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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

NCT03019406 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 22

Last updated 2026-01-30

Study results available
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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.

Conditions

  • Glycogen Storage Disease Type II-Pompe's Disease

Interventions

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

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
6 Months
Max Age
17 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2017-10-12
Primary Completion
2019-09-30
Completion
2027-08-10
FDA Drug
Yes

Countries

  • United States
  • France
  • Japan
  • Taiwan
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03019406 on ClinicalTrials.gov