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| ID | Type | Description | Link |
|---|---|---|---|
| LTS13930 | Other Identifier | Other company study code |
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To collect uniform and meaningful data on patients with Pompe disease who experience anaphylaxis, severe allergic reactions, and/or signals of severe cutaneous and/or systemic immune complex-mediated reactions following treatment with alglucosidase alfa.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| pompe safety sub-registry | patients are selected from those who are enrolled in the Pompe Registry, and will be followed for safety evaluation in this sub-registry |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| alglucosidase alfa | Biological | Alglucosidase alfa IV infusion of 20 mg/kg; qow |
|
| Measure | Description | Time Frame |
|---|---|---|
| number of patients experience anaphylaxis, severe allergic reactions and/or signals of severe cutaneous and/or systematic immune complex-mediated reactions | collect meaningful data on patients with these outcomes following treatment with alglucosidase alfa | 4 Years |
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Inclusion Criteria:
Exclusion Criteria:
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Patients who are enrolled in the Pompe Registry will be enrolled in this sub-registry include patients with infantile-onset Pompe disease, as well as those with late-onset Pompe disease.
An approximately equal proportion of currently treated and treatment-naïve patients is targeted for enrollment at each site.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor | Genzyme, a Sanofi Company | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigational Site Number 840016 | Phoenix | Arizona | 85013 | United States | ||
| Investigational Site Number 840002 |
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| ID | Term |
|---|---|
| D006009 | Glycogen Storage Disease Type II |
| D000707 | Anaphylaxis |
| ID | Term |
|---|---|
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
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| ID | Term |
|---|---|
| C509951 | GAA protein, human |
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| Durham |
| North Carolina |
| 27710 |
| United States |
| Investigational Site Number 840004 | Hershey | Pennsylvania | 17033 | United States |
| Investigational Site Number 840014 | Pittsburgh | Pennsylvania | 15213 | United States |
| Investigational Site Number 840008 | Salt Lake City | Utah | 84113 | United States |
| Investigational Site Number 840001 | Fairfax | Virginia | 22030 | United States |
| Investigational Site Number 056001 | Ghent | 9000 | Belgium |
| Investigational Site Number 056002 | Leuven | 3000 | Belgium |
| Investigational Site Number 056003 | Leuven | 3000 | Belgium |
| Investigational Site Number 203001 | Prague | 12808 | Czechia |
| Investigational Site Number 276002 | Halle | 06120 | Germany |
| Investigational Site Number 380001 | Brescia | 25123 | Italy |
| Investigational Site Number 380002 | Cagliari | 09126 | Italy |
| Investigational Site Number 380006 | Messina | 98125 | Italy |
| Investigational Site Number 380005 | Milan | 20133 | Italy |
| Investigational Site Number 380004 | Padova | 35128 | Italy |
| Investigational Site Number 380003 | Roma | 00168 | Italy |
| Investigational Site Number 158001 | Taipei | 10043 | Taiwan |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006008 | Glycogen Storage Disease |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D006969 | Hypersensitivity, Immediate |
| D006967 | Hypersensitivity |
| D007154 | Immune System Diseases |