NCT01302860已完成3 期
A One-year Open-label, Multicenter Trial to Assess Efficacy, Safety and Tolerability of Canakinumab (ACZ885) and the Efficacy and Safety of Childhood Vaccinations in Patients Aged 4 Years or Younger With Cryopyrin Associated Periodic Syndromes (CAPS)
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 17
- 试验地点
- 1
- 主要终点
- Percentage of Participants Aged 4 Years or Younger With at Least One Complete Response at Week 56
研究概览
简要总结
This trial will assess the safety, efficacy and tolerability of ACZ885 in patients aged 4 years and younger with cryopyrin associated periodic syndromes (CAPS)
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
- 1 Month 至 60 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female patients that are 28 days up to 60 months of age at the time of the screening visit.
- •Body weight > or = 2.5 kg.
- •Parent or legal guardian's written informed consent is required before any assessment is performed for patients.
- •At study entry, patients should have a clinical diagnosis of FCAS, MWS, or NOMID and symptoms requiring pharmacological intervention. Prior agreement between the Investigator and Novartis for study eligibility is required for patients who do not have a molecular diagnosis of NALP3 mutations available (either testing not performed, or testing performed but negative) upon study entry. For those patients who have not been molecularly tested for NALP3 mutations, molecular testing should be performed during the course of the study.
- •For patients treated with an IL-1 blocking agent (i.e. anakinra, rilonacept), these treatments should be discontinued prior to the baseline visit and patients must demonstrate active disease prior to treatment.
- •Patients who are scheduled to receive an immunization, according to their local vaccination guidelines, with an inactivated vaccine must be willing to participate in the assessment schedule for vaccinated patients.
排除标准
- •Preterm neonates for whom, in the Investigator's judgment, participation in the study is not deemed appropriate.
- •History of recurrent and/or evidence of active bacterial, fungal, or viral infections (including HIV).
- •Patients with immunodeficiency or treatment with immunosuppressive drugs.
- •Live vaccinations within < or = 3 months prior to screening. No live vaccinations will be allowed throughout the course of this study and up to 3 months following the last dose.
- •Patients with an increased risk of tuberculosis (TB) infection according to following risk factors:
- •Patients with recent close contact with persons known to have active pulmonary TB disease
- •Foreign-born patients from countries with a high prevalence of tuberculosis
- •Patients with recent tuberculosis infection (including children > 6 months with a positive PPD test [defined as an induration of at least 10mm])
- •Patients with end-stage renal disease
- •Patients with diabetes mellitus
- •Patients receiving immunosuppressive therapy
- •Patients with hematologic cancers.
- •Participation in another trial within the last 30 days or 5 half-lives of the investigational compound (whichever is longer).
- •Familial and social conditions rendering regular medical assessment not possible.
- •Pediatric patients with neutropenia (absolute neutrophil count [ANC] < 1.5 x 10 to the 9th/l)
- •Other protocol defined inclusion/exclusion criteria may apply
研究组 & 干预措施
Canakinumab
Experimental
Canakinumab s.c. injection (2 mg/kg) was administered every 8 weeks.
干预措施: ACZ885 (Drug)
结局指标
主要结局
Percentage of Participants Aged 4 Years or Younger With at Least One Complete Response at Week 56
时间窗: Week 56
Complete response was defined as clinical remission and serological remission. Clinical remission was defined as Physician global assessment of auto-inflammatory disease activity as absent or minimal (using a 5-point scale ranging from absent to severe) and assessment of skin disease as absent or minimal (using a 5-point scale ranging from absent to severe). Serological remission was defined as C reactive protein (CRP) or Serum amyloid A protein (SAA) to be less than (\<) 15 milligram per liter (mg/L) and \<10 mg/L respectively.
次要结局
- Percentage of Participants Aged 2 Years or Younger With at Least One Complete Response at Week 56(Week 56)
- Percentage of Participants With Defined Grades in Physician's Global Assessment Score at Week 56(Week 56)
- Percentage of Participants With Defined Grades in Physician Assessment of Skin Disease at Week 56(Week 56)
- Change From Baseline in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations at Week 56(Baseline, Week 56)
- Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)(Day 1 (start of study treatment) up to Week 56 (end of study))
- Percentage of Participants Receiving a Concomitant Vaccination During the Study(Day 1 (start of study treatment) to Week 56 (end of study))
- Number of Vaccination Cases With Protective Antibody Levels Following Immunization With Inactivated Vaccines(Day -14 (prior-vaccination), Day 0 (vaccination), Day 28, Day 57 (post-vaccination))
- Number of Participants With Anti-canakinumab Antibodies at Week 56(Week 56 (End of study))
研究者
研究点 (1)
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