Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis
|ClinicalTrials.gov Identifier: NCT00872729|
Recruitment Status : Completed
First Posted : March 31, 2009
Results First Posted : October 28, 2014
Last Update Posted : May 19, 2017
|Condition or disease||Intervention/treatment||Phase|
|Cystinosis||Drug: Cystagon® Drug: RP103||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||9 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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 Patients With Nephropathic Cystinosis|
|Study Start Date :||May 2009|
|Actual Primary Completion Date :||October 2009|
|Actual Study Completion Date :||October 2009|
Active Comparator: Cystagon®
Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg
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
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.
- 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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00872729
|United States, California|
|University of California San Diego Medical Center|
|San Diego, California, United States, 92103|
|Study Director:||Evelyn Olson, BS||Horizon Pharma USA, Inc.|