Clinical Trials Logo

Clinical Trial 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.


Clinical Trial 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. ;


Study Design

Time Perspective: Cross-Sectional


Related Conditions & MeSH terms


NCT number NCT01327807
Study type Observational
Source Cystinosis Research Foundation
Contact Betty L Cabrera, M.P.H
Phone 858-822-3747
Email curator@cystinosisregistry.org
Status Recruiting
Phase N/A
Start date August 2010
Completion date December 2022

See also
  Status Clinical Trial Phase
Recruiting NCT05545774 - Neuromuscular Characterisation in Late Adolescent and Adult Cystinosis Patients
Completed NCT01432561 - Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagonâ„¢ N/A
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Completed NCT04125927 - Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2) Phase 3
Completed NCT00071903 - The Role of Susceptibility to Thrombosis in the Pseudotumor Cerebri of Nephropathic Cystinosis: A Case-Control Study N/A
Recruiting NCT05994534 - PK and PD Study of NPI-001 and Cysteamine Bitartrate Phase 1/Phase 2
Completed NCT00004312 - Establishment of a Database for Long-Term Monitoring of Patients With Nephropathic Cystinosis N/A
Completed NCT00004350 - Evaluation of Fanconi Syndrome and Cystinosis N/A
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Recruiting NCT06065852 - National Registry of Rare Kidney Diseases
Completed NCT02533076 - The Functional Consequences of the CTNS-deletion for the TRPV1-receptor in Cystinosis Patients Phase 0
Enrolling by invitation NCT05146830 - A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04
Completed NCT00872729 - Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis Phase 1/Phase 2
Completed NCT00001736 - New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis Phase 1
Recruiting NCT05959668 - Development of Health-related Quality of Life Instrument for Patients With Cystinosis
Withdrawn NCT02124070 - Therapeutic Effect of Recombinant Human Growth Hormone (rhGH) on the Myopathy of Cystinosis Phase 1/Phase 2
Completed NCT01614431 - N Acetyl Cysteine for Cystinosis Patients Phase 4
Recruiting NCT05843851 - Genetic Newborn Screening for Cystinosis and Primary Hyperoxaluria N/A
Completed NCT01000961 - Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis Phase 3
Active, not recruiting NCT03897361 - Stem Cell Gene Therapy for Cystinosis Phase 1/Phase 2