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NCT03019406 · ClinicalTrials.gov registry record · Phase 2
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
A Phase 2 study of Glycogen Storage Disease Type II-Pompe's Disease, sponsored by Genzyme, a Sanofi Company.
- Active
- Registry status
- Phase 2
- Development phase
- 22
- Enrollment target
- 12
- Study locations
NCT03019406: Active Phase 2 study of Glycogen Storage Disease Type II-Pompe's Disease, sponsored by Genzyme, a Sanofi Company.
NCT03019406 is a Phase 2 study of Glycogen Storage Disease Type II-Pompe's Disease that is active but no longer recruiting, run by Genzyme, a Sanofi Company. The registered enrollment target is 22 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (83% lower). The trial reports 12 study locations across 6 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03019406, a Phase 2 study of Glycogen Storage Disease Type II-Pompe's Disease, is active but no longer recruiting, sponsored by Genzyme, a Sanofi Company.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 22 participants
- Enrollment target
- 12
- Study locations
Study 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.
Primary Outcome
Adverse event (AE): any untoward medical occurrence in participant received study drug \& did not necessarily had to have causal relationship with treatment. TEAEs: AEs developed/worsened in grade/become serious during PAP period (from the time of 1st study drug dose up to Week 25). Serious AE(SAE): any untoward medical occurrence at any dose resulted in death, was life-threatening, required inpatient hospitalization, prolongation of existing hospitalization, resulted in persistent or significan
Conditions Studied
Interventions
- DRUG Avalglucosidase alfa (GZ402666)
- DRUG Alglucosidase alfa (GZ419829)
Study Locations (12)
Other
- Investigational Site Number : 2500004 - Nantes
- Investigational Site Number : 2500003 - Paris
- Investigational Site Number : 2500002 - Paris
- Investigational Site Number : 2500001 - Tours
- Investigational Site Number : 1580001 - Hsinchu
- Investigational Site Number : 8260002 - Manchester
Tokyo
- Investigational Site Number : 3920001 - Fuchu-shi
- Investigational Site Number : 3920002 - Fuchu-shi
New York
- Regional Medical Genetics Center of New York Site Number : 8400002 - Valhalla
North Carolina
- Duke University Medical Center Site Number : 8400001 - Durham
Washington
- Seattle Childrens Hospital and Regional Medical Center- Site Number : 8400005 - Seattle
London, City of
- Investigational Site Number : 8260001 - London
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 22 participants |
| Start Date | 2017-10-12 |
| Est. Completion | 2027-08-10 |
| Phase | Phase 2 |
What the registry record for NCT03019406 still lists
NCT03019406 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 22 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (83% lower).
The record links to 1 condition, with Glycogen Storage Disease Type II-Pompe's Disease appearing as the primary indexed condition, and to 2 interventions - of which Avalglucosidase alfa (GZ402666) is the first listed.
NCT03019406 lists 12 locations in 6 states (Other, Tokyo, New York).
Frequently Asked Questions
What is clinical trial NCT03019406 about?
NCT03019406 is a clinical study titled "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". 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 ...
What is the current status of trial NCT03019406?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 22 participants. The study started on 2017-10-12. Estimated completion is 2027-08-10.
What conditions does trial NCT03019406 study?
This clinical trial studies the following conditions: Glycogen Storage Disease Type II-Pompe's Disease.
What interventions are being tested in trial NCT03019406?
The interventions under investigation include: Avalglucosidase alfa (GZ402666) (DRUG), Alglucosidase alfa (GZ419829) (DRUG).
Who is sponsoring clinical trial NCT03019406?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03019406 being conducted?
This trial has 12 study locations across New York, North Carolina, Washington, Tokyo, London, City of. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03019406's enrollment target sits among peer trials
22 1790th of 2000 higher than 207 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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