Clinical Trial Phase I / II to evaluate the safety and efficacy of the infusion of autologous CD34+ cells transduced with a lentiviral vector carrying the FANCA gene (orphan drug) for patients with Fanconi Anemia Subtype A. - Fancolen-1

FUNDACION PARA LA INVESTIGACION BIOMEDICA DEL HOSPITAL UNIVERSITARIO NIÑO JESUS0 个研究点目标入组 5 人开始时间: 2012年1月27日最近更新: 2013年5月13日

试验速览

暂无简介。

•- Patients diagnosed with the Fanconi anemia complementation group-A
•- At least one of the following parameters must be less than the indicated values?: hemoglobin 8.0 g / dL, neutrophil count: 750/mm3, platelets: 30.000/mm3
•- Age> 1 year
•- Lansky Index> 60%
•- Mild functional impairment of organs
•- To provide informed consent in accordance with current legislation
•- Number of transduced CD34+ cells: At least 3x100000 purified CD34 + / kg body weight
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 5
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 5
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 5

•- Patients with HLA-identical family donor.
•- Evidence of leukemia or myelodysplastic syndrome, or cytogenetic abnormalities in bone marrow aspirates predictive of those. In this case studies performed two months before the entry of the patient in the clinical trial will be considered as valid
•- Evidence that the patient has signs of somatic mosaicism associated to hematologic improvement.
•- Any concomitant disease or condition in the investigator's opinion incapacitate the subject for their participation in the study.
•- Effect on pre-existing sensory or motor> = grade 2 according to the criteria of the National Cancer Institute (NCI).

发起方

FUNDACION PARA LA INVESTIGACION BIOMEDICA DEL HOSPITAL UNIVERSITARIO NIÑO JESUS