Cystinosis is an inherited disease that if untreated, results in kidney failure as early as the first decade of life. The current marketed therapy is Cystagon® (cysteamine bitartrate) which must be taken every six hours for the rest of the patient's life to prevent complications of cystinosis. RP103 is a formulation of cysteamine bitartrate that is being studied to see if it may be able to be given less frequently, once every 12 hours, and have similar results to four times a day Cystagon®.
This is a multi-center, open-label, randomized, cross-over study to determine whether steady-state, twice a day treatment with Cysteamine Bitartrate Delayed-release Capsules(RP103) results in comparable depletion of white blood cell (WBC) cystine levels compared to the existing four times a day cysteamine treatment. It will involve up to 20 clinic visits plus intermittent home use of the RP103. Most of these clinic visits occur in clusters of 3-4 consecutive days. Eligible patients will be offered enrollment into a long-term follow up study. Study with completed results acquired from Horizon in 2024.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
43
Run-in Period (Weeks 1, 2, 3) and Period 1 (Weeks 4, 5, 6) or Period 2 (Weeks 7, 8, 9); Immediate crossover to opposite treatment than taken during Period 1: Every 6H, supplied in 150 and 50mg capsules/Duration of Treatment: 3 weeks each period used
Period 1 (Weeks 4, 5, 6) or Period 2 (Weeks 7, 8, 9); Immediate crossover to opposite treatment than taken during Period 1: Every 12H, supplied in 75 and 25mg capsules/Duration of Treatment: 3 weeks
Stanford University Medical School
Stanford, California, United States
Emory Children's Center
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago (formerly Children's Memorial Hospital)
Chicago, Illinois, United States
Hospices Civils de Lyon
Lyon, France
The Steady-state White Blood Cell Cystine Levels of RP103 Compared to Cystagon®
Time frame: 4 weeks after the last subject has completed the study
Comparison of Cysteamine PK Profiles, Steady State Cmax, Between RP103 and Cystagon®.
Time frame: 4 weeks after the last subject has completed the study
Comparison of Cysteamine PK Profiles, Steady State Tmax, Between RP103 and Cystagon®.
Time frame: 4 weeks after the last subject has completed the study
Comparison of Cysteamine PK Profiles, AUC(0-t), Between RP103 and Cystagon®.
Time frame: 6 hours post dosing for Cystagon®; 12 hours post dosing for RP103.
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Villeneuve-Lapeyronie Hospital
Montpellier, France
Necker Hospital
Paris, France
Robert Debre Hospital
Paris, France
Radboud University Nijmegen Medical Center
Nijmegen, Netherlands