Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
RPH-104 · 2 trials · 2 indications
Incidence rate, expressed as the number of events per 100 patient-years of follow-up, for SAEs
Incidence rate, expressed as the number of events per 100 patient-years of follow-up, for AESI
Complete response defined as resolution of "marker" attack by Visit 2 (Day 7) and lack of recurrent attacks during the treatment period up to Visit 10 (Day 112). Criteria of resolution of a "marker" FMF attack include simultaneous clinical and laboratory signs of the attack resolution: * Physician Global Assessment (PGA) score \< 2 (i.e. minimum or complete lack of clinical signs and symptoms) AND * C-reactive protein (CRP) level ≤ 10 mg/L OR CRP reduction by ≥ 70% compared to baseline defined at enrollment to treatment period (Visit 1). Criteria of a recurrent FMF attack development after resolution of "marker" attack include simultaneous development of clinical and laboratory signs of the attack: * PGA score ≥ 2 assuming mild, moderate or severe disease activity (i.e. clinical signs), AND * CRP level ≥ 30 mg/L (serological signs). PGA is a 5-point scale: from 0 = no disease-related clinical signs and symptoms to 4 = severe clinical signs and symptoms of the disease.
| Arm | Type | Description |
|---|---|---|
| RPH-104 q2w | EXPERIMENTAL | RPH-104 80 mg once every 2 weeks subcutaneously or RPH-104 160 mg once every 2 weeks subcutaneously |
| RPH-104 | EXPERIMENTAL | Test product group receiving RPH-104 subcutaneous (s.c.) injections:160 mg on Day 0, 80 mg on Day 7, Day 14 and every 2 weeks (q2w) thereafter. In case "marker" attack does not resolve at Visit 2 - the treatment group will be unblinded: patients will receive planned RPH-104 80 mg administration. In case of a new attack on further days of treatment period until Visit 10 inclusive - the treatment group will be unblinded: the dose of RPH-104 could be escalated to 160 mg q2w; The patients already receiving RPH-104 160 mg q2w will continue to receive RPH-104 at this dose. Further dose escalation is forbidden. |
| Placebo | PLACEBO_COMPARATOR | Placebo group receiving the equivalent placebo dose also as s.c. injections on Day 0, Day 7, Day 14 and q2w thereafter. In case the "marker" attack does not resolve at Visit 2 - the treatment group will be unblinded: patients will be switched to active treatment with RPH-104 in SC injections at a dose of 160 mg followed by administration of 80 mg in 7 days at the Attack + 7 days Visit, and 80 mg at the next Visits. In a case of a new attack the patients switching from placebo and receiving RPH-104 at 80 mg dose could be escalated to RPH-104 160 mg q2w; Further dose escalation is forbidden. |
| Name | Type | Description |
|---|---|---|
| RPH-104 | BIOLOGICAL | solution for subcutaneous administration 40 mg/mL, 2 mL in the 4-mL glass vial |
| Placebo | DRUG | Normal Saline (0.9% Sodium Chloride solution for subcutaneous Injection), 2 mL in the 4 mL-glass vial. The placebo will contain no active pharmaceutical ingredients. |
Inclusion Criteria: 1. The patient with Familial Mediterranean Fever (FMF) with resistance to or intolerance of colchicine, who completed the core study, during which he/she received at least one dose of RPH-104. 2. Voluntarily signed and dated Patient Informed Consent Form (ICF) for participation ...