NL-OMON50760尚未招募2 期
A pilot, open-label, single arm, multicenter study to evaluate safety, tolerability, pharmacokinetics and efficacy of intravenous administrations of emapalumab, an anti-interferon gamma (anti-IFNγ) monoclonal antibody, in patients with systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still*s Disease (AOSD) developing Macrophage Activation Syndrome/ secondary HLH (MAS/sHLH) - NI-0501-06
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 3
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 99（—）
入选标准
- •1. Patients of both genders 2. For sJIA patients: confirmed sJIA diagnosis. For
- •patients presenting with MAS in the context of the onset of sJIA high
- •presumption of sJIA (as per Appendix A) will suffice for eligibility.AOSD
- •patients: confirmed AOSD diagnosis as per Yamaguchi criteria (Appendix E) 3.
- •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.
- •(see Appendix B), 4 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 PDN
- •equivalent in 2 divided doses, (or at least 60 mg/day in patients of 30 kg or
- •more) 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. 5.Tocilizumab, TNF inhibitors and canakinumab, if
- •administered, have to be discontinued before emapalumab initiation. 6. 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. 7. 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.
排除标准
- •1. Diagnosis of suspected or confirmed primary HLH or HLH consequent to a
- •neoplastic disease. 2.Active mycobacteria (typical and atypical), Histoplasma
- •Capsulatum, Shigella, Salmonella, Campylobacter and Leishmania infections., 3.
- •Clinical suspicion of latent tuberculosis., 4. Positive serology for HIV
- •antibodies., 5. Presence of malignancy., 6. 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 emapalumab safety., 7. History of hypersensitivity or allergy to any
- •component of the study drug., 8. Receipt of a BCG vaccine within 12 weeks prior
- •to screening., 9. Receipt of live or attenuated live vaccines (other than BCG)
- •within 6 weeks prior to screening., 10. Pregnant or lactating female patients.
研究者
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