Fabry DiseaseNCT00343577
Antiproteinuric Agents and Fabry Disease
An observational study of Fabry Disease and Proteinuria, run by University of Alabama at Birmingham at 1 site in United States.
Trial summary
Fabry disease is a rare disorder that often has kidney involvement with increased urine protein excretion. Proteinuria is recognized as an important risk factor for progression of chronic kidney disease. Read more about this study
- Who is it for?
- Ages 14 to 95 years
- Conditions the study is for
- Fabry Disease, Proteinuria
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2001to Dec 2006
- 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 14 to 95 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- genetically confirmed Fabry disease
- institution of commercially available agalsidase-beta
Who cannot join
- s/p kidney transplant
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Alabama at Birmingham
Birmingham, Alabama, 35294-0006
About this study
Official title: Observational Study of Antiproteinuric Agents in Patients With Fabry Disease Treated With Enzyme Replacement Therapy
Fabry disease is a rare disorder that often has kidney involvement with increased urine protein excretion. Proteinuria is recognized as an important risk factor for progression of chronic kidney disease. Our hypothesis is that using drugs that reduce urine protein excretion (ACE inhibitors and ARBs) will have a beneficial effect on patients with Fabry disease who already are receiving enzyme replacement therapy. A longitudinal, observational study is being undertaken to determine the utility of these agents in Fabry disease, realizing that these agents are primarily indicated for reducing systemic blood pressure, and most patients with Fabry disease have relatively low blood pressures at baseline.
Read the detailed description
Fabry disease is a rare disorder that often has kidney involvement with increased urine protein excretion. Proteinuria is recognized as an important risk factor for progression of chronic kidney disease. Our hypothesis is that using drugs that reduce urine protein excretion (ACE inhibitors and ARBs) will have a beneficial effect on patients with Fabry disease who already are receiving enzyme replacement therapy. A longitudinal, observational study is being undertaken to determine the utility of these agents in Fabry disease, realizing that these agents are primarily indicated for reducing systemic blood pressure, and most patients with Fabry disease have relatively low blood pressures at baseline.
- Conditions
- Fabry Disease, Proteinuria
- Keywords
- proteinuria, ARBs, agalsidase-beta, MDRD estimated GFR, Chronic kidney disease
- Medical subject headings
- Fabry Disease, Proteinuria, Renal Insufficiency, Chronic
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jun 2006 (estimated)
- Study startJan 2001
- Study completionDec 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- More trials run by University of Alabama at BirminghamThe 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
- Time perspective
- Prospective
- Enrolment
- 12 participants
Sponsor and investigators
- Lead sponsor
- University of Alabama at Birmingham
- Sponsor type
- University, hospital or other
- Investigators
- David G Warnock, MD, Principal investigator, University of Alabama at Birmingham
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00343577
- Sponsor's protocol ID
- X050202007
- Status last verified
- Jun 2006
- First submitted
- 21 Jun 2006
- Last updated
- 19 Nov 2013
- Results posted
- No
Publications
Tahir H, Jackson LL, Warnock DG. Antiproteinuric therapy and fabry nephropathy: sustained reduction of proteinuria in patients receiving enzyme replacement therapy with agalsidase-beta. J Am Soc Nephrol. 2007 Sep;18(9):2609-17. doi: 10.1681/ASN.2006121400. Epub 2007 Jul 26.
PubMed 17656478
Study at a glance
From the registry record, last updated by the sponsor on 19 Nov 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Observational Study of Antiproteinuric Agents in Patients With Fabry Disease Treated With Enzyme Replacement Therapy
- ClinicalTrials.gov ID
- NCT00343577
- Acronym / sponsor ID
- X050202007
- Conditions
- Fabry Disease, Proteinuria
- Keywords
- proteinuria, ARBs, agalsidase-beta, MDRD estimated GFR, Chronic kidney disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 12 participants
- Lead sponsor
- University of Alabama at BirminghamUniversity, hospital or other
- Study start
- Jan 2001
- Study completion
- Dec 2006
- First posted
- 23 Jun 2006 (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.
Fabry disease is a rare disorder that often has kidney involvement with increased urine protein excretion. Proteinuria is recognized as an important risk factor for progression of chronic kidney disease. Our hypothesis is that using drugs that reduce urine protein excretion (ACE inhibitors and ARBs) will have a beneficial effect on patients with Fabry disease who already are receiving enzyme replacement therapy. A longitudinal, observational study is being undertaken to determine the utility of these agents in Fabry disease, realizing that these agents are primarily indicated for reducing systemic blood pressure, and most patients with Fabry disease have relatively low blood pressures at baseline.
Detailed description
Fabry disease is a rare disorder that often has kidney involvement with increased urine protein excretion. Proteinuria is recognized as an important risk factor for progression of chronic kidney disease. Our hypothesis is that using drugs that reduce urine protein excretion (ACE inhibitors and ARBs) will have a beneficial effect on patients with Fabry disease who already are receiving enzyme replacement therapy. A longitudinal, observational study is being undertaken to determine the utility of these agents in Fabry disease, realizing that these agents are primarily indicated for reducing systemic blood pressure, and most patients with Fabry disease have relatively low blood pressures at baseline.
Study design
- Time perspective
- Prospective
Who may qualify
- Ages
- 14 to 95 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- genetically confirmed Fabry disease
- institution of commercially available agalsidase-beta
Key exclusion criteria (1 listed)
- s/p kidney transplant
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Not reported in the source record.
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 19 Nov 2013.
- Results publication: PubMed 17656478Tahir H, Jackson LL, Warnock DG. Antiproteinuric therapy and fabry nephropathy: sustained reduction of proteinuria in patients receiving enzyme replacement therapy with agalsidase-beta. J Am Soc Nephrol. 2007 Sep;18(9):2609-17. doi: 10.1681/ASN.2006121400. Epub 2007 Jul 26.
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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.
David G Warnock, MD principal investigator
University of Alabama at Birmingham
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 and Proteinuria, run by University of Alabama at Birmingham at 1 site in United States.
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 2 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 19 Nov 2013. 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 United States. 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.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00343577, which also lists 1 linked publication.
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 19 Nov 2013.