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients With Infantile-onset Pompe Disease Treated With Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 22 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 6 Months – 17 Years
• Healthy volunteers: Accepted

Summary

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.

Detailed description

The duration of the study for each participant will be up to 9 years and 4 months that will consist of a 14-day screening period, that may be extended to up to 4 weeks in pre-specified situations. This is followed by a 25-week treatment period and an up to a 462-week treatment extension period and a 4-week post-treatment observation period. Cohort 1 and 2 (Cohort 1: avalglucosidase alfa 20 milligrams per kilogram \[mg/kg\], Cohort 2: avalglucosidase alfa 40 mg/kg) will be non-randomized and Cohort 3 (Cohort 3a: avalglucosidase alfa 40 mg/kg \[maximum tolerated dose\] and Cohort 3b: alglucosidase alfa) will be randomized.

Conditions

• Glycogen Storage Disease Type II-Pompe's Disease

Interventions

Type	Name	Description
DRUG	Avalglucosidase alfa (GZ402666)	Pharmaceutical form: powder for concentrate for solution for infusion, Route of administration: IV
DRUG	Alglucosidase alfa (GZ419829)	Pharmaceutical form: powder for concentrate for solution for infusion, Route of administration: IV

Timeline

• Start date: 2017-10-12
• Primary completion: 2019-09-30
• Completion: 2027-08-10
• First posted: 2017-01-12
• Last updated: 2026-01-30
• Results posted: 2021-11-22

Locations

12 sites across 5 countries: United States, France, Japan, Taiwan, United Kingdom

Regulatory

• FDA-regulated drug study