NL-OMON52297招募中2 期
A two-cohort, open-label, single arm, multicenter study to evaluate efficacy, safety and tolerability, pharmacokinetics and pharmacodynamics, of emapalumab in children and adults with macrophage activation syndrome (MAS) in Still*s disease (including systemic juvenile idiopathic arthritis and Adult onset Still*s disease) or with MAS in Systemic lupus erythematous - NI-0501-14
Swedish Orphan International0 个研究点目标入组 5 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 5
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
- 0 至 99（—）
入选标准
- •Inclusion criteria
- •Run-in phase in all cohorts
- •1. Informed consent provided by the patient or by the patient's legally
- •authorized representative(s) with the assent of patients who are legally
- •capable of providing it, as required by local law.
- •2. Male and female patients aged between 6 months and 80 years of age at the
- •time of diagnosis of active MAS.
- •3. MAS defined as per the criteria defined below for each cohort and requiring
- •treatment with GCs as per standard of care.
- •Interventional phase in all cohorts
- •1. Informed consent provided by the patient or by the patient's legally
- •authorized representative(s) with the assent of patients who are legally
- •capable of providing it, as required by local law.
- •2. Male and female patients aged between 6 months and 80 years of age at the
- •time of diagnosis of active MAS.
- •3. Patients who have shown an inadequate response to high dose intravenous
- •(i.v.) GCs administered for at least 3 days according to local standard
- •clinical practice, including but not limited to pulses of 30 mg/kg
- •methylprednisolone (mPDN) on 3 consecutive days. High i.v. GCs dose is
- •recommended not to be lower than 2 mg/kg/ day PDN equivalent (or at least 60
- •mg/day in pediatric patients of 30 kg or more and at least 1 g/day in adult MAS
- •patients). In case of rapid worsening of the patient*s condition and/or
- •laboratory parameters, as per Investigator judgment, inclusion may occur within
- •less than 3 days from starting high dose GCs.
- •4. Diagnosis of active MAS confirmed by the treating rheumatologist, having
- •ascertained the followings:
- •a. Febrile patients presenting with ferritin > 684 ng/mL.
- •b. and any 2 of:
- •i. Platelet count <= 181 x109/L
- •ii. Aspartate aminotransferase (AST) -level > 48 U/L
- •iii. Triglycerides > 156 mg/dL
- •iv. Fibrinogen level <= 360 mg/dL
- •5. Female patients of child-bearing potential (sexually or non sexually
- •active). Female patients who are sexually active must be willing to use highly
- •effective methods of contraception from study drug initiation to 6 months after
- •the last dose of study drug.
- •Specific inclusion criteria for Cohort 1 and Cohort 2
- •6. Cohort 1:
- •a. Confirmed sJIA diagnosis. For patients presenting with MAS in the context of
- •the onset of sJIA, high presumption of sJIA will suffice for eligibility.
- •b. Confirmed diagnosis of AOSD as per Yamaguchi criteria (Yamaguchi et al.
- •7. Cohort 2:
- •a. Confirmed diagnosis of SLE as per Systemic Lupus International Collaborating
- •Clinics (SLICC) 2012-criteria.
排除标准
- •1. Primary hemophagocytic lymphohistiocytosis (pHLH) documented by either the
- •presence of a known causative genetic mutation or abnormal perforin expression
- •or CD107a degranulation assay as described with pHLH or by the presence of
- •family history.
- •2. Confirmed malignancy. Note: patients with a suspected malignancy should have
- •mononuclear cells typed by flow cytometry and/or tissue biopsy, as applicable,
- •to rule out malignancy.
- •3. Treatment with canakinumab, Janus kinase (JAK) inhibitors, tumor necrosis
- •factor (TNF) inhibitors and tocilizumab at the time of emapalumab initiation.
- •4. Ongoing treatment with anakinra at a dose above 4 mg/kg/day at time of
- •emapalumab initiation.
- •5. Patients treated with etoposide for MAS in the last 1 month.
- •6. Presence of any medical or psychological condition or laboratory result that
- •in the opinion of the Investigator can interfere with the patient's ability to
- •comply with the protocol requirements or makes the patient not appropriate for
- •inclusion to the study and treatment with emapalumab.
- •7. Foreseeable inability to cooperate with given instructions or study
- •procedures.
- •8. Clinically active mycobacteria (typical and atypical), Histoplasma
- •Capsulatum, or Salmonella infections.
- •9. Evidence of leishmania infections.
- •10. Evidence of latent TB.
- •11. History of hypersensitivity or allergy to any component of the study drug.
- •12. Receipt of a Bacillus Calmette-Guerin (BCG) vaccine within 12 weeks prior
- •to Screening.
- •13. Receipt of a live or attenuated live (other than BCG) vaccine within 4
- •weeks prior to Screening.
- •14. Pregnancy or lactating female patients.
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