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Acute Myeloid LeukemiaPhase 2NCT03286530

Ruxolitinib + Allogeneic Stem Cell Transplantation in AML

A Phase 2 study testing Ruxolitinib in people with Acute Myeloid Leukemia and Acute Myeloid Leukemia in Remission, run by Massachusetts General Hospital at 6 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

This research study is studying a drug that may help decrease the chances of relapse after Allogeneic Stem Cell transplantation for Acute Myeloid Leukemia. The name of the study drug involved in this study is: Ruxolitinib Read more about this study

Who is it for?
Ages 60 to 80 years
What is being tested?
Ruxolitinib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
64 participants
Where
6 sitesUnited States
Study dates
3 Nov 2017to Dec 2026
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 60 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Participants must have pathologically confirmed AML in CR1 as defined by:
  • Bone marrow biopsy with < 5% blasts
  • No clusters or collections of blast cells
  • No extramedullary leukemia
  • Absolute neutrophil count ≥ 1000/µL (achieved post-induction at some point)
  • Please note that full platelet recovery is not necessary, and thus, patients achieving CRp are eligible.
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  • ---Or participants have pathologically confirmed MDS as defined by:
  • Bone marrow biopsy with <10% blasts
  • Patients receiving MDS-directed therapy must be off treatment for > 2 weeks prior to start of conditioning.
  • Participants must be designated to undergo reduced intensity allogeneic peripheral blood hematopoietic stem cell transplantation (HCT). Consent will be obtained prior to admission for HCT. The following HCT conditions must be planned:
  • Donors must be 8/8 HLA-matched (at the allele level) as defined by matching at HLA-A, -B, -DR and -C who pass institutional standard to serve as a peripheral blood stem cell donor
  • Donor grafts must be G-CSF mobilized peripheral blood stem cells with dose and apheresis logistics at the discretion of institutional standard
  • Conditioning therapy will be one of the following 3 options:
  • Fludarabine / Melphalan where fludarabine is ≥ 90 mg/m2 IV total dose and melphalan is 100-140 mg/m2 IV total dose. Exact logistics of administration are at the discretion of institutional standard.
  • Fludarabine / Busulfan where fludarabine is ≥ 90 mg/m2 IV total dose and busulfan = 6.4 mg/kg IV total dose. Exact logistics of administration are at the discretion of institutional standard.
  • Fludarabine / Busulfan where fludarabine is ≥ 90 mg/m2 IV total dose and busulfan is dosed to achieve AUC of 4000 µmol/min based on a pharmacokinetics determined from a test dose. Exact logistics are at the discretion of institutional standard.
  • GVHD prophylaxis is comprised of tacrolimus / short course methotrexate as defined by tacrolimus started prior to day 0 of HCT and methotrexate given after HCT on days +1, +3 and +6 ± +11 at a dose of 5-10 mg/m2 IV. Exact logistics are at the discretion of the treating institution.
  • Age ≥ 60 and ≤ 80 years old
  • ECOG performance status 0-2
  • Male participants must agree to use an acceptable method for contraception during the entire study treatment period and through 6 months after the last dose of treatment.
  • Ability to understand and the willingness to sign a written informed consent document

Who cannot join

  • Have had a prior allogeneic HSCT.
  • Patients without normal organ function defined as follows:
  • AST (SGOT), ALT (SGPT) and Alkaline Phosphatase >3 × institutional Upper Limit of Normal (ULN)
  • Direct bilirubin >2.0 mg/dL
  • Adequate renal function as defined by calculated creatinine clearance ≤ 40 mL/min (Cockcroft-Gault formula)
  • Have a history of other malignancy(ies) unless:
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  • They have been disease-free for at least 5 years and are deemed by the treating investigator to be at low risk for recurrence of that malignancy,
  • --- or
  • The only cancer they have had is cervical cancer in situ, or basal cell or squamous cell carcinoma of the skin
  • Have a chronic or active infection that requires systemic antibiotics, antifungal or antiviral treatment.
  • Have current or a history of congestive heart failure New York Heart Association (NYHA) class 3 or 4, or any history of documented diastolic or systolic dysfunction (LVEF < 40%, as measured by MUGA scan or echocardiogram)
  • Have an uncontrolled intercurrent illness including, but not limited to, ongoing infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
  • Have active uncontrolled infection. An active uncontrolled infection is defined as hemodynamic instability attributable to sepsis or new symptoms, worsening physical signs, or radiographic findings attributable to infection. Persisting fever without signs or symptoms will not be interpreted as an active uncontrolled infection.
  • Be HIV-positive
  • Have a systemic infection requiring IV antibiotic therapy, nor any other severe infection
  • Planned use of ex vivo or in vivo T-cell depletion
  • Have current or a history of ventricular or life-threatening arrhythmias or diagnosis

Trial locations and contacts

6 sites in 1 country.

6 sites in 1 country

United States6 sites

  • Beth Israel Deaconess Medical Center

    Boston, Massachusetts, 02115

  • Massachusetts General Hospital

    Boston, Massachusetts, 02115

  • The Ohio State University

    Columbus, Ohio, 43210

  • Vanderbilt University

    Nashville, Tennessee, 37235

  • Washington University

    St Louis, Missouri, 63130

  • Medical College of Wisconsin

    Wauwatosa, Wisconsin, 53226

Other trials in United States

About this study

Official title: Phase II Study of Maintenance Ruxolitinib After Allogeneic Stem Cell Transplantation for Older Patients With Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) in Complete Remission

This research study is studying a drug that may help decrease the chances of relapse after Allogeneic Stem Cell transplantation for Acute Myeloid Leukemia. The name of the study drug involved in this study is:

  • Ruxolitinib
Read the detailed description

This research study is a Phase II clinical trial. Phase II clinical trials test the safety and effectiveness of an investigational drug to learn whether the drug works in treating a specific disease. "Investigational" means that the drug is being studied.

The FDA (the U.S. Food and Drug Administration) has not approved ruxolitinib for this specific disease but it has been approved for other blood diseases.

In this research study, investigators are trying to discover if ruxolitinib will decrease chances of relapse after having an allogeneic stem cell transplantation.

Ruxolitinib is a medication that blocks certain proteins called tyrosine kinases. Specifically, it blocks tyrosine kinases called JAK2. Many cancers have over active "cell signaling." What this means is that certain functions in the cancer cells never turn off and this makes them grow in an uncontrolled way. Ruxolitinib, shuts down the pathway that depends on the JAK2 tyrosine kinases. The JAK2 pathway is over active with acute myeloid leukemia. Ruxolitinib has also been shown to lower the rates of graft versus host disease, a complication of transplant. The exact way ruxolitinib does this is not yet clear but it may have to do with its ability to block the JAK2 pathway since this pathway can also lead to inflammation in the body.

Being tested
Ruxolitinib
Keywords
Acute Myeloid Leukemia, Acute Myeloid Leukemia in Remission, Allogenic Stem Cell Transplantation
Medical subject headings
Leukemia, Myeloid, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • RuxolitinibDrug

    Also called Jakafi

    Patients who fulfill eligibility criteria will be entered into the trial to receive Ruxolitinib.

    After the screening procedures confirm participation in the research study. The participant will be given a drug diary. The participant will be asked to document information in the drug diary about the study treatment.

Study groups in NCT03286530
Study groupRoleWhat they receive
RuxolitinibExperimentalFollowing a standard of care allogeneic stem cell transplantation, participants will be started on Ruxolitinib. Ruxolitinib is administered orally 2 times per day at a fixed dose. Each study treatment cycle lasts 28 days. Up to 24 cycles.
Receives: Drug: Ruxolitinib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 1-year GVHD/relapse free survival rate (GRFS rate)

    The number of participants surviving after one year that have not experienced graft-versus-host disease (GVHD) or graft relapse (GRFS rate).

    Measured: 1 Year

Secondary outcomes (5)

  1. Progression Free Survival

    Kaplan-Meier estimates of progression free survival (PFS) will be calculated, with patients without an event being censored at last date of contact

    Measured: Until disease progression or death from any cause, approximately 5 years

  2. Overall Survival

    Overall survival is measured as the time from the hematopoietic stem cell transplantation (HSCT) until death. Participants without an event will be censored at the date of last contact.

    Measured: Until death, approximately 5 years

  3. Cumulative incidence of drug related toxicities

    Cumulative incidence of treatment related adverse events as assessed by Common Terminology Criteria for Adverse Events (CTCAE 4). Early deaths from all other causes are considered a competing risk.

    Measured: 2 Years

  4. Time to Relapse

    The amount of time from the hematopoietic stem cell transplantation (HSCT) until disease relapse. Relapse is the recurrence of cancer after having a bone marrow biopsy without evidence of cancer. Time to treatment-related mortality is considered a competing risk.

    Measured: 2 Years

  5. Time to treatment-related mortality (TRM)

    The amount of time between receiving the HSCT and death due to a treatment related cause. Time to relapse is considered a competing risk.

    Measured: 2 Years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Sep 2017
  2. Study start3 Nov 2017
  3. Primary completionDec 2026 (estimated)
  4. Study completionDec 2026 (estimated)

Resources

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
64 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03286530
Sponsor's protocol ID
17-273
Status last verified
Mar 2026
First submitted
9 Aug 2017
Last updated
27 Mar 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • DeFilipp Z, Kim HT, Knight LW, O'Connor SM, Dhaver SE, White M, Dholaria B, Schroeder MA, Vasu S, Abedin S, Chung J, El-Jawahri A, Frigault MJ, McAfee S, Newcomb RA, O'Donnell PV, Spitzer TR, Chen YB, Hobbs GS. Low rates of chronic graft-versus-host disease with ruxolitinib maintenance following allogeneic HCT. Blood. 2025 May 15;145(20):2312-2316. doi: 10.1182/blood.2024028005.

    PubMed 40106768

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Ruxolitinib in people with Acute Myeloid Leukemia and Acute Myeloid Leukemia in Remission, run by Massachusetts General Hospital at 6 sites 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 21 inclusion and 17 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: under way, not looking for participants, as of the sponsor's last update on 27 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites 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?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

1-year GVHD/relapse free survival rate (GRFS rate) (time frame: 1 Year). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03286530, which also lists 1 linked publication.

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 27 Mar 2026.

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