A Pilot Study to Assess the Safety, Tolerability and
Pharmacokinetics (PK) and Pharmacodynamics (PD) of Cysteamine Bitartrate
Delayed-release Capsules (RP103), Compared to Cysteamine Bitartrate Capsules, (CystagonŽ)
in Patients with Nephropathic Cystinosis.
The University of California, San Diego (UCSD)
will be studying an investigational cysteamine drug (RP103) for the potential
treatment of cystinosis. RP103 will be compared to the existing treatment,
CystagonŽ. Dr. Bruce Barshop is the principal investigator for this study.
The study requires a 5 day/4 night in-patient stay in the Clinical Research
Center and involves blood draws and other evaluations. Travel arrangements will
be made at no expense to the participant and compensation for participating in
the study will be provided. Daily living expenses for a family member or
guardian accompanying a minor will also be provided.
Eligible patients must be on a stable dose of CystagonŽ for 21 days prior to
starting the study, be able to swallow the 150 mg capsules whole, and not have
received a transplant. More information is available at clinicaltrials.gov [link
to:
http://clinicaltrials.gov/ct2/show/NCT00872729?term=cystinosis&rank=3].
Raptor Pharmaceuticals is sponsoring this study. It is the first clinical study
planned with Cysteamine Bitartrate Delayed-release Capsules.
Participation in research is always entirely voluntary. If you are interested in
participating and would like more information, please contact Betty Cabrera
B.S., M.P.H. at University of California San Diego Medical Center, San Diego,
California, United States, 92103. Phone: (619) 471-9554 or email:
blcabrera@ucsd.edu.