Phase II clinical trial to evaluate safety and efficacy of mobilisation and collection of CD34+ cells after treatment with plerixafor and filgrastim in patients with Fanconi anaemia for subsequent transduction with a lentiviral vector carrying the FANCA gene and reinfusion into the patient - Fancostem-Plerixafor & Filgrastim mobilisation in Fanconi Anaemia

Great Ormond Street Hospital NHS foundation Trust0 个研究点目标入组 20 人开始时间: 2015年7月15日最近更新: 2019年2月28日

试验速览

暂无简介。

•Inclusion criteria
•Patients diagnosed with FA confirmed by chromosomal instability test with diepoxybutane or mitomycin C.
•Age 1-15 years
•At least one of the following parameters must exceed the values indicated: haemoglobin: 8.0 g/dL; neutrophils: 750/mm3; platelets: 30.000/mm3.
•Lansky index > 60%.
•Left ventricular ejection fraction > 50%.
•Provide informed consent in accordance with current legislation. Parent/Guardian consent and assent where appropriate
•Women of childbearing potential must have a negative pregnancy test in serum or urine at the screening visit, and accept the use of adequate contraception method from, at least, the 14 days prior to the first dose of mobilisation treatment until 14 days after the last.(N/A for children)
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 5
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range 0
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range 0

•Exclusion criteria
•Evidence of myelodysplastic syndrome or leukaemia, or cytogenetic abnormalities predictive of these conditions in bone marrow aspirate analysis. This assessment should be made by valid studies two months before the initial assessment.
•Patients with active infectious process or other serious underlying medical condition including malignancy.
•Severe (= grade 3) functional organ impairment (liver, kidney, respiratory) according to the criteria of the National Cancer Institute (NCI CTCAE v4.03).
•Previously received haematopoietic transplantation.
•Any concomitant disease or condition that, in the opinion of the investigator, deems the subject unfit to participate in the study.
•Patients who, after a psychosocial assessment, are censored as unfit for the procedure.
•Patients who have received blood transfusions in the previous three months.
•Pregnant or breastfeeding women (N/A for children)

发起方

Great Ormond Street Hospital NHS foundation Trust