Registry of breastfeeding women with Pompe disease treated with Myozyme

Start Date
01/12/2007
Expected end Date
01/12/2021
Location
USA
Status
Recruiting

What is the aim of the trial?

This study is an observational study and no new or experimental drugs or treatments will be given to the participants.

Pompe disease (also known as glycogen storage disease type II) is caused by a lack of a critical enzyme in the body called ‘acid alpha-glucosidase’. A lack of this enzyme causes glycogen (a type of sugar) to accumulate in the tissues of the body, especially heart and skeletal muscle, stopping them from working properly.

Myozyme is a treatment for Pompe disease which replaces the missing enzyme. The main aim of this study is to test for the presence of Myozyme in the breast milk of women with Pompe disease who have been treated with Myozyme. This study will also look at breast milk production and composition.

Who can be involved in the trial?

This trial is for women with Pompe disease who are either pregnant and intending to breast-feed or currently lactating. Participants must receive at least one infusion of Myozyme while lactating.

Participants in the trial must also be participants in the Pompe Registry, a database of patients with Pompe disease that was created to aid understanding of and research into the condition and its treatment.

What happens during the trial?

Participants in the trial will be asked to provide samples of blood and breast milk.

Where is the study taking place?

People who live anywhere in the world can participate in this study.

How could the results of the trial benefit patients?

This study will enable researchers to find out what effect taking Myozyme has on breast milk and milk production. The findings from this study will help ensure that breast-feeding children are not put at undue risk if their mother is administered Myozyme.

Contact Details:

Medical Information, Genzyme

Tel: +1 800 745 4447

Email: medinfo@genzyme.com

Official name of the Trial:

Pompe Lactation Sub-Registry

Trial study number:

NCT00566878

Further trial details:

For further information on the trial and detailed inclusion and exclusion please click on the link below. Sometimes these details can be quite technical. If you have any questions please discuss this with your clinician or contact the clinical trial organisers.

http://clinicaltrials.gov/ct2/show/study/NCT00566878

Background information and related links:

This study is an observational study and no new or experimental drugs or treatments will be given to the participants.

Pompe disease (also known as glycogen storage disease type II) is caused by a lack of a critical enzyme in the body called ‘acid alpha-glucosidase’. A lack of this enzyme causes glycogen (a type of sugar) to accumulate in the tissues of the body, especially heart and skeletal muscle, stopping them from working properly.

Myozyme is a treatment for Pompe disease which replaces the missing enzyme. The main aim of this study is to test for the presence of Myozyme in the breast milk of women with Pompe disease who have been treated with Myozyme. This study will also look at breast milk production and composition.

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