Actively Recruiting

Phase 2
Age: 0 - 75Years
All Genders
ID05805605

Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning With Post-Transplant Cyclophosphamide for Treating Hematological Diseases

Led by Masonic Cancer Center, University of Minnesota · Updated on 2025-07-01

56

Participants Needed

1

Research Sites

52 weeks

Total Duration

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AI-Summary

What this Trial Is About

Researchers are evaluating a Phase II study focused on patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT) using a reduced intensity conditioning (RIC) regimen. This regimen includes cyclophosphamide, fludarabine, and total body irradiation (TBI) followed by stem cell infusion from related, unrelated, or partially matched family donors. The study aims to assess the outcomes of this treatment approach, including rates of acute and chronic graft-versus-host disease (GVHD) over 12 months. The treatment involves a preparative regimen with cyclophosphamide, fludarabine, and a single fraction of total body irradiation given prior to transplant. On day 0, patients receive an infusion of peripheral blood stem cells or bone marrow cells according to donor preference. After transplant, patients receive post-transplant cyclophosphamide, sirolimus starting from day +5 through day +60 with a loading dose and maintenance dosing, and mycophenolate mofetil from day +5 through day +35. Dosing varies by patient characteristics such as weight and age. Participants will be closely monitored throughout the study with evaluations of acute and chronic GVHD at 12 months, relapse rates at 100 days, overall survival up to 72 months, and transplant-related mortality at 12 months. Organ function, infection status, and other safety parameters will be assessed. The study includes ongoing follow-up to observe treatment effects and safety outcomes over time.

CONDITIONS

Brief Title

Allo HSCT Using RIC and PTCy for Hematological Diseases

Who Can Participate

Age: 0 - 75Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age 0 to 75 years with Karnofsky score 270% if 16 years or older, or Lansky score 250 if under 16 years
  • Suitable donor: 5/6 or 6/6 related donor, 7-8/8 HLA matched donor, or haplotype matched related donor
  • Diagnosed with eligible hematological diseases including certain leukemias, lymphomas, myelodysplastic syndromes, myeloproliferative neoplasms, and related disorders
  • For acute leukemias, must be in remission by morphology and without minimal residual disease by certain tests
  • Must meet disease-specific remission and risk criteria as detailed in the study
  • Adequate organ function including liver, kidney, heart, and lung function within specified limits
  • HIV infection allowed if viral load undetectable and management plan established
  • Females and males of childbearing potential must agree to use birth control during treatment
  • Voluntary written consent obtained
Not Eligible

You will not qualify if you...

  • Pregnant or breastfeeding women
  • Untreated active infections
  • Active central nervous system malignancy
  • CML in blast crisis phase
  • Progressive intermediate or high grade non-Hodgkin lymphoma, mantle cell lymphoma, or Hodgkin disease on salvage therapy
  • Less than 3 months since prior myeloablative transplant
  • Evidence of progressive disease by imaging or biopsy, except certain PET activity without CT changes
  • Other conditions or features that may increase risk or interfere with treatment as per study protocol

AI-Screening

AI-Powered Screening

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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)

Reduced Intensity Conditioning

Duration - 8 days

Participants receive conditioning treatment including allopurinol from day -7 to day 0, fludarabine from day -6 to day -2, cyclophosphamide on day -6, and total body irradiation on day -1 to prepare for stem cell transplant.

Daily visits for treatment administration from day -7 to day 0

Stem Cell Transplant

Duration - 1 day

Participants receive peripheral blood stem cell or bone marrow cell transplant on day 0.

1 visit (in-person) on transplant day

Post-Transplant Cyclophosphamide and Immunosuppression

Duration - 31 days

Participants receive cyclophosphamide on days +3 and +4, and begin immunosuppressive medications including sirolimus and mycophenolate mofetil from day +5 through day +35 to prevent graft-versus-host disease.

Visits on days +3, +4, and daily or frequent visits from day +5 to day +35

Immunosuppression Maintenance

Duration - 56 days

Participants continue sirolimus treatment daily from day +5 to day +60 to maintain immunosuppression.

Regular visits during days +5 to +60

Follow-up and Monitoring

Duration - Up to 12 months

Participants are monitored for transplant outcomes including graft-versus-host disease, relapse, and survival for up to 12 months and beyond.

Regular follow-up visits during the first year after transplant

Trial Site Locations

Total: 1 location

1

Masonic Cancer Center at University of Minnesota

Minneapolis, Minnesota, United States, 55455

Actively Recruiting

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Research Team

M

Mark Juckett

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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