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Pulmonary Involvement in Patients With Fabry Disease

This study has been completed.
Information provided by (Responsible Party):
University of Zurich Identifier:
First received: June 28, 2012
Last updated: May 26, 2014
Last verified: May 2014
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.

Condition Intervention
Fabry Disease Other: Lung function measurement

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Retrospective
Official 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

Resource links provided by NLM:

Further study details as provided by University of Zurich:

Primary Outcome Measures:
  • 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

Enrollment: 110
Study Start Date: July 2012
Study Completion Date: May 2014
Primary Completion Date: May 2014 (Final data collection date for primary outcome measure)
Intervention Details:
    Other: Lung function measurement
    Other Name: FEV1, FVC
Detailed Description:
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).

Ages Eligible for Study:   16 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Ambulatory patients at University Hospital of Zurich with Fabry disease

Inclusion criteria:

  • All patients with established diagnosis of Fabry disease

Exclusion criteria:

  • Missing informed consent
  Contacts and Locations
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Please refer to this study by its identifier: NCT01632111

University Hospital Zurich, Division of Internal Medicine
Zurich, Switzerland
Sponsors and Collaborators
University of Zurich
Principal Investigator: Daniel Franzen, MD University Hospital Zurich, Division of Internal Medicine
  More Information

Responsible Party: University of Zurich Identifier: NCT01632111     History of Changes
Other Study ID Numbers: Fabry
Study First Received: June 28, 2012
Last Updated: May 26, 2014

Additional relevant MeSH terms:
Fabry Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders processed this record on August 18, 2017