NCT01303380已完成2 期
An Open-label, Multicenter, Efficacy and Safety Pilot Study of 6-month Canakinumab Treatment With up to 6-month Follow-up in Patients With Active Hyper-IgD Syndrome (HIDS)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- Number of Flares Per Participant During Historical Period and Treatment Period
研究概览
简要总结
This pilot study is designed to evaluate the efficacy, the safety, and the pharmacokinetics (PK) / pharmacodynamics (PD) of canakinumab treatment in patients with HIDS.
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
- 盲法
- None
入排标准
- 年龄范围
- 24 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with a diagnosis of HIDS proven by DNA analysis and/or enzymatic studies.
- •At time of start of drug treatment: active HIDS as evidenced by a physician global assessment of HIDS flare severity ≥ 2 and CRP values >10 mg/L (normal CRP < or = 10 mg/L).
- •Patients who have a history of > or = 3 febrile acute HIDS flares in a 6-month period when not receiving prophylaxis treatment (e.g. anakinra daily treatment) with a duration of each flare lasting > or = 4 days and limiting the normal daily activities.
排除标准
- •Pregnant or nursing (lactating) women.
- •History of being immunocompromised, including a positive HIV at screening (ELISA and Western blot) test result.
- •Positive Hepatitis B or Hepatitis C.
- •Live vaccinations within 3 months prior to the start of the trial
- •Positive tuberculosis screening test.
- •Other protocol-defined inclusion/exclusion criteria may apply
研究组 & 干预措施
Canakinumab
Experimental
干预措施: Canakinumab (Drug)
结局指标
主要结局
Number of Flares Per Participant During Historical Period and Treatment Period
时间窗: Historical period, Month 6 (End of treatment period)
A flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a C-reactive protein (CRP) value \> 10 mg/L. Flares during a historical period were defined as most recent 6-months in which the participant has not received treatment for their HIDS other than symptomatic treatment with NSAIDs and/or corticosteroids.
次要结局
- Number of Flares Per Participant at During Treatment Period and 24 Month Extension Period(Month 6 (End of treatment period), Month 36 (End of Long term treatment Period 2))
- Number of Participants Who Flared at Month 6, Month 24 and Month 36(Baseline, Month 6 (End of treatment period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2))
- Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score(Any flare event [Baseline up to Month 36 (End of long term treatment period 2)])
- Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score(Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2))
- Percentage of Participants With Defined Grades of Participants Assessed Symptom Control(Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2))
- Percentage of Participants With Defined Grades of Physician Assessed Symptom Control(Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2))
- Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment(Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2))
- Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment(Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2))
- Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment(Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2))
- Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment(Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2))
- Time to Resolution of the Initial Flare After First Canakinumab Treatment(Day 1 (Baseline), Day 28)
- Change From Baseline in Inflammation Markers Over Time up to Month 24(Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2))
- Health Assessment Questionnaire (HAQ) Global Score in Adults Over Time(Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2))
- Childhood Health Assessment Questionnaire (CHAQ) Global Score in Children Over Time(Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2))
- Percentage of Participants Who Received Dose Up-titration During 6-month Treatment Period(Day 1 up to Month 6 (End of follow up))
- Duration of Flares Experienced During the Study(Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2))
- Time to Flare After the Last Dose of Canakinumab During the Follow-up Period(Last dose of canakinumab treatment in follow-up period to end of follow-up period (Day 337))
- Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)(Day 1 (Start of study treatment) up to Month 36 (End of study))
- Participants Who Received Rescue Treatment(Baseline up to Month 36 (End of study))
- Serum Concentration-time Profile of Canakinumab(Day 1 (Pre-dose), Day 4, Day 15, Day 43, Day 85, Day 127, Day 169 (End of treatment period), Day 197, Day 225, Day 253, Day 281, Day 309, and Day 337 (End of follow-up period) (Post-dose))
- Serum Concentration of Total Interleukin-1β Antibody (IL-1β)(Day 1 (Pre-dose), Day 4, Day 15, Day 43, Day 85, Day 127, Day 169 (End of treatment period), Day 197, Day 225, Day 253, Day 281, Day 309, and Day 337 (End of follow-up period) (Post-dose))
- Number of Participants Exhibiting Anti-canakinumab Antibodies at Any Visit(Baseline up to Month 36 (End of study))
研究者
研究点 (1)
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