Conditions: Cystinosis
Keywords: cystinosis cysteamine inheritable disease orphan disease CTNS protein, human metabolic disease nephropathic cystinosis
Allocation: RANDOMIZED | Intervention model: CROSSOVER
Enrollment: 43
Healthy volunteers: no

Brief summary:
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®.

Detailed description:
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.

Arms:
arm 1: None arm 2: None

Interventions: intervention 1: Cystagon® (Cysteamine Bitartrate) intervention 2: Cysteamine Bitartrate Delayed-release Capsules (RP103)
Intervention descriptions: intervention 1: 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 intervention 2: 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

Protocol document: