Actively Recruiting

Phase 2
Age: 0 - 60Years
All Genders
ID03314974

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

Led by Masonic Cancer Center, University of Minnesota · Updated on 2025-06-24

300

Participants Needed

1

Research Sites

52 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are evaluating a Phase II study of allogeneic hematopoietic stem cell transplantation (HCT) using a myeloablative preparative regimen for patients with various hematological diseases. The study compares two conditioning approaches: total body irradiation (TBI) or a chemotherapy regimen of fludarabine and busulfan for patients unable to receive radiation. This is followed by a post-transplant graft-versus-host disease (GVHD) prevention treatment using cyclophosphamide, tacrolimus, and mycophenolate mofetil. Participants receive one of two treatment regimens: the TBI regimen or the non-TBI regimen. In the TBI group, patients undergo total body irradiation from day -5 to -2 followed by stem cell transplantation on day 0. In the non-TBI group, busulfan and fludarabine are given from day -5 to -2 prior to transplantation. Both groups receive cyclophosphamide on days +3 and +4, and begin tacrolimus and mycophenolate mofetil on day +5 with dosing durations dependent on GVHD presence and engraftment status. Throughout the study, participants are monitored for acute and chronic GVHD, relapse, survival, graft function, and treatment-related mortality at various time points up to two years post-transplant. Key assessments include neutrophil and platelet engraftment by days 42 and six months, along with ongoing evaluations of GVHD and survival outcomes. The total participation time spans up to two years, with detailed follow-up for safety and effectiveness of the transplant approaches.

CONDITIONS

Brief Title

Myeloablative Allo HSCT With Related or Unrelated Donor for Heme Disorders

Who Can Participate

Age: 0 - 60Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age 60 years or younger
  • Karnofsky or Lansky performance status of 70% or higher
  • Voluntary written consent given by participant or guardian
  • Adequate kidney function: creatinine less than twice normal or creatinine clearance at least 40 ml/min/1.73m2
  • Adequate liver function: bilirubin, AST, alkaline phosphatase less than 4 times normal
  • Adequate lung function: oxygen diffusion capacity over 50% predicted or oxygen saturation over 95% on room air for children unable to do lung tests
  • Adequate heart function: no severe heart failure, no uncontrolled arrhythmia, and ejection fraction over 45%
  • HIV infection allowed only if viral load is undetectable and managed by infectious disease specialists
  • Agreement to use birth control if sexually active with potential to conceive
  • Sufficient donor graft availability
  • Specific disease remission or risk criteria for various leukemias, lymphomas, myeloproliferative neoplasms, myelodysplasia, and bone marrow failure syndromes
  • Patients not eligible for higher priority protocols
Not Eligible

You will not qualify if you...

  • Chemotherapy refractory large cell and high grade non-Hodgkin lymphoma with progressive disease after two or more salvage treatments
  • Chronic myelogenous leukemia in blast crisis phase
  • Large cell lymphoma, mantle cell lymphoma, and Hodgkin disease progressing on salvage therapy
  • Evidence of disease progression by imaging or biopsy
  • Active central nervous system malignancy
  • Prior myeloablative transplant within 6 months for patients 18 years or younger; for older patients, prior myeloablative allotransplant or autologous transplant
  • Active HIV infection or positive HIV serology without viral control
  • Active uncontrolled infection
  • Pregnant or breastfeeding females; females must have negative pregnancy test before treatment

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - Approximately 100 days of treatment and medication tapering

Participants receive myeloablative allogeneic hematopoietic stem cell transplantation with either a Total Body Irradiation (TBI) or Non-TBI regimen, followed by immunosuppressive medications to prevent graft-versus-host disease.

Multiple visits during the first 100 days after transplantation

Follow-up

Duration - Up to 2 years

Participants are monitored for graft-versus-host disease, relapse, survival, and engraftment up to 2 years after transplantation.

Regular follow-up visits during 2 years post-treatment

Trial Site Locations

Total: 1 location

1

Masonic Cancer Center at University of Minnesota

Minneapolis, Minnesota, United States, 55337

Actively Recruiting

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Research Team

T

Tamy Grainger

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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