NCT01000961

Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis

A Randomized, Crossover Pharmacokinetic and Pharmacodynamic Study to Determine the Safety and Efficacy of Cysteamine Bitartrate Delayed-release Capsules (RP103), Compared to Cystagon® in Patients With Nephropathic Cystinosis

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 43 (actual)
• Sponsor: Amgen · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

Summary

Cystinosis is an inherited 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 to four times a day Cystagon®.

Detailed description

This is a multi-center, open-label, randomized, cross-over study to determine whether steady-state, twice a day treatment with Cysteamine Bitartrate Delayed-release Capsules(RP103) results in comparable depletion of white blood cell (WBC) cystine levels compared to the existing four times a day cysteamine treatment. It will involve up to 20 clinic visits plus intermittent home use of the RP103. Most of these clinic visits occur in clusters of 3-4 consecutive days. Eligible patients will be offered enrollment into a long-term follow up study. Study with completed results acquired from Horizon in 2024.

Conditions

• Cystinosis

Interventions

Type	Name	Description
DRUG	Cystagon® (Cysteamine Bitartrate)	Run-in Period (Weeks 1, 2, 3) and Period 1 (Weeks 4, 5, 6) or Period 2 (Weeks 7, 8, 9); Immediate crossover to opposite treatment than taken during Period 1: Every 6H, supplied in 150 and 50mg capsules/Duration of Treatment: 3 weeks each period used
DRUG	Cysteamine Bitartrate Delayed-release Capsules (RP103)	Period 1 (Weeks 4, 5, 6) or Period 2 (Weeks 7, 8, 9); Immediate crossover to opposite treatment than taken during Period 1: Every 12H, supplied in 75 and 25mg capsules/Duration of Treatment: 3 weeks

Timeline

• Start date: 2010-06-01
• Primary completion: 2011-06-01
• Completion: 2011-08-01
• First posted: 2009-10-23
• Last updated: 2024-12-19
• Results posted: 2014-11-19

Locations

8 sites across 3 countries: United States, France, Netherlands