ClinicalTrials.gov record

Active, not recruiting Phase 2 Interventional Accepts healthy volunteers Results available

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

ClinicalTrials.gov ID: NCT03019406

Public ClinicalTrials.gov record NCT03019406. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

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ClinicalTrials.gov public records Last synced May 20, 2026, 1:05 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

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

Study identification

NCT ID: NCT03019406
Recruitment status: Active, not recruiting
Study type: Interventional
Phase: Phase 2
Lead sponsor: Genzyme, a Sanofi Company; Industry
Enrollment: 22 participants

Conditions and interventions

Conditions

Glycogen Storage Disease Type II-Pompe's Disease

Interventions

Alglucosidase alfa (GZ419829) Drug
Avalglucosidase alfa (GZ402666) Drug

Drug

Eligibility (public fields only)

Age range: 6 Months to 17 Years
Sex: All
Healthy volunteers: Accepts healthy volunteers

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date: Oct 11, 2017
Primary completion: Sep 29, 2019
Completion: Aug 9, 2027
Last update posted: Jan 29, 2026

2017 – 2027

United States locations

U.S. sites: 3
U.S. states: 3
U.S. cities: 3

Facility	City	State	ZIP	Site status
Regional Medical Genetics Center of New York Site Number : 8400002	Valhalla	New York	10595	—
Duke University Medical Center Site Number : 8400001	Durham	North Carolina	27710-4000	—
Seattle Childrens Hospital and Regional Medical Center- Site Number : 8400005	Seattle	Washington	98040	—

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 9 non-U.S. sites.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT03019406, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do: No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from: Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh: Last update posted Jan 29, 2026 · Synced May 20, 2026

Open the official record

The complete protocol, eligibility criteria, and contact information for NCT03019406 live on ClinicalTrials.gov.

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