Clinical Trial: Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Pilot Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Cysteamine Bitartrate Delayed-release Capsules (RP103), Compared to Cysteamine Bitartrate (Cystagon®) in
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 Summary: 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.
Sponsor: Horizon Pharma USA, Inc.
Current Primary Outcome:
- 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 [ Time Frame: 12 hours post RP103 dosing and 6 hours post 1st Cystagon® dosing ]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.
- Pharmacodynamic Parameter: Changes of White Blood Cell (WBC) Cystine Level From Baseline [ Time Frame: up to 12 hours post Cystagon® dosing and RP103 dosing ]
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.
Original Primary Outcome: To assess the safety and tolerability of RP103 compared to Cystagon® [ Time Frame: 24 hours per period ]
Current Secondary Outcome:
Original Secondary Outcome:
- Pharmacodynamic profile of white cell cystine depletion of RP103 compared to Cystagon® [ Time Frame: 24 hours per period ]
- Comparison of plasma PK parameters of cysteamine between RP103 and Cystagon® [ Time Frame: 24 hours per period ]
Information By: Horizon Pharma USA, Inc.
Dates:
Date Received: March 27, 2009
Date Started: May 2009
Date Completion:
Last Updated: April 11, 2017
Last Verified: April 2017
