Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A

• Status: Active, not recruiting
• Trial end date: 2022-04-01
• Target enrollment: 0
• Participant gender: All

Summary

This is an open, Phase I / II clinical trial to evaluate the safety and efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene for patients with Fanconi Anemia of Subtype A .

CD34 + cells derived from bone marrow and / or mobilized peripheral blood (fresh and / or cryopreserved) from patients with Fanconi subtype A (FA-A), will be transduced ex vivo with a lentiviral vector carrying the gene FANCA (orphan drug) . After transduction the cells will be inoculated in patients in order to restore their hematopoiesis with genetically corrected stem cells.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Hospital Infantil Universitario Niño Jesús, Madrid, Spain
• Collaborators: Centro de Investigación en Red de Enfermedades Raras (CIBERER); Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT); Hospital Vall d'Hebron; Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz; Universitat Autonoma de Barcelona
• Treatments: JM 3100; Lenograstim; Plerixafor; Sargramostim

Criteria

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.