Fabry DiseaseNCT01632111
Pulmonary Involvement in Patients With Fabry Disease
An observational study of Fabry Disease, run by University of Zurich at 1 site in Switzerland.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Lung function measurement
- How many people
- 110 participants
- Where
- 1 siteSwitzerland
- Study dates
- Jul 2012to May 2014
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Ambulatory patients at University Hospital of Zurich with Fabry disease
Who may be able to join
- All patients with established diagnosis of Fabry disease
Who cannot join
- Missing informed consent
Trial locations and contacts
1 site in 1 country.
Switzerland1 site
University Hospital Zurich, Division of Internal Medicine
Zurich
About this study
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
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.
Read the 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).
- Conditions
- Fabry Disease
- Being tested
- Lung function measurement
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lung function measurementOther
Also called FEV1, FVC
Spirometry
What is being measured
The primary outcome is the main question the study is designed to answer.
Lung function measurements
Retrospective analysis of yearly measured lung function parameters to investigate on the lung function changes in patients with Fabry disease
Measured: From first consultation until present
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jun 2012 (estimated)
- Study startJul 2012
- Primary completionMay 2014
- Study completionMay 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- More trials run by University of ZurichThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 110 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Zurich
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Daniel Franzen, MD, Principal investigator, University Hospital Zurich, Division of Internal Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01632111
- Sponsor's protocol ID
- Fabry
- Status last verified
- May 2014
- First submitted
- 28 Jun 2012
- Last updated
- 28 May 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 May 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01632111
- Acronym / sponsor ID
- Fabry
- Conditions
- Fabry Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 110 participants (actual)
- Lead sponsor
- University of ZurichUniversity, hospital or other
- Study start
- Jul 2012
- Primary completion
- May 2014 (actual)
- Study completion
- May 2014 (actual)
- First posted
- 29 Jun 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Ambulatory patients at University Hospital of Zurich with Fabry disease
Key inclusion criteria (1 listed)
- All patients with established diagnosis of Fabry disease
Key exclusion criteria (1 listed)
- Missing informed consent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Lung function measurementAlso: FEV1, FVCSpirometry | Other | – |
Primary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 May 2014.
Related trials for Fabry Disease
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A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
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Evaluation of the Effect of a Low-FODMAP Diet on Gastrointestinal Symptoms and Inflammatory Markers in Fabry Patients
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A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Daniel Franzen, MD principal investigator
University Hospital Zurich, Division of Internal Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fabry Disease, run by University of Zurich at 1 site in Switzerland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 May 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Switzerland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Lung function measurements (time frame: From first consultation until present). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 May 2014.