Significance and Purpose:
Many different resources and tools are necessary to make significant advances in medical
research. Progress in rare diseases such as cystinosis can often be impeded by the lack of
information available about the disease and limited access to volunteers eligible for
clinical trials. Therefore, patients who are willing to provide information about how the
disease has affected them and also make themselves available to participate in trials are
among the most valuable resources the investigators have to fight a disease. However, the
research community desperately needs the right tool that will permit access to these
resources.
A tool widely used to conveniently collect both data about a disease and information about
potential clinical trial participants is a patient registry. A patient registry is any system
that allows for the organized collection of data about disease outcomes in affected
populations for a scientific, clinical, or policy purpose. The Cystinosis Research Foundation
(CRF) has aligned itself with cystinosis medical experts and organizations worldwide to
create the first ever international, online patient registry for cystinosis, Cure Cystinosis
International Registry (CCIR). The express purpose of CCIR is to make anonymous information
available to the research community and thus promote accelerated research in advanced
treatments and ultimately a cure for cystinosis.
Objectives:
The objectives of CCIR are:
Evaluate epidemiology and clinical characteristics of cystinosis around the world.
Evaluate and compare the diagnosis, treatment, and kidney transplant rates among
cystinosis communities from different geographical areas.
Enhance the understanding of how cystinosis affects quality of life.
CCIR Registration:
Interested cystinosis patients may register themselves with CCIR online at
http://www.cystinosisregistry.org. Registration is easy and secure. Simply go to the website
and create a CCIR account and complete a survey. The CCIR website is currently available in
English and Spanish, and will soon be available in French, Portuguese, and possibly other
languages.
Benefits to CCIR participants include instant access to the registry's accumulated survey
results (reported as anonymous group data), and opportunities to submit questions to
cystinosis experts and to learn of clinical trial opportunities. No personal information is
shared outside of CCIR. Individual identities are known only to appropriate CCIR staff.
