Trial record 11 of 16 for:
Open Studies | "Anaphylaxis"
Alglucosidase Alfa Pompe Safety Sub-Registry
Verified September 2014 by Sanofi
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
First received: October 17, 2012
Last updated: September 29, 2014
Last verified: September 2014
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.
Biological: Alglucosidase alfa
||Observational Model: Cohort
Time Perspective: Prospective
||A Prospective Safety Sub-Registry to Assess Anaphylaxis and Severe Allergic Reactions, and Severe Cutaneous and Systemic Immune Complex Mediated Reactions With Alglucosidase Alfa Treatment
Primary Outcome Measures:
| Estimated Enrollment:
| Study Start Date:
| Estimated Study Completion Date:
| Estimated Primary Completion Date:
||August 2022 (Final data collection date for primary outcome measure)
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
Biological: Alglucosidase alfa
IV infusion of 20 mg/kg; qow
Other Name: Myozyme; Lumizyme
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
Patients who 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.
- the patient must be enrolled in the Pompe Registry;
- have a confirmed diagnosis of Pompe disease (confirmation of diagnosis is defined as documented GAA enzyme deficiency from any tissue source and/or documentation of 2 GAA gene mutations);
- be naïve to treatment with alglucosidase alfa (4000-L), plan to be treated with alglucosidase alfa, or have been treated with alglucosidase alfa for less than 2 years.
- if the patient does not plan to receive treatment with alglucosidase alfa (4000-L scale)
- patients who have received an investigational drug (excluding alglucosidase alfa) within 30 days prior to signing a sub-registry consent form
- if they are taking or plan to take any investigational product while enrolled in the sub-registry.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01710813
|Cambridge, Massachusetts, United States |
Genzyme, a Sanofi Company
||Genzyme, a Sanofi Company
No publications provided
||Sanofi ( Genzyme, a Sanofi Company )
History of Changes
|Other Study ID Numbers:
|Study First Received:
||October 17, 2012
||September 29, 2014
||United States: Food and Drug Administration
European Union: European Medicines Agency
Keywords provided by Sanofi:
systemic immune complex-mediated reactions
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on November 20, 2014
Glycogen Storage Disease Type II
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Carbohydrate Metabolism, Inborn Errors
Central Nervous System Diseases
Genetic Diseases, Inborn
Glycogen Storage Disease
Lysosomal Storage Diseases
Lysosomal Storage Diseases, Nervous System
Metabolism, Inborn Errors
Nervous System Diseases