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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 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. The trial reports 12 study locations across 6 states.
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.
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03019406
The ClinicalTrials.gov registry entry for NCT03019406 describes a study currently listed as active not recruiting, categorized as Phase 2. The registered enrollment target is 22 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Genzyme, a Sanofi Company, which has 196 total studies on file at ClinicalTrials.gov.
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 reports 12 study locations spanning 6 distinct geographic areas - top geographies include 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.
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