Critical Issues

Enrolling patients in the registry

Alport syndrome is not a common disease. Recruitment of sufficient numbers of participants for meaningful therapeutic trials will require a multicenter effort. ASTOR will work with patients, families and physicians on Informed Consent, HIPAA consent, IRB processing, and related issues. Regardless of enrollment status, ASTOR will work with families and physicians on any questions related to Alport syndrome.

Treatment options

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 trials 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 physicians and patients at multiple centers.