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Pulmonary Involvement in Patients With Fabry Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01632111
Recruitment Status : Completed
First Posted : June 29, 2012
Last Update Posted : May 28, 2014
Information provided by (Responsible Party):
University of Zurich

Brief Summary:
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 or disease Intervention/treatment
Fabry Disease Other: Lung function measurement

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).

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Study Type : Observational
Actual Enrollment : 110 participants
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
Study Start Date : July 2012
Actual Primary Completion Date : May 2014
Actual Study Completion Date : May 2014

Resource links provided by the National Library of Medicine

Intervention Details:
  • Other: Lung function measurement
    Other Name: FEV1, FVC

Primary Outcome Measures :
  1. 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

Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years and older   (Child, Adult, Older Adult)
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01632111

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University Hospital Zurich, Division of Internal Medicine
Zurich, Switzerland
Sponsors and Collaborators
University of Zurich
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Principal Investigator: Daniel Franzen, MD University Hospital Zurich, Division of Internal Medicine
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Responsible Party: University of Zurich Identifier: NCT01632111    
Other Study ID Numbers: Fabry
First Posted: June 29, 2012    Key Record Dates
Last Update Posted: May 28, 2014
Last Verified: May 2014
Additional relevant MeSH terms:
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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