Clinical Studies Volunteers

The Cystinosis Research Foundation is dedicated to funding research that will lead to improved treatments and a cure for cystinosis. The following studies are being funded by the CRF to advance knowledge in key areas of cystinosis treatment.  Click the following links to see the full information for each study.

The Pediatric Neurology Research Group at the University of California, San Diego (UCSD) is conducting an onlinle research cystinosis adult outcome study.   Funding for this research grant was provided by the Cystinosis Research Foundation.
 

Update Phase 3 Clinical Trials - Raptor Pharmaceuticals DR Cysteamine
We are proud to report that the Cystinosis Research Foundation provided funding for every bench and clinical research study leading to the discovery of the delayed-release medication.  
Raptor Pharmaceuticals has completed the Phase 3 clinical trials of RP103, the delayed-release oral formulation of cysteamine, (DR Cysteamine) in 41 patients with cystinosis.  A report on the data from the trial was announced in 2011.  The extension phase of the clinical trial, in which all patients completing the Phase 3 study may elect to continue on DR Cysteamine therapy, is ongoing. Raptor anticipates FDA approval of the new drug in early 2012.  The slow-release medication will be a reality for all of our children in the near future.  The Cystinosis Research Foundation's primary focus is to find a cure for cystinosis. 

"Stress and Coping in Cystinosis," UCSD Pediatric Neurology Research Group 

This study will examine stress reactions, coping responses, quality of life, and medical adherence in individuals with cystinosis.  Individuals with cystinosis ages 12 years through adulthood and their parents will be asked to complete several questionnaires.