Low-Dose Oral Imatinib in the Treatment of Scleroderma Pulmonary Involvement: A Phase II Pilot Study
试验速览
- 阶段
- 2 期
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Improvement of the pulmonary interstitial disease, evaluated by spirometry and DLCO, lung HRCTscan, emogasanalysis.
研究概览
简要总结
The purpose of this study is to verify effect and tolerability of imatinib on pulmonary and skin fibrosis in patients affected by systemic sclerosis.
详细描述
Low-dose Oral Imatinib in the Treatment of Scleroderma Pulmonary Involvement: a Phase II pilot study.
Background and rationale
Systemic sclerosis (SSc; scleroderma) is a rare, multisystem connective tissue disease characterised by widespread microvascular lesions and by the increased deposition of matrix components in the skin and internal organs, with varying degrees of severity.
The incidence of scleroderma appears to have increased from 0.6 new cases annually per million in 1947 to 19 new cases annually in 1991 .
The median survival have been calculated to be approximately 60% at 5 years and 50% at 10 years. The U.S. collaborative study reported a 12 years survival of 30% .
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
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •SSc with active pulmonary involvement refractory to conventional immunosuppressive regimen
- •Presence of active interstitial alveolitis (presence of grade 2 exertional dyspnea according to Mahler Dyspnea Index AND worsening of lung function tests PLUS interstitial alveolitis assessed by HRCTscan, ground-glass pattern involving at least two lung segments OR dyspnea (as defined above)PLUS Interstitial alveolitis assessed by HRCTscan (AS DEFINED ABOVE) AND neutrophilic or eosinophilic alveolitis detected through broncoalveolar lavage
- •Resistance to conventional immunosuppressive treatment (worsening or lack of improvement of lung function tests after cyclophosphamide therapy, conducted for at least three months, with a cumulative dosage > or = 6 g
- •Age 18-80 years
- •Ability ti give an informed consent
- •Use of an acceptable method of birth control; pregnancy will be ruled out before study beginning
排除标准
- •Connective tissue diseases other than SSc
- •Smoking Habit
- •Pregnancy or lactation
- •HBV or HCV infection
- •Severe anaemia (Hb< or = 8g/dl)
- •Hepatic disease (ALT or ALP>1.5 fold above normal levels)
- •Moderate or severe renal failure (creatinine clearance<59ml/min)
- •Severe heart failure, with ejection fraction < or = 35% measured by echocardiography
- •thyroid disease with clinical evidence of hyperthyroidism treated with substitutive therapy
研究组 & 干预措施
1
Imatinib 200 mg p.o. once a day for 6 months
干预措施: Imatinib (Drug)
结局指标
主要结局
Improvement of the pulmonary interstitial disease, evaluated by spirometry and DLCO, lung HRCTscan, emogasanalysis.
时间窗: 1 year
次要结局
- Cutaneous Involvement, evaluated by the modified Rodnan skin score; quality of life and patient physical and emotional well being, evaluated by SF-36 score and HAQ(1 year)