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NCT04848779 · ClinicalTrials.gov registry record
A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)
A clinical trial of Glycogen Storage Disease Type II, sponsored by Sanofi.
- Active
- Registry status
- 16
- Enrollment target
- 15
- Study locations
NCT04848779: Active study of Glycogen Storage Disease Type II, sponsored by Sanofi.
NCT04848779 is a study of Glycogen Storage Disease Type II that is active but no longer recruiting, run by Sanofi. The registered enrollment target is 16 participants, below the 674-participant average among 3 other Glycogen Storage Disease Type II trials with a reported enrollment target (98% lower). The trial reports 15 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04848779, a study of Glycogen Storage Disease Type II, is active but no longer recruiting, sponsored by Sanofi.
- ACTIVE NOT RECRUITING
- Registry status
- 16 participants
- Enrollment target
- 15
- Study locations
Study Summary
Primary Objective: To describe the effect of routine practice with alglucosidase alfa in patients with IOPD ≤6 months of age, on invasive ventilation-free survival after 52 weeks of treatment. Secondary Objectives: * To describe the effect of routine practice with alglucosidase alfa on invasive ventilation-free survival and survival at 12 and 18 months of age, as well as on change in left ventricular mass (LVM) Z score, Alberta Infant Motor Scale (AIMS) score, body weight, body length, and head circumference Z scores, and urinary glucose tetrasaccharide (Hex4), at Week 52 of treatment. * To describe the safety, tolerability, and immunogenicity of alglucosidase alfa in the routine practice of IOPD treatment.
Conditions Studied
Interventions
- DRUG Alglucosidase alfa GZ419829
Study Locations (15)
Other
- Investigational Site Number : 0560001 - Leuven
- Investigational Site Number : 2500001 - Tours
- Investigational Site Number : 2760001 - Giessen
- Investigational Site Number : 3800001 - Florence
- Investigational Site Number : 5280001 - Rotterdam
- Investigational Site Number : 1580001 - Taipei
- Investigational Site Number : 8260002 - Manchester
New York
- Advanced Medical Genetics- Site Number : 8400002 - Hawthorne
North Carolina
- Duke University Medical Center- Site Number : 8400004 - Durham
Ohio
- Cincinnati Children's Hospital Medical Center- Site Number : 8400001 - Cincinnati
Tennessee
- Le Bonheur Children's Hospital- Site Number : 8400005 - Memphis
Washington
- Seattle Children's Hospital- Site Number : 8400003 - Seattle
Monza E Brianza
- Investigational Site Number : 3800002 - Monza
Catalunya [Cataluña]
- Investigational Site Number : 7240001 - Esplugues de Llobregat
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 16 participants |
| Start Date | 2021-06-10 |
| Est. Completion | 2026-10-28 |
What the registry record for NCT04848779 still lists
NCT04848779 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 16 participants, a relatively small participant target, below the 674-participant average among 3 other Glycogen Storage Disease Type II trials with a reported enrollment target (98% lower).
The record links to 1 condition, with Glycogen Storage Disease Type II appearing as the primary indexed condition, and to 1 intervention - of which Alglucosidase alfa GZ419829 is the first listed.
NCT04848779 lists 15 locations in 9 states (Other, New York, North Carolina).
Frequently Asked Questions
What is clinical trial NCT04848779 about?
NCT04848779 is a clinical study titled "A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)". Primary Objective: To describe the effect of routine practice with alglucosidase alfa in patients with IOPD ≤6 months of age, on invasive ventilation-free survival after 52 weeks of treatment. Secondary Objectives: * To describe the effect of routine practice with alglucosidase alfa on invasive v...
What is the current status of trial NCT04848779?
This trial is currently active not recruiting. The enrollment target is 16 participants. The study started on 2021-06-10. Estimated completion is 2026-10-28.
What conditions does trial NCT04848779 study?
This clinical trial studies the following conditions: Glycogen Storage Disease Type II.
What interventions are being tested in trial NCT04848779?
The interventions under investigation include: Alglucosidase alfa GZ419829 (DRUG).
Who is sponsoring clinical trial NCT04848779?
This trial is sponsored by Sanofi, which has 713 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04848779 being conducted?
This trial has 15 study locations across New York, North Carolina, Ohio, Tennessee, Washington. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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