RecruitingInterventionalPhase 2

MT2025-35 Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning Treosulfan and Fludarabine, With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases

NCT ID: NCT07493538Sponsor: Masonic Cancer Center, University of MinnesotaLast updated: 2026-04-16

Summary

This is a Phase II study following subjects proceeding with Treosulfan (36g/m2) preparative regimen followed by a related, unrelated, or partially matched family donor stem cell infusion, with post-transplant cyclophosphamide (PTCy) at 40mg/kg, tacrolimus and MMF for GVHD prophylaxis.

Arms & interventions

DrugTreosulfan
12 g/m2 administered intravenously over 2 hours on days -4, -3, and -2.
DrugFludarabine
Fludarabine will be administered intravenously over 1 hour, every 24 hours on days -6 to -2. The daily dose of fludarabine will be determined by model-based dosing utilizing Bayesian methodology .
RadiationTotal Body Irradiation
TBI 200 cGy will be administered as a single treatment on day -1 per current institutional guidelines.
DrugTacrolimus
Tacrolimus may be initiated on day +5 either PO or IV gtt , with a goal trough level of 5-10mg/mL and avoiding higher levels for the first two weeks post-transplant, as recent evidence demonstrated increased adverse events for levels over 10 mg/mL.
DrugMycophenolate Mofetil
All patients begin mycophenolate mofetil (MMF) day +5 through day +35 if no acute GVHD or 7 days after engraftment, whichever is later.
DrugCyclophosphamid
Cyclophosphamide 40 mg/kg will be given as an IV infusion over 1-2 hours (depending on volume) on Days +3 post-transplant (between 60 and 72 hours after stem cell infusion) and on Day +4 post-transplant (approximately 24 hours after Day +3 cyclophosphamide).
BiologicalStem Cell Infusion
Given on day 0.

Outcome measures

Primary

Overall Survival
Evaluate rates of overall survival at 100 days after transplant.
Time frame: Day 100

Secondary

Transplant Related Mortality
Time frame: Day 100 and 1 year
Overall Survival
Time frame: 1 and 2 years

Eligibility criteria

Sex: AllAge: 2 Years to 75 YearsHealthy volunteers: No

Inclusion Criteria: * Patients 2-75 years of age * ≤7 5 years of age: Karnofsky score ≥ 70% (≥ 16 years) or Lansky play score ≥ 50 (\< 16 years) with appropriate organ criteria as below (in other inclusion criteria) * 5/6 or 6/6 related donor, OR a 5-8/8 HLA-A, B, C, DRB1 allele match unrelated donor, OR a haplotype (at least 5/10) related donor * adequate liver (no decompensated liver failure, Child Pugh A, AST/ALT \<5X ULN) and renal function (creatinine \<2.0) * absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction ≥ 40% * DLCO FEV1, FVC ≥ 40% predicted, and absence of O2 requirement Exclusion Criteria: * Pregnant or breastfeeding * Evidence of untreated/uncontrolled HIV infection * Untreated active serious infection * Active CNS malignancy * CML in blast crisis not in a complete remission by abnormal blast count. * Less than 3 months since prior myeloablative transplant

Study locations (1)

Masonic Cancer Center at University of Minnesota

Minneapolis, Minnesota, 55455

Recruiting