Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Cystagon · 2 trials · 1 indication
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...
Cystagon is a small molecule drug used for cystinosis, a rare disease. It is being developed by Amgen Inc. (AMGN) and is currently in Phase 3 clinical development. Cystagon is an investigational drug and has not been approved by the FDA.
Cystagon is being developed by Amgen Inc., a biopharmaceutical company traded on NASDAQ under the ticker AMGN. The drug is currently in Phase 3 clinical development for the treatment of cystinosis, a rare disease.
Cystagon is in Phase 3 clinical development. It is an investigational drug for the treatment of cystinosis, a rare disease. The drug has not been approved by the FDA and is still undergoing clinical trials to evaluate its safety and efficacy.
Cystagon has been studied in two completed clinical trials. NCT00872729 was a Phase 1 pilot study with 9 patients, and NCT01000961 was a Phase 3 study with 43 patients. Both trials were conducted in patients with cystinosis, with the Phase 3 study including sites in the United States, France, and the Netherlands.
Cystagon is compared to RP103 in clinical trials, but they are not the same drug. RP103 is a delayed-release formulation of cysteamine bitartrate, while Cystagon is the reference drug used as an active control in these studies. Both are being evaluated for the treatment of cystinosis.