New drug combo aims to make stem cell transplants safer for blood cancer patients
NCT ID NCT03314974
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This Phase 2 trial is testing a stem cell transplant from a donor for people with various blood cancers and bone marrow disorders. The transplant uses strong chemotherapy or radiation to wipe out the diseased marrow, then replaces it with healthy donor cells. A new combination of three drugs (cyclophosphamide, tacrolimus, and mycophenolate mofetil) is given after the transplant to prevent graft-versus-host disease, a serious complication where donor cells attack the patient's body. The study aims to see if this approach reduces chronic graft-versus-host disease while still controlling the cancer. About 300 participants up to age 60 are being enrolled.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Hematopoietic stem cell transplant (bone marrow or blood stem cells from a donor) followed by cyclophosphamide, tacrolimus, and mycophenolate mofetil
- What this could lead to
- If successful, this approach could improve survival and reduce severe side effects for people with blood cancers needing a stem cell transplant.
- What could go wrong
- This is a mid-stage trial, so results are not yet proven. Risks include graft failure, serious infections, and graft-versus-host disease, which can be life-threatening.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 300 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Mar 2018
- Expected to finish
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Jun 2028
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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Up to 60 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
-Inclusion Criteria: * Age: ≤ 60 years of age * Performance Status: Karnofsky ≥ 70%, Lansky play score ≥ 70 * Consent: Voluntary written consent (adult or legally authorized representative; or parental/guardian) * Adequate Organ Function: * Renal: Creatinine \<2x upper limit of normal. Patients above this limit must have creatinine clearance ≥ 40 ml/min/1.73m2 as determined by an age-appropriate method, such as cystatin C GFR. * Hepatic: Bilirubin, AST, alkaline phosphatase \<4 times the upper limit of institutional normal * Pulmonary: Diffusion capacity of oxygen, corrected for hemoglobin, \> 50% of predicted. For pediatric patients not able to undergo PFTs or diffusion testing: O2 sat of \>95% on room air * Cardiac: Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction \> 45%. For children not able to cooperate with MUGA or echocardiography, such should be clearly stated in the physician's documentation * HIV Status: HIV infection with undetectable viral load. All HIV+ patients must be evaluated by Infectious Disease (ID) and a HIV management plan establish prior to transplantation Other Inclusion Criteria: * Women of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control for the duration of treatment. * Donor Availability: Patients considered for transplantation must have a sufficient graft as based on current criteria of the University of Minnesota Blood and Marrow Transplantation Program * Eligible Diseases and Status: Patients are eligible unless their treatment is to be guided by a higher priority protocol. * Acute Leukemias: Must be in remission by morphology (≤5% blasts). Also a small percentage of blasts that is equivocal between marrow regeneration vs. early relapse are acceptable provided there are no associated cytogenetic markers consistent with relapse. * Acute Myeloid Leukemia (AML) and related precursor neoplasms: 2nd or greater complete remission (CR); first complete remission (CR1) in patients \> 60 years old; CR1 in ≤ 60 years old that is NOT considered as favorable-risk. * Favorable risk AML is defined as having one of the following: * t(8,21) without cKIT mutation * inv(16) or t(16;16) without cKIT mutation * Normal karyotype with mutated NPM1 and wild type FLT-ITD * Normal karyotype with double mutated CEBPA * Acute prolymphocytic leukemia (APL) in first molecular remission at the end of consolidation * Very high risk pediatric patients with AML: Patients \<21 years, however, are eligible with (M2 marrow) with \< 25% blasts in marrow after having failed one or more cycles of chemotherapy. * Acute lymphoblastic leukemia (ALL)/lymphoma: second or greater CR; CR1 unable to tolerate consolidation chemotherapy due to chemotherapy-related toxicities; CR1 high-risk ALL. * High risk ALL is defined as having one of the following: * Evidence of high risk cytogenetics, e.g. t(9;22), t(1;19), t(4;11), other MLL rearrangements, IKZF1 * 30 years of age or older at diagnosis * White blood cell counts of greater than 30,000/mcL (B-ALL) or greater than 100,000/mcL (T-ALL) at diagnosis * CNS leukemia involvement during the course of disease * Slow cytologic response (\>10% lymphoblasts in bone marrow on Day 14 of induction therapy) * Evidence of persistent immonophenotypic or molecular minimal residual disease (MRD) at the end of induction and consolidation therapy * Very high risk pediatric patients with ALL: patients \<21 years are also considered high risk CR1 if they had M2 or M3 marrow at day 42 from the initiation of induction or M3 marrow at the end of induction. They are eligible once they achieve a complete remission. * Chronic Myelogenous Leukemia excluding refractory blast crisis: To be eligible in first chronic phase (CP1) patient must have failed or be intolerant to one or more tyrosine kinase inhibitors. * Plasma Cell Leukemia after initial therapy, in patients who have achieved at least a partial remission * Myeloproliferative Neoplasms/Myelofibrosis, either primary as a result of polycythemia vera or essential thrombocythemia, with disease risk of intermediate or high-risk according to DIPSS criteria. Blasts must be \<10% by bone marrow aspirate morphology. * Myelodysplasia (MDS) IPSS INT-2 or High Risk (i.e. RAEB, RAEBt) or Refractory Anemia with severe pancytopenia, transfusion dependence, or high risk cytogenetics or molecular features. Blasts must be \< 10% by a representative bone marrow aspirate morphology. * Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL), Marginal Zone B-Cell Lymphoma or Follicular Lymphoma are eligible if there was disease progression/relapse within 12 of achieving a partial or complete remission. Patients who had remissions lasting \> 12 months, are eligible after at least two prior therapies. Patients with bulky disease (nodal mass greater than 5 cm) should be considered for debulking chemotherapy before transplant. * Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia are eligible after initial therapy in CR1+ or PR1+. * Diffuse large Cell NHL \> CR/\> PR: Patients in CR/PR with initial short remission (\<6 months) are eligible, or those who have failed/or are not eligible for autologous transplant. * Lymphoblastic Lymphoma, Burkitt's Lymphoma, and other high-grade NHL after initial therapy if stage III/IV in CR1/PR1 or after progression if stage I/II \< 1 year. * Multiple Myeloma beyond PR2: Patients with chromosome 13 abnormalities, first response lasting less than 6 months, or β-2 microglobulin \> 3 mg/L, may be considered for this protocol after initial therapy. * Juvenile myelomonocytic leukemia * Biphenotypic/Undifferentiated/Prolymphocytic Leukemias in first or subsequent CR. * MRD positive leukemia (AML, ALL or accelerated/blast phase CML). Selected patients in morphologic CR, but with positive immunophenotypic (flow cytometry) or molecular evidence of MRD may be eligible if recent chemotherapy has not resulted in MRD negative status. * Natural Killer Cell Malignancies * Acquired Bone Marrow Failure Syndromes except for Fanconi Anemia or Dyskeratosis Congenita * Other Leukemia Subtypes: A major effort in the field of hematology is to identify patients who are of high risk for treatment failure so that patients can be appropriately stratified to either more (or less) intensive therapy. This effort is continually ongoing and retrospective studies identify new disease features or characteristics that are associated with treatment outcomes. Therefore, if new features are identified after the writing of this protocol, patients can be enrolled with the approval of two members of the study committee. Exclusion Criteria: * Chemotherapy refractory large cell and high grade NHL (i.e., progressive disease after \> 2 salvage regimens) * CML in blast crisis * Large cell lymphoma, mantle cell lymphoma and Hodgkin disease that is progressing on salvage therapy. * Evidence of progressive disease by imaging modalities or biopsy - persistent PET activity, though possibly related to lymphoma, is not an exclusion criterion in the absence of CT changes indicating progression. * Active central nervous system malignancy * if ≤ 18 years old, prior myeloablative transplant within the last 6 months. If \>18 years old prior myeloablative allotransplant or autologous transplant * Active HIV infection or known HIV positive serology * active uncontrolled infection * Pregnant or breastfeeding. The agents used in this study include Pregnancy Category D: known to cause harm to a fetus. Females of childbearing potential must have a negative pregnancy test prior to starting therapy.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Masonic Cancer Center at University of Minnesota
RECRUITINGMinneapolis, Minnesota, 55337, United States
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