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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01000961 | Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis | PHASE3 | COMPLETED | 43 | — | — | Jun 1, 2010 | Aug 1, 2011 | Dec 19, 2024 | 8 | United States, France +1 |
| NCT00872729 | Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis | PHASE1 | COMPLETED | 9 | — | — | May 1, 2009 | Oct 1, 2009 | Dec 19, 2024 | 1 | United States |
t = 6 for Cystagon and t = 12 for RP103. Cystagon is dosed every 6 hours and there is no measurement after 6 hours and up to 12 hours.
The pharmacodynamic (PD) parameter measures the changes of WBC cystine level from the baseline. Cystine is a disulfide amino acid formed through oxidation of two molecules of cysteine; hence, cystine's concentration is commonly given in half-cystine equivalents to avoid confusion. The level of cystine in WBC/leukocytes is expressed in units of nmol half-cystine/mg protein (nmol ½ cystine/mg protein). Half-cystine is quantified by a reduction of cystine followed by an assay for cysteine, which is then normalized by the total cellular protein content within the sample using methods of such as Lowry assay, bicinchoninic acid assay, or Bradford.
| Arm | Type | Description |
|---|---|---|
| RP103 Q12H | EXPERIMENTAL | - |
| Cystagon® Q6H | ACTIVE_COMPARATOR | - |
| Cystagon® | ACTIVE_COMPARATOR | Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg |
| RP103 | EXPERIMENTAL | Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg |
| Name | Type | Description |
|---|---|---|
| Cystagon® (Cysteamine Bitartrate) | DRUG | Run-in Period (Weeks 1, 2, 3) and Period 1 (Weeks 4, 5, 6) or Period 2 (Weeks 7, 8, 9); Immediate crossover to opposite treatment than taken during Period 1: Every 6H, supplied in 150 and 50mg capsules/Duration of Treatment: 3 weeks each period used |
| Cysteamine Bitartrate Delayed-release Capsules (RP103) | DRUG | Period 1 (Weeks 4, 5, 6) or Period 2 (Weeks 7, 8, 9); Immediate crossover to opposite treatment than taken during Period 1: Every 12H, supplied in 75 and 25mg capsules/Duration of Treatment: 3 weeks |
| Cystagon® | DRUG | Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg. Duration of Treatment and Dose: Reference Period up to four doses Q6H. |
| RP103 | DRUG | Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg. Duration of treatment and Dose: Single dose of Test Product at dose equivalent to Reference Product. |
Inclusion Criteria: * Male and female subjects must have nephropathic cystinosis. * Subjects must be on a stable dose of Cystagon® sufficient to maintain their white blood cell (WBC) cystine level at ≤ 1.0 nmol/half-cystine/mg protein. * Subjects must be able to swallow their typically administered...