The objective of this clinical trial is to develop a cell therapy for Fanconi Anemia which enables enhanced donor hematopoietic and immune reconstitution with decreased toxicity by transplanting depleted stem cells from a donor with and without using an experimental antibody treatment called JSP-191 as a part of conditioning. This experimental treatment will hopefully cause fewer side effects than chemotherapy (the current standard of care method).
Participants will be administered the conditioning regimen, are assessed until they receive the depleted stem cell infusion, and will be followed for up to 2 years after the cell infusion.
Fanconi Anemia diagnosis as demonstrated by abnormal chromosome breakage studies with increased sensitivity to mitomycin-C (MMC) or diepoxybutane (DEB) and at least one mutation in a known Fanconi-associated gene
Bone marrow failure (defined by reduction in at least one cell line on two separate occasions at least one month apart (e.g., platelet count of <100,000 per cubic millimeter, hemoglobin <9 gm/dl and/or absolute neutrophil count (ANC) of <1000/mm)
Age of ≥2 years
Consenting ≥5/10 HLA-matched related or unrelated donor available for apheresis
Patients with available and consenting 10/10 HLA-identical sibling donor for apheresis
Patients with any acute or uncontrolled infections at the time of enrollment, including bacterial, fungal or viral
Patients who are seropositive for HIV-I/II or HTLV-I/II.
Patients receiving any other investigational agents or other biological, chemotherapy, or radiation therapy within 14 days of enrollment
Porteus, Matthew, MD
Lead sponsor
Stanford University
Sponsor institution