NCT00872729

Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis

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

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Amgen · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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

Conditions

• Cystinosis

Interventions

Type	Name	Description
DRUG	Cystagon®	Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg. Duration of Treatment and Dose: Reference Period up to four doses Q6H.
DRUG	RP103	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.

Timeline

• Start date: 2009-05-01
• Primary completion: 2009-10-01
• Completion: 2009-10-01
• First posted: 2009-03-31
• Last updated: 2024-12-19
• Results posted: 2014-10-28

Locations

1 site across 1 country: United States