Recruiting
Phase 2

Treosulfan with PTCy

Sponsor:

Masonic Cancer Center, University of Minnesota

Code:

NCT07493538

Conditions

AML

MDS

Acute Leukemia

Acute Myeloid Leukemia

Myelodysplastic Syndromes

Eligibility Criteria

Sex: All

Age: 2 - 70+

Healthy Volunteers: Not accepted

Interventions

Treosulfan

Fludarabine

Total Body Irradiation

Tacrolimus

Mycophenolate Mofetil

Study Details

Brief 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.

Conditions

AML

MDS

Acute Leukemia

Acute Myeloid Leukemia

Myelodysplastic Syndromes

Study ID

NCT07493538

Start date

Apr 10, 2026

Status verified date

Apr, 2026

Completion date

Mar, 2035

Anticipated

Primary completion date

Mar, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 70+

Healthy Volunteers: Not accepted

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 Design

Enrollment

132 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Treo/Flu with PTCy

Subjects treated with Treosulfan and Fludarabine preparative regimen with TBI for AML and MDS patients followed by a related or unrelated donor stem cell infusion utilizing PTCy, tacrolimus and MMF as GVHD prophylaxis.

Interventions

Treosulfan

12 g/m2 administered intravenously over 2 hours on days -4, -3, and -2.

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 .

Total Body Irradiation

TBI 200 cGy will be administered as a single treatment on day -1 per current institutional guidelines.

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.

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.

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).

Stem Cell Infusion

Given on day 0.

Primary outcome measure

  • Overall Survival [ Time Frame: Day 100 ]

Central Contacts and Locations

Central contacts

Christopher Graham, MD

612-625-3051grah0329@umn.edu

Locations

Masonic Cancer Center at University of Minnesota

Recruiting

Minneapolis, Minnesota, United States, 55455

More Information

Sponsor

Masonic Cancer Center, University of Minnesota

Last update posted

Apr 16, 2026

Last verified

Apr, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Masonic Cancer Center, University of Minnesota on 2026-04-16.