The purpose of this study is to assess safety and efficacy of the long-term treatment with RPH-104 at doses of 80 or 160 mg once every 2 weeks (q2w) in patients with familial Mediterranean fever (FMF) with colchicine resistance or intolerance (i.e. colchicine resistant, crFMF), who completed the core study, during which they received at least one dose of RPH-104 (i.e. study patient population).
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.
Voluntarily signed and dated Patient Informed Consent Form (ICF) for participation in this study.
to visit the study site every 2 weeks for RPH-104 administration by qualified study site personnel
Any medically significant event that was observed in a patient during his/her participation in the core study, as well as any other medical conditions (including psychiatric disorders) or laboratory abnormalities, which may increase the potential risk associated with participation in the study and treatment with RPH-104, or may affect the interpretation of the study results, and which, according to the Investigator's opinion, may lead to the patient's non-compliance with the study inclusion criteria.
Pregnant and/or lactating women or women planning pregnancy during the study or within 2 months after the last RPH-104 dose.
Women of childbearing potential, i.e. all females with physiological ability to conceive except for those with final cessation of menses, which should be determined retrospectively after 12 months of natural amenorrhea, i.e. amenorrhea with an appropriate clinical status, for example, at respective age, who do not agree to use highly effective contraceptives throughout the study, starting from the moment of signing the ICF and for at least 8 weeks after the last RPH-104 dose or Men who are sexually active and do not agree to use highly effective contraceptives throughout the study, starting from the moment of signing the ICF and for at least 8 weeks after the last RPH-104 dose.
complete abstinence: if it corresponds to the preferred and conventional lifestyle of the subject. Periodic abstinence (e.g. calendar, ovulation, symptothermal, postovulation method) and interrupted coitus are not considered acceptable contraceptive methods;
Single group
RPH-104
Biological/Vaccinesolution for subcutaneous administration 40 mg/mL, 2 mL in the 4-mL glass vial
Incidence of Treatment-Emergent Adverse Events (AEs), by System Organ Class and Preferred Term
Incidence of Treatment-Emergent Serious Adverse Events (SAEs), by System Organ Class and Preferred Term
Incidence of Treatment-Emergent Adverse Events of Special Interest (AESI), by System Organ Class and Preferred Term
Incidence rate for serious adverse events (SAEs)
Incidence rate, expressed as the number of events per 100 patient-years of follow-up, for SAEs
Percentage of patients with physician global assessment of disease activity scale (PGA) <2
PGA of disease activity involves a 5-point system: from 0 = no clinical signs and symptoms associated with the disease to 4 = severe clinical signs and symptoms associated with the disease.
Percentage of patients with serological remission
Serological remission is defined as C-reactive (CRP) level ≤10 mg/L.
Percentage of patients whose Serum amyloid A (SAA) levels returned to normal values
SAA normal levels are defined as SAA \<10 mg/L
Percentage of patients who have experienced ≥ 1 attacks per month (since Day 0)
Criteria for the diagnosis of an attack are defined as the simultaneous development of clinical and serological signs of an attack, including: * the score on the scale of global physician's assessment of disease activity (PGA) ≥ 2, suggesting "mild", "moderate" or "severe" activity of the disease (i.e., clinical signs), AND * CRP level ≥ 30 mg/L (i.e. serological signs)
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R-Pharm International, LLC
Lead sponsor
Atlant Clinical LLC
Collaborator
Unimed Laboratories
Collaborator
Data Management 365
Collaborator
R-Pharm JSC
Collaborator
TRPharm İlaç Sanayi Ticaret Anonim Şirketi and ASCOT SCIENCE Education and Consulting Ltd.
Collaborator
Exacte Labs LLC
Collaborator
Key Stat LLC
Collaborator
