Conditions: Cystinosis
Keywords: cystinosis cysteamine inheritable disease orphan disease CTNS protein, human nephropathic cystinosis
Allocation: NON_RANDOMIZED | Intervention model: SINGLE_GROUP
Enrollment: 9
Healthy volunteers: no

Brief summary:
Cystinosis is an inheritable 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.

Detailed description:
This is a single-dose, open-labeled, non-randomized, two-period study of Cysteamine Bitartrate Delayed-release Capsules (RP103) and Cystagon® in up to 10 patients (male or female) with nephropathic cystinosis under fasting conditions. It will involve a 4 night check-in to a clinical research center.

Study with completed results acquired from Horizon in 2024

Arms:
arm 1: Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg arm 2: Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg

Interventions: intervention 1: Cystagon® intervention 2: RP103
Intervention descriptions: intervention 1: Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg.

Duration of Treatment and Dose: Reference Period up to four doses Q6H. intervention 2: Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg.

Duration of treatment and Dose: Single dose of Test Product at dose equivalent to Reference Product.

Protocol document: