A pilot study on the safety and efficacy of haemopoietic stem cell mobilization (CD34+ cells) with MOZOBIL ± G-CSF, in adult patients diagnosed with beta-thalassaemia major.

niversity of Washington0 个研究点开始时间: 2010年6月15日最近更新: 2012年3月19日

适应症

试验速览

暂无简介。

•a) ?eta- thalassemia major
•b) Age >18<50
•c) Karnofsky performance status ³80%
•d) Splenectomized patients or patients with spleen volume <800cm3 (only for the non splenectomized patients
•who will receive Mozobil + G-CSF) [V=0.523 x length x thickness x width (De Odorico I et al, 1999)]
•e) Compliant with regular transfusions and regular chelation
•f) Liver iron by MRI <280µmol/gr or 1.7msec by T2*MRI
•g) Heart iron by MRI >2.8 (SI/SD) or 9msec by T2*MRI
•h) Hepatitis B or C virus load negative by PCR
•i) Left ventricular ejection fraction (LVEF) >45% by echocardiogram
•j) Adequate respiratory function with DLCO >50%
•k) Negative pregnancy test, if female
•l) Ability to give informed consent and willingness to meet all the expected requirements of the protocol for the
•duration of the study
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years)
•F.1.3.1 Number of subjects for this age range

•a) History of thrombosis or known thrombophilia
•b) Symptomatic viral, bacterial or fungal infection within 6 weeks prior eligibility evaluation
•c) Pregnancy or lactation
•d) HIV positivity
•e) History of malignancy, other than local skin cancer
•f) Other systematic disease non thalassemia-associated
•g) Splenectomized patients with platelet count >900,000 (only for the splenectomized patients who will receive
•low dose G-CSF+ Mozobil)
•h) Additional risk factors for thrombosis, including Factor V Leiden; antiphospholipid antibodies and less than
•50% of the lowest normal value for the following procoagulants: antithrombin 3, protein C, or protein S.

发起方

niversity of Washington