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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01733316 | Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis | PHASE3 | COMPLETED | 41 | — | — | Jan 31, 2013 | Jul 10, 2017 | Dec 27, 2024 | 14 | United States, Belgium +4 |
| NCT01744782 | Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis | PHASE3 | COMPLETED | 17 | — | — | Dec 20, 2012 | Dec 13, 2016 | Dec 27, 2024 | 2 | United States, Brazil |
The primary analysis of WBC cystine was performed using the natural log transformed WBC cystine level; the log transformation is a normalizing transformation. For each participant, the difference between the morning and corresponding non-morning log WBC cystine value (non-morning minus morning) at each monthly visit during the Cystagon® phase (Months 1, 2, and 3) was computed and these differences were averaged. The average difference between morning and non-morning log WBC cystine value was similarly computed for each participant during the RP103 phase (Months 5, 6, and 7). The primary analysis compared within-subject pairs (Cystagon® phase paired with RP103 phase) of non-morning minus morning average differences of log WBC cystine level.
Blood samples were taken 30 minutes after the morning RP103 dose at each study visit to determine White Blood Cell (WBC) cystine concentration. WBC cystine concentrations were determined using liquid chromatography.
| Arm | Type | Description |
|---|---|---|
| All Participants | EXPERIMENTAL | Cystagon® Phase: From Screening and during Months 1, 2, 3 participants receive their usual dose of Cystagon® every 6 hours (Q6H). RP103 Phase: During Months 3.5, 4, 5, 6, 7 participants receive RP103 every 12 hours (Q12H). Long Term Phase: On or after Month 7, for the remainder of study participants receive RP103 Q12H. |
| RP103 | EXPERIMENTAL | From Day 1 and throughout the duration of participation, RP103 (Cysteamine Bitartrate Delayed-release Capsules) was administered every 12 hours (Q12H), supplied as 75 mg and 25 mg capsules. |
| Name | Type | Description |
|---|---|---|
| RP103 | DRUG | - |
| Cystagon® | DRUG | - |
INCLUSION CRITERIA: * Male or female with a documented diagnosis of cystinosis * On a stable dose of Cystagon® at least 21 days prior to Screening * WBC cystine level \> 1 nmol 1/2 cystine/mg of protein, on average over at least 2 measurements collected during the 2 years prior to Screening * No cl...