Busulfan, Fludarabine Phosphate, and Post-Transplant Cyclophosphamide in Treating Patients With Blood Cancer Undergoing Donor Stem Cell Transplant
NCT ID: NCT02861417
Last Updated: 2025-08-08
Study Results
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Basic Information
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ACTIVE_NOT_RECRUITING
PHASE2
204 participants
INTERVENTIONAL
2016-08-05
2026-08-31
Brief Summary
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Detailed Description
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I. To assess safety of timed sequential busulfan and fludarabine conditioning regimen and post transplant cyclophosphamide as determined by day 100 non-relapse mortality in patients undergoing allogeneic transplantation: from matched donors; from mismatched (haploidentical) donors.
SECONDARY OBJECTIVE:
I. To evaluate efficacy of this therapy and to compare outcomes between recipients of matched and mismatched donors by studying the following endpoints: graft versus host disease (GVHD)-free/relapse free survival; relapse-free survival; overall survival; non-relapse mortality; relapse rate; time to neutrophil and platelet engraftment; incidence of acute and chronic GVHD; grade 3 and 4 adverse events.
TERTIARY OBJECTIVE:
I. To study impact of timed sequential busulfan therapy and post-transplant cyclophosphamide on immune reconstitution and cytokines levels post-transplant.
OUTLINE: Patients are assigned to 1 of 4 groups.
GROUP I (FROM HAPLOIDENTICAL DONOR): Patients receive busulfan intravenously (IV) over 3 hours on days -13, -12, and -6 to -3, thiotepa IV over 4 hours on day -7, fludarabine phosphate IV over 1 hour on days -6 to -3. Patients undergo stem cell transplantation IV on day 0. Patients then receive cyclophosphamide IV over 3 hours on days 3 and 4. Beginning on day 5, patients receive tacrolimus IV continuously or orally (PO) twice daily (BID) for up to 3 months and mycophenolate mofetil PO thrice daily (TID).
GROUP II (FROM MATCHED DONOR): Patients receive busulfan IV over 3 hours on days -13, -12, and -6 to -3, fludarabine phosphate IV over 1 hour on days -6 to -3. Patients undergo stem cell transplantation IV on day 0. Patients then receive cyclophosphamide IV over 3 hours on days 3 and 4. Beginning on day 5, patients receive tacrolimus IV continuously or PO BID for up to 3 months.
GROUP III and GROUP IV: Patients receiving haploidentical related donor transplant, diagnosis of myelofibrosis, \> 60 years old, or patients with comorbidity scores \> 3 will go in Group 3 or 4. If patients with comorbidity score \> 3, then the principal investigator is the final arbiter of eligibility for comorbidity score \> 3. Busulfan is administered at the dose calculated to achieve a total (including first two doses delivered on day -20 and day -13) system exposure of 20,000 +/- 12% uMol-min based on the pharmacokinetic studies.
GROUP V and GROUP VI: Patients receive busulfan IV over 3 hours on days -20, -13, and -6 to -3, a lower dose of thiotepa IV over 4 hours on day -7, fludarabine phosphate IV over 1 hour on days -6 to -3. Patients undergo stem cell transplantation IV on day 0. Patients then receive cyclophosphamide IV over 3 hours on days 3 and 4. Beginning on day 5, patients receive tacrolimus IV continuously or PO BID for up to 3 months and mycophenolate mofetil PO TID.
Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Group I (haploidentical donor transplant, chemotherapy)
Patients receive busulfan IV over 3 hours on days -13, -12, and -6 to -3, thiotepa IV over 4 hours on day -7, fludarabine phosphate IV over 1 hour on days -6 to -3. Patients undergo stem cell transplantation IV on day 0. Patients then receive cyclophosphamide IV over 3 hours on days 3 and 4. Beginning on day 5, patients receive tacrolimus IV continuously or PO BID for up to 3 months and mycophenolate mofetil PO TID.
Allogeneic Hematopoietic Stem Cell Transplantation
Undergo stem cell transplantation
Busulfan
Given IV
Cyclophosphamide
Given IV
Fludarabine
Given IV
Fludarabine Phosphate
Given IV
Laboratory Biomarker Analysis
Correlative studies
Mycophenolate Mofetil
Given PO
Pharmacological Study
Correlative studies
Tacrolimus
Given IV or PO
Thiotepa
Given IV
Group II (matched donor transplant, chemotherapy)
Patients receive busulfan IV over 3 hours on days -13, -12, and -6 to -3, fludarabine phosphate IV over 1 hour on days -6 to -3. Patients undergo stem cell transplantation IV on day 0. Patients then receive cyclophosphamide IV over 3 hours on days 3 and 4. Beginning on day 5, patients receive tacrolimus IV continuously or PO BID for up to 3 months.
Allogeneic Hematopoietic Stem Cell Transplantation
Undergo stem cell transplantation
Busulfan
Given IV
Cyclophosphamide
Given IV
Fludarabine
Given IV
Fludarabine Phosphate
Given IV
Laboratory Biomarker Analysis
Correlative studies
Pharmacological Study
Correlative studies
Tacrolimus
Given IV or PO
Group III (haploidentical donor transplant, chemotherapy)
Patients receiving haploidentical related donor transplant, diagnosis of myelofibrosis, \> 60 years old, or patients with comorbidity scores \> 3 will go in Group 3 or 4. If patients with comorbidity score \> 3, then the principal investigator is the final arbiter of eligibility for comorbidity score \> 3. Busulfan is administered at the dose calculated to achieve a total (including first two doses delivered on day -20 and day -13) system exposure of 20,000 +/- 12% uMol-min based on the pharmacokinetic studies.
Allogeneic Hematopoietic Stem Cell Transplantation
Undergo stem cell transplantation
Busulfan
Given IV
Cyclophosphamide
Given IV
Fludarabine
Given IV
Fludarabine Phosphate
Given IV
Laboratory Biomarker Analysis
Correlative studies
Mycophenolate Mofetil
Given PO
Pharmacological Study
Correlative studies
Tacrolimus
Given IV or PO
Thiotepa
Given IV
Group IV (matched donor transplant, chemotherapy)
Patients receiving haploidentical related donor transplant, diagnosis of myelofibrosis, \> 60 years old, or patients with comorbidity scores \> 3 will go in Group 3 or 4. If patients with comorbidity score \>3, then the principal investigator is the final arbiter of eligibility for comorbidity score \> 3. Busulfan is administered at the dose calculated to achieve a total (including first two doses delivered on day -20 and day -13) system exposure of 20,000 +/- 12% uMol-min based on the pharmacokinetic studies.
Allogeneic Hematopoietic Stem Cell Transplantation
Undergo stem cell transplantation
Busulfan
Given IV
Cyclophosphamide
Given IV
Fludarabine
Given IV
Fludarabine Phosphate
Given IV
Laboratory Biomarker Analysis
Correlative studies
Pharmacological Study
Correlative studies
Tacrolimus
Given IV or PO
Group V (haploidentical donor transplant, chemotherapy)
Patients receive busulfan IV over 3 hours on days -20, -13, and -6 to -3, a lower dose of thiotepa IV over 4 hours on day -7, fludarabine phosphate IV over 1 hour on days -6 to -3. Patients undergo stem cell transplantation IV on day 0. Patients then receive cyclophosphamide IV over 3 hours on days 3 and 4. Beginning on day 5, patients receive tacrolimus IV continuously or PO BID for up to 3 months and mycophenolate mofetil PO TID.
Allogeneic Hematopoietic Stem Cell Transplantation
Undergo stem cell transplantation
Busulfan
Given IV
Cyclophosphamide
Given IV
Fludarabine
Given IV
Fludarabine Phosphate
Given IV
Laboratory Biomarker Analysis
Correlative studies
Mycophenolate Mofetil
Given PO
Pharmacological Study
Correlative studies
Tacrolimus
Given IV or PO
Thiotepa
Given IV
Group VI (matched or haploidentical transplant, chemotherapy)
Patients receiving fully matched or haploidentical donor transplant receive busulfan IV over 3 hours on days -20, -13, and -6 to -3, a lower dose of thiotepa IV over 4 hours on day -7, fludarabine phosphate IV over 1 hour on days -6 to -3. Patients undergo stem cell transplantation IV on day 0. Patients then receive cyclophosphamide IV over 3 hours on days 3 and 4. Beginning on day 5, patients receive tacrolimus IV continuously or PO BID for up to 3 months and mycophenolate mofetil PO TID.
Allogeneic Hematopoietic Stem Cell Transplantation
Undergo stem cell transplantation
Busulfan
Given IV
Cyclophosphamide
Given IV
Fludarabine
Given IV
Fludarabine Phosphate
Given IV
Laboratory Biomarker Analysis
Correlative studies
Mycophenolate Mofetil
Given PO
Pharmacological Study
Correlative studies
Tacrolimus
Given IV or PO
Thiotepa
Given IV
Interventions
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Allogeneic Hematopoietic Stem Cell Transplantation
Undergo stem cell transplantation
Busulfan
Given IV
Cyclophosphamide
Given IV
Fludarabine
Given IV
Fludarabine Phosphate
Given IV
Laboratory Biomarker Analysis
Correlative studies
Mycophenolate Mofetil
Given PO
Pharmacological Study
Correlative studies
Tacrolimus
Given IV or PO
Thiotepa
Given IV
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Patients must have a haploidentical related donor or a fully matched related or unrelated donor
* Performance score of \>= 70 by Karnofsky/Lansky or performance score (PS) 0 to 1 (Eastern Cooperative Oncology Group \[ECOG\] =\< 1)
* Left ventricular ejection fraction \>= 50%
* Adequate pulmonary function with forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC) and diffusion capacity of the lung for carbon monoxide (DLCO) \>= 50% of expected corrected for hemoglobin and/or volume; children unable to perform pulmonary function tests (e.g., less than 7 years old) pulse oximetry of \>= 92% on room air
* Creatinine clearance (calculated creatinine clearance by Cockcroft-Gault using adjusted body weight if actual body weight is 20% greater than ideal is permitted) should be \> 50 ml/min
* Bilirubin =\< 2 x the upper limit of normal (except with patients high indirect bilirubin due to Gilbert's syndrome, hypersplenism, or hemolysis)
* Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) \< 200
* Negative beta human chorionic gonadotropin (HCG) test in a woman with child bearing potential, defined as not post-menopausal for 12 months or no previous surgical sterilization; women of child bearing potential must be willing to use an effective contraceptive measure while on study
* Patient or patient's legal representative able to sign informed consent
Exclusion Criteria
* Uncontrolled infections
* Patients with comorbidity score \> 3; the principal investigator is the final arbiter of eligibility for comorbidity score \> 3
* Prior allogeneic transplant
* Patients with active hepatitis B and C
* Patients with prior coronary artery disease
* Patients who received inotuzumab and/or gemtuzumab in the past
12 Years
75 Years
ALL
No
Sponsors
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National Cancer Institute (NCI)
NIH
M.D. Anderson Cancer Center
OTHER
Responsible Party
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Principal Investigators
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Uday R Popat, MD
Role: PRINCIPAL_INVESTIGATOR
M.D. Anderson Cancer Center
Locations
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M D Anderson Cancer Center
Houston, Texas, United States
Countries
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References
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Popat UR, Pasvolsky O, Bassett R Jr, Mehta RS, Olson A, Chen J, Alousi AM, Al-Atrash G, Bashir Q, Gulbis AM, Hosing CM, Im JS, Kebriaei P, Khouri I, Marin D, Nieto Y, Oran B, Saini N, Shigle TL, Srour SA, Ramdial JL, Rezvani K, Qazilbash MH, Andersson BS, Champlin RE, Shpall EJ. Myeloablative fractionated busulfan for allogeneic stem cell transplant in older patients or patients with comorbidities. Blood Adv. 2023 Oct 24;7(20):6196-6205. doi: 10.1182/bloodadvances.2023010850.
Related Links
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MD Anderson Cancer Center
Other Identifiers
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NCI-2017-00614
Identifier Type: REGISTRY
Identifier Source: secondary_id
2016-0137
Identifier Type: OTHER
Identifier Source: secondary_id
2016-0137
Identifier Type: -
Identifier Source: org_study_id
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