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Cure Cystinosis International Registry (CCIR)

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2014 by Cystinosis Research Foundation
Sponsor:
Information provided by (Responsible Party):
Jerry A. Schneider, Cystinosis Research Foundation
ClinicalTrials.gov Identifier:
NCT01327807
First received: March 29, 2011
Last updated: March 10, 2014
Last verified: March 2014

March 29, 2011
March 10, 2014
August 2010
December 2020   (final data collection date for primary outcome measure)
Age at treatment initiation and the effect on disease outcomes [ Time Frame: Lifetime ] [ Designated as safety issue: No ]
Not Provided
Complete list of historical versions of study NCT01327807 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
 
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.

Observational
Time Perspective: Cross-Sectional
Not Provided
Not Provided
Non-Probability Sample

International cystinosis community

  • Cystinosis
  • Nephropathic Cystinosis
  • Renal Fanconi Syndrome
Not Provided
Cystinsosis patients
Those with a diagnosis of cystinosis.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
750
December 2022
December 2020   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Diagnosis of cystinosis
Both
Not Provided
No
Contact: Betty L Cabrera, M.P.H 858-822-3747 curator@cystinosisregistry.org
United States
 
NCT01327807
CCIR100913
No
Jerry A. Schneider, Cystinosis Research Foundation
Cystinosis Research Foundation
Not Provided
Principal Investigator: Jerry A Schneider, M.D. University of California, San Diego
Cystinosis Research Foundation
March 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP