EUCTR2016-004223-23-FR进行中（未招募）1 期
A pilot, open-label, single arm, multicenter study to evaluate safety, tolerability, pharmacokinetics and efficacy of intravenous administrations of NI-0501, an anti-interferon gamma (anti-IFN?) monoclonal antibody, in patients with systemic Juvenile Idiopathic Arthritis (sJIA) developing Macrophage Activation Syndrome/secondary HLH (MAS/sHLH)
ovimmune SA0 个研究点目标入组 10 人开始时间: 2017年2月13日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 10
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Patients of both genders, aged from birth to <18 years
- •Confirmed sJIA or high presumption of sJIA
- •Diagnosis of active MAS confirmed by the treating rheumatologist, having ascertained the followings:
- •Febrile patient presenting with:
- •- Ferritin > 684 ng/mL
- •and any two of
- •- Platelet count = 181 x10^9/L
- •- AST levels > 48 U/L
- •- Triglycerides > 156 mg/dL
- •- Fibrinogen levels = 360 mg/dL.
- •Patient presenting an inadequate response to high dose i.v. glucocorticoid treatment administered for at least 3 days as per local standard of care (including but not limited to pulses of 30 mg/kg methylprednisolone (mPDN) on 3 consecutive days).
- •High i.v. glucocorticoid dose should not be lower than 2 mg/kg/ day of mPDN equivalent in 2 divided doses, up to a total of 60 mg/day. In case of rapid worsening of the patient’s condition and/or lab parameters, inclusion may occur within less than 3 days from starting high dose i.v. glucocorticoids.
- •Informed consent provided by the patient (as required by local law), or by the patient’s legally authorized representative(s) with the assent of patients who are legally capable of providing it, as applicable.
- •Having received guidance on contraception for both male and female patients sexually active and having reached puberty:
- •Females of child-bearing potential require use of highly effective contraceptive measures (failure rate of less than 1% per year) from screening until 6 months after receiving last dose of the study drug.
- •Highly effective contraceptive measures include:
- •o Sexual abstinence
- •o Hormonal contraceptives: combination or progesterone only
- •o Intrauterine methods: intrauterine devices or systems
- •o Bilateral tubal occlusion
- •o Vasectomised partner
- •Males with partners(s) of child-bearing potential must agree to take appropriate precautions (such as sexual abstinence, barrier contraception, vasectomy) to avoid fathering a child from screening until 6 months after receiving last dose of the study drug.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 10
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Diagnosis of suspected or confirmed primary HLH or HLH consequent to a neoplastic disease.
- •Patients treated with Tocilizumab, Canakinumab or TNF inhibitors within 5 times of their defined half-life.
- •Active mycobacteria (typical and atypical), Histoplasma Capsulatum, Shigella, Salmonella, Campylobacter and Leishmania infections.
- •Clinical suspicion of latent tuberculosis.
- •Positive serology for HIV antibodies.
- •Presence of malignancy.
- •Patients who have another concomitant disease or malformation severely affecting the cardiovascular, pulmonary, CNS, liver or renal function that in the opinion of the Investigator may significantly affect likelihood to respond to treatment and/or assessment of NI-0501 safety.
- •History of hypersensitivity or allergy to any component of the study drug.
- •Receipt of a BCG vaccine within 12 weeks prior to screening.
- •Receipt of live or attenuated live vaccines (other than BCG) within 6 weeks prior to screening.
- •Pregnant or lactating female patients.
研究者
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