AMLPhase 2NCT06973668
A Randomized Study to Compare Post-transplant Cyclophosphamide, Sirolimus, Ruxolitinib and Post-transplant Cyclophosphamide, Sirolimus, Mycophenolate Mofetil to Prevent Graft Versus Host Disease
A Phase 2 study testing Mycophenolate Mofetil and Ruxolitinib in people with AML, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
The goal of this clinical research study is to compare the effects of these drug combinations (cyclophosphamide, sirolimus, and MMF vs cyclophosphamide, sirolimus, and ruxolitinib) on the prevention of GVHD after a stem cell transplant. Read more about this study
- Who is it for?
- Ages 65 to 75 years
- Conditions the study is for
- AML
- What is being tested?
- Mycophenolate Mofetil and Ruxolitinib
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 80 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Jul 2025to 31 Jan 2030
- 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 65 to 75 years
- All sexes
- No healthy volunteers
- Older adult
Who may be able to join
- Age ≥ 65 and < 75 years are eligible if they have one of the following diseases.
- Acute Myeloid Leukemia
- Myelodysplastic syndrome
- Chronic myelomonocytic leukemia
- Available HLA-identical or haploidentical related donor or a 7/8 or 8/8 HLA matched unrelated donor.
- Peripheral blood stem cells as a graft source
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- Subject must voluntarily sign an informed consent.
- Adequate organ function per local laboratory reference range as follows: - Aspartate transaminase (AST) and alanine transaminase (ALT) < 3.0X ULN - Total Bilirubin <1.5 x ULN (unless bilirubin rise is due to Gilbert's syndrome or of nonhepatic origin) - Subject must have adequate renal function as demonstrated by a creatinine clearance ≥ 40 mL/min/1.73 m2 (as reported in epic using 2021 CKD-EPI creatinine equation)
- DLCO corrected for Hgb, if applicable) ≥ 50% of predicted
- Ejection Fraction ≥ 50%
- The effects on the developing human fetus are unknown. For this reason and as well as other therapeutic agents used in this trial are known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. (Refer to Pregnancy Assessment Policy MD Anderson Institutional Policy # CLN1114). This includes all female patients, between the onset of menses (as early as 8 years of age) and 55 years unless the patient presents with an applicable exclusionary factor which may be one of the following:
- Postmenopausal (no menses in greater than or equal to 12 consecutive months).
- History of hysterectomy or bilateral salpingo-oophorectomy.
- Ovarian failure (Follicle Stimulating Hormone and Estradiol in menopausal range, who have received Whole Pelvic Radiation Therapy).
- History of bilateral tubal ligation or another surgical sterilization procedure.
- Approved methods of birth control are as follows: Hormonal contraception (i.e. birth control pills, injection, implant, transdermal patch, vaginal ring), Intrauterine device (IUD), Tubal Ligation or hysterectomy, Subject/Partner post vasectomy, Implantable or injectable contraceptives, and condoms plus spermicide. Not engaging in sexual activity for the total duration of the trial and the drug washout period is an acceptable practice; however periodic abstinence, the rhythm method, and the withdrawal method are not acceptable methods of birth control. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
- Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of study agent administration.
Who cannot join
- Subject is known to be positive for HIV.
- Subject has acute promyelocytic leukemia.
- Subject has known active CNS involvement with AML.
- Hematopoietic Cell Transplantation Comorbidity Index (HCT-CI) score of >5
- Evidence of other clinically significant uncontrolled condition(s) including, but not limited to:
- Uncontrolled and/or active systemic infection (viral, bacterial or fungal)
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- Chronic hepatitis B virus (HBV) or hepatitis C (HCV) requiring treatment. Note: subjects with serologic evidence of prior vaccination to HBV (i.e. hepatitis B surface (HBs) antigen negative-, anti-HBs antibody positive and anti-hepatitis B core (HBc) antibody negative) or subjects with positive anti-HBc antibody but negative Hep B DNA may participate.
- Cardiac history of CHF requiring treatment or Ejection Fraction < 50% or unstable angina or MI within 1 year of study entry
- Major adverse cardiac events such as MI/stroke and pulmonary embolism (PE)/deep vein thrombosis (DVT) within 6 months. Recent history of Central line-associated DVT may be allowed after discussion with PI.
- Current and/or history of active TB
- White Blood Cell count > 25 X 109 /L.
- Pregnant women are excluded from this study because the study agent has unknown potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother, breastfeeding should be discontinued if the mother is treated with the study agent. These potential risks may also apply to other agents used in this study.
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
The University of Texas M. D. Anderson Cancer Center
Houston, Texas, 77030
Uday Popat, MBA,MD, 713-563-0812, upopat@mdanderson.org
Uday Popat, MBA,MD
About this study
The goal of this clinical research study is to compare the effects of these drug combinations (cyclophosphamide, sirolimus, and MMF vs cyclophosphamide, sirolimus, and ruxolitinib) on the prevention of GVHD after a stem cell transplant.
Read the detailed description
Primary Objective:
To compare Grade 2-4 acute graft versus host disease-free survival (GFS) between the MMF and Ruxolitinib arms
Secondary Objectives:
To compare the following between treatment arms:
- Grade 3-5 adverse event of this regimen as per CTCAE v5.0 criteria.
- Graft versus host disease-free, relapse-free survival (GRFS)
- Time to neutrophil and platelet engraftment
- Incidence of acute and chronic GVHD
- Relapse incidence
- Non relapse mortality
- Overall survival
- Progression-free survival
- Chimerism
- Immunosuppression cessation time
- Rate of graft failure
Exploratory Objective:
To conduct mechanistic studies to compare immune recovery between arms and correlate these with disease control and GVHD prophylaxis.
- Conditions
- AML
- Being tested
- Mycophenolate Mofetil, Ruxolitinib
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
15 mg/kg/dose (max: 1,000mg/dose) IV/PO three times daily
RuxolitinibDrug
Also called Jakafi®
5 mg PO every 12 hours
| Study group | Role | What they receive |
|---|---|---|
| 01A:Treatment With Post-Transplant Cyclophosphamide (PTCy), Sirolimus and MMF | Experimental | 15 mg/kg/dose (max: 1,000mg/dose) IV/PO three times daily Receives: Drug: Mycophenolate Mofetil |
| 01B:Treatment With Post-Transplant Cyclophosphamide (PTCy),Sirolimus and Ruxolitinib | Experimental | 5 mg PO every 12 hours Receives: Drug: Ruxolitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Adverse Events (AEs)
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Measured: Through study completion; an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 May 2025
- Study start22 Jul 2025
- Primary completion31 Jan 2028 (estimated)
- Study completion31 Jan 2030 (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 M.D. Anderson Cancer CenterThe 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
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Uday Popat, MBA,MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06973668
- Sponsor's protocol ID
- 2024-1940
- Other IDs
- NCI-2025-03239
- Status last verified
- May 2026
- First submitted
- 6 May 2025
- Last updated
- 20 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Study to Compare Post-transplant Cyclophosphamide, Sirolimus, Ruxolitinib and Post-transplant Cyclophosphamide, Sirolimus, Mycophenolate Mofetil to Prevent Graft Versus Host Disease
- ClinicalTrials.gov ID
- NCT06973668
- Acronym / sponsor ID
- 2024-1940
- Conditions
- AML
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 22 Jul 2025 (actual)
- Primary completion
- 31 Jan 2028 (estimated)
- Study completion
- 31 Jan 2030 (estimated)
- First posted
- 15 May 2025 (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.
The goal of this clinical research study is to compare the effects of these drug combinations (cyclophosphamide, sirolimus, and MMF vs cyclophosphamide, sirolimus, and ruxolitinib) on the prevention of GVHD after a stem cell transplant.
Detailed description
Primary Objective:
To compare Grade 2-4 acute graft versus host disease-free survival (GFS) between the MMF and Ruxolitinib arms
Secondary Objectives:
To compare the following between treatment arms:
- Grade 3-5 adverse event of this regimen as per CTCAE v5.0 criteria.
- Graft versus host disease-free, relapse-free survival (GRFS)
- Time to neutrophil and platelet engraftment
- Incidence of acute and chronic GVHD
- Relapse incidence
- Non relapse mortality
- Overall survival
- Progression-free survival
- Chimerism
- Immunosuppression cessation time
- Rate of graft failure
Exploratory Objective:
To conduct mechanistic studies to compare immune recovery between arms and correlate these with disease control and GVHD prophylaxis.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 01A:Treatment With Post-Transplant Cyclophosphamide (PTCy), Sirolimus and MMF | Experimental | Drug: Mycophenolate Mofetil15 mg/kg/dose (max: 1,000mg/dose) IV/PO three times daily |
| 01B:Treatment With Post-Transplant Cyclophosphamide (PTCy),Sirolimus and Ruxolitinib | Experimental | Drug: Ruxolitinib5 mg PO every 12 hours |
Who may qualify
- Ages
- 65 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Older adult
Key inclusion criteria (17 listed)
- Age ≥ 65 and < 75 years are eligible if they have one of the following diseases.
- Available HLA-identical or haploidentical related donor or a 7/8 or 8/8 HLA matched unrelated donor.
- Peripheral blood stem cells as a graft source
- Subject must voluntarily sign an informed consent.
- Adequate organ function per local laboratory reference range as follows: - Aspartate transaminase (AST) and alanine transaminase (ALT) < 3.0X ULN - Total Bilirubin <1.5 x ULN (unless bilirubin rise is due to Gilbert's syndrome or of nonhepatic origin) - Subject must have adequate renal function as demonstrated by a creatinine clearance ≥ 40 mL/min/1.73 m2 (as reported in epic using 2021 CKD-EPI creatinine equation)
Key exclusion criteria (12 listed)
- Subject is known to be positive for HIV.
- Subject has acute promyelocytic leukemia.
- Subject has known active CNS involvement with AML.
- Hematopoietic Cell Transplantation Comorbidity Index (HCT-CI) score of >5
- Evidence of other clinically significant uncontrolled condition(s) including, but not limited to:
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 |
|---|---|---|
| Mycophenolate Mofetil15 mg/kg/dose (max: 1,000mg/dose) IV/PO three times daily | Drug | 01A:Treatment With Post-Transplant Cyclophosphamide (PTCy), Sirolimus and MMF |
| RuxolitinibAlso: Jakafi®5 mg PO every 12 hours | Drug | 01B:Treatment With Post-Transplant Cyclophosphamide (PTCy),Sirolimus and Ruxolitinib |
Primary outcome
Safety and Adverse Events (AEs)
Time frame: Through study completion; an average of 1 year
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
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 20 May 2026.
- MD Anderson Cancer Center WebsiteLink provided by the sponsor.
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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.
Uday Popat, MBA,MD principal investigator
M.D. Anderson Cancer Center
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 Mycophenolate Mofetil and Ruxolitinib in people with AML, run by M.D. Anderson Cancer Center 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 17 inclusion and 12 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 20 May 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 Uday Popat, MBA,MD ((713) 563-0812, upopat@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and Adverse Events (AEs) (time frame: Through study completion; an average of 1 year). 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 20 May 2026.