Acute Lymphoblastic LeukemiaPhase 2OPTIMIZENCT06001385
HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation With Reduced Dose Post Transplantation Cyclophosphamide GvHD Prophylaxis
A Phase 2 study testing Busulfan, Fludarabine and PBSC Hematopoietic Stem Cell Transplantation (HSCT) in people with Acute Lymphoblastic Leukemia and Acute Myeloid Leukemia, run by Center for International Blood and Marrow Transplant Research at 32 sites in United States.
Trial summary
The goal of this clinical trial is to determine the effectiveness of Reduced Dose Post-Transplant Cyclophosphamide (PTCy) in patients with hematologic malignancies after receiving an HLA-Mismatched Unrelated Donor (MMUD) . Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Leukemia
- What is being tested?
- Busulfan, Fludarabine and PBSC Hematopoietic Stem Cell Transplantation (HSCT)
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 313 participants
- Where
- 32 sitesUnited States
- Study dates
- 8 Dec 2023to 30 Dec 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Stratum 1 Recipient Inclusion Criteria:
- Age ≥ 18 years and < 66 years (chemotherapy-based conditioning) or < 61 years (total body irradiation [TBI]-based conditioning) at the time of signing informed consent
- Patient or legally authorized representative has the ability to provide informed consent according to the applicable regulatory and institutional requirements.
- Stated willingness to comply with all study procedures and availability for the duration of the study.
- Planned MAC regimen as defined per study protocol
- Available partially HLA-MMUD (4/8-7/8 at HLA-A, -B, -C, and -DRB1 is required) with age ≥ 18 and ≤ 40 years (≤ 35 preferred).
- Product planned for infusion is MMUD T-cell replete PBSC allograft
- HCT-CI < 5. The presence of prior malignancy will not be used to calculate HCT-CI for this trial to allow for the inclusion of patients with secondary or therapy-related AML or MDS.
- One of the following diagnoses:
- Acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), or other acute leukemia in 1st remission or beyond with ≤ 5% marrow blasts and no circulating blasts or evidence of extra-medullary disease. Documentation of bone marrow assessment will be accepted within 45 days prior to the anticipated start of conditioning.
- Patients with MDS with no circulating blasts and with < 10% blasts in the bone marrow (higher blast percentage allowed in MDS due to lack of differences in outcomes with < 5% or 5-10% blasts in MDS). Documentation of bone marrow assessment will be accepted within 45 days prior to the anticipated start of conditioning.
- Cardiac function: Left ventricular ejection fraction ≥ 45% based on most recent echocardiogram or multi-gated acquisition scan (MUGA) results.
- Estimated creatinine clearance ≥ 45mL/min calculated by equation.
- Pulmonary function: diffusing capacity of the lungs for carbon monoxide (DLCO) corrected for hemoglobin > 50% and forced expiratory volume in first second (FEV1) predicted > 50% based on most recent pulmonary function test (PFT) results
- Liver function acceptable per local institutional guidelines
- KPS of ≥ 70%
Stratum 2 Recipient Inclusion Criteria:
- Age ≥18 years at the time of signing informed consent
- Patient or legally authorized representative has the ability to provide informed consent according to the applicable regulatory and local institutional requirements.
- Stated willingness to comply with all study procedures and availability for the duration of the study.
- Planned NMA/RIC regimen per study protocol
- Available partially HLA-MMUD (4/8-7/8 at HLA-A, -B, -C, and -DRB1 is required) with age ≥ 18 and ≤ 40 years (≤ 35 preferred).
- Product planned for infusion is MMUD T-cell replete PBSC allograft
- One of the following diagnoses:
- Patients with acute leukemia or chronic myeloid leukemia (CML) with no circulating blasts, no evidence of extramedullary disease, and with < 5% blasts in the bone marrow. Documentation of bone marrow assessment will be accepted within 45 days prior to the anticipated start of conditioning.
- Patients with MDS with no circulating blasts and with < 10% blasts in the bone marrow (higher blast percentage allowed in MDS due to lack of differences in outcomes with < 5% or 5-10% blasts in MDS.) Documentation of bone marrow assessment will be accepted within 45 days prior to the anticipated start of conditioning.
- Patients with chronic lymphocytic leukemia (CLL) or other leukemias (including prolymphocytic leukemia) with chemosensitive disease at time of transplantation
- Higher risk CMML according to CMML-specific prognostic scoring system or high risk MDS/MPN not otherwise specified are eligible, provided there is no evidence of high-grade bone marrow fibrosis or massive splenomegaly at the time of enrollment.
- Patients with lymphoma with chemosensitive disease at the time of transplantation
- Cardiac function: Left ventricular ejection fraction ≥ 40% based on most recent echocardiogram or MUGA results with no clinical evidence of heart failure
- Estimated creatinine clearance ≥ 45mL/min calculated by equation
- Pulmonary function: DLCO corrected for hemoglobin > 50% and FEV1 predicted >50% based on most recent PFT results
- Liver function acceptable per local institutional guidelines
- KPS of ≥ 60%
Stratum 3 Recipient Inclusion Criteria:
- Age ≥18 years at the time of signing informed consent
- Patient or legally authorized representative has the ability to provide informed consent according to the applicable regulatory and local institutional requirements.
- Stated willingness to comply with all study procedures and availability for the duration of the study.
- Planned NMA/RIC regimen per study protocol
- Available partially HLA-MMUD (4/8-7/8 at HLA-A, -B, -C, and -DRB1 is required) with age ≥ 18 and ≤ 40 years (≤ 35 preferred).
- Product planned for infusion is MMUD T-cell replete PBSC allograft
- Diagnosis of primary myelofibrosis with risk features making them eligible for HCT. Myelofibrosis secondary to essential thrombocythemia, polycythemia vera, or MDS with grade 4 fibrosis are also eligible. Patients with a myelofibrosis diagnosis require sponsor approval before enrolling.
- Cardiac function: Left ventricular ejection fraction ≥ 40% based on most recent echocardiogram or MUGA results with no clinical evidence of heart failure
- Estimated creatinine clearance ≥ 45 mL/min calculated by equation
- Pulmonary function: DLCO corrected for hemoglobin > 50% and FEV1 predicted >50% based on most recent PFT results
- Liver function acceptable per local institutional guidelines
- KPS of ≥ 60%
Donor Inclusion Criteria (note: donors are not research subjects):
- Must be unrelated to the subject and high-resolution HLA-matched at 4/8, 5/8, 6/8, or 7/8 (HLA-A, -B, -C, and -DRB1.
- Donor must be typed at high-resolution for a minimum of HLA-A, -B, -C, -DQB1, and -DPB1.
- Age ≥ 18 years and ≤ 40 years at the time of signing informed consent for PBSC donation. Note: donors are preferred to be ≤ 35.
- Meet the donor registries' medical suitability requirements for PBSC donation.
- Must undergo eligibility screening according to current Food and Drug Administration (FDA) requirements. Donors who do not meet one or more of the donor screening requirements may donate under urgent medical need.
- Must agree to donate PBSC.
- Must have the ability to give informed consent according to standard (non-study) informed consent according to applicable donor regulatory requirements.
Recipient Exclusion Criteria (Strata 1, 2, and 3):
- Suitable HLA-matched related or 8/8 high-resolution matched unrelated donor available
- Subject unwilling or unable to give informed consent, or unable to comply with the protocol including required follow-up and testing
- Subjects with a prior allogeneic transplant
- Subjects with an autologous transplant within the past 3 months
- Females who are breast-feeding or pregnant
- Uncontrolled bacterial, viral, or fungal infection at the time of the transplant preparative regimen
- Concurrent enrollment on a preventative GvHD and/or infectious disease prevention clinical trial.
- Subjects who undergo desensitization to reduce anti-donor HLA antibody levels prior to transplant.
- Patients who are HIV+ with persistently positive viral load. HIV-infected patients on effective anti-retroviral therapy (ART) with undetectable viral load within 6 months are eligible for this trial. Patients with well controlled HIV are eligible provided resistance panels are negative, the patient is compliant with ART, and their disease remains well controlled.
Donor Exclusion Criteria:
- Donor unwilling or unable to donate.
- Recipient positive for HLA antibodies against a mismatched HLA in the selected donor determined by the presence of donor specific HLA antibodies (DSA) to any mismatched HLA allele/antigen at any of the following loci (HLA-A, -B, -C, -DRB1, DRB3, DRB4, DRB5, -DQA1, - DQB1, -DPA1, -DPB1) with median fluorescence intensity (MFI) >3000 by microarray-based single antigen bead testing. In patients receiving red blood cell or platelet transfusions, DSA evaluation must be performed or repeated post-transfusion and prior to donor mobilization and initiation of recipient preparative regimen.
Trial locations and contacts
32 sites in 1 country.
32 sites in 1 country
United States32 sites
Emory University
Atlanta, Georgia, 30322
St. David's South Austin Medical Center
Austin, Texas, 78704
Greenbaum Cancer Center University of Maryland
Baltimore, Maryland, 21201
Tufts University
Boston, Massachusetts, 02155
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
University of North Carolina
Chapel Hill, North Carolina, 27599
University of Virginia Health System
Charlottesville, Virginia, 22908
Ohio State Medical Center
Columbus, Ohio, 43210
The Center for Bone Marrow Transplantation at Geisinger
Danville, Pennsylvania, 17822
Colorado Blood Cancer Institute at Presbyterian St. Luke's
Denver, Colorado, 80218
Karmanos Cancer Institute
Detroit, Michigan, 48201
City of Hope
Duarte, California, 91010
MD Anderson Cancer Center
Houston, Texas, 77030
Mayo Clinic Jacksonville
Jacksonville, Florida, 32224
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72205
University of Miami Sylvester Cancer Center
Miami, Florida, 33136
Froedtert & the Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
University of Minnesota
Minneapolis, Minnesota, 55455
TriStar Centennial
Nashville, Tennessee, 37203
Memorial Sloan Kettering Cancer Center - Adults
New York, New York, 10065
Abramson Cancer Center
Philadelphia, Pennsylvania, 19104
Mayo Clinic Arizona
Phoenix, Arizona, 85054
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15232
Oregon Health & Science University
Portland, Oregon, 97239
Mayo Clinic
Rochester, Minnesota, 55902
Methodist Hospital San Antonio
San Antonio, Texas, 78229
University of California San Francisco
San Francisco, California, 94158
Honor Health
Scottsdale, Arizona, 85258
Fred Hutchinson Cancer Center
Seattle, Washington, 98109
Barnes Jewish Hospital / Washington University
St Louis, Missouri, 63110
Stanford University
Stanford, California, 94305
Moffitt Cancer Center
Tampa, Florida, 33612
About this study
Official title: A Phase II Study of Reduced Dose Post Transplantation Cyclophosphamide as GvHD Prophylaxis in Adult Patients With Hematologic Malignancies Receiving HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation
The goal of this clinical trial is to determine the effectiveness of Reduced Dose Post-Transplant Cyclophosphamide (PTCy) in patients with hematologic malignancies after receiving an HLA-Mismatched Unrelated Donor (MMUD) . The main question[s] it aims to answer are:
- Does a reduced dose of PTCy reduce the occurrence of infections in the first 100 days after transplant?
- Does a reduced dose of PTCy maintain the same level of protection against Graft Versus Host Disease (GvHD) as the standard dose of PTCy?
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Leukemia, Myelodysplastic Syndromes, Chronic Myeloid Leukemia, Chronic Lymphocytic Leukemia
- Being tested
- Busulfan, Fludarabine, PBSC Hematopoietic Stem Cell Transplantation (HSCT), Post-Transplant Cyclophosphamide, Mesna
- Keywords
- Lymphoma, Leukemia, Hematologic Diseases, Myelodysplastic Syndromes, Preleukemia, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia , Lymphocytic, Chronic, B-Cell, Leukemia, Myeloid, Acute, Leukemia, Biphenotypic, Acute, Neoplasms by Histologic Type, Neoplasms, Lymphoproliferative Disorders, Lymphatic Diseases, Immunoproliferative Disorders, Immune System Disorders, Bone Marrow Diseases, Precancerous Conditions, Leukemia, Lymphoid, Leukemia, B-Cell, Leukemia, Myeloid
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Lymphocytic, Chronic, B-Cell, Myeloproliferative Disorders, Lymphoma, Leukemia, Myelomonocytic, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BusulfanDrug
Also called Busulfex®
Given IV or PO pre-transplant as part of conditioning regimen
FludarabineDrug
Also called Fludara®
Given IV pre-transplant as part of conditioning regimen
PBSC Hematopoietic Stem Cell Transplantation (HSCT)Procedure or surgery
Also called PBSC HSCT, PBSC HCT, PBSC Transplantation, PBSCT
Peripheral blood stem cell graft is infused from a mismatched unrelated donor on Day 0
Post-Transplant CyclophosphamideDrug
Also called Cytoxan®, PTCy
Cyclophosphamide (25mg/kg) is administered on Day 3 and Day 4 post-transplant as an IV infusion over 1-2 hours.
First 20 subjects with a 4-6/8 HLA mismatched unrelated donor will receive an intermediate dose of post-transplant cyclophosphamide of 37.5 mg/kg Day 3 and Day 4 post-transplant.
MesnaDrug
Also called Mesnex®
Mesna is given in divided doses IV 30 min pre- and at 3, 6, and 8 hours post cyclophosphamide.
TacrolimusDrug
Tacrolimus is given at a dose of 0.05 mg/kg PO or an IV dose of 0.03 mg/kg of ideal body weight (IBW) starting on Day +5 post-transplant with taper recommended at Day + 90 and finished by Day +180.
Also called MMF, Cellcept®
Mycophenolate mofetil (MMF) is given at a dose of 15 mg/kg three times daily IV or PO from Day +5 to Day +35 post-transplant.
Patient Reported OutcomesOther
Also called PRO
Survey assessments will be administered to study participants pre transplant, at Day + 100, Day + 180, and Day +365 post transplant.
MelphalanDrug
Given IV pre transplant as part of conditioning regimen
Total-body irradiationRadiation
Also called TBI
Administered pre-transplant as part of conditioning regimen
CyclophosphamideDrug
Also called Cytoxan®
Given IV pre-transplant as part of conditioning regimen
| Study group | Role | What they receive |
|---|---|---|
| Regimen A (MAC: Busulfan and Fludarabine, PBSC HCT; Reduce Dose PTCy | Experimental | Patients Receive:
Patients receive:
Busulfan (≥ 9 mg/kg total dose) IV or PO on days -6 to -3 Fludarabine (150 mg/m2 total dose) IV on days -6 to -2
Patients receive a peripheral blood stem cell (PBSC) graft infusion from a mismatched unrelated donor on Day 0.
Patients receive a reduced dose of cyclophosphamide (25mg/kg per dose) on Day 3 and Day 4 post-transplant.
First 20 patients with a 4-6/8 mismatched unrelated donor will receive an alternate dose of post-transplant cyclophosphamide of 37.5 mg/kg on Days 3 and Day 4 post-transplant. Receives: Drug: Busulfan; Drug: Fludarabine; Procedure: PBSC Hematopoietic Stem Cell Transplantation (HSCT); Drug: Post-Transplant Cyclophosphamide; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Other: Patient Reported Outcomes |
| Regimen B (MAC: Fludarabine and TBI, PBSC HCT; Reduce Dose PTCy | Experimental | Patients receive:
Fludarabine (90 mg/m2 total dose) IV on days -7 to -5 Total body irradiation (TBI) (1200 cGy total dose) on days -4 to -1
Patients receive a peripheral blood stem cell (PBSC) graft infusion from a mismatched unrelated donor on Day 0.
Patients receive a reduced dose of cyclophosphamide (25mg/kg per dose) on Day 3 and Day 4 post-transplant.
First 20 patients with a 4-6/8 mismatched unrelated donor will receive an alternate dose of post-transplant cyclophosphamide of 37.5 mg/kg on Days 3 and Day 4 post-transplant. Receives: Drug: Fludarabine; Procedure: PBSC Hematopoietic Stem Cell Transplantation (HSCT); Drug: Post-Transplant Cyclophosphamide; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Other: Patient Reported Outcomes; Radiation: Total-body irradiation |
| Regimen C (RIC: Fludarabine and Busulfan; PBSCT HCT; Reduced Dose PTCy | Experimental | Patients receive:
Fludarabine (150-180 mg/m2 total dose) IV on days -6 to -2 Busulfan (less than or equal to 8 mg/kg PO or 6.4 mg/kg IV) on days -5 and -4
Patients receive a peripheral blood stem cell (PBSC) graft infusion from a mismatched unrelated donor on Day 0.
Patients receive a reduced dose of cyclophosphamide (25mg/kg per dose) on Day 3 and Day 4 post-transplant.
First 20 patients with a 4-6/8 mismatched unrelated donor will receive an alternate dose of post-transplant cyclophosphamide of 37.5 mg/kg on Days 3 and Day 4 post-transplant. Receives: Drug: Busulfan; Drug: Fludarabine; Procedure: PBSC Hematopoietic Stem Cell Transplantation (HSCT); Drug: Post-Transplant Cyclophosphamide; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Other: Patient Reported Outcomes |
| Regimen D (RIC: Fludarabine and Melphalan; PBSCT HCT; Reduced Dose PTCy | Experimental | Patients receive:
Fludarabine (125-150 mg/m2 total dose) IV on days -7 to -3 Melphalan (100-140 mg/m2) IV on day -1
Patients receive a PBSC graft infusion from a mismatched unrelated donor on Day 0.
Patients receive a reduced dose of cyclophosphamide (25mg/kg per dose) on Day 3 and Day 4 post-transplant.
First 20 patients with a 4-6/8 mismatched unrelated donor will receive an alternate dose of post-transplant cyclophosphamide of 37.5 mg/kg on Days 3 and Day 4 post-transplant. Receives: Drug: Fludarabine; Procedure: PBSC Hematopoietic Stem Cell Transplantation (HSCT); Drug: Post-Transplant Cyclophosphamide; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Other: Patient Reported Outcomes; Drug: Melphalan |
| Regimen E (NMA: Fludarabine, Cyclophosphamide, and TBI; PBSCT HCT; Reduced Dose PTCy | Experimental | Patients receive:
Fludarabine (150mg/m2 total dose) IV on days -6 to -2 Cyclophosphamide (29-50mg/kg) IV on days -6 and -5 TBI (200cGy) on day -1
Patients receive a PBSC graft infusion from a mismatched unrelated donor on Day 0.
Patients receive a reduced dose of cyclophosphamide (25mg/kg per dose) on Day 3 and Day 4 post-transplant.
First 20 patients with a 4-6/8 mismatched unrelated donor will receive an alternate dose of post-transplant cyclophosphamide of 37.5 mg/kg on Days 3 and Day 4 post-transplant. Receives: Drug: Fludarabine; Procedure: PBSC Hematopoietic Stem Cell Transplantation (HSCT); Drug: Post-Transplant Cyclophosphamide; Drug: Mesna; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Other: Patient Reported Outcomes; Radiation: Total-body irradiation; Drug: Cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Infection Free Survival
Survival at 100 days without grades 2-3 infections (per BMT CTN grading criteria)
Measured: 100 days post-HCT
Secondary outcomes (16)
Overall Survival
Defined as time interval between date of transplant and death from any cause
Measured: 1-year post-HCT
Progression-free survival
Defined as disease relapse or progression, or death by any cause
Measured: 1-year post-HCT
Infection-free survival
Defined as death and grades II-III infection (per BMT CTN criteria)
Measured: 1-year post-HCT
Graft versus host disease relapse free survival
Defined as relapse or progression of underling disease (by 1 year), grade III-IV acute GvHD (by 6 months), chronic GvHD requiring systemic immune suppression (by 1 year), or death by any cause (by 1 year).
Measured: 1-year post-HCT
Non-relapse mortality
Defined as death without evidence of disease progression or recurrence
Measured: 1-year post-HCT
Cumulative incidence of neutrophil recovery
Defined as achieving an absolute neutrophil count (ANC) greater than or equal to 500/mm3 for three consecutive measurements on three different days
Measured: Day 28 post-HCT
Cumulative incidence of platelet recovery
Defined as platelet count ≥20,000/mm^3 or ≥50,000/mm^3 with no platelet transfusions within seven days.
Measured: Day 28 post-HCT
Cumulative incidence of primary and secondary graft failure
Primary graft failure is defined as no neutrophil recovery to ≥ 500 cells/mm3 by Day 28 post HCT.
Secondary graft failure is defined as as initial neutrophil count recovery followed by subsequent decline in absolute neutrophil counts <500 cells/mm3, unresponsive to growth factor therapy, but cannot be explained by infection, disease relapse, or medications.
Measured: Day 28 and 1-year post-HCT
Donor T-Cell Chimerism
Defined as percent of donor chimerism via peripheral blood
Measured: Day 28, 100 and 365 post-HCT
Cumulative incidence of acute GvHD
Defined as cumulative incidence of grades II-IV acute GvHD
Measured: Day 100 and Day 180 post-HCT
Cumulative incidence of chronic GvHD
Measured: 1-year post-HCT
Cumulative incidence of grade 2-3 bacterial, fungal, and viral infections
Defined as grades 2-3 infection as defined by BMT CTN grading criteria.
Measured: Day 100 and 1-year post-HCT
Cumulative incidence of grades 2-3 BK virus hemorrhagic cystitis
Defined as incidence of BK virus hemorrhagic cystitis per BMT CTN grading criteria
Measured: 1-year post-HCT
Cumulative incidence of relapse/progression
Defined as disease relapse or progression from Day 0 to 1-year post-HCT
Measured: 1-year post-HCT
Overall Toxicity
To tabulate adverse events (AEs, experienced by recipients, defined as grade 3-5 unexpected and grade 5 expected AEs according to CTCAE v5
Measured: 1-year post-HCT
Incidence and Severity of cytokine release syndrome
Defined and graded using the ASTCT grading criteria.
Measured: within 14 days post-HCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Aug 2023
- Study start8 Dec 2023
- Primary completion24 Dec 2026 (estimated)
- Study completion30 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Lymphoblastic LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 313 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Center for International Blood and Marrow Transplant Research
- Sponsor type
- Research network
- Collaborators
- National Marrow Donor Program
- Responsible party
- Sponsor
- Investigators
- Steven Devine, MD, Principal investigator, NMDPGinna Laport, MD, Study chair, NMDP
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06001385
- Sponsor's protocol ID
- OPTIMIZE
- Acronym
- OPTIMIZE
- Status last verified
- Aug 2026
- First submitted
- 14 Aug 2023
- Last updated
- 17 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Reduced Dose Post Transplantation Cyclophosphamide as GvHD Prophylaxis in Adult Patients With Hematologic Malignancies Receiving HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT06001385
- Acronym / sponsor ID
- OPTIMIZE / OPTIMIZE
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Leukemia, Myelodysplastic Syndromes, Chronic Myeloid Leukemia, Chronic Lymphocytic Leukemia
- Keywords
- Lymphoma, Leukemia, Hematologic Diseases, Myelodysplastic Syndromes, Preleukemia, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia , Lymphocytic, Chronic, B-Cell, Leukemia, Myeloid, Acute
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 313 participants (estimated)
- Lead sponsor
- Center for International Blood and Marrow Transplant ResearchResearch network
- Collaborators
- National Marrow Donor Program
- Study start
- 8 Dec 2023 (actual)
- Primary completion
- 24 Dec 2026 (estimated)
- Study completion
- 30 Dec 2026 (estimated)
- First posted
- 21 Aug 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical trial is to determine the effectiveness of Reduced Dose Post-Transplant Cyclophosphamide (PTCy) in patients with hematologic malignancies after receiving an HLA-Mismatched Unrelated Donor (MMUD) . The main question[s] it aims to answer are:
- Does a reduced dose of PTCy reduce the occurrence of infections in the first 100 days after transplant?
- Does a reduced dose of PTCy maintain the same level of protection against Graft Versus Host Disease (GvHD) as the standard dose of PTCy?
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Regimen A (MAC: Busulfan and Fludarabine, PBSC HCT; Reduce Dose PTCy | Experimental | Drug: Busulfan, Drug: Fludarabine, Procedure: PBSC Hematopoietic Stem Cell Transplantation (HSCT), Drug: Post-Transplant Cyclophosphamide, Drug: Tacrolimus, Drug: Mycophenolate Mofetil, Other: Patient Reported OutcomesPatients Receive: Patients receive: Busulfan (≥ 9 mg/kg total dose) IV or PO on days -6 to -3 Fludarabine (150 mg/m2 total dose) IV on days -6 to -2 Patients receive a peripheral blood stem cell (PBSC) graft infusion from a mismatched unrelated donor on Day 0. Patients receive a reduced dose of cyclophosphamide (25mg/kg per dose) on Day 3 and Day 4 post-transplant. First 20 patients with a 4-6/8 mismatched unrelated donor will receive an alternate dose of post-transplant cyclophosphamide of 37.5 mg/kg on Days 3 and Day 4 post-transplant. |
| Regimen B (MAC: Fludarabine and TBI, PBSC HCT; Reduce Dose PTCy | Experimental | Drug: Fludarabine, Procedure: PBSC Hematopoietic Stem Cell Transplantation (HSCT), Drug: Post-Transplant Cyclophosphamide, Drug: Tacrolimus, Drug: Mycophenolate Mofetil, Other: Patient Reported Outcomes, Radiation: Total-body irradiationPatients receive: Fludarabine (90 mg/m2 total dose) IV on days -7 to -5 Total body irradiation (TBI) (1200 cGy total dose) on days -4 to -1 Patients receive a peripheral blood stem cell (PBSC) graft infusion from a mismatched unrelated donor on Day 0. Patients receive a reduced dose of cyclophosphamide (25mg/kg per dose) on Day 3 and Day 4 post-transplant. First 20 patients with a 4-6/8 mismatched unrelated donor will receive an alternate dose of post-transplant cyclophosphamide of 37.5 mg/kg on Days 3 and Day 4 post-transplant. |
| Regimen C (RIC: Fludarabine and Busulfan; PBSCT HCT; Reduced Dose PTCy | Experimental | Drug: Busulfan, Drug: Fludarabine, Procedure: PBSC Hematopoietic Stem Cell Transplantation (HSCT), Drug: Post-Transplant Cyclophosphamide, Drug: Tacrolimus, Drug: Mycophenolate Mofetil, Other: Patient Reported OutcomesPatients receive: Fludarabine (150-180 mg/m2 total dose) IV on days -6 to -2 Busulfan (less than or equal to 8 mg/kg PO or 6.4 mg/kg IV) on days -5 and -4 Patients receive a peripheral blood stem cell (PBSC) graft infusion from a mismatched unrelated donor on Day 0. Patients receive a reduced dose of cyclophosphamide (25mg/kg per dose) on Day 3 and Day 4 post-transplant. First 20 patients with a 4-6/8 mismatched unrelated donor will receive an alternate dose of post-transplant cyclophosphamide of 37.5 mg/kg on Days 3 and Day 4 post-transplant. |
| Regimen D (RIC: Fludarabine and Melphalan; PBSCT HCT; Reduced Dose PTCy | Experimental | Drug: Fludarabine, Procedure: PBSC Hematopoietic Stem Cell Transplantation (HSCT), Drug: Post-Transplant Cyclophosphamide, Drug: Tacrolimus, Drug: Mycophenolate Mofetil, Other: Patient Reported Outcomes, Drug: MelphalanPatients receive: Fludarabine (125-150 mg/m2 total dose) IV on days -7 to -3 Melphalan (100-140 mg/m2) IV on day -1 Patients receive a PBSC graft infusion from a mismatched unrelated donor on Day 0. Patients receive a reduced dose of cyclophosphamide (25mg/kg per dose) on Day 3 and Day 4 post-transplant. First 20 patients with a 4-6/8 mismatched unrelated donor will receive an alternate dose of post-transplant cyclophosphamide of 37.5 mg/kg on Days 3 and Day 4 post-transplant. |
| Regimen E (NMA: Fludarabine, Cyclophosphamide, and TBI; PBSCT HCT; Reduced Dose PTCy | Experimental | Drug: Fludarabine, Procedure: PBSC Hematopoietic Stem Cell Transplantation (HSCT), Drug: Post-Transplant Cyclophosphamide, Drug: Mesna, Drug: Tacrolimus, Drug: Mycophenolate Mofetil, Other: Patient Reported Outcomes, Radiation: Total-body irradiation, Drug: CyclophosphamidePatients receive: Fludarabine (150mg/m2 total dose) IV on days -6 to -2 Cyclophosphamide (29-50mg/kg) IV on days -6 and -5 TBI (200cGy) on day -1 Patients receive a PBSC graft infusion from a mismatched unrelated donor on Day 0. Patients receive a reduced dose of cyclophosphamide (25mg/kg per dose) on Day 3 and Day 4 post-transplant. First 20 patients with a 4-6/8 mismatched unrelated donor will receive an alternate dose of post-transplant cyclophosphamide of 37.5 mg/kg on Days 3 and Day 4 post-transplant. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BusulfanAlso: Busulfex®Given IV or PO pre-transplant as part of conditioning regimen | Drug | Regimen A (MAC: Busulfan and Fludarabine, PBSC HCT; Reduce Dose PTCy, Regimen C (RIC: Fludarabine and Busulfan; PBSCT HCT; Reduced Dose PTCy |
| FludarabineAlso: Fludara®Given IV pre-transplant as part of conditioning regimen | Drug | Regimen A (MAC: Busulfan and Fludarabine, PBSC HCT; Reduce Dose PTCy, Regimen B (MAC: Fludarabine and TBI, PBSC HCT; Reduce Dose PTCy, Regimen C (RIC: Fludarabine and Busulfan; PBSCT HCT; Reduced Dose PTCy, Regimen D (RIC: Fludarabine and Melphalan; PBSCT HCT; Reduced Dose PTCy, Regimen E (NMA: Fludarabine, Cyclophosphamide, and TBI; PBSCT HCT; Reduced Dose PTCy |
| PBSC Hematopoietic Stem Cell Transplantation (HSCT)Also: PBSC HSCT, PBSC HCT, PBSC Transplantation, PBSCTPeripheral blood stem cell graft is infused from a mismatched unrelated donor on Day 0 | Procedure or surgery | Regimen A (MAC: Busulfan and Fludarabine, PBSC HCT; Reduce Dose PTCy, Regimen B (MAC: Fludarabine and TBI, PBSC HCT; Reduce Dose PTCy, Regimen C (RIC: Fludarabine and Busulfan; PBSCT HCT; Reduced Dose PTCy, Regimen D (RIC: Fludarabine and Melphalan; PBSCT HCT; Reduced Dose PTCy, Regimen E (NMA: Fludarabine, Cyclophosphamide, and TBI; PBSCT HCT; Reduced Dose PTCy |
| Post-Transplant CyclophosphamideAlso: Cytoxan®, PTCyCyclophosphamide (25mg/kg) is administered on Day 3 and Day 4 post-transplant as an IV infusion over 1-2 hours. First 20 subjects with a 4-6/8 HLA mismatched unrelated donor will receive an intermediate dose of post-transplant cyclophosphamide of 37.5 mg/kg Day 3 and Day 4 post-transplant. | Drug | Regimen A (MAC: Busulfan and Fludarabine, PBSC HCT; Reduce Dose PTCy, Regimen B (MAC: Fludarabine and TBI, PBSC HCT; Reduce Dose PTCy, Regimen C (RIC: Fludarabine and Busulfan; PBSCT HCT; Reduced Dose PTCy, Regimen D (RIC: Fludarabine and Melphalan; PBSCT HCT; Reduced Dose PTCy, Regimen E (NMA: Fludarabine, Cyclophosphamide, and TBI; PBSCT HCT; Reduced Dose PTCy |
| MesnaAlso: Mesnex®Mesna is given in divided doses IV 30 min pre- and at 3, 6, and 8 hours post cyclophosphamide. | Drug | Regimen E (NMA: Fludarabine, Cyclophosphamide, and TBI; PBSCT HCT; Reduced Dose PTCy |
| TacrolimusTacrolimus is given at a dose of 0.05 mg/kg PO or an IV dose of 0.03 mg/kg of ideal body weight (IBW) starting on Day +5 post-transplant with taper recommended at Day + 90 and finished by Day +180. | Drug | Regimen A (MAC: Busulfan and Fludarabine, PBSC HCT; Reduce Dose PTCy, Regimen B (MAC: Fludarabine and TBI, PBSC HCT; Reduce Dose PTCy, Regimen C (RIC: Fludarabine and Busulfan; PBSCT HCT; Reduced Dose PTCy, Regimen D (RIC: Fludarabine and Melphalan; PBSCT HCT; Reduced Dose PTCy, Regimen E (NMA: Fludarabine, Cyclophosphamide, and TBI; PBSCT HCT; Reduced Dose PTCy |
| Mycophenolate MofetilAlso: MMF, Cellcept®Mycophenolate mofetil (MMF) is given at a dose of 15 mg/kg three times daily IV or PO from Day +5 to Day +35 post-transplant. | Drug | Regimen A (MAC: Busulfan and Fludarabine, PBSC HCT; Reduce Dose PTCy, Regimen B (MAC: Fludarabine and TBI, PBSC HCT; Reduce Dose PTCy, Regimen C (RIC: Fludarabine and Busulfan; PBSCT HCT; Reduced Dose PTCy, Regimen D (RIC: Fludarabine and Melphalan; PBSCT HCT; Reduced Dose PTCy, Regimen E (NMA: Fludarabine, Cyclophosphamide, and TBI; PBSCT HCT; Reduced Dose PTCy |
| Patient Reported OutcomesAlso: PROSurvey assessments will be administered to study participants pre transplant, at Day + 100, Day + 180, and Day +365 post transplant. | Other | Regimen A (MAC: Busulfan and Fludarabine, PBSC HCT; Reduce Dose PTCy, Regimen B (MAC: Fludarabine and TBI, PBSC HCT; Reduce Dose PTCy, Regimen C (RIC: Fludarabine and Busulfan; PBSCT HCT; Reduced Dose PTCy, Regimen D (RIC: Fludarabine and Melphalan; PBSCT HCT; Reduced Dose PTCy, Regimen E (NMA: Fludarabine, Cyclophosphamide, and TBI; PBSCT HCT; Reduced Dose PTCy |
| MelphalanGiven IV pre transplant as part of conditioning regimen | Drug | Regimen D (RIC: Fludarabine and Melphalan; PBSCT HCT; Reduced Dose PTCy |
| Total-body irradiationAlso: TBIAdministered pre-transplant as part of conditioning regimen | Radiation | Regimen B (MAC: Fludarabine and TBI, PBSC HCT; Reduce Dose PTCy, Regimen E (NMA: Fludarabine, Cyclophosphamide, and TBI; PBSCT HCT; Reduced Dose PTCy |
| CyclophosphamideAlso: Cytoxan®Given IV pre-transplant as part of conditioning regimen | Drug | Regimen E (NMA: Fludarabine, Cyclophosphamide, and TBI; PBSCT HCT; Reduced Dose PTCy |
Primary outcome
Infection Free Survival
Time frame: 100 days post-HCT
Survival at 100 days without grades 2-3 infections (per BMT CTN grading criteria)
16 secondary outcomes
Overall Survival
Time frame: 1-year post-HCT
Progression-free survival
Time frame: 1-year post-HCT
Infection-free survival
Time frame: 1-year post-HCT
Graft versus host disease relapse free survival
Time frame: 1-year post-HCT
Non-relapse mortality
Time frame: 1-year post-HCT
Cumulative incidence of neutrophil recovery
Time frame: Day 28 post-HCT
Cumulative incidence of platelet recovery
Time frame: Day 28 post-HCT
Cumulative incidence of primary and secondary graft failure
Time frame: Day 28 and 1-year post-HCT
Donor T-Cell Chimerism
Time frame: Day 28, 100 and 365 post-HCT
Cumulative incidence of acute GvHD
Time frame: Day 100 and Day 180 post-HCT
Cumulative incidence of chronic GvHD
Time frame: 1-year post-HCT
Cumulative incidence of grade 2-3 bacterial, fungal, and viral infections
Time frame: Day 100 and 1-year post-HCT
Cumulative incidence of grades 2-3 BK virus hemorrhagic cystitis
Time frame: 1-year post-HCT
Cumulative incidence of relapse/progression
Time frame: 1-year post-HCT
Overall Toxicity
Time frame: 1-year post-HCT
Incidence and Severity of cytokine release syndrome
Time frame: within 14 days post-HCT
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Aug 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Steven Devine, MD principal investigator
NMDP
Ginna Laport, MD study chair
NMDP
32 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Busulfan, Fludarabine and PBSC Hematopoietic Stem Cell Transplantation (HSCT) in people with Acute Lymphoblastic Leukemia and Acute Myeloid Leukemia, run by Center for International Blood and Marrow Transplant Research at 32 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 17 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Infection Free Survival (time frame: 100 days post-HCT). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Aug 2026.