Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)
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|ClinicalTrials.gov Identifier: NCT01197378|
Recruitment Status : Completed
First Posted : September 9, 2010
Results First Posted : July 24, 2018
Last Update Posted : July 24, 2018
|Condition or disease||Intervention/treatment||Phase|
|Cystinosis||Drug: Cysteamine Bitartrate Delayed-release Capsules||Phase 3|
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 Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||60 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients With Cystinosis|
|Actual Study Start Date :||August 27, 2010|
|Actual Primary Completion Date :||June 26, 2017|
|Actual Study Completion Date :||June 26, 2017|
Experimental: Cysteamine Bitartrate
Cysteamine bitartrate delayed-release capsules were administered twice daily for up to 96 months.
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.
- Number of Participants With Treatment-emergent Adverse Events [ Time Frame: From first dose of study drug to 7 days after the last dose; median duration of treatment was 1461 days. ]
Drug-related adverse events (AEs) are AEs the investigator assessed as having relation to drug of 'possibly', 'probably' or 'definitely'.
The severity of AEs was categorized according to the Common Terminology Criteria for Adverse Events (CTCAE), Version 3.0 as follows:
- Mild (Grade 1): experience is minor and does not cause significant discomfort to subject or change in activities of daily living (ADL); subject is aware of symptoms but symptoms are easily tolerated;
- Moderate (Grade 2): experience is an inconvenience or concern to the subject and causes interference with ADL, but the subject is able to continue with ADL.
- Severe (Grade 3): experience significantly interferes with ADL and the subject is incapacitated and/or unable to continue with ADL
- Life-threatening (Grade 4): experience that, in the view of the Investigator, places the subject at immediate risk of death from the event as it occurred.
- Trough Plasma Cysteamine Concentration [ Time Frame: Day 1 (predose) and Month 6, Years 1, 1.5, 2, 3, 4 and 5 at 0.5 hours post-dose ]Plasma cysteamine concentration was determined using methods employing Hydrophilic Interaction Liquid Chromatography (HILC) high pressure liquid chromatography (HPLC) tandem mass spectrometry (HPLC-MS/MS).
- White Blood Cell Cystine Concentration [ Time Frame: Day 1 (predose) and Month 6, Years 1, 1.5, 2, 3, 4 and 5 at 0.5 hours post-dose ]White blood cell (WBC) cystine concentration was determined using high performance liquid chromatography-electrospray ionization tandem mass spectrometry (LC-ESI-MS/MS).
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01197378
|United States, California|
|California Pacific Medical Center (CPMC) Research Institute|
|San Francisco, California, United States, 94115|
|Stanford University Medical School|
|Stanford, California, United States, 94305|
|United States, Georgia|
|Emory Children's Center|
|Atlanta, Georgia, United States, 30322|
|United States, Illinois|
|Ann & Robert H. Lurie Children's Hospital of Chicago|
|Chicago, Illinois, United States, 60614|
|United States, Texas|
|Texas Children's Hospital/Baylor University|
|Houston, Texas, United States, 77030|
|Hospices Civils de Lyon|
|Hôpital Arnaud Villeneuve ‐ CHU Montpellier|
|Robert Debre Hospital|
|Radboud University Nijmegen Medical Center|
|Study Director:||Evelyn Olson, BS||Horizon Pharma USA, Inc.|