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Multi-center Controlled Clinical Trials in Alport Syndrome-A Feasibility Study

NCT01696253 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 360

Last updated 2018-03-22

No results posted yet for this study

Summary

Over the past 30 years much has been learned about the molecular genetics and natural history of familial forms of hematuria. However, enhanced understanding of these conditions has yet to generate effective therapies for Alport syndrome(AS), the form of familial hematuria associated with end-stage renal disease. Males with AS inevitably develop end-stage kidney failure, with a 50% likelihood of dialysis or kidney transplantation by age 25 years. There is no proven treatment for AS, although studies in animals have suggested several promising potential therapies. Pharmacological or biological treatments that might delay or prevent the development of kidney failure exist, but need to be evaluated through clinical trials. Researchers interested in implementing clinical trials in AS will face several challenges, the foremost of which is the relative rarity of the disease, necessitating aggressive efforts to identify and recruit potential subjects for multi-center collaborative clinical trials. The Alport Syndrome Research Collaborative (ARC) was established in 2009 as a partnership of the Alport Syndrome Treatments and Outcomes Registry (ASTOR), the European Alport Registry and centers of AS research in Canada, China and France with the objective of testing potential treatments to delay or prevent terminal renal failure in people with AS. In this feasibility study the five ARC centers will interrogate existing AS registries and databases, and monitor accrual of new AS cases over an 18-month period, in order to quantify subjects in the disease categories of interest. As part of this project we will examine the utility of urinary uromodulin excretion as a marker of kidney injury and potential trial endpoint in AS clinical trials. Our goals are to (1) demonstrate that participating centers have access to sufficient numbers of males and females with AS to populate adequately-powered clinical trials focused on two clinical targets, microalbuminuria and overt proteinuria, and (2) to test the hypothesis that in males with AS urinary uromodulin excretion decreases as albuminuria and proteinuria increase and that uromodulin offers an independent and insightful measure of renal fibrosis and response to therapy.

Conditions

  • Alport Syndrome

Sponsors & Collaborators

  • University of Utah

    collaborator OTHER

  • Peking University First Hospital

    collaborator OTHER

  • University of Toronto

    collaborator OTHER

  • University of Göttingen

    collaborator OTHER

  • Hôpital Necker-Enfants Malades

    collaborator OTHER

  • University of Minnesota

    lead OTHER

Principal Investigators

  • Clifford E Kashtan, MD · University of Minnesota

Eligibility

Min Age
1 Year
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-09-30
Primary Completion
2014-08-31
Completion
2017-12-31

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01696253 on ClinicalTrials.gov