Clinical Research Directory

Inclusion Criteria - Transplant Recipient * Age ≥2 months old at the time of chemotherapy administration * A proven mutation as defined by direct sequencing of patient DNA * Has a suitable matched sibling donor or matched unrelated donor (8/8) or single haplotype matched (≥3 of 6) family member donor * Patient must fulfill pre-transplant evaluation: * Left ventricular ejection fraction \>40% and no evidence of uncorrected congenital malformation with clinical symptomatology * Creatinine clearance (CrCl) or glomerular filtration rate (GFR) ≥ 50 ml/min/1.73m2 or serum Creatinine ≤1.2mg/dL * Resting pulse oximetry ≥90% on room or ≥95% on oxygen supplementation * Lansky (age-dependent) performance score ≥50 * Bilirubin ≤3 times the upper limit of normal for age * Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≤ 5 times the upper limit of normal for age Exclusion Criteria - Transplant Recipient * Positive for HIV infection by genome PCR * Presence of active malignancy * A social situation indicating that the family may not be able to comply with protocol procedures and recommended medical care * Presence of a medical condition indicating that survival will be dismal such as the requirement for mechanical ventilation, severe failure of a major organ system, or evidence of a serious, progressive infection that is refractory to medical therapy Inclusion Criteria - Matched Sibling Donor and Haplocompatible Donor * Fully matched sibling donor (8/8), or matched unrelated donor (8/8), or at least single haplotype matched (≥3 of 6) family member * At least 1 year old (MSD) and at least 18 years of age (Haplocompatible) * HIV negative * Not pregnant as confirmed by negative serum or urine pregnancy test within 14 days prior to enrollment (if female) * Not breast feeding * Regarding donation eligibility, is identified as either: * Completed the process of donor eligibility determination as outlined in 21 CFR 1271 and agency guidance; OR * Does not meet 21 CFR 1271 eligibility requirements, but has a declaration of urgent medical need completed by the principal investigator or physician sub-investigator per 21 CFR 1271