Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01197378 | Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) | PHASE3 | COMPLETED | 60 | — | — | Aug 27, 2010 | Jun 26, 2017 | Dec 27, 2024 | 10 | United States, France +1 |
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.
| Arm | Type | Description |
|---|---|---|
| Cysteamine Bitartrate | EXPERIMENTAL | Cysteamine bitartrate delayed-release capsules were administered twice daily for up to 96 months. |
| Name | Type | Description |
|---|---|---|
| Cysteamine Bitartrate Delayed-release Capsules | DRUG | 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. |
Inclusion Criteria: * Male and female subjects must have completed the last visit of Study RP103-03 and be willing to continue with RP103 treatment. OR for patients who did not complete the RP103-03 study: * Male and female subjects must have cystinosis. * Subjects must be on a stable dose of Cys...