Clinical Trial: Cure Cystinosis International Registry

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: Cure Cystinosis International Registry

Brief Summary:

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.

Detailed Summary:

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 cyst
  Sponsor: Cystinosis Research Foundation
  

  Current Primary Outcome: Age at treatment initiation and the effect on disease outcomes [ Time Frame: Lifetime ]
  
  

  Original Primary Outcome:
  
  

  Current Secondary Outcome:
  
  

  Original Secondary Outcome:
  
  Information By: Cystinosis Research Foundation
  

  Dates:
  Date Received: March 29, 2011
  Date Started: August 2010
  Date Completion: December 2022
  Last Updated: March 10, 2014
  Last Verified: March 2014