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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
  Purpose

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.


Condition
Cystinosis
Nephropathic Cystinosis
Renal Fanconi Syndrome

Study Type: Observational
Study Design: Time Perspective: Cross-Sectional
Official Title: Cure Cystinosis International Registry

Resource links provided by NLM:


Further study details as provided by Cystinosis Research Foundation:

Primary Outcome Measures:
  • Age at treatment initiation and the effect on disease outcomes [ Time Frame: Lifetime ] [ Designated as safety issue: No ]

Estimated Enrollment: 750
Study Start Date: August 2010
Estimated Study Completion Date: December 2022
Estimated Primary Completion Date: December 2020 (Final data collection date for primary outcome measure)
Groups/Cohorts
Cystinsosis patients
Those with a diagnosis of cystinosis.

Detailed Description:

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.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

International cystinosis community

Criteria

Inclusion Criteria:

  • Diagnosis of cystinosis
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01327807

Contacts
Contact: Betty L Cabrera, M.P.H 858-822-3747 curator@cystinosisregistry.org

Locations
United States, California
University of California, San Diego Recruiting
San Diego, California, United States, 92103-8450
Contact: Betty L Cabrera, M.P.H.    619-471-9554    curator@cystinosisregistry.org   
Principal Investigator: Jerry A Schneider, M.D.         
Sponsors and Collaborators
Cystinosis Research Foundation
Investigators
Principal Investigator: Jerry A Schneider, M.D. University of California, San Diego
  More Information

Additional Information:
Publications:
Responsible Party: Jerry A. Schneider, Professor Emeritus of Pediatrics, Cystinosis Research Foundation
ClinicalTrials.gov Identifier: NCT01327807     History of Changes
Other Study ID Numbers: CCIR100913
Study First Received: March 29, 2011
Last Updated: March 10, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by Cystinosis Research Foundation:
Registries
Cystinosis
Nephropathic Cystinosis
Renal Fanconi Syndrome
Cystine
Kidney
Rare Diseases

Additional relevant MeSH terms:
Cystinosis
Fanconi Anemia
Fanconi Syndrome
Syndrome
Anemia
Anemia, Aplastic
Anemia, Hypoplastic, Congenital
Bone Marrow Diseases
DNA Repair-Deficiency Disorders
Disease
Genetic Diseases, Inborn
Hematologic Diseases
Kidney Diseases
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Pathologic Processes
Renal Tubular Transport, Inborn Errors
Urologic Diseases

ClinicalTrials.gov processed this record on November 25, 2014