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Alglucosidase Alfa Pompe Safety Sub-Registry

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01710813
Recruitment Status : Active, not recruiting
First Posted : October 19, 2012
Last Update Posted : May 18, 2020
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Brief Summary:
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.

Condition or disease Intervention/treatment
Pompe Disease Biological: alglucosidase alfa

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Study Type : Observational [Patient Registry]
Actual Enrollment : 110 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 4 Years
Official Title: 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
Study Start Date : March 20, 2015
Estimated Primary Completion Date : March 2021
Estimated Study Completion Date : April 2021


Group/Cohort Intervention/treatment
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




Primary Outcome Measures :
  1. number of patients experience anaphylaxis, severe allergic reactions and/or signals of severe cutaneous and/or systematic immune complex-mediated reactions [ Time Frame: 4 Years ]
    collect meaningful data on patients with these outcomes following treatment with alglucosidase alfa



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

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.

Criteria

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01710813


Locations
Show Show 18 study locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
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Study Director: Medical Monitor Genzyme, a Sanofi Company
Additional Information:

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Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT01710813    
Other Study ID Numbers: AGLU06909
LTS13930 ( Other Identifier: Other company study code )
First Posted: October 19, 2012    Key Record Dates
Last Update Posted: May 18, 2020
Last Verified: May 2020
Keywords provided by Sanofi ( Genzyme, a Sanofi Company ):
pompe disease
alglucosidase alfa
anaphylaxis
severe cutaneous
systemic immune complex-mediated reactions
Additional relevant MeSH terms:
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Glycogen Storage Disease Type II
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Glycogen Storage Disease
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases