pfront autologous hematopoietic stem cell transplantation versus immunosuppressive medication in early diffuse cutaneous systemic sclerosis: an international multicentre, open-label, randomized con-trolled trial

niversity Medical Centre Utrecht0 sites120 target enrollmentTBD

Overview

No summary available.

Registry

who.int

Start Date

TBD

End Date

TBD

Last Updated

last year

Study Type

Interventional

Sponsor

niversity Medical Centre Utrecht

•1\. Age between 18 and 65 years.
•2\. Fulfilling the 2013 ACR\-EULAR classification criteria for SSc (appendix B).
•3\. Disease duration \= 2 years (from onset of first non\-Raynaud’s symptoms) and diffuse cutaneous disease with
•\- mRSS \= 15 and/or
•\- clinically significant organ involvement as defined by either:
•a)respiratory involvement \=
•i. DLCO and/or (F)VC \= 85% (of predicted) and evidence of interstitial lung disease on HR\-CT scan with clinically relevant obstructive disease and emphysema excluded.
•ii. Patients with a DCLO and/or FVC \> 85%, but with a progressive course of lung disease: defined as relative decline of \>10% in FVC predicted and/or TLC predicted, or \>15% in DLCO predicted and evidence of interstitial lung disease on HR\-CT scan with clinically relevant obstructive disease and emphysema excluded, within 12 months. Intercurrent infections excluded.
•b)renal involvement \= any of the following criteria: hypertension (two successive BP readings
•of either systolic \= 160 mm Hg or diastolic \> 110 mm Hg, at least 12 hours apart), persistent

•A potential subject who meets any of the following criteria will be excluded from participation in this study:
•1\. Pregnancy or unwillingness to use adequate contraception during study
•2\. Concomitant severe disease \=
•a)respiratory: resting mean pulmonary artery pressure (mPAP) \> 20 mmHg (by right heart catheterisa
•tion), DLCO \< 40% predicted, respiratory failure as defined by the primary endpoint
•b)renal: creatinine clearance \< 40 ml/min (measured or estimated)
•c)cardiac: clinical evidence of refractory congestive heart failure; LVEF \< 45% by cardiac echo or cardiac
•MR; chronic atrial fibrillation necessitating oral anticoagulation; uncontrolled ventricular
•arrhythmia; pericardial effusion with hemodynamic consequences
•d)liver failure as defined by a sustained 3\-fold increase in serum transaminase or bilirubin, or a Child\-