AMLPhase 2NCT07493538
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
A Phase 2 study testing Treosulfan, Fludarabine and Total Body Irradiation in people with AML and MDS, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 75 years
- Conditions the study is for
- AML, MDS, Acute Leukemia
- What is being tested?
- Treosulfan, Fludarabine and Total Body Irradiation
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 132 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Apr 2026to Mar 2035
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Masonic Cancer Center at University of Minnesota
Minneapolis, Minnesota, 55455
About this study
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
- Being tested
- Treosulfan, Fludarabine, Total Body Irradiation, Tacrolimus, Mycophenolate Mofetil
- Medical subject headings
- Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TreosulfanDrug
12 g/m2 administered intravenously over 2 hours on days -4, -3, and -2.
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 .
Total Body IrradiationRadiation
Also called TBI
TBI 200 cGy will be administered as a single treatment on day -1 per current institutional guidelines.
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.
Also called MMF
All patients begin mycophenolate mofetil (MMF) day +5 through day +35 if no acute GVHD or 7 days after engraftment, whichever is later.
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).
Stem Cell InfusionBiological
Given on day 0.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Treosulfan; Drug: Fludarabine; Radiation: Total Body Irradiation; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Drug: Cyclophosphamid; Biological: Stem Cell Infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Survival
Evaluate rates of overall survival at 100 days after transplant.
Measured: Day 100
Secondary outcomes (2)
Transplant Related Mortality
● Estimate transplant related mortality (TRM) at 100 days and 1 year with this conditioning and GVHD prophylaxis combination
Measured: Day 100 and 1 year
Overall Survival
Estimate overall survival at 1 and 2 years
Measured: 1 and 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2026
- Study start10 Apr 2026
- Primary completionMar 2030 (estimated)
- Study completionMar 2035 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AMLStudies of the same condition, open and under way.
- More trials run by Masonic Cancer Center, University of MinnesotaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 132 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07493538
- Sponsor's protocol ID
- 2025LS162
- Status last verified
- Apr 2026
- First submitted
- 20 Mar 2026
- Last updated
- 16 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07493538
- Acronym / sponsor ID
- 2025LS162
- Conditions
- AML, MDS, Acute Leukemia, Acute Myeloid Leukemia, Myelodysplastic Syndromes
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 132 participants (estimated)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- 10 Apr 2026 (actual)
- Primary completion
- Mar 2030 (estimated)
- Study completion
- Mar 2035 (estimated)
- First posted
- 25 Mar 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treo/Flu with PTCy | Experimental | Drug: Treosulfan, Drug: Fludarabine, Radiation: Total Body Irradiation, Drug: Tacrolimus, Drug: Mycophenolate Mofetil, Drug: Cyclophosphamid, Biological: Stem Cell InfusionSubjects 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. |
Who may qualify
- Ages
- 2 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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%
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Treosulfan12 g/m2 administered intravenously over 2 hours on days -4, -3, and -2. | Drug | Treo/Flu with PTCy |
| FludarabineFludarabine 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 . | Drug | Treo/Flu with PTCy |
| Total Body IrradiationAlso: TBITBI 200 cGy will be administered as a single treatment on day -1 per current institutional guidelines. | Radiation | Treo/Flu with PTCy |
| TacrolimusTacrolimus 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 | Treo/Flu with PTCy |
| Mycophenolate MofetilAlso: MMFAll patients begin mycophenolate mofetil (MMF) day +5 through day +35 if no acute GVHD or 7 days after engraftment, whichever is later. | Drug | Treo/Flu with PTCy |
| CyclophosphamidCyclophosphamide 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). | Drug | Treo/Flu with PTCy |
| Stem Cell InfusionGiven on day 0. | Biological | Treo/Flu with PTCy |
Primary outcome
Overall Survival
Time frame: Day 100
Evaluate rates of overall survival at 100 days after transplant.
2 secondary outcomes
Transplant Related Mortality
Time frame: Day 100 and 1 year
Overall Survival
Time frame: 1 and 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Apr 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Treosulfan, Fludarabine and Total Body Irradiation in people with AML and MDS, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Christopher Graham, MD (612-625-3051, grah0329@umn.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Survival (time frame: Day 100). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Apr 2026; AzTrials last refreshed its copy on 1 Nov 2026.