ClinicalTrials.gov record NCT03314974

Myeloablative Allo HSCT With Related or Unrelated Donor for Heme Disorders

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

Myeloablative Allogeneic Hematopoietic Cell Transplantation Using a Related or Unrelated Donor for the Treatment of Hematological Diseases

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
SexAll
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Phase II interventional treatment study testing a myeloablative allogeneic hematopoietic stem cell transplant approach. Patients receive either total body irradiation (TBI) or a fludarabine+busulfan preparative regimen (for those who cannot receive radiation), then receive post-transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil for graft-versus-host disease (GVHD) prevention. The study enrolls patients up to 60 years old. It is non-randomized with two parallel treatment arms.

What it is studying. The study tests outcomes after two different myeloablative preparative regimens (TBI vs fludarabine+busulfan) followed by post-transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil for GVHD prevention.

What the researchers hope to learn. Primary scientific goal: measure the incidence of chronic graft-versus-host disease (GVHD) at 1 year. Secondary scientific goals: measure rates of grade II–IV acute GVHD (by Day +100), chronic GVHD at 2 years, relapse, overall survival, treatment-related mortality, graft-versus-host disease-free, relapse-free survival (GRFS) at 1 and 2 years, and neutrophil and platelet engraftment at Day 42 and 6 months.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

This is a Phase II study of allogeneic hematopoietic stem cell transplant (HCT) using a myeloablative preparative regimen (of either total body irradiation (TBI); or, fludarabine/busulfan for patients unable to receive further radiation). followed by a post-transplant graft-versus-host disease (GVHD) prophylaxis regimen of post-transplant cyclophosphamide (PTCy), tacrolimus (Tac), and mycophenolate mofetil (MMF).

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Biological
  • HSCT with TBI Regimen
  • HSCT with Non-TBI Regimen

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive a myeloablative preparative regimen (either total body irradiation or busulfan + fludarabine), followed by an allogeneic hematopoietic stem cell infusion. After transplant they receive post‑transplant graft‑versus‑host disease (GVHD) prophylaxis with post‑transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil, and are followed with regular clinic visits and tests to monitor engraftment, GVHD, relapse, and organ function. Pregnancy testing and contraception requirements apply; HIV‑positive patients (see eligibility notes) need Infectious Disease evaluation and a management plan prior to transplant.

How the study is structured. Phase II interventional treatment study. Non-randomized, parallel design with two experimental arms: a TBI-based preparative regimen or a non-TBI (busulfan+fludarabine) preparative regimen. No masking. Participants are followed for measures including acute and chronic GVHD, engraftment, relapse, and survival at specified time points (up to 2 years for some outcomes).

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Waldenström macroglobulinemia

Clinical trials63 trials listed for Waldenström macroglobulinemiaEvery trial the registry lists for Waldenström macroglobulinemia, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Waldenström macroglobulinemia researchLatest monthly update: September 2026. Includes: Upfront treatment outcomes. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Mar 30, 2018
Primary completion
Jun 10, 2027 (estimated)
Study completion
Jun 10, 2028 (estimated)
Target enrollment
300 participants (estimated)
Last updated on registry
Jun 25, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Masonic Cancer Center, University of Minnesota

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

hematopoietic stem cell transplant
A procedure that replaces blood-forming cells after high-dose treatment. 'Autologous' uses the person's own cells collected beforehand; 'allogeneic' uses cells from a donor.
Performance Status
A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation.
Karnofsky
A scale from 100 (no complaints) down to 0 that describes how well a person can carry out daily activities. Studies often require a minimum score.
legally authorized representative
A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
upper limit of normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
creatinine clearance
A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
GFR
A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
echocardiography
An ultrasound scan of the heart. A transesophageal echocardiogram is done through a probe passed into the food pipe for a closer view.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
relapse
A condition that improved with treatment and has since returned or worsened.
complete remission
No sign of the condition can be found with the tests used in the study. It does not always mean the condition is gone permanently, and the definition depends on the condition and the study.
minimal residual disease
Very small amounts of disease that remain after treatment and can only be found with highly sensitive tests. 'MRD-negative' means none was detected at the test's limit. The exact test and threshold depend on the condition and the study.
refractory
A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
stratified
Randomisation done separately within subgroups, such as age bands or disease severity, so that each group contains a similar mix.
biopsy
Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.
PET
A scan in which a small amount of a radioactive tracer is injected so that activity in tissues can be seen. Often combined with a CT or MRI scan.
CT
An X-ray scan that builds detailed cross-section images of the body. 'High-resolution' CT (HRCT) is used to look closely at the lungs.
childbearing potential
Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

← Back to clinical trials