PLERIXAFOR MOBILIZED STEM CELLS AS SOURCE FOR GENE THERAPY OF BETA-THALASSEMIA AMD-THAL .

FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR0 个研究点目标入组 6 人开始时间: 2011年12月12日最近更新: 2021年10月26日

适应症

试验速览

暂无简介。

•Transfusion-dependent beta-thalassemia (any genotype) • = 18 years • Karnofsky Index > 80 % • Adequate cardiac, renal, hepatic and pulmonary functions as evidenced by: o LVEF greater than 45% by echo and normal ECG or presence of abnormalities not significant for cardiac disease o DLCO diffusing capacity > 50% and FEV1 and FVC > 60% predicted o Serum creatinine < 1.5 upper limit normal o Absent-mild-moderate liver iron overload on T2*MRI (less than 12 months before enrolment) o Absent-mild-moderate cardiac iron overload T2*MRI (less than 12 Sample size months before enrolment) o absence of severe fibrosis or cirrhosis on fibroscan or liver biopsy (less than 12 months before enrolment) • Negative thrombophilic screen and negative history for previous thrombotic events • For all patients, agreement to use highly effective and adequate method of contraception while receiving treatment and for at least 3 months following drug administration (including both females of child bearing potential and males with partners of child bearing potential) • Good adherence to transfusion and chelation programme • Written informed consent.
•Are the trial subjects under 18? no
•Number of subjects for this age range: 0
•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) yes
•F.1.3.1 Number of subjects for this age range

•Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long-acting agents) • Symptomatic viral, bacterial, or fungal infection within 6 weeks of eligibility evaluation or active infection (included fever of unknown origin) • Neoplasia (except local skin cancer or cervical intraepithelial neoplasia) or history of familial” cancer • Myelodysplasia or other serious hematological disorder than thalassemia • History of uncontrolled seizures • Other systemic disease judged non compatible with the procedure • Positivity for HIV and/or HCV RNA and/or HbsAg and/or HBV DNA • Active alcohol or substance abuse within 6 months of the study • Pregnancy or lactation.

发起方

FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR