Pulmonary Involvement in Patients With Fabry Disease

The objective of this study is to investigate whether Agalsidase alpha, a drug commonly prescribed in patients with Fabry disease, is associated with improvement of the pulmonary involvement. According to the Global Initiative for Obstructive Lung Disease (GOLD), the surrogate markers for obstructive lung diseases are a decrease in both forced expiratory volume in one second (FEV1) and FEV1/FVC ratio, whereas FVC is the forced vital capacity. However, the measurement of these lung function parameters is indicated as yearly follow-up examinations with or without the treatment of Agalsidase alpha in patients with Fabry disease.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Fabry Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Cardiovascular Diseases Central Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses Vascular Diseases

Age range

16 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

University Hospital Zurich, Division of Internal Medicine

Zurich, Switzerland

About this study

Pulmonary function tests und DLCO measurements are performed yearly in relation with yearly follow up examinations in the Department of Internal Medicine from the University Hospital of Zurich. We will retrospectively collect the results of the pulmonary functions test (spirometry).

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

All patients with established diagnosis of Fabry disease

Exclusion criteria

Missing informed consent

Treatment and study plan

Lung function measurement

Other

Spirometry

Other names: FEV1, FVC

Primary outcomes

Lung function measurements

Time frame: From first consultation until present

Retrospective analysis of yearly measured lung function parameters to investigate on the lung function changes in patients with Fabry disease

Sponsors and collaborators

Lead sponsor

University of Zurich

Other

Registry information

Official study title

Impact of the Treatment With Agalsidase Alpha on Lung Function and on Pulmonary Involvement in Patients With Fabry Disease. A Multicenter, Retrospective Observational Study

Important dates

Study start: 2012
Primary completion: 2014
Study completion: 2014
First posted: Jun 29, 2012
Registry last updated: May 28, 2014

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