NCT07493538

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
132

participants targeted

Target at P75+ for phase_2

Timeline
106mo left

Started Apr 2026

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress2%
Apr 2026Mar 2035

First Submitted

Initial submission to the registry

March 20, 2026

Completed
5 days until next milestone

First Posted

Study publicly available on registry

March 25, 2026

Completed
16 days until next milestone

Study Start

First participant enrolled

April 10, 2026

Completed
3.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2030

Expected
5 years until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2035

Last Updated

April 16, 2026

Status Verified

April 1, 2026

Enrollment Period

3.9 years

First QC Date

March 20, 2026

Last Update Submit

April 13, 2026

Conditions

Myelodysplastic SyndromesAMLMDSAcute LeukemiaAcute Myeloid Leukemia

Outcome Measures

Primary Outcomes (1)

  • Overall Survival

    Evaluate rates of overall survival at 100 days after transplant.

    Day 100

Secondary Outcomes (2)

  • Transplant Related Mortality

    Day 100 and 1 year

  • Overall Survival

    1 and 2 years

Study Arms (1)

Treo/Flu with PTCy

EXPERIMENTAL

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.

Drug: TreosulfanDrug: FludarabineRadiation: Total Body IrradiationDrug: TacrolimusDrug: Mycophenolate MofetilDrug: CyclophosphamidBiological: Stem Cell Infusion

Interventions

FludarabineDRUG

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 .

Treo/Flu with PTCy
Total Body IrradiationRADIATION

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

Also known as: TBI
Treo/Flu with PTCy
TacrolimusDRUG

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.

Treo/Flu with PTCy
Mycophenolate MofetilDRUG

All patients begin mycophenolate mofetil (MMF) day +5 through day +35 if no acute GVHD or 7 days after engraftment, whichever is later.

Also known as: MMF
Treo/Flu with PTCy
CyclophosphamidDRUG

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).

Treo/Flu with PTCy
Stem Cell InfusionBIOLOGICAL

Given on day 0.

Treo/Flu with PTCy
TreosulfanDRUG

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

Treo/Flu with PTCy

Eligibility Criteria

Age2 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Patients 2-75 years of age
  • /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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Masonic Cancer Center at University of Minnesota

Minneapolis, Minnesota, 55455, United States

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, AcuteMyelodysplastic Syndromes

Interventions

treosulfanfludarabineWhole-Body IrradiationTacrolimusMycophenolic AcidCyclophosphamide

Condition Hierarchy (Ancestors)

Leukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Diseases

Intervention Hierarchy (Ancestors)

RadiotherapyTherapeuticsInvestigative TechniquesMacrolidesLactonesOrganic ChemicalsCaproatesAcids, AcyclicCarboxylic AcidsFatty AcidsLipidsPhosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsPhosphoramidesOrganophosphorus Compounds

Central Study Contacts

Christopher Graham, MD

CONTACT

612-625-3051grah0329@umn.edu

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 20, 2026

First Posted

March 25, 2026

Study Start

April 10, 2026

Primary Completion (Estimated)

March 1, 2030

Study Completion (Estimated)

March 1, 2035

Last Updated

April 16, 2026

Record last verified: 2026-04

Locations

US(1)