Trial record 11 of 16 for:
Open Studies | "Anaphylaxis"
Alglucosidase Alfa Pompe Safety Sub-Registry
Verified March 2015 by Sanofi
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
First received: October 17, 2012
Last updated: March 24, 2015
Last verified: March 2015
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
|Target Follow-Up Duration:
||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
Alglucosidase alfa IV infusion of 20 mg/kg; qow
Other Name: Myozyme; Lumizyme
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
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.
- The patient must be enrolled in the Pompe Registry;
- Provide a signed patient information and authorization form;
- 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 and plan to be treated with alglucosidase alfa at or prior to enrollment, or are being treated with alglucosidase alfa.
- Patients will be excluded if they have received an investigational drug (excluding alglucosidase alfa) within 30 days prior to signing a Safety Sub-Registry Patient Information and Authorization form, or if they are taking or plan to take any investigational product while enrolled in the Safety 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
|Investigational Site Number 056001
|Gent, Belgium, 9000 |
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
||March 24, 2015
||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 April 16, 2015
Glycogen Storage Disease Type II
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Central Nervous System Diseases
Genetic Diseases, Inborn
Glycogen Storage Disease
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Metabolism, Inborn Errors
Nervous System Diseases