ClinicalTrials.gov record NCT05190991

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

An International Multicenter Open-label Clinical Study of the Safety and Efficacy of RPH-104 for Prevention of Recurring Attacks in Adult Subjects With Familial Mediterranean Fever With Resistance to or Intolerance of Colchicine

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
Age18 years old to 80 years old
SexAll
LocationArmenia, Russia, Turkey
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is a phase 2, open-label extension testing long-term use of RPH-104 in adults with Familial Mediterranean Fever (FMF) who were resistant or intolerant to colchicine and who completed a prior core study. Participants receive subcutaneous RPH-104 every two weeks for up to 198 weeks, with an 8-week safety follow-up. The study tracks adverse events, blood markers of inflammation, disease activity, attack frequency, and symptom severity.

What it is studying. The study is testing the long-term safety and activity of subcutaneous RPH-104 given every two weeks at doses of 80 mg or 160 mg in adults with colchicine-resistant or colchicine-intolerant FMF who completed the core study.

What the researchers hope to learn. Collect long-term safety data (treatment-emergent adverse events, serious adverse events, adverse events of special interest) and measure effects on disease activity and inflammation (physician global assessment, CRP, SAA), attack frequency, symptom severity, renal function, and use of additional symptomatic therapy.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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).

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Biological
  • RPH-104

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • 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
  • 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

  • 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.
  • 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;
  • intramuscular, intra-articular or peri-articular administration of glucocorticoids;
  • anakinra;
  • tofacitinib, baricitinib;
  • any experimental drugs (except for RPH-104)
  • 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
  • Positive results of tuberculosis screening performed at Visit 10 of the core study (QuantiFERON-Tuberculosis(TB)/T-Spot.TB test, chest X-ray).
  • Participation in other experimental studies (except for the core study).

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Show the detailed description from the registry

This study is a long-term open-label extension (OLE) of the core double blind randomized placebo-controlled study CL04018065. This OLE study will have the following periods:

  • Screening period - 2 weeks (from signing an Informed Consent Form [ICF] at Visit 10 of the core study to Visit 1, Day 0 of this OLE study) - includes unblinding of the treatment groups determined in the core study (unless was unblinded previously) and determination of the eligibility criteria compliance for this study (all other screening procedures are performed after the completion of Visit 11 of the core study, which is also a Day 0 of this OLE study); All patients meeting the study inclusion/exclusion criteria according to the screening results will be transferred to the treatment period at the same day (Day 0) - the beginning of this period will be considered Visit 1 of this study
  • Treatment period - All patients will be receiving RPH-104 treatment for 198 weeks in the open-label regimen (both patient and the Investigator, along with all study team will know which treatment is used) at the doses determined for them in the core study. Thus, the possible RPH-104 dose will be:
  • 80 mg q2w subcutaneously (SC);
  • 160 mg q2w SC

The first RPH-104 administration in this study will be performed at Visit 1. To maintain the q2w dosage regimen, the screening and the first open-label injection of RPH-104 (if the patient meets the study eligibility criteria) must be performed on the same day - Day 0 (which is also the day of Visit 11 of the core study) - i.e. the first administration in this study will be performed 2 weeks (±3 days) after the last administration in the core study. Further, RPH-104 will be administered to patients q2w both during each scheduled visit to the study site (with safety and efficacy assessments according to the visits schedule), and during Drug Administration Visits (DAVs) at the study site or at the patient's accommodation An injection of the study drug to patients is performed by qualified medical personnel every 2 weeks when the patient visits the study site; it is also possible for the patients to self-administer the drug at home (for which patients will be appropriately trained and provided with the necessary quantity of the drug, materials for the injection (including special containers for their disposal) and proper drug transportation). Safety and efficacy assessments are performed at Visit 1, Visit 2 (in 2 weeks), Visit 3 (in 4 weeks), Visit 4 (in 4 weeks), Visit 5 (in 8 weeks) and thereafter every 12 weeks (Visit 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19 and 20) according to the visits schedule. In case of RPH-104 administration in patients' accommodation, telephone contacts with a patient will be conducted by the Investigator every 4 weeks in between the visits starting from the Visit 4.In case of FMF attack development, patients who receive 80 mg of the drug may be switched to the increased maximum drug dose160 mg based at the discretion of the investigator. The drug is administered only at scheduled visits. Dose reduction of RPH-104 is not allowed in this study. After patients receive the last dose of RPH-104 at Week 198 of the study, the treatment period will be considered completed and an 8-week safety follow-up will start

  • Safety follow-up period - 8 weeks. During this period the patients will have to visit the study site twice in 4 weeks (Visit 21) and 8 weeks (Visit 22) after the last RPH-104 dose for safety assessments.

The last visit of the safety follow-up period (Visit 22) is the End of Study Visit; after completion of all procedures of this visit, patients will be considered to have completed the study.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive RPH‑104 by subcutaneous injection every 2 weeks for up to 198 weeks in an open‑label extension; injections are given at clinic visits or at home after training (or by trained staff visiting the home). The study begins with screening, has several visits in the first month, then scheduled visits about every 12 weeks with safety and efficacy checks; telephone contact occurs every 4 weeks between visits if dosing at home. Participants will have blood tests for inflammatory markers (for example CRP and serum amyloid A), symptom assessments, and monitoring for adverse events; investigators may increase dose from 80 mg to 160 mg if attacks occur (dose reduction is not allowed). After the last dose there is an 8‑week safety follow‑up with two visits.

How the study is structured. Open-label, single-group phase 2 extension: all participants receive RPH-104 by subcutaneous injection every two weeks (80 mg or 160 mg) for 198 weeks. Doses were set based on the prior core study and patients on 80 mg may be increased to 160 mg if new attacks occur. Injections are given at clinic visits or by trained self-administration; early visits are frequent then move to about every 12 weeks, and safety follow-up lasts 8 weeks after the last dose.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The listed sites are in Armenia, Russia, Turkey. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on familial Mediterranean fever

Clinical trials62 trials listed for familial Mediterranean feverEvery trial the registry lists for familial Mediterranean fever, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in familial Mediterranean fever researchLatest monthly update: September 2026. Includes: Colchicine form change. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Oct 5, 2021
Primary completion
December 2028 (estimated)
Study completion
January 2029 (estimated)
Target enrollment
60 participants (estimated)
Last updated on registry
Dec 16, 2024
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
R-Pharm International, LLC
Collaborators
  • Atlant Clinical LLC
  • Unimed Laboratories
  • Data Management 365
  • R-Pharm JSC
  • TRPharm İlaç Sanayi Ticaret Anonim Şirketi and ASCOT SCIENCE Education and Consulting Ltd.
  • Exacte Labs LLC
  • Key Stat LLC

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (8)

  • Center of Medical Genetics and Primary Health Care LLCRecruiting

    Yerevan, 0001 · Armenia

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

    Moscow, 119048 · Russia

  • Moscow Multidisciplinary Scientific and Clinical Center named after S.P. BotkinRecruiting

    Moscow, 125284 · Russia

  • Medical Technologies Ltd.Recruiting

    Saint Petersburg, 191025 · Russia

  • Terafarm, LlcRecruiting

    Stavropol, 355000 · Russia

  • Hacettepe University Faculty of MedicineRecruiting

    Ankara, 06230 · Turkey (Türkiye)

  • Istanbul University Istanbul Faculty of MedicineRecruiting

    Istanbul, 34093 · Turkey (Türkiye)

  • Istanbul University Cerrahpasa Faculty of MedicineRecruiting

    Istanbul, 34098 · Turkey (Türkiye)

Terms used on this page · explanations by MedicalUpdates.ai

Informed Consent Form
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
inclusion criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Women of childbearing potential
Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
glucocorticoids
Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
immunosuppressants
A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
open-label extension
A continuation study in which participants who completed an earlier study can keep receiving the treatment, usually with everyone knowing what they are given.
double blind
Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
randomized
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
placebo-controlled
A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
Screening period
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
unblinded
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
eligibility criteria
The rules the study team uses to decide who may take part. They are usually split into inclusion criteria and exclusion criteria.
study drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
safety follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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