Pulmonary Involvement in Patients With Fabry Disease | NCT01632111

Studies
Pulmonary Involvement in Patients With Fabry Disease

NCT01632111

Sponsor

University of Zurich

Status

Completed

Last Update Posted

May 28, 2014Estimated

Enrollment

110Actual

Phase

Not provided

Conditions

Fabry Disease

Interventions

Lung function measurement

Countries

Switzerland

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

May 2014

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jul 2012

Primary Completion Date

May 2014Actual

Completion Date

May 2014Actual

First Submitted Date

Jun 28, 2012

First Submission Date that Met QC Criteria

Jun 28, 2012

First Posted Date

Jun 29, 2012Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

May 26, 2014

Last Update Posted Date

May 28, 2014Estimated

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

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).

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

110Actual

Name	Type	Description	Arm Group Labels	Other Names
Lung function measurement	Other	Spirometry		FEV1, FVC

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion criteria:

All patients with established diagnosis of Fabry disease

Exclusion criteria:

Missing informed consent

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

16 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Ambulatory patients at University Hospital of Zurich with Fabry disease

Sampling Method

Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Daniel Franzen, MD	University Hospital Zurich, Division of Internal Medicine	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
University Hospital Zurich, Division of Internal Medicine		Zurich			Switzerland

Condition Browse Module

MeSH Terms

ID	Term
D000795	Fabry Disease

Ancestor Terms

ID	Term
D013106	Sphingolipidoses
D020140	Lysosomal Storage Diseases, Nervous System
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
D014797	Vital Capacity

Ancestor Terms

ID	Term
D014109	Total Lung Capacity
D008176	Lung Volume Measurements
D012129	Respiratory Function Tests
D003948	Diagnostic Techniques, Respiratory System

Browse Leaves

Not provided

Browse Branches

Not provided