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)
试验速览
- 阶段
- 4 期
- 状态
- 撤回
- 发起方
- 入组人数
- 3
- 主要终点
- Change from baseline in the number of FMF attacks per month.
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
- •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.
研究组 & 干预措施
Anakinra
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).
干预措施: Anakinra (Drug)
结局指标
主要结局
Change from baseline in the number of FMF attacks per month.
时间窗: Up to 6 Months
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.
次要结局
- 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.)
- Number of Participants with Adverse Events (AEs), Serious Adverse Events (SAEs), deaths, and AEs leading to study drug discontinuation(Up to 7 months)
- Changes over time in vital signs(Up to 7 months)
- Changes over time in body weight and height(Up to 7 months)
- Changes over time in Laboratory safety assessment(Up to 7 months)