Pulmonary Involvement in Patients With Fabry Disease

NCT01632111 | Completed

• 1 other identifier: Fabry
• Study Type: observational
• Target: 110
• Locations: 1 country
• Sites: 1

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.

Conditions

Fabry Disease

Outcome Measures

Primary Outcomes (1)

• Lung function measurements

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

  From first consultation until present

Interventions

Lung function measurement OTHER

Spirometry

Also known as: FEV1, FVC

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Ambulatory patients at University Hospital of Zurich with Fabry disease

You may qualify if:

• All patients with established diagnosis of Fabry disease

You may not qualify if:

• Missing informed consent

Sponsors & Collaborators

• University of Zurich: lead

Study Sites (1)

University Hospital Zurich, Division of Internal Medicine

Zurich, Switzerland

Location

MeSH Terms

Conditions

Fabry Disease

Interventions

Vital Capacity

Condition Hierarchy (Ancestors)

Sphingolipidoses; 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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

Total Lung Capacity; Lung Volume Measurements; Respiratory Function Tests; Diagnostic Techniques, Respiratory System; Diagnostic Techniques and Procedures; Diagnosis; Respiratory Physiological Phenomena; Circulatory and Respiratory Physiological Phenomena

Study Officials

• Daniel Franzen, MD

  University Hospital Zurich, Division of Internal Medicine

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 28, 2012
• First Posted: June 29, 2012
• Study Start: July 1, 2012
• Primary Completion: May 1, 2014
• Study Completion: May 1, 2014
• Last Updated: May 28, 2014

Record last verified: 2014-05

Locations

CH (1)