A single-arm, open-label, phase 2 study of autologous CD34+ stem cells-selected transplantation for severe systemic sclerosis - A single-arm, open-label, phase 2 study of autologous CD34+ stem cells-selected transplantation for severe systemic sclerosis

Akashi Koichi0 个研究点目标入组 12 人开始时间: 2020年1月9日最近更新: 2023年10月17日

适应症

试验速览

暂无简介。

•A patient aged 16 to 64 who were diagnosed as systemic sclerosis with performance status (ECOG) 0 to 2. a) Within 4 years after onset and modified Rodnan total skin thickness score (mRSS) is 15 or more , and applicable to one of the following organ complication 1) Pulmonary complications: interstitial pneumonia diagnosed by chest X-ray examination or chest CT, and %FVC or %DLCO is less than 80%. 2) Renal complications: systolic blood pressure is 160 mmHg or more or diastolic blood pressure is 110 mmHg or more, urinalysis abnormality (urine protein, urine occult blood, urinary sediment ), microangiopathic hemolytic anemia, or elevated serum creatinine. 3) cardiac complications: reversible heart failure, or arrhythmia (supraventricular, ventricular, AV block), or pericardial effusion b) 1) Involvement of sclerosis to the trunk within 2 years after onset, mRSS is 20 or more, and 2) ESR is 25 mm/1h or more and/or Hb is 11 g/dL or less.

•A patient with one of the following severe organ complication, a) Heart: uncontrollable arrhythmia, uncontrollable heart failure, LVEF is less than 50% in cardiac ultrasound examination, moderate or more pulmonary hypertension (mean pulmonary artery pressure is 40 mmHg or more) b) Lung: PaO2 is less than 60 mmHg at room air, %VC is less than 50%. %DLCO is less than 20%. c) Kidney: renal glomerular filtration rate is less than 40 ml/min, serum creatinine is 2 mg/dl or more. d) Uncontrolled malignant neoplasm. e) Uncontrollable infection. f) Cyclophosphamide is used more than 10 g in total.

发起方

Akashi Koichi