Efficacy of Tocilizumab in a Patient With Relapsing Polychondritis
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Physician Global Assessment of Disease Activity
研究概览
简要总结
Relapsing polychondritis (RP) is a rare, immune-mediated disease associated with inflammation in cartilaginous structures and other tissues throughout the body. Prognosis can be poor, especially in cases where there is acute involvement of the laryngotracheal cartilages leading to airway destruction, which are resistant to treatments such as corticosteroids, immunosuppressive or cytotoxic drugs. The pathogenesis remains unclear although it is thought that autoimmune reactions to antigens present in cartilages, such as type II collagen and matrilin may evoke symptoms. There are no known clinical or laboratory measures that predict the expression of specific disease manifestations or the overall disease course. Two recently published case reports have shown an association with elevated serum IL-6 levels and relapsing polychondritis. In these case reports, both patients with refractory relapsing polychondritis were treated with tocilizumab, a humanized monoclonal antibody to the Interleukin 6 receptor, and achieved sustained response to the drug. This single patient trial aims to evaluate the response to Tocilizumab in an eight year old boy with relapsing polychondritis who has been shown to have elevated serum IL-6 levels and who has responded poorly to conventional therapies. The study hypothesis is that Tocilizumab will be able to control the disease in this patient.
详细描述
In this N = 1 study a single known patient with relapsing polychondritis who has failed methotrexate, various anti TNF medications, anti IL1 medication and prolongued glucocorticosteroids will be recruited to receive Tocilizumab 8 mg /kg q 2 weeks iv.
The objective is to assess efficacy of tociliuzmab in combination with stable ongoing therapy. Our patient received tocilizumab 8 mg/kg over 1 hour by intravenous infusion every 2 weeks throughout the course of the study. To assess tocilizumab efficacy, the primary objective is the change in physician global assessment on a 100-mm horizontal visual analogue scale (VAS) of disease activity.
The secondary objectives were the change in parent global assessment of disease activity on a 100 mm VAS and the glucocorticoid dose in mg per day. Frequency of adverse events was also measured at baseline and after each biweekly tocilizumab infusion.
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
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Refractory relapsing polychondritis
- •Failed glucocorticoid and methotrexate therapy
排除标准
- •This is an N=1 clinical trial with a known patient, therefore, exclusion criteria are non-applicable.
研究组 & 干预措施
Tocilizumab
Single arm open label study. In this arm patient will receive 8mg/kg of Tocilizumab q 2 weeks iv.
干预措施: Tocilizumab (Drug)
结局指标
主要结局
Physician Global Assessment of Disease Activity
时间窗: Baseline and then every 2 weeks prior to each infusion for total duration of 30 weeks
Physician global assessment of disease activity was assessed on a 100 mm Visual Analogue Scale where 0 would be no disease activity and 100 would be the maximum disease activity. Higher values therefore indicate higher disease activity and therefore a worse outcome. Change of this outcome measure over time was documented.
次要结局
- Prednisone Dose(30 weeks)
- Parent/Patient Global Assessment of Overall Well Being(30 weeks)
研究者
Johannes Roth
Pediatric Rheumatologist
Children's Hospital of Eastern Ontario