Cure Cystinosis International Registry
Cure Cystinosis International Registry
Cure Cystinosis International Registry (CCIR) is an online, patient self-identifying registry
 developed by medical and scientific experts specifically for the cystinosis community.

 CCIR's sole purpose is to identify people with cystinosis worldwide in an effort to
 accelerate novel treatments and a cure for cystinosis.

 CCIR provides a safe and secure platform for:

 -  sharing anonymous medical information about cystinosis with researchers, clinicians and
 patients

 -  disseminating information about research opportunities

 -  connecting researchers/investigators and prospective participants *

 Interested cystinosis patients may register themselves with CCIR online at
 http://www.cystinosisregistry.org.

 * No personal information is shared outside of CCIR. Individual identities are known only to
 appropriate CCIR staff. If a participant is matched to a clinical trial, the participant
 receives a notice from CCIR, after which they can decide whether they wish to contact the
 study sponsor.
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.
Inclusion Criteria:

 -  Diagnosis of cystinosis