An Open-label Extension Study to Assess Efficacy, Safety and Tolerability of Canakinumab and the Efficacy and Safety of Childhood Vaccinations in Patients With Cryopyrin Associated Periodic Syndromes (CAPS)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 17
- 试验地点
- 12
- 主要终点
- The Percentage of Participants Without Disease Relapse as Determined by the Physician's Global Assessment of Autoinflammatory Disease Activity, Assessment of Skin Disease and Serological Inflammation Markers.
研究概览
简要总结
This trial will provide long-term safety, efficacy and tolerability of ACZ885 in CAPS patients that completed the CACZ885D2307 study
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
入排标准
- 年龄范围
- 1 Year 至 4 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who completed the core CACZ885D2307 study (a patient is defined as having completed the core study if they completed the study up to and including the EOS visit with no major protocol deviations in the core).
- •Male and female patients that are ≥ 1 year of age at the time of the roll-over visit.
- •Parent or legal guardian written informed consent must be obtained before any assessment in the extension CACZ885D2307E1 study is performed.
排除标准
- •Patients for who continued treatment in the CACZ885D2307E1 extension study is not considered appropriate by the treating physician.
- •Patients who discontinued from the core CACZ885D2307 study
- •Other protocol-defined inclusion/exclusion criteria may apply.
研究组 & 干预措施
canakinumab
Patients will receive a standard dose at an equivalent of 2 mg/kg s.c. of canakinumab (ACZ885) every 8 weeks. Possible dose and/or dosing regimen adjustments that can be administered include: 4 mg/kg s.c. (every 4 to 8 weeks) 6 mg/kg s.c. (every 4 to 8 weeks) 8 mg/kg s.c. (every 4 to 8 weeks)
干预措施: ACZ885 (Biological)
结局指标
主要结局
The Percentage of Participants Without Disease Relapse as Determined by the Physician's Global Assessment of Autoinflammatory Disease Activity, Assessment of Skin Disease and Serological Inflammation Markers.
时间窗: Week /80, 104, 128, and 152 (A minimum of 6 months and maximum of 24 months)
Disease relapse following complete response is defined as inflammation markers: C-Reactive Protein (CRP) and/or Serum Amyloid A (SAA) result \> 30 mg/L AND Physician's Global Assessment of Autoinflammatory Disease Activity \> minimal or Physician's Global Assessment \>= minimal AND Skin Disease Assessment \> minimal. Physician's Global Assessment of Autoinflammatory Disease Activity and Skin Disease Assessment (urticarial skin rash) are completed by the investigator using a 5 point rating scale: absent, minimal, mild, moderate and severe.
次要结局
- Change From Baseline (Core Study Baseline) in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations(Week 0, 80, 104, 128 and 152, last assessment)
- Frequency Counts of Physician's Global Assessment of Autoinflammatory Disease and Skin Disease(minimum of 6 months and maximum of 24 months)
- Immunogenicity of Canakinumab (ACZ885). Number of Participants With Anti-canakinumab Antibodies(minimum of 6 months and maximum of 24 months)
- Number of Vaccination Cases With Protective Antibody Levels Following Immunization With Inactivated Vaccines(pre-vaccine dose, Day 28 post-vaccine)