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

Launched by R-PHARM INTERNATIONAL, LLC · Jan 12, 2022

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ClinConnect Summary

This clinical trial is studying a medication called RPH-104 to see if it can help prevent fever attacks in adults who have a condition known as Familial Mediterranean Fever (FMF) and who cannot tolerate or do not respond to a common treatment called colchicine. The trial aims to evaluate how safe RPH-104 is when taken over a longer period and whether it effectively reduces fever attacks. Participants will receive either 80 mg or 160 mg of the medication every two weeks. Researchers will also look at how the drug affects patients' quality of life and its overall performance in the body.

To be eligible for this study, participants must have previously taken RPH-104 in a related study and be able to attend appointments every two weeks for treatment or self-administer the medication at home. It's important that participants agree to use effective birth control methods during the study and for a period after it ends. Those with certain medical conditions, pregnant or breastfeeding women, and individuals needing specific medications may not qualify. If you decide to participate, you'll be closely monitored, and your experiences will contribute to understanding how RPH-104 may help others with FMF.

Gender

ALL

Eligibility criteria

• 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 in this study.
• 3. The patient's ability and desire, according to the Investigator's discretion, to follow the schedule of visits, follow the study procedures and follow the Protocol requirements, including the following:
• • to visit the study site every 2 weeks for RPH-104 administration by qualified study site personnel
• • or
• • • to learn the subcutaneous (SC) injection technique and self-administer RPH-104 at his/her accommodation as per the study Protocol
• • or
• • • to agree with the qualified medical personnel visits to his/her accommodation for RPH-104 administration.
• Exclusion Criteria:
• • 1. 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.
• • 2. Pregnant and/or lactating women or women planning pregnancy during the study or within 2 months after the last RPH-104 dose.
• • 3. 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.
• Highly effective contraception methods include:
• • 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;
• • female sterilization: surgical bilateral ovariectomy (with/without hysterectomy) or tubal ligation at least 6 weeks before the start of the core study. In a case of ovariectomy only, the female reproductive status should be verified by further hormonal test;
• • male sterilization (with documented absence of sperm in ejaculate post vasectomy) at least 6 months before the start of the core study. Vasectomized male partner should be the only partner of the participating female subject;
• * combination of any two of the following methods (a+b or a+c or b+c):
• • 1. use of oral, injectable or implanted hormonal contraceptives; in a case of oral contraceptives, the woman should constantly use the same product as was used during the core study;
• • 2. installation of an intrauterine device or contraceptive system;
• • 3. use of barrier contraceptives: condom or occlusive cap (diaphragm or cervical cap/contraceptive vaginal ring) with spermicidal foam/gel/film/cream/vaginal suppository
• 4. The need for a therapy with any of the following products from the moment of signing the ICF till the study treatment period completion:
• • systemic glucocorticoids at a dose exceeding 0,2 mg/kg/day of prednisolone (or 0,16 mg/kg/day of methylprednisolone, or an equivalent dose of another glucocorticoid) orally;
• • rilonacept, tocilizumab, rituximab, canakinumab, tumor necrosis factor alpha (TNF-a) inhibitors (TNFi) and other biological products (except for RPH-104);
• • immunosuppressants (cyclosporine, methotrexate, leflunomide, thalidomide, azathioprine, 6-mercaptopurine, cyclophosphamide, etc.);
• • methylprednisolone (or an equivalent) at a dose of more than 40 mg/day parenterally;
• • intramuscular, intra-articular or peri-articular administration of glucocorticoids;
• • anakinra;
• • tofacitinib, baricitinib;
• • any experimental drugs (except for RPH-104)
• • 5. The need to use a live (attenuated) vaccine during the study or within 3 months after the last RPH-104 dose. 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-Tuberculosis(TB)/T-Spot.TB test, chest X-ray).
• • 7. Participation in other experimental studies (except for the core study).