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Familial Mediterranean FeverPhase 3NCT07880886

Goflikicept for Resolution and Prevention of Attacks in Pediatric and Adolescent Patients With Colchicine-Resistant or Colchicine-Intolerant Familial Mediterranean Fever

A Phase 3 study testing RPH-104 in people with Familial Mediterranean Fever and FMF, run by R-Pharm at 3 sites in Russia.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
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Trial summary

The primary objective is to evaluate the main pharmacokinetic (PK) parameters, efficacy, and safety of goflikicept (GFC) in children and adolescents with colchicine-resistant or colchicine-intolerant familial Mediterranean fever (FMF). Read more about this study

Who is it for?
Ages 5 to 17 years
Conditions the study is for
Familial Mediterranean Fever, FMF
What is being tested?
RPH-104
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
18 participants
Where
3 sitesRussia
Study dates
Dec 2026to Apr 2030
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 5 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Confirmed diagnosis of Familial Mediterranean Fever (FMF) based on new diagnostic criteria of the Eurofever/PRINTO expert group (2019)
  • Male and female participants aged ≥ 5 and < 18 years at the time of signing Informed Consent Form (ICF) and Visit 1, respectively:
  • 2.1. In Kingdom of Saudi Arabia, the upper age limit is 14 years (i.e. the participants should be between ≥ 5 and ≤ 14 years old)
  • 2.2. In other countries, the upper age limit is 18 years (i.e. the participants should be between ≥ 5 and < 18 years old)
  • Availability of a voluntarily signed and dated ICF for participation in this study: the informed consent of the parent or legal representative and the consent of the child
  • Availability of one of the documented data listed below (at the time of the start of screening) (records of medical examinations/consultations including those done by the investigator, hospital discharges) confirming:
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  • 4.1. colchicine resistance at the maximum tolerated therapeutic dose (depending on age and weight) confirmed by the persistence of at least one attack of the disease per month on average, despite the indicated therapy for at least the last 3 months before the start of screening. Colchicine administration will be continued at stable dose if it is not associated with unacceptable adverse drug reactions
  • 4.2. intolerance to therapeutic or subtherapeutic doses of colchicine (presence of unacceptable adverse drug reactions). The dose of colchicine should be stable for at least 5 days before the participant is enrolled (before screening)
  • Ability of the participant and the parent (legal representative) and willingness, in the reasoned opinion of the investigator, to visit the study site during all scheduled visits, to perform the study-related procedures and comply with the Protocol requirements including consent to undergo subcutaneous injections by qualified personnel of the site
  • One active attack with a duration of no more than 2 days (48 hours) at the time of Visit 1 defined as the development of both clinical and serological signs of an attack including:
  • Physician's Global Assessment (PGA) score ≥ 2 suggesting "mild", "moderate", or "severe" disease activity (i.e. clinical signs)
  • C-reactive protein (CRP) level > 10 mg/L (i.e. serological signs)
  • The Russian Federation, the Hashemite Kingdom of Jordan and the Republic of Turkey: Consent of female adolescents, after menarche, to use highly effective contraceptives throughout the study, starting from the screening (signing ICF) and for at least 8 weeks after completion (termination) of the study therapy; as well as the presence of a negative pregnancy test (serum test for chorionic human gonadotropin (HCG)) OR Consent of sexually active male adolescents to use highly effective contraceptives throughout the study, starting from the screening (signing ICF) and for at least 8 weeks after completion (termination) of the study therapy

Who cannot join

  • Hypersensitivity to GFC and/or its components/excipients
  • Administration of live (attenuated) vaccine less than 3 months prior to Visit 1 (treatment period initiation) and/or necessity to use such vaccine within 3 months after GFC discontinuation
  • Conditions or signs which, according to the investigator, suggest impaired (reduced) immune response and/or significantly increase the risk of immunomodulating therapy including (but not limited to):
  • 3.1. any acute or chronic active infection at the time of screening, an infection requiring antimicrobial therapy within 4 weeks prior to screening, or a history of serious, opportunistic, or frequently recurring infection within 6 months prior to screening
  • 3.2. presence of infection caused by the human immunodeficiency virus (HIV), hepatitis B virus or hepatitis C virus
  • History of active or latent tuberculosis (TB) or existing risk factors or signs suggestive of active or latent M. tuberculosis including (not limited to) the following:
Show all 36Show fewer
  • 4.1. living or working in specific conditions that increase the risk of contact with participants with TB, such as residential care, places of deprivation of liberty, communities of persons without a special place of residence, etc. during the last year before the start of the treatment period
  • 4.2. close contact, i.e. living at the same premises (at home or in another enclosed environment) for a prolonged period of time (several days or weeks, rather than minutes or hours) with a person with active pulmonary TB during the last year before the start of the treatment period
  • 4.3. results of examinations indicating active or latent infection with M. tuberculosis: positive QuantiFERON-TB / T-SPOT.TB test at screening; chest Xray/CT findings confirming pulmonary TB during screening
  • Any other relevant concomitant diseases or conditions that, in the Investigator's judgment, may affect the participant's participation or well-being in the study and/or interfere with the assessment of study results
  • History of organ transplantation or necessity in transplantation at the screening onset
  • Surgical intervention within 8 weeks prior to the screening or planned for the period of participation in the study, which, in the investigator's opinion, agreed with the Sponsor, will generate an unacceptable risk for the participant
  • Any existing malignant neoplasm during the screening period or within 5 years prior to screening, including blood and lymphatic neoplasm, except non-metastatic basal cell and squamous cell skin cancer after complete resection or carcinoma in situ of any type after complete resection
  • Pregnancy or breast-feeding
  • History of alcohol or psychoactive substances abuse, according to the investigator's evaluation
  • Previous administration of:
  • rilonacept - less than 6 weeks prior to Day 0 of the study
  • canakinumab - less than 12 weeks prior to Day 0 of the study
  • anakinra - less than 72 hours prior to Day 0 of the study
  • rituximab - less than 24 weeks before day 0
  • tumor necrosis factor (TNF) inhibitors, IL-6 inhibitors and other biological medications with an immunosuppressive mechanism of action - less than 6 weeks or 5 half-lives (whichever is longer) before study day 0
  • immunosuppressants (azathioprine, leflunomide, dapsone, cyclosporine, mycophenolate mofetil, tacrolimus, sirolimus, mercaptopurine, methotrexate, etc.) - less than 4 weeks or 5 elimination half-lives (whichever is longer) before study day 0. In case of leflunomide administration complete elimination course using cholestyramine should be documented
  • intravenous (IV) immunoglobulin (Ig) - less than 8 weeks before day 0;
  • intravenous administration of glucocorticoids - less than 1 week (from the end of therapy) to study day 0
  • intramuscular, intra-articular or periarticular administration of glucocorticoids - less than 4 weeks before day 0
  • systemic oral glucocorticoid therapy at doses > 0.2 mg/kg/day of prednisolone equivalent on day 0
  • changes in the dose/treatment regimen of oral glucocorticoids during the 4 weeks prior to the screening
  • Any of the deviations in the laboratory tests below:
  • neutrophil count < 1.5×10^9/L (< 1500/mm^3)
  • white blood cell (WBC) count < 3×10^9/L (< 3000/mm^3)
  • platelet count < 100×10^9/L (< 100 000/mm^3 or < 100 000×10^6/L)
  • ALT and/or AST ≥ 2.0 × upper limit of normal (ULN); if ALT and/or AST is ≥ 2 × ULN but < 3 × ULN at the screening, retest is allowed
  • bilirubin > 1.5 x ULN (except for confirmed Gilbert's disease)
  • Concomitant participation in other clinical studies at the screening onset or administration of any unauthorized (investigational) medications less than 4 weeks or 5 half-life periods (whichever is longer) before Visit 1 (treatment initiation)
  • Prior participation in this clinical study in case of at least one GFC dose
  • Severe renal failure: Glomerular filtration rate (GFR) calculated by Schwartz formula is < 45 mL/min/1.73 m^2

Trial locations and contacts

3 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Yulia Karpova contact

+7 (495) 956-79-37 ext. 1197

jn.karpova@rpharm.ru

Darya Bukhanova contact

+7 (495) 956-79-37

bukhanova@rpharm.ru

Russia3 sites

  • Federal State Budgetary Scientific Institution "V.A. Nasonova Research Institute of Rheumatology

    Moscow, 115522

    Irina Nikishina, +7 (495) 109-29-10, sokrat@irramn.ru

  • State Budgetary Institution of Healthcare of the City of Moscow "Morozovskaya Children's City Clinical Hospital of the Moscow City Health Department" (GBUZ "Morozovskaya DGBK DZM")

    Moscow, 119049

    Elena ZHolobova, +7 (495) 870-55-22, mdgkb@zdrav.mos.ru

  • LLC "Medical Technologies"

    Saint Petersburg, 191025

    Mikhail Kostik, +7 (495) 721-84-22, info@metg.ru

Other trials in Russia

About this study

Official title: International, Multicenter, Open-label Clinical Study of Goflikicept for Resolution and Prevention of Attacks in Pediatric and Adolescent Participants With Familial Mediterranean Fever With Resistance to or Intolerance of Colchicine

The primary objective is to evaluate the main pharmacokinetic (PK) parameters, efficacy, and safety of goflikicept (GFC) in children and adolescents with colchicine-resistant or colchicine-intolerant familial Mediterranean fever (FMF).

Read the detailed description

This is an open-label, non-randomized clinical study in pediatric and adolescent participants.

The study consists of three periods:

  • the screening period (up to 12 weeks) which includes a period for evaluating eligibility criteria and monitoring "marker" attacks
  • treatment period, up to 56 weeks including:
  • 16-week main treatment period
  • 40-week long-term treatment period
  • 8-week safety follow-up

The total maximum duration of participation of one participant in the study is up to 76 weeks, i.e. approximately 1 year and 6 months.

The study enrolls participants in two sequential cohorts. Cohort 1 includes 6 adolescents aged 12-14 years in the Kingdom of Saudi Arabia (KSA) and 12-17 years in other countries. An interim safety and pharmacokinetic (PK) analysis will be performed. If enrollment of participants aged 5-17 years is considered feasible, Cohort 2 enrolls participants aged 5-14 years in the KSA and 5-17 years in other countries, until at least 18 participants receive the investigational treatment.

Being tested
RPH-104
Keywords
RPH-104, colchicine inefficacy, colchicine intolerance, colchicine resistance, FMF, interleukin-1 inhibitor, Pediatric and adolescent patients
Medical subject headings
Familial Mediterranean Fever

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • RPH-104Biological

    Also called Arcerix, goflikicept

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

Study groups in NCT07880886
Study groupRoleWhat they receive
RPH-104ExperimentalRPH-104 subcutaneously at 2.2 mg/kg on Day 0, followed by 1.1 mg/kg on Days 7 and 14 and then every 2 weeks (q2w). The dose may be increased to 2.2 mg/kg q2w (maximum 160 mg) in non-responders or participants with recurrent familial Mediterranean fever (FMF) attacks.
Receives: Biological: RPH-104

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Maximum serum concentration of goflikicept (Cmax (W16))

    Measured: Visit 1 (Baseline) to Visit 10 (Week 16)

  2. Area under serum concentration-time curve for goflikicept (AUC(0-W16))

    Measured: Visit 1 (Baseline) to Visit 10 (Week 16)

Secondary outcomes (23)

  1. Changes in trough serum goflikicept concentration

    Measured: Visit 1 (Baseline) to Visit 10 (Week 16), and Visit 16 (Week 56)

  2. Trough serum steady-state concentration of goflikicept (Cmin,ss)

    Measured: Visit 1 (Baseline) to Visit 10 (Week 16), and Visit 16 (Week 56)

  3. Area under curve for the dosing interval (t) at steady state (ss) after repeated injections (AUC0-t,ss)

    Measured: Visit 1 (Baseline) to Visit 10 (Week 16), and Visit 16 (Week 56)

  4. Time to steady state

    Measured: Visit 1 (Baseline) to Visit 10 (Week 16), and Visit 16 (Week 56)

  5. Percentage of participants with a complete response to treatment

    Complete response is defined as resolution of a "marker" familial Mediterranean fever (FMF) attack by day 7 and the absence of recurrent (new) attacks during the treatment period.

    Measured: Up to Visit 16 (Week 56)

  6. Change from baseline in PGA (Physician Global Assessment) score

    The Physician's Global Assessment (PGA) is an investigator-rated 5-point scale used to evaluate overall disease activity. Scores range from 0 to 4, where: 0 = none; 1 = minimum; 2 = mild; 3 = moderate; 4 = severe signs and symptoms. Higher scores indicate greater disease severity.

    Measured: at Visit 1 (Baseline) and Visit 16 (Week 56)

  7. Change from baseline in C-reactive protein (CRP) level

    Measured: at Visit 1 (Baseline) and Visit 16 (Week 56)

  8. Change from baseline in serum amyloid A (SAA) level

    Measured: at Visit 1 (Baseline) and Visit 16 (Week 56)

  9. Percentage of participants with serological remission (C-reactive protein < 10 mg/L)

    Serological remission is defined as a C-reactive protein (CRP) level of less than 10 mg/L.

    Measured: Up to Visit 16 (Week 56)

  10. Time to a recurrent (new) FMF attack in participants with a resolved "marker" attack

    Recurrent (new) Familial Mediterranean Fever (FMF) attack is defined as the presence of both clinical and serological signs of an attack after resolution of the "marker" attack.

    Measured: Visit 2 (Week 1) to Visit 16 (Week 56)

  11. Percentage of participants who were administered an increased dose of goflikicept 2.2 mg/kg once every 2 weeks

    Measured: Up to Visit 16 (Week 56)

  12. Frequency, Severity, Nature, and Outcomes of Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Adverse event (AE) severity is assessed using the Common Terminology Criteria for Adverse Events (CTCAE) version 6.0.

    Measured: Up to Visit 16 (Week 56)

  13. Percentage of participants with anti-drug antibodies (ADA)

    Measured: Up to Visit 16 (Week 56)

  14. Number of participants with clinically significant laboratory abnormalities

    Laboratory parameters include:

    • Hematology
    • Blood Biochemistry
    • Measurement of the concentration of immunoglobulin type G (IgG)
    • Urinalysis

    Measured: Up to Visit 16 (Week 56)

  15. Number of participants with clinically significant vital sign abnormalities

    Measurement of vital signs includes:

    • body temperature (in degrees Celsius)
    • blood pressure (BP) (one arm, mm Hg)
    • heart rate (HR) (bpm)
    • respiratory rate (RR) (per min)

    Measured: Up to Visit 16 (Week 56)

  16. Percentage of participants with Physician Global Assessment (PGA) score < 2 at each visit

    The Physician's Global Assessment (PGA) is an investigator-rated 5-point scale used to evaluate overall disease activity. Scores range from 0 to 4, where: 0 = none; 1 = minimum; 2 = mild; 3 = moderate; 4 = severe signs and symptoms. Higher scores indicate greater disease severity.

    Measured: Up to Visit 16 (Week 56)

  17. Сhange from baseline in the overall disease activity assessed by the participant/parent (Visual analogue scale)

    Assessment of the disease activity by the subject/legal representative is performed using a visual analog scale (VAS) in mm (0-100), where 0 mm = very good, 100 mm = very bad.

    Measured: at Visit 1 (Baseline) and Visit 16 (Week 56)

  18. Percentage of participants with a partial response

    Partial response is defined as resolution of a "marker" familial Mediterranean fever (FMF) attack by day 7 and the presence of recurrent (new) attacks during the treatment period.

    Measured: Up to Visit 16 (Week 56)

  19. Percentage of participants with normal serum amyloid A levels

    Percentage of participants whose serum amyloid A (SAA) levels return to normal (<10 mg/L) at each study visit.

    Measured: Up to Visit 16 (Week 56)

  20. Percentage of participants receiving additional symptomatic therapy

    Includes additional symptomatic therapy with nonsteroidal anti-inflammatory drugs (NSAIDs), paracetamol (acetaminophen), or glucocorticoids.

    Measured: Up to Visit 16 (Week 56)

  21. Percentage of participants who prematurely discontinued therapy due to Adverse Events (AEs)

    Measured: Up to Visit 16 (Week 56)

  22. Percentage of participants with neutralizing antibodies (NAb)

    Measured: Up to Visit 16 (Week 56)

  23. Number of participants with clinically significant electrocardiogram (ECG) abnormalities

    Measured: Up to Visit 16 (Week 56)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted19 Oct 2026
  2. Study startDec 2026 (estimated)
  3. Primary completionMay 2029 (estimated)
  4. Study completionApr 2030 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential
Model description
Sequential enrollment in 2 age-based cohorts with an interim safety and pharmacokinetics analysis after Cohort 1
Masking
None (open label)
Enrolment
18 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07880886
Sponsor's protocol ID
CL04018389
Status last verified
Oct 2026
First submitted
12 Oct 2026
Last updated
19 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing RPH-104 in people with Familial Mediterranean Fever and FMF, run by R-Pharm at 3 sites in Russia.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 13 inclusion and 36 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 19 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in Russia. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Yulia Karpova (+7 (495) 956-79-37 ext. 1197, jn.karpova@rpharm.ru), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Maximum serum concentration of goflikicept (Cmax (W16)) (time frame: Visit 1 (Baseline) to Visit 10 (Week 16)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07880886.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Oct 2026.

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