This study looks at giving genetically engineered blood stem cells (a gene therapy) intravenously to patients diagnosed with Fanconi anemia complementation group A (FA-A). It generally enrolled patients aged 1 to 21 years who did not have a matched family donor. It was carried out in Phase 1/Phase 2 and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Patients diagnosed with Fanconi Anemia complementation group A (FA-A) * Minimum age 1 year * Maximum age 21 years * Lansky Index\> 60%. * Informed consent in accordance with current legal regulations. * Number of cells to be transduced: At least 3x10\^5 purified CD34+ / kg body weight. * Negative result in the urine pregnancy test at the baseline visit for women of childbearing age, who should be committed to using an effective contraceptive method during the period of study participation. Exclusion Criteria: * Patients with an human leukocyte antigen (HLA) identical family donor. * Evidence of myelodysplastic syndrome or leukemia, or cytogenetic abnormalities predicting the same in bone marrow aspirates. In this case, the studies carried out two months in advance of the patient's entry into the clinical trial will be considered valid. * Evidence that the patient to be infused has signs of somatic mosaicism, with hematologic improvement. * Any illness or concomitant process that in the opinion of the investigator incapacitates the subject for their participation in the study. * Pre-existing sensory or motor impairment\> = grade 2 according to the National Cancer Institute (NCl) criteria. * Pregnant or lactating women.