NCT01197378

Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)

A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients With Cystinosis

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 60 (actual)
• Sponsor: Amgen · Industry
• Sex: All
• Age: 1 Year
• 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 immediate release) which must be taken every six hours for the rest of the patient's life to prevent complications of cystinosis. Cysteamine bitartrate delayed-release capsules (RP103) is a formulation of cysteamine bitartrate that is being studied to see if it can be given less frequently, once every 12 hours, and have similar results to four times a day Cystagon®.

Detailed description

This is a long-term, open-label, study to determine the safety and tolerability of twice a day treatment with cysteamine bitartrate delayed-release capsules (RP103). It will involve 6-9 monthly clinic visits followed by quarterly clinic visits for the duration of the study and home use of cysteamine bitartrate delayed-release capsules. Initially, enrollment was open to those patients who had completed the previous Phase 3 Study (RP103-03, NCT01000961). Subsequently enrollment in Study RP103-04 was opened to additional participants, including children aged 1 to 6 years and renal transplant recipients, who had previously been on a stable dose of Cystagon® for at least 21 days. Study with completed results acquired from Horizon in 2024.

Conditions

• Cystinosis

Interventions

Type	Name	Description
DRUG	Cysteamine Bitartrate Delayed-release Capsules	Participants who entered the trial from the RP103-03 study continued treatment with cysteamine bitartrate every 12 hours at the last dose level prescribed during their participation in that study. Participants not entering the trial from Study RP103-03 were started on twice a day administration of cysteamine bitartrate at a total daily RP103 dose of 70% of their pre-study total daily stable Cystagon® dose.

Timeline

• Start date: 2010-08-27
• Primary completion: 2017-06-26
• Completion: 2017-06-26
• First posted: 2010-09-09
• Last updated: 2024-12-27
• Results posted: 2018-07-24

Locations

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