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

NCT06666335 | Not Yet Recruiting Phase 4

• 1 other identifier: Sobi.ANAKIN-701
• Study Type: interventional
• Target: 3
• Locations: 0 countries
• Sites: N/A

Brief 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 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.

Conditions

Familial Mediterranean Fever (FMF )

Outcome Measures

Primary Outcomes (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.

  Up to 6 Months

Secondary Outcomes (9)

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

  Up to 6 months.

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

  Up to 6 months.

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

  Up to 6 months.

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

  Up to 6 months.

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

  Up to 6 months.

Study Arms (1)

Anakinra

EXPERIMENTAL

Anakinra, 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).

Drug: Anakinra

Interventions

Anakinra DRUG

Subcutaneous injections once daily for 6 months

Also known as: Kineret

Anakinra

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• \. Live vaccines within 4 weeks prior to enrollment.
• \. 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.
• \. Clinical evidence of liver disease or liver injury as indicated by presence of abnormal liver tests
• \. Presence of severe chronic kidney disease
• \. Diagnosis of amyloidosis at baseline.

Sponsors & Collaborators

• Swedish Orphan Biovitrum: lead
• Tigermed Consulting Co., Ltd: collaborator

MeSH Terms

Conditions

Familial Mediterranean Fever

Interventions

Interleukin 1 Receptor Antagonist Protein

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Central Study Contacts

Sobi Clinical Trials Contact

CONTACT

+46(0)86972000; medical.info@sobi.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 8, 2024
• First Posted: October 30, 2024
• Study Start: September 1, 2025
• Primary Completion (Estimated): August 30, 2027
• Study Completion (Estimated): November 30, 2027
• Last Updated: June 15, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share