Clinical Trial: Alport Syndrome Treatments and Outcomes Registry
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational [Patient Registry]
Official Title: Alport Syndrome Treatments and Outcomes Registry
Brief Summary:
Over the past 30 years much has been learned about the molecular genetics and natural history of familial forms of hematuria. However, the enhanced understanding of these conditions has yet to generate effective therapies for Alport syndrome, the form of familial hematuria associated with end-stage renal disease. Males with Alport syndrome 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 Alport syndrome, although studies in animals have suggested several promising potential therapies. Potential drug treatments that might delay or prevent the development of kidney failure exist, but need to be evaluated through clinical trails. Conducting clinical trails for proposed treatments for Alport syndrome present many challenges. Because Alport syndrome is not a common disease, informative clinical trials will require the collaboration of investigators at multiple centers.
The University of Minnesota, Department of Pediatrics, proposes to create the Alport Syndrome Treatments and Outcomes Registry (ASTOR) in order to facilitate clinical trials for the treatment of Alport Syndrome. This registry will be the first of its kind in North America. Because Alport syndrome is a rare disorder, recruitment of sufficient participants for meaningful therapeutic trials will require a multicenter effort. The primary objective of establishing and sustaining this registry is to enable clinical natural history studies and therapeutic trials to be conducted in children and adolescents with Alport syndrome.
Detailed Summary:
Detailed Description:
ASTOR is envisioned as a permanent organization sustained by private philanthropic and public funding sources. Recruitment of participants for the registry will consist of three approaches. First, pediatric nephrologists in the United States and Canada will be invited to participate in ASTOR. Activities of participating investigators will include identification and recruitment of potential study participants, collection and transmission of participant data to the ASTOR central office at the University of Minnesota, and implementation of study protocols.
Participating physicians will inform their participants and parents about ASTOR and invite them to participate in writing. The correspondence will include instructions on how to contact ASTOR personal about formal participation. Participants and their parents will be asked to permit sharing of their personal health information with the ASTOR central office.
Second, the ASTOR central office will develop a website for participants and families. The website will provide participants and families with medical and new research information and instructions on how to contact registry staff about registry participation. Potential participants who contact the central office will be asked to give consent for ASTOR staff to contact their health care providers.
Third, the University of Utah School of Medicine's Department of Nephrology, currently holds data pertaining to approximately 350 families with a reported history of Alport syndrome. Department investigators have agreed to provide information that will ultimately assist ASTOR staff in executing recruitment activities by providing historical medical health information pertaining the natural progression of the
Sponsor: University of Minnesota - Clinical and Translational Science Institute
Current Primary Outcome: Data Collection: natural history study [ Time Frame: Ongoing ]
Original Primary Outcome:
Current Secondary Outcome:
Original Secondary Outcome:
Information By: University of Minnesota - Clinical and Translational Science Institute
Dates:
Date Received: May 30, 2007
Date Started: September 2007
Date Completion: January 2022
Last Updated: January 24, 2017
Last Verified: January 2017
