NCT07493538 · Masonic Cancer Center, University of Minnesota
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
What this study is about
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.
View original scientific description
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.
Interventions
DRUG
Treosulfan
12 g/m2 administered intravenously over 2 hours on days -4, -3, and -2.
DRUG
Fludarabine
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 .
RADIATION
Total Body Irradiation
TBI 200 cGy will be administered as a single treatment on day -1 per current institutional guidelines.
DRUG
Tacrolimus
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.
DRUG
Mycophenolate Mofetil
All patients begin mycophenolate mofetil (MMF) day +5 through day +35 if no acute GVHD or 7 days after engraftment, whichever is later.
DRUG
Cyclophosphamid
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).
BIOLOGICAL
Stem Cell Infusion
Given on day 0.
Primary outcome measures
Overall Survival
Time frame: Day 100
Evaluate rates of overall survival at 100 days after transplant.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
Where
- Minneapolis, Minnesota
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 16, 2026 · Source of record for eligibility and locations