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

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

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

Brief 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 Summary: 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 RP103. Enrollment will be offered first to those patients who have completed the previous Phase 3 Study (RP103-03, NCT01000961) and won't be offered to others until data analysis of the RP103-03 subjects has been completed.
Sponsor: Horizon Pharma USA, Inc.

Current Primary Outcome: Safety and tolerability of long-term repeat dosing of RP103 in patients with nephropathic cystinosis. [ Time Frame: 30 days after Last Patient Last Visit ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Steady-state pharmacokinetics (PK) and pharmacodynamics (PD) of RP103. [ Time Frame: 30 days after Last Patient Last Visit ]
  • Long term quality of life using either PedsQL™ or SF-36® instruments. [ Time Frame: 30 days after Last Patient Last Visit ]


Original Secondary Outcome: Same as current

Information By: Horizon Pharma USA, Inc.

Dates:
Date Received: September 3, 2010
Date Started: August 2010
Date Completion: June 2017
Last Updated: April 11, 2017
Last Verified: April 2017