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.
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
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
9
Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg. Duration of Treatment and Dose: Reference Period up to four doses Q6H.
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.
University of California San Diego Medical Center
San Diego, California, United States
Plasma Pharmacokinetic Parameter: Cmax of Cysteamine
Time frame: 12 hours post RP103 dosing and 7 hours post 1st Cystagon® dosing
Plasma Pharmacokinetic Parameter: Tmax of Cysteamine
Time frame: 12 hours post RP103 dosing and 7 hours post 1st Cystagon® dosing
Plasma Pharmacokinetic Parameter: AUC(0-t) of Cysteamine
t = 6 for Cystagon and t = 12 for RP103. Cystagon is dosed every 6 hours and there is no measurement after 6 hours and up to 12 hours.
Time frame: 12 hours post RP103 dosing and 6 hours post 1st Cystagon® dosing
Pharmacodynamic Parameter: Changes of White Blood Cell (WBC) Cystine Level From Baseline
The pharmacodynamic (PD) parameter measures the changes of WBC cystine level from the baseline. Cystine is a disulfide amino acid formed through oxidation of two molecules of cysteine; hence, cystine's concentration is commonly given in half-cystine equivalents to avoid confusion. The level of cystine in WBC/leukocytes is expressed in units of nmol half-cystine/mg protein (nmol ½ cystine/mg protein). Half-cystine is quantified by a reduction of cystine followed by an assay for cysteine, which is then normalized by the total cellular protein content within the sample using methods of such as Lowry assay, bicinchoninic acid assay, or Bradford.
Time frame: up to 12 hours post Cystagon® dosing and RP103 dosing
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