Pulmonary Disease and Exercise Tolerance in Boys With Fabry Disease

Status unconfirmed

Conditions studied: Fabry Disease

In brief

When to start children with Fabry disease on therapy is controversial because of its expense and inconvenience. Many Fabry children complain of exercise intolerance. In adults, the investigators have found decreased lung function and ability to exercise on a treadmill. Whether or not lung function and exercise capacity is abnormal in children is unknown. While lung function and exercise tests are commonly part of routine evaluations for adults with Fabry, they are not yet for children. The objective of the proposed study is to more accurately define the lung and exercise abnormalities in a group of 20 boys from 8-18 years of age with Fabry disease who have not been treated with enzyme replacement therapy (Fabrazyme).

Key facts

Study ID

NCT01304875

Run by

Cedars-Sinai Medical Center

People needed

20

Starts

2010-04-01

Last updated by the study team

2013-06-27

Who can join

Age: 8 and older, up to 18. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Fabry disease
• Male
• Between 8-18 years of age
• Enrolled in Fabry registry and have standard assessments

You may not qualify if…

• Enzyme replacement therapy or an experimental therapy
• Inability to perform the tests
• Other, serious medical conditions that would impact the tests

Where it is running

• Cedars-Sinai Medical Center — Los Angeles, California, United States (enrolling)

Full record on ClinicalTrials.gov

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