Safety and Efficacy of RPH-104 Used to Prevent Recurrent Fever Attacks in Adult Patients With Colchicine Resistant or Colchicine Intolerant Familial Mediterranean Fever

This is an interventional treatment trial for Familial Mediterranean Fever focused on measuring RPH-104, colchicine inefficacy, colchicine intolerance, colchicine resistance, subcutaneous Read More

Inclusion Criteria:

1. The patient who completed the last visit of the treatment period in the core study, according to the protocol, during which he/she received at least one dose of RPH-104.
2. Voluntarily signed and dated Patient Informed Consent Form for participation in this study.

3. The patient's ability and desire, according to the investigator, to follow the schedule of visits, follow the study procedures and follow the protocol requirements, i.e they agree to:

   • come to the study site every 2 weeks for the investigational product administration by qualified site staff. OR
   • learn the subcutaneous injection technique and self-administer the investigational product at home as per protocol of this study.

Exclusion Criteria:

1. Any medically important event that was observed in a patient during his/her participation in the core study, and, in the opinion of the investigator, is a reason for not including this patient in the present study, and any other medical (including psychiatric) conditions or laboratory abnormalities, which may increase the potential risk associated with participation in the study and receiving the investigational product, or may affect the interpretation of the results of the study, and which, in the Investigator's reasonable opinion, result in the patient's non-compliance with the inclusion criteria
2. Pregnant and/or lactating women or women planning pregnancy during the study or within 2 months after the last dose of the study drug.

3. Women of childbearing potential, i.e. all women with the physiological ability to become pregnant (except for women with a final cessation of menstruation, which should be determined retrospectively after 12 months of natural amenorrhea, i.e. amenorrhea with an appropriate clinical status, for example, an appropriate age), who DO NOT agree to use highly effective contraception for of the entire study period, starting from the beginning of the screening phase (signing informed consent) and for a minimum of 8 weeks after the last dose of the study drug. OR

   Men who are sexually active and do NOT agree to use highly effective contraceptives throughout the study, starting from the beginning of the screening phase and for at least 8 weeks after the last dose of the study drug.

   Highly effective contraception methods include:

   • sterilization in women: surgical bilateral removal of the ovaries (with or without removal of the uterus) or ligation of the fallopian tubes at least 6 weeks before the start of the study therapy. In the case of removal of only the ovaries, the reproductive status of a woman should be confirmed by a subsequent assessment of hormone level;
   • sterilization in men, at least 6 months before the start of the study therapy with proper documentation of the absence of sperm in the ejaculate after vasectomy. For the women participating in the study, the sexual partner after a vasectomy should be the only partner;

   • using a combination of any two of the following methods (a+b or a+c or b+c):

     1. the use of oral, injectable or implanted hormonal contraceptives; in the case of the use of oral contraceptives, women should constantly use the same drug for at least 3 months before the start of the study therapy;
     2. the installation of an intrauterine device or contraceptive system;
     3. barrier methods of contraception: condom or occlusive cap (diaphragm or cervical cap/contraceptive vaginal ring) with spermicidal foam/gel/film/cream/vaginal suppository.
4. The need for systemic glucocorticoid therapy at doses > 0.2 mg/kg/day of prednisolone (0.16 mg/kg/day of methylprednisolone or an equivalent dose of another glucocorticoid) orally from the moment of signing the Informed Consent Form to the end of the period of therapy with the study drug.
5. The need to use a live (attenuated) vaccine during the study or within 3 months after the last dose of the study drug. Live attenuated vaccines include vaccines against viruses: measles, rubella, mumps, chickenpox, rotavirus, flu (as a nasal spray), yellow fever, polio (oral polio vaccine); vaccines against tuberculosis (BCG), typhoid fever (oral typhoid vaccine) and typhus (typhus vaccine). Immunocompetent family members of the patient should not be vaccinated with the oral polio vaccine during the patient's participation in the study.
6. Positive results of tuberculosis screening performed at Visit 10 of the core study (QuantiFERON-TB/T-SPOT.TB test, chest x-ray).