Donor Stem Cell Transplant With Treosulfan Fludarabine and Thiotepa in Treating Patients With Non-malignant Disorders

  • End date
    Jul 1, 2027
  • participants needed
  • sponsor
    Fred Hutchinson Cancer Research Center
Updated on 5 August 2021
bone marrow procedure
primary cancer


This phase II trial studies how well donor stem cell transplant, treosulfan, fludarabine, and thiotepa work in treating patients with non-cancerous disorders. Giving chemotherapy before a donor transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient's immune cells and help destroy any remaining cancer cells.



Patients receive thiotepa intravenously (IV) twice daily (BID) on day -7, treosulfan IV on days -6 to -4, fludarabine phosphate IV on days -6 to -2, and rabbit anti-thymocyte globulin IV on days -4 to -2. Patients then undergo allogeneic hematopoietic cell transplant via infusion on day 0.

After completion of study treatment, patients are followed up at 1 year and then annually thereafter.

Condition Cancer, Cancer/Tumors, Ewing's Family Tumors, Cancer (Pediatric), Neoplasms, Non-Neoplastic Hematologic and Lymphocytic Disorder, Non-Neoplastic Hematologic and Lymphocytic Disorder, Non-Neoplastic Hematologic and Lymphocytic Disorder, Non-Neoplastic Hematologic and Lymphocytic Disorder, Non-Neoplastic Hematologic and Lymphocytic Disorder, Non-Neoplastic Hematologic and Lymphocytic Disorder
Treatment fludarabine phosphate, allogeneic hematopoietic stem cell transplantation, treosulfan, thiotepa, Rabbit Anti-thymocyte Globulin
Clinical Study IdentifierNCT03980769
SponsorFred Hutchinson Cancer Research Center
Last Modified on5 August 2021


Yes No Not Sure

Inclusion Criteria

Patient with nonmalignant disease treatable by allogenic HCT
Patient with a nonmalignant disease that is not clearly defined (a patient with a non-malignant disease HCT for whom a genetic mutation responsible for their non-malignant disease phenotype has not been identified) are eligible for the study following discussion with and approval by the protocol principal investigator (PI) (Dr. Lauri Burroughs)
DONOR: For the very few occasions where we identify a donor hematopoietic progenitor cell (HPC)-A from a non-NMDP source, we have procedures in place through our unrelated donor office to collect the information necessary to comply with donor testing, screening, and declaration of donor eligibility according to 21 Code of Federal Regulations (CFR) 1271. We require that the donor testing be performed by a United States Clinical Laboratory Improvement Amendment (CLIA) approved laboratory. In the very rare case where the donor testing is not able to be performed in a CLIA-approved laboratory, or there is confirmatory testing that needs to be performed, or for any donor identified from Europe and at risk for Creutzfeldt-Jakob disease (CJD), we note this on the donor screening form and require that the unrelated donor medical director or the attending physician approves the use of the donor HPC-A product under urgent medical need
DONOR: Human leukocyte antigen (HLA)-identical related donor OR unrelated donor matched for HLA-A, B, C, DRB1 and DQB1 or mismatched for a single allele at HLA-A, B, C, or a single DQB1 antigen or allele mismatch by high resolution deoxyribonucleic acid (DNA) typing
DONOR: Bone marrow is the preferred cell source (when feasible). However, peripheral blood stem cells (PBSC) is also allowed and the PI may determine if PBSC is preferred for certain patients
The recommended total nucleated cell count (TNC) for bone marrow grafts is >= 4.0 x 10^8 TNC/kg (actual recipient weight)
The recommended CD34 cell count for PBSC grafts is >= 5 x 10^6 CD34/kg (actual recipient weight) and the recommended maximum CD34 cell count for PBSC grafts is 10 x 10^6 CD34/kg (actual recipient weight)
DONOR: HLA-matched sibling bone marrow in combination with HLA-matched sibling umbilical cord blood if the HLA-matched sibling umbilical cord blood was collected and stored. The HLA-matched sibling bone marrow and cord blood would be matched for HLA-A, B, C, DRB1 and DQB1

Exclusion Criteria

Patients with idiopathic aplastic anemia and Fanconi anemia; patients with aplastic anemia associated with paroxysmal nocturnal hemoglobinuria (PNH) or inherited marrow failure syndromes (except Fanconi anemia) will be allowed
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 of < 26% may be enrolled if approved by a cardiologist
Impaired pulmonary function as evidenced by carbon monoxide diffusing capability (DLCO) < 50% of predicted (or, if unable to perform pulmonary function tests, then oxygen [O2] saturation < 92% on room air)
Impaired renal function as evidenced by
Estimated creatinine clearance < 60 mL/min/1.73m^2 using either the Chronic Kidney Disease Epidemiology Collaboration (CKD EPI) equation for adult patients (> 18 years old), or the updated Schwartz formula for pediatric patients (< 18 years old). If the estimated creatinine clearance is < 60 mL/min/1.73m^2, then renal function must be measured by 24-hour creatinine clearance, Iothalamate, Iohexol or nuclear GFR and the patient is excluded if their measured creatinine clearance is < 50 mL/min/1.73 m^2, OR
Serum creatinine > 2 x upper limit of normal, OR
Dialysis dependent
Evidence of synthetic dysfunction or severe cirrhosis requiring deferral of conditioning as recommended by a gastroenterology specialist
Active infectious disease requiring deferral of conditioning as recommended by an infectious disease specialist
Positive for HIV (human immunodeficiency virus)
Females who are pregnant or breast-feeding
Known hypersensitivity to treosulfan, fludarabine, and/or thiotepa
DONOR: Donors deemed unable to undergo marrow harvesting of PBSC mobilization and leukapheresis
DONOR: HIV-positive donors
DONOR: Donors with active infectious hepatitis
DONOR: Female donor with positive pregnancy test
DONOR: Donors are excluded if the patient has an identified antibody against a donor-specific HLA locus as specified in standard practice
DONOR: HLA-matched sibling cord blood units that have not passed donor screening for infectious disease markers as recommended by the National Marrow Donor Project (NMDP) will not be used unless a waiver is signed by the clinical attending allowing use of cord blood unit. Cord blood units are presumed to be cytomegalovirus (CMV) negative regardless of serologic testing due to passive transmission of maternal CMV antibodies
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