Alglucosidase Alfa Pompe Safety Sub-Registry

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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Key information

Conditions

Pompe Disease Anaphylaxis Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Carbohydrate Metabolism, Inborn Errors Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Glycogen Storage Disease Glycogen Storage Disease Type II Hypersensitivity Hypersensitivity, Immediate Immune System Diseases Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Investigational Site Number 056001, Ghent, Belgium

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

alglucosidase alfa

Biological

Alglucosidase alfa IV infusion of 20 mg/kg; qow

Other names: Myozyme; Lumizyme

Primary outcomes

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

Sponsors and collaborators

Lead sponsor

Genzyme, a Sanofi Company

Industry

Registry information

Official study 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

Important dates

Study start: 2015
Primary completion: 2021
Study completion: 2021
First posted: Oct 19, 2012
Registry last updated: Jul 13, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed