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

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.

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.

Inclusion Criteria:

1. Informed consent form signed by the patient or a legal guardian representative.
2. Male or female patients, 2 years of age or older with a body weight ≥ 10 kg.
3. 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).
4. Patient must have an estimated mean of at least 2 acute FMF attacks per month within 2 months prior to enrollment to the study.
5. Patient must be resistant to colchicine treatment.
6. 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.
7. 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.

Exclusion Criteria:

1. Previous enrollment to this study.
2. Participation in another clinical interventional study 30 days prior to enrollment.
3. Treatment with an investigational drug within 5 half-lives prior to enrollment.
4. 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.

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.