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RP103 · 2 trials · 1 indication
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...
RP103 is an investigational small molecule being developed for cystinosis, a rare disease. It is formulated as cysteamine bitartrate delayed-release capsules and is intended to treat the condition. The drug is currently in Phase 3 clinical development, though it is not yet approved.
RP103 is a cysteamine bitartrate formulation. Cysteamine works by depleting cystine within cells, which is the underlying metabolic defect in cystinosis. This mechanism addresses the accumulation of cystine crystals that cause tissue damage in patients with the disease.
RP103 is being developed by Amgen Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol AMGN. Amgen is conducting the clinical development program for this cysteamine bitartrate delayed-release capsule.
RP103 is in Phase 3 clinical development. Two Phase 3 trials have been completed, with a total of 58 patients enrolled. The drug is still investigational and has not been approved by regulatory authorities.
RP103 has been studied in two completed Phase 3 trials. NCT01733316 evaluated safety and effectiveness in 41 patients with cystinosis across the US, Belgium, France, Italy, Netherlands, and UK. NCT01744782 studied 17 cysteamine treatment-naive patients in the US and Brazil.
RP103 is a specific delayed-release capsule formulation of cysteamine bitartrate. The drug name RP103 refers to this particular product being developed by Amgen. Cysteamine bitartrate is the active pharmaceutical ingredient, and RP103 is the branded investigational formulation.