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

NCT05190991 | Recruiting Phase 2

• 1 other identifier: CL04018071
• Study Type: interventional
• Target: 60
• Locations: 3 countries
• Sites: 8

Brief Summary

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).

Conditions

Familial Mediterranean Fever; FMF

Keywords

RPH-104; colchicine inefficacy; colchicine intolerance; colchicine resistance; subcutaneous; FMF; interleukin-1 inhibitor

Outcome Measures

Primary Outcomes (5)

• Incidence of Treatment-Emergent Adverse Events (AEs), by System Organ Class and Preferred Term

  Up to 62 weeks

• Incidence of Treatment-Emergent Serious Adverse Events (SAEs), by System Organ Class and Preferred Term

  Up to 62 weeks

• Incidence of Treatment-Emergent Adverse Events of Special Interest (AESI), by System Organ Class and Preferred Term

  Up to 62 weeks

• Incidence rate for serious adverse events (SAEs)

  Incidence rate, expressed as the number of events per 100 patient-years of follow-up, for SAEs

  Up to 62 weeks

• Incidence rate for adverse events of special Interest (AESI)

  Incidence rate, expressed as the number of events per 100 patient-years of follow-up, for AESI

  Up to 62 weeks

Secondary Outcomes (16)

• Percentage of patients with physician global assessment of disease activity scale (PGA) <2

  Up to 62 weeks

• Percentage of patients with serological remission

  Up to 62 weeks

• Percentage of patients whose Serum amyloid A (SAA) levels returned to normal values

  Up to 62 weeks

• Percentage of patients who have experienced ≥ 1 attacks per month (since Day 0)

  Up to 54 weeks

• Percentage of patients who have not had a single attack

  Up to 54 weeks

Study Arms (1)

RPH-104 q2w

EXPERIMENTAL

RPH-104 80 mg once every 2 weeks subcutaneously or RPH-104 160 mg once every 2 weeks subcutaneously

Biological: RPH-104

Interventions

RPH-104 BIOLOGICAL

solution for subcutaneous administration 40 mg/mL, 2 mL in the 4-mL glass vial

RPH-104 q2w

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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
• to learn the subcutaneous (SC) injection technique and self-administer RPH-104 at his/her accommodation as per the study Protocol
• to agree with the qualified medical personnel visits to his/her accommodation for RPH-104 administration.

You may not qualify if:

• 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.
• 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):
• 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;
• installation of an intrauterine device or contraceptive system;
• use of barrier contraceptives: condom or occlusive cap (diaphragm or cervical cap/contraceptive vaginal ring) with spermicidal foam/gel/film/cream/vaginal suppository
• 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;

Sponsors & Collaborators

• R-Pharm International, LLC: lead
• 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

Study Sites (8)

Center of Medical Genetics and Primary Health Care LLC

Yerevan, 0001, Armenia

RECRUITING

FSBEI HE First Moscow State Medical University named after I.M. Sechenov

Moscow, 119048, Russia

RECRUITING

Moscow Multidisciplinary Scientific and Clinical Center named after S.P. Botkin

Moscow, 125284, Russia

RECRUITING

Medical Technologies Ltd.

Saint Petersburg, 191025, Russia

RECRUITING

Terafarm, Llc

Stavropol, 355000, Russia

RECRUITING

Hacettepe University Faculty of Medicine

Ankara, 06230, Turkey (Türkiye)

RECRUITING

Istanbul University Istanbul Faculty of Medicine

Istanbul, 34093, Turkey (Türkiye)

RECRUITING

Istanbul University Cerrahpasa Faculty of Medicine

Istanbul, 34098, Turkey (Türkiye)

RECRUITING

MeSH Terms

Conditions

Familial Mediterranean Fever

Condition Hierarchy (Ancestors)

Hereditary Autoinflammatory Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Mikhail Samsonov

  R-Pharm

  STUDY DIRECTOR

Central Study Contacts

Yulia Karpova

CONTACT

+7 495 956 79 37; jn.karpova@inbox.ru

Marina Beloukhova

CONTACT

+7 495 956 79 37; beloukhova@partners.rpharm.ru

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 3, 2021
• First Posted: January 13, 2022
• Study Start: October 5, 2021
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): January 1, 2029
• Last Updated: December 16, 2024

Record last verified: 2024-12

Locations

RU (4); TU (3); AR (1)