Clinical Trial Phase I / II to evaluate the safety and efficacy of the infusion of cells transduced with a therapeutic lentiviral vector for patients with Fanconi Anemia Subtype A.

Conditions: Fanconi anemia (Subtype A)
MedDRA version: 15.1Level: LLTClassification code 10055206Term: Fanconi's anemiaSystem Organ Class: 100000004850
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2011-006100-12-ES

Lead Sponsor: FUNDACION PARA LA INVESTIGACION BIOMEDICA DEL HOSPITAL UNIVERSITARIO NIÑO JESUS

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 5

Inclusion Criteria

- 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

Exclusion Criteria

- 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).