Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab

Eligibility Criteria

• 1. 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
• 2. 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)
• 3. Age of ≥2 years
• 4. Consenting ≥5/10 HLA-matched related or unrelated donor available for apheresis
• 5. Organ function defined as:
• 1. Serum Creatinine \<2.0 mg/dL and corrected creatinine clearance/cystatin cL \>60 mL/min/1.73m\^2 without dialysis
• 2. Forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), and diffusing capacity of the lung for carbon monoxide (DLCO) corrected for hemoglobin and volume, \>50% predicted by pulmonary function tests (PFTs)
• 3. For patients unable to cooperate for PFTs, criteria are no evidence of dyspnea at rest, no exercise intolerance, and no requirement for supplemental oxygen with spO2 \>93%
• 4. Shortening fraction of ≥29% or ejection fraction of ≥45% by echocardiogram
• 5. Serum total bilirubin of \<4 x ULN
• 6. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \< 5 x ULN
• 7. Prothrombin time international normalized ratio (PT INR) and partial thromboplastin time (PTT) \<1.5 x ULN
• 6. Life expectancy of at least 2 years
• 7. Patients of childbearing potential must be willing to use an effective contraceptive method for the duration of the peri-transplant conditioning through hematopoietic recovery
• 8. Patients and/or parents or legal guardians must be able to provide written informed consent and authorize use and disclosure of personal health information in accordance with Health Insurance Portability and Accountability Act
• 1. Patients with available and consenting 10/10 HLA-identical sibling donor for apheresis
• 2. Patients with any acute or uncontrolled infections at the time of enrollment, including bacterial, fungal or viral
• 3. Patients who are seropositive for HIV-I/II or HTLV-I/II.
• 4. Patients receiving any other investigational agents or other biological, chemotherapy, or radiation therapy within 14 days of enrollment
• 5. Patients with any active malignancies, myelodysplastic syndrome or other concerns for high-risk bone marrow disease
• 6. Patients who received androgens in last 3 months
• 7. Pregnant or lactating women
• 8. Women who are nursing and do not wish to discontinue breastfeeding
• 9. Lansky/Karnofsky performance score \<50%.
• 10. Any other medical condition or history that, in the opinion of the Principal Investigator, could pose a significant safety risk to the participant or jeopardize the integrity of the study
• 11. Patients who, in the opinion of the Principal Investigator, may not be able to comply with the safety monitoring requirements of the study