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Complete title: Allogeneic Hematopoietic Cell Transplantation for Patients with Nonmalignant Inherited Disorders Using a Treosulfan Based Preparative Regimen
|Research Study Number||2256.00|
|Principal Investigator||Lauri Burroughs, MD|
Research Study Description
Eligibility Criteria (must meet the following to participate in this study)
Genders Eligible for Study: Both
- Patients with a nonmalignant disease treatable by allogeneic HCT
- Patients with a known nonmalignant disease that is not clearly defined will need to be discussed with the protocol principal investigator (PI) (Dr. Lauri Burroughs) and potentially the nonmalignant board to determine if they are eligible for HCT on this study
- DONOR: Human leukocyte antigens (HLA)-identical related donors or unrelated donors matched for HLA-A, B, C, DRB1, and DQB1 or mismatched for a single allele at HLA-A, B, C, DRB1 or a single DQB1 antigen or allele mismatch by high resolution deoxyribonucleic acid (DNA) typing
- DONOR: Bone marrow is the preferred cell source; PBSC are allowed if donor refuses or is unable to give marrow
- DONOR: Umbilical Cord Blood: Unit selection is based on the cryopreserved total nucleated cell (TNC) dose and matching at HLA-A, B antigen level and DRB1 allele level typing; while HLA-C antigen/allele level typing is not considered in the matching criteria, if available, may be used to optimize unit selection
- DONOR: Umbilical Cord Blood: The patient and the cord blood unit(s) must be matched for at least 4 of 6 loci as defined above
- DONOR: Umbilical Cord Blood: Selection of two UCB units is allowed to provide sufficient cell dose
Other eligibility criteria may apply.
Exclusions (conditions that would prevent participation in this study)
- Patients with impaired cardiac function as evidenced by ejection fraction < 35% (or, if unable to obtain ejection fraction, shortening fraction of < 26%) or cardiac insufficiency requiring treatment or symptomatic coronary artery disease; patients with a shortening fraction < 26% may be enrolled if approved by a cardiologist
- Patients with impaired pulmonary function as evidenced by diffusion capacity of the lung for carbon monoxide (DLCO) < 50% of predicted (or, if unable to perform pulmonary function tests, then oxygen [O2] saturation < 92% on room air)
- Patients with impaired renal function as evidenced by creatinine-clearance < 50% for age, weight, height or serum creatinine > 2X upper normal limit or dialysis-dependent
- Patients with evidence of synthetic dysfunction or severe cirrhosis requiring deferral of conditioning as recommended by a gastroenterology specialist
- Patients with an active infectious disease requiring deferral of conditioning; as recommended by an infectious disease specialist
- Patients who are seropositive for human immunodeficiency virus (HIV)
- Females who are pregnant or breast-feeding
- Patients with a known hypersensitivity to treosulfan and/or fludarabine
- Receiving another experimental drug within 4 weeks of initiation of conditioning (day -6)
- DONOR: Deemed unable to undergo marrow harvesting or PBSC mobilization and leukapheresis
- DONOR: HIV-positive
- DONOR: With active infectious hepatitis
- DONOR: Females with a positive pregnancy test
Other exclusion criteria may apply.
Bone Marrow and Hematopoietic Stem Cell Transplant (BMT and HSCT); Diamond Blackfan Anemia; Immunodeficiency Syndromes; Sickle Cell Anemia; Umbilical Cord Blood Transplant (UCBT); Non-malignant Condition
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