Pediatric Fanconi Anemia Subtype A - Healing Genes

Pediatric Fanconi Anemia Subtype A

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A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A

A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects With Fanconi Anemia Subtype A

Phase 1


Doctors at Stanford University Institute for Stem Cell Biology and Regenerative Medicine Lucille Packard Children’s Hospital (CA) are recruiting pediatric participants with Fanconi Anemia Subtype A for a study on how well a gene-modified immunotherapy correct the FANCA gene mutation that causes Fanconi anemia. The treatment consists of a self-transplant of the patient’s own stem cells, after they have been drawn and gene-modified in a lab setting to impart the correct gene, then multiplied in number. The goal is to have the stem cells populate the bone marrow and compensate for their genetic mutation.

The study will require leukapheresis (drawing of the stem cells) and, several days after they have been gene-modified, infusion of the  stem cells. Follow up will continue for 3 years and consist of lab and physical assessments.


  • Be 1 to 12 years of age
  • Be diagnosed with Fanconi anemia, as diagnosed by chromosomal fragility assay per the study’s lab criteria
  • Patient must meet not have an available and medically eligible human leukocyte antigen (HLA)-identical sibling donor.


  1. Prescreening tests to confirm eligibility of the patient to participate.
  2. White blood cells will be drawn and over several weeks, the patient will receive no treatment while the cells are gene-modified in a lab.
  3. The investigatory treatment is infused once.
  4. Follow up will continue for at least 3 years.


The study site is at Stanford University Institute for Stem Cell Biology and Regenerative Medicine of Lucille Packard Children’s Hospital, Stanford, California. Map.


Contact: Sandeep Soni, MD  |  1-650-725-9250  |  [email protected]

Contact: Agnieszka Czechowicz, MD, PhD  |  1-650-497-2218  |  [email protected]



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