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Familial Mediterranean Fever (FMF )Phase 4NCT06666335

A Study to Evaluate Efficacy and Safety of Anakinra in Chinese Patients With Colchicine-resistent FMF

A Phase 4 study testing Anakinra in people with Familial Mediterranean Fever (FMF ), run by Swedish Orphan Biovitrum.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

The purpose of this study is to evaluate the efficacy and safety of anakinra in Chinese patients with colchicine-resistand Familial Mediterranian Fever (FMF). The study consists of up to one month screening, to see if a patient is suitable to the study, 6 months of treatment with anakinra and one month safety follow… Read more about this study

Who is it for?
Ages 2 years and older
Conditions the study is for
Familial Mediterranean Fever (FMF )
What is being tested?
Anakinra
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
No sites listed yet
Study dates
1 Mar 2026to 30 Nov 2027
Phase
Phase 4
Are there results?
None posted in the registry

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 2 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Informed consent form signed by the patient or a legal guardian representative.
  • Male or female patients, 2 years of age or older with a body weight ≥ 10 kg.
  • Diagnosis of FMF confirmed by a positive genetic testing i.e., mutations in both alleles of the MEFV gene (i.e. homozygous or compound heterozygous).
  • Patient must have an estimated mean of at least 2 acute FMF attacks per month within 2 months prior to enrollment to the study.
  • Patient must be resistant to colchicine treatment.
  • Female patients of childbearing potential and male patients with female partners of childbearing potential must use an effective method of contraception during the study (abstinence being a possible option) as well as a negative pregnancy test prior to enrollment for females of childbearing potential and participating in the study.
  • Negative tuberculosis screening confirmed at screening visit by the Mantoux Tuberculin skin test (TST) using purified protein derivative (PPD), or by Interferon-Gamma-Release Assays (IGRAs) e.g., QuantiFERON® TB Gold Plus (QFT-Plus) or T-Spot® (TB Test) within 8 weeks prior to enrollment. Negative results must be complemented by the medical history, physical examination, and Chest X-Ray. Patients presenting positive TST or IGRA with or without active or clinical suspicion of latent tuberculosis are not eligible to enter the study. Previously vaccinated for Tuberculosis patients: IGRA positive patients are not eligible to enter the study; TST positive patients with an induration of 15 mm and more are also not eligible to enter the study, TST positive patients (with an induration up to 15 mm) are also not eligible to enter the study, unless an IGRA test is subsequently performed and provides a negative result.

Who cannot join

  • Previous enrollment to this study.
  • Participation in another clinical interventional study 30 days prior to enrollment.
  • Treatment with an investigational drug within 5 half-lives prior to enrollment.
  • Previous or current treatment with anakinra, or any other IL-1 inhibitor.
  • 6. Live vaccines within 4 weeks prior to enrollment.
  • 7. Known presence or suspicion of active, chronic or recurrent bacterial, fungal or viral infections, including but not limited to tuberculosis, HIV infection, Covid-19 infection, hepatitis B or C infection at baseline.
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  • 8. Clinical evidence of liver disease or liver injury as indicated by presence of abnormal liver tests
  • 9. Presence of severe chronic kidney disease
  • 10. Diagnosis of amyloidosis at baseline.
  • Other protocol defined inclusion/exclusion criteria may apply.

About this study

Official title: A Prospective, Open-label, Single Arm, Multicenter, Post-authorization Efficacy and Safety Study of Subcutaneous Anakinra in Chinese Patients With Colchicine-resistant Familial Mediterranean Fever (FMF)

The purpose of this study is to evaluate the efficacy and safety of anakinra in Chinese patients with colchicine-resistand Familial Mediterranian Fever (FMF). The study consists of up to one month screening, to see if a patient is suitable to the study, 6 months of treatment with anakinra and one month safety follow up after last dose of anakinra. In total 3 patients, male and female from 2 years of age (minimum 10kg weight), will be enrolled to the study.

Read the detailed description

This is a prospective, open-label, single-arm, multi-center study, and this study consists of a 6-month treatment period with anakinra followed by a 4-Week period to evaluate safety of anakinra after the last dose of study drug i.e. at Month 7. The study is divided into three parts: screening, treatment period, and safety follow-up.

The patient will enter screening after informed consent is obtained and will undergo screening assessments to confirm eligibility. Duration of the screening period will be kept as short as possible and should not exceed 4 weeks.

Patients will be assigned to study drug after they have met all of the inclusion criteria and none of the exclusion criteria. Patients will receive daily subcutaneous treatment with anakinra for 6 months.

After the last dose of anakinra at Month 6, the safety will continue to be evaluated at a Safety Follow-up visit., at Month 7.

The primary endpoint will be change in the number of FMF attacks per month per patient from baseline to subsequent study visits, up to Month 6.

Being tested
Anakinra
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.

  • AnakinraDrug

    Also called Kineret

    Subcutaneous injections once daily for 6 months

Study groups in NCT06666335
Study groupRoleWhat they receive
AnakinraExperimentalAnakinra, once daily s.c. injection, starting dose of 100 mg/day for patients with body weight ≥ 50 kg and 1-2 mg/kg/day for patients with body weight < 50 kg for 6 months. For patients < 16 years and < 50 kg not responding sufficiently, the dose can be increased up to 4 mg/kg/day (max of 200 mg/day).
Receives: Drug: Anakinra

What is being measured

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

  1. Change from baseline in the number of FMF attacks per month.

    An FMF attack is defined as fever ≥ 38°C lasting between 6 hours to 7 days and accompanied by painful serositis manifestations in any of the following sites: abdomen, chest, joints and skin.

    Measured: Up to 6 Months

Secondary outcomes (9)

  1. The number of months free of FMF attacks will be evaluated in each patient.

    An FMF attack is defined as fever ≥ 38°C lasting between 6 hours to 7 days and accompanied by painful serositis manifestations in any of the following sites: abdomen, chest, joints and skin.

    Measured: Up to 6 months.

  2. Change from baseline in C-reactive protein (CRP) concentrations.

    CRP levels (measured in mg/L) will be determined in serum.

    Measured: Up to 6 months.

  3. Change from baseline in Serum Amyloid A (SAA) concentrations.

    SAA levels (measured in mg/L) will be determined in serum.

    Measured: Up to 6 months.

  4. Change from baseline in the dose of analgesic agents used for the treatment of FMF symptoms.

    Reduction in the dose (measured in mg/kg) of analgesic treatments for FMF attacks will be evaluated during the study.

    Measured: Up to 6 months.

  5. Number of patients responding to study drug over time using the Modified FMF50 score.

    Modified FMF50 score response is defined by the to the presence of 3 of the 4 following criteria: decrease by ≥ 50% in the frequency of all FMF attacks; decrease by ≥ 50% in the frequency of FMF attacks in the joints; decrease by ≥ 50% in the levels of either SAA or CRP (measured as mg/L) with target level concentrations for CRP and SAA ≤10 mg/L; Increase by ≥ 50% in QoL values assessed using the VAS scale (0-10cm), where scores of 0 and 10 indicate the lowest and highest QoL scores, respectively.

    Measured: Up to 6 months.

  6. Number of Participants with Adverse Events (AEs), Serious Adverse Events (SAEs), deaths, and AEs leading to study drug discontinuation

    An AE is any untoward medical occurrence in a study patient to whom a medicinal product is administered, and which does not necessarily have a causal relationship with this treatment.

    AEs include abnormal test findings, clinically significant signs and symptoms, changes in physical examination findings, progression or worsening of underlying disease.

    Serious adverse events (SAEs) were defined as any untoward medical occurrences that result in death, are life threatening, require (or prolong) hospitalization, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgement of investigators represent significant hazards.

    Measured: Up to 7 months

  7. Changes over time in vital signs

    Vital signs change of each patients are evaluated over time. The vital sign evaluated are blood pressure (mmHg), heart rate (beats per minute), and body temperature (Celsius degrees).

    Measured: Up to 7 months

  8. Changes over time in body weight and height

    Body weight (kilograms) and height (centimeters) of each patients are evaluated over time.

    Measured: Up to 7 months

  9. Changes over time in Laboratory safety assessment

    Number of patients with abnormal laboratory test results

    Measured: Up to 7 months

Timeline

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

  1. First posted30 Oct 2024
  2. Study start1 Mar 2026 (estimated)
  3. Primary completion30 Aug 2027 (estimated)
  4. Study completion30 Nov 2027 (estimated)

Why it stopped: Business decision.

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 4
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06666335
Sponsor's protocol ID
Sobi.ANAKIN-701
Status last verified
Jun 2026
First submitted
8 Oct 2024
Last updated
29 Jun 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Anakinra in people with Familial Mediterranean Fever (FMF ), run by Swedish Orphan Biovitrum.

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 7 inclusion and 10 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: withdrawn, as of the sponsor's last update on 29 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

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

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

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

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change from baseline in the number of FMF attacks per month. (time frame: Up to 6 Months). 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 NCT06666335.

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 29 Jun 2026.

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