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

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborators:
University of Utah
Peking University First Hospital
University of Toronto
University of Göttingen
Hôpital Necker-Enfants Malades
Information provided by (Responsible Party):
University of Minnesota - Clinical and Translational Science Institute
ClinicalTrials.gov Identifier:
NCT01696253
First received: September 20, 2012
Last updated: June 19, 2014
Last verified: June 2014
  Purpose

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.


Condition
Alport Syndrome

Study Type: Observational
Study Design: Observational Model: Case-Only
Official Title: Multi-center Controlled Clinical Trials in Alport Syndrome-A Feasibility Study

Resource links provided by NLM:


Further study details as provided by University of Minnesota - Clinical and Translational Science Institute:

Primary Outcome Measures:
  • Multi-center Controlled Clinical Trials in Alport Syndrome-A Feasibility Study [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    The primary outcome to be measured is the number of subjects with Alport syndrome who would meet eligibility criteria for an anticipated clinical treatment trial. Power calculations indicate that the trial would require recruitment of 90 total subjects, stratified into 4 groups based on urine albumin and protein excretion.


Secondary Outcome Measures:
  • Multi-center Controlled Clinical Trials in Alport Syndrome-A Feasibility Study [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
    The primary outcome to be measured is the differences in urinary uromodulin excretion in Alport subjects stratified by urinary albumin excretion.


Biospecimen Retention:   Samples Without DNA

First morning clean catch urine sample.


Estimated Enrollment: 360
Study Start Date: September 2012
Estimated Study Completion Date: August 2014
Estimated Primary Completion Date: August 2014 (Final data collection date for primary outcome measure)
Groups/Cohorts
Subjects at risk for Alport sydrome
Newly identified subjects with Alport syndrome

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   1 Year and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

A family is eligible for this multi-center feasibility study if a firm diagnosis of AS in a family member has been made by skin biopsy, kidney biopsy, or molecular genetic analysis.

In order to enhance identification of subjects with early stage AS (normalbuminuria or MA) and subjects with untreated proteinuria, investigators at each recruitment site will contact all known AS families in their databases, seeking at-risk children whose disease status is unknown. The family populations to be queried at each site are estimated in the table below:

Criteria

Inclusion Criteria: Study subjects under the age of 18 in all Study groups will require parental consent. The Principal Investigator is applying for a waiver of assent agreements by all subjects under 13 years of age. Parents will sign a formal parental consent form provided by each regional site research staff.

Exclusion Criteria: Subjects under the age of 5 years and those subjects without Alport syndrome.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01696253

Locations
United States, Minnesota
University Of Minnesota
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
University of Minnesota - Clinical and Translational Science Institute
University of Utah
Peking University First Hospital
University of Toronto
University of Göttingen
Hôpital Necker-Enfants Malades
Investigators
Principal Investigator: Clifford E Kashtan, MD University of Minnesota - Clinical and Translational Science Institute
  More Information

Additional Information:
No publications provided

Responsible Party: University of Minnesota - Clinical and Translational Science Institute
ClinicalTrials.gov Identifier: NCT01696253     History of Changes
Other Study ID Numbers: 1206M15384
Study First Received: September 20, 2012
Last Updated: June 19, 2014
Health Authority: United States: Institutional Review Board

Additional relevant MeSH terms:
Nephritis, Hereditary
Urogenital Abnormalities
Nephritis
Kidney Diseases
Urologic Diseases
Congenital Abnormalities
Collagen Diseases
Connective Tissue Diseases

ClinicalTrials.gov processed this record on July 31, 2014