臨床研究等提出・公開システム

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)

A clinical trial of autologous stem cell transplantation for severe systemic sclerosis (A clinical trial of autologous stem cell transplantation for severe systemic sclerosis)

Nakamura Masafumi

Akashi Koichi

Kyushu University Hospital

3-1-1 Maidashi, Higashi-ku, Fukuoka 812-8582, Japan

akashi@med.kyushu-u.ac.jp

kawano sachiko

Kyushu University Hospital

3-1-1 Maidashi, Higashi-ku, Fukuoka 812-8582, Japan

+81-92-642-5082

byssien@jimu.kyushu-u.ac.jp

Interventional

single arm study

open(masking not used)

historical control

single assignment

treatment purpose

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.

Both

systemic sclerosis

D036102

After peripheral blood stem cell mobilization and aphereisi with cyclophosphamide 4 g/m2 and G-CSF, CD34+ cells were enriched by using anti-CD34 immunomagnetic beads. Conditioning was performed using high-dose cyclophosphamide (50 mg/kg) for 4 days, and freeze-thawed CD34+ cells were transplanted.

D012595

The mean change in modified Rodnan skin score at 12 months after transplantation

The proportion of 25% improvement in skin score at 12 months. The mean change in forced vital capacity at 12 months. The mean change in serum KL-6 levels at 12 months. Event-free survival. Overall survival Incidence of adverse events.

Not Recruiting

+81-92-642-5063

Dec. 05, 2016