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 Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)
Active, not recruiting · NCT04848779 · Registry updated 2026-06-24Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD) | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, disorder of glycogen metabolism, glycogen storage disease II, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, nervous system disorder | Active, not recruiting | Not stated | 2026-06-24 | NCT04848779 |
1 results · Page 1 of 1
Organizations
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| Organizations | Linked trials |
|---|---|
| Sanofi | 254 |
5 results · Page 1 of 1
Research sites
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| Research sites | City | Country | Linked trials |
|---|---|---|---|
| Advanced Medical Genetics- Site Number : 8400002 | Hawthorne | United States | 2 |
| Cincinnati Children's Hospital Medical Center- Site Number : 8400001 | Cincinnati | United States | 2 |
| Duke University Medical Center- Site Number : 8400004 | Durham | United States | 2 |
| Le Bonheur Children's Hospital- Site Number : 8400005 | Memphis | United States | 1 |
| Seattle Children's Hospital- Site Number : 8400003 | Seattle | United States | 2 |