Blasts More Than 20 Percent of Bone Marrow Nucleated CellsPhase 1/2NCT02257138
Ruxolitinib Phosphate and Decitabine in Treating Patients With Relapsed or Refractory or Post Myeloproliferative Acute Myeloid Leukemia
A Phase 1/2 study testing Decitabine, Laboratory Biomarker Analysis and Ruxolitinib Phosphate in people with Blasts More Than 20 Percent of Bone Marrow Nucleated Cells and Blasts More Than 20 Percent of Peripheral Blood White Cells, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
This phase I/II trial studies the side effects and best dose of ruxolitinib phosphate when given together with decitabine and to see how well they work in treating patients with acute myeloid leukemia that has come back or is not responding to treatment, or has developed from a type of bone marrow diseases called… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Blasts More Than 20 Percent of Bone Marrow Nucleated Cells, Blasts More Than 20 Percent of Peripheral Blood White Cells, Myelodysplastic/Myeloproliferative Neoplasm
- What is being tested?
- Decitabine, Laboratory Biomarker Analysis and Ruxolitinib Phosphate
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- 12 Feb 2015to 19 Mar 2021
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of AML (World Health Organization [WHO] classification definition of >= to 20% blasts)
- In the phase I portion of the study all patients with relapsed or refractory AML are eligible; for the Phase II portion of the study, patients must have AML progressing from prior MPN (MPN-BP) or have myelodysplastic syndrome (MDS)/MPN with more than 20% blasts; temporary prior measures to control blood counts, such as apheresis or Hydrea are allowed; patients with newly diagnosed or previously treated disease are eligible as long as prior therapy does not include hypomethylating agents; prior therapy for ruxolitinib for MPN is allowed
- Serum biochemical values with the following limits unless considered due to leukemia:
- Creatinine =< 1.5 mg/dl
- Total bilirubin =< 1.5 mg/dL, unless increase is due to hemolysis or congenital disorder
- Transaminases (serum glutamate pyruvate transaminase [SGPT]) =< 2.5 x upper limit of normal (ULN)
Show all 9Show fewer
- Ability to take oral medication
- Ability to understand and provide signed informed consent
- Performance status =< 3, unless directly related to disease process as determined by the principal investigator
Who cannot join
- Any coexisting medical condition that in the judgment of the treating physician is likely to interfere with study procedures or results including uncontrolled severe infections, as well as uncontrolled cardiac disease, or other organ dysfunction; patients with history of tuberculosis, human immunodeficiency virus (HIV) or hepatitis B and C are excluded
- Nursing women, women of childbearing potential with positive blood pregnancy test within 30 days of study start, or women of childbearing potential who are not willing to maintain adequate contraception (such as birth control pills, intrauterine device [IUD], diaphragm, abstinence, or condoms by their partner) over the entire course of the study
- Incomplete recovery from any prior surgical procedures or had surgery within 4 weeks prior to study entry, excluding the placement of vascular access
- Active clinically serious and uncontrolled infection
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase I/II Study of Ruxolitinib Plus Decitabine in Patients With Post Myeloproliferative Neoplasm - Acute Myeloid Leukemia (AML)
This phase I/II trial studies the side effects and best dose of ruxolitinib phosphate when given together with decitabine and to see how well they work in treating patients with acute myeloid leukemia that has come back or is not responding to treatment, or has developed from a type of bone marrow diseases called myeloproliferative neoplasms. Ruxolitinib phosphate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as decitabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving ruxolitinib phosphate together with decitabine may be an effective treatment for acute myeloid leukemia.
Read the detailed description
PRIMARY OBJECTIVES:
- To determine the tolerability of the combination of decitabine and ruxolitinib phosphate (ruxolitinib [DI]) in patients with leukemia. (Phase I) II. To determine the efficacy of ruxolitinib in increasing and prolonging response induced by decitabine alone in patients with post myeloproliferative neoplasm acute myeloid leukemia (AML) (post MPN-AML) alternatively referred to as (myeloproliferative neoplasm - blast phase; MPN-BP). (Compared to historical response rate with decitabine alone) (Phase II)
SECONDARY OBJECTIVES:
- To compare whether there is a difference in response rate patients with post-MPN AML with janus kinase 2 (JAK2) mutations and patients without JAK2 mutations.
OUTLINE: This is a phase I, dose-escalation study of ruxolitinib phosphate followed by a phase II study.
Patients receive ruxolitinib phosphate orally (PO) twice daily (BID) on days 1-28 and decitabine intravenously (IV) over 1-2 hours on days 1-5. Treatment repeats every 4-6 weeks for up to 24 courses in the absence of disease progression or unacceptable toxicity.
- Conditions
- Blasts More Than 20 Percent of Bone Marrow Nucleated Cells, Blasts More Than 20 Percent of Peripheral Blood White Cells, Myelodysplastic/Myeloproliferative Neoplasm, Recurrent Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
- Being tested
- Decitabine, Laboratory Biomarker Analysis, Ruxolitinib Phosphate
- Medical subject headings
- Myelodysplastic-Myeloproliferative Diseases, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DecitabineDrug
Also called 5-Aza-2'-deoxycytidine, Aza-TdC, Dacogen, Decitabine for Injection
Given IV
Laboratory Biomarker AnalysisOther
Correlative studies
Ruxolitinib PhosphateDrug
Also called INCB-18424 Phosphate, Jakafi
Given PO
| Study group | Role | What they receive |
|---|---|---|
| Treatment (ruxolitinib phosphate, decitabine) Ph1 | Experimental | Patients receive ruxolitinib phosphate PO BID on days 1-28 and decitabine IV on days 1-5. Treatment repeats every 4-6 weeks for up to 24 courses in the absence of disease progression or unacceptable toxicity. Receives: Drug: Decitabine; Other: Laboratory Biomarker Analysis; Drug: Ruxolitinib Phosphate |
| Treatment (ruxolitinib phosphate, decitabine) Ph2 | Experimental | Patients receive 50mg ruxolitinib phosphate PO BID on days 1-28 and decitabine IV on days 1-5. Treatment repeats every 4-6 weeks for up to 24 courses in the absence of disease progression or unacceptable toxicity. Receives: Drug: Decitabine; Other: Laboratory Biomarker Analysis; Drug: Ruxolitinib Phosphate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose of Ruxolitinib Phosphate (Phase I)
Maximum tolerated dose (MTD) is defined as the highest dose studied for which the incidence of dose-limiting toxicity (DLT) is less than or equal to 17% (1 out of 6).
Measured: Up to 6 weeks
Number of Participants With a Response (Complete Response [CR] + CR With Incomplete Blood Count Recovery) (Phase 2)
Complete Response (CR) is defined as - The participant must be free of all symptoms related to leukemia and have an absolute neutrophil count >/= 1 z 10^9/L, no need for red blood cell transfusion, platelet count>/+ 100 x 10^9/L, and normal marrow differential (</= 5 % blasts) in a normo- or hypercellular marrow. Complete Response with incomplete blood count recovery (CRi) is defined as - Same as CR but incomplete count recovery.
Measured: Up to 6 years
Number of Participants With Post-MPN Acute Myeloid Leukemia (AML) With JAK2 Mutations (Phase 2)
JAK2 mutations were assessed with the baseline Bone Marrow or Peripheral Blood.
Measured: Baseline
Secondary outcomes (1)
Number of JAK2 Positive+ and JAK2 Negative- Participants With a Response (Phase 2)
JAK2 mutations were assessed with the baseline Bone Marrow or Peripheral Blood. Complete Response (CR) is defined as - The participant must be free of all symptoms related to leukemia and have an absolute neutrophil count >/= 1 z 10^9/L, no need for red blood cell transfusion, platelet count>/+ 100 x 10^9/L, and normal marrow differential (</= 5 % blasts) in a normo- or hypercellular marrow. Complete Response with incomplete blood count recovery (CRi) is defined as - Same as CR but incomplete count recovery.
Measured: up to 6 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Oct 2014 (estimated)
- Study start12 Feb 2015
- Primary completion19 Mar 2021
- Study completion19 Mar 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Blasts More Than 20 Percent of Bone Marrow Nucleated CellsStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Farhad Ravandi-Kashani, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02257138
- Sponsor's protocol ID
- 2014-0344
- Other IDs
- NCI-2014-02299, 2014-0344
- Status last verified
- May 2025
- First submitted
- 26 Sep 2014
- Last updated
- 8 Jun 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II Study of Ruxolitinib Plus Decitabine in Patients With Post Myeloproliferative Neoplasm - Acute Myeloid Leukemia (AML)
- ClinicalTrials.gov ID
- NCT02257138
- Acronym / sponsor ID
- 2014-0344
- Conditions
- Blasts More Than 20 Percent of Bone Marrow Nucleated Cells, Blasts More Than 20 Percent of Peripheral Blood White Cells, Myelodysplastic/Myeloproliferative Neoplasm, Recurrent Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 12 Feb 2015 (actual)
- Primary completion
- 19 Mar 2021 (actual)
- Study completion
- 19 Mar 2021 (actual)
- First posted
- 6 Oct 2014 (estimated)
- Results
- Posted 15 Aug 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase I/II trial studies the side effects and best dose of ruxolitinib phosphate when given together with decitabine and to see how well they work in treating patients with acute myeloid leukemia that has come back or is not responding to treatment, or has developed from a type of bone marrow diseases called myeloproliferative neoplasms. Ruxolitinib phosphate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as decitabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving ruxolitinib phosphate together with decitabine may be an effective treatment for acute myeloid leukemia.
Detailed description
PRIMARY OBJECTIVES:
- To determine the tolerability of the combination of decitabine and ruxolitinib phosphate (ruxolitinib [DI]) in patients with leukemia. (Phase I) II. To determine the efficacy of ruxolitinib in increasing and prolonging response induced by decitabine alone in patients with post myeloproliferative neoplasm acute myeloid leukemia (AML) (post MPN-AML) alternatively referred to as (myeloproliferative neoplasm - blast phase; MPN-BP). (Compared to historical response rate with decitabine alone) (Phase II)
SECONDARY OBJECTIVES:
- To compare whether there is a difference in response rate patients with post-MPN AML with janus kinase 2 (JAK2) mutations and patients without JAK2 mutations.
OUTLINE: This is a phase I, dose-escalation study of ruxolitinib phosphate followed by a phase II study.
Patients receive ruxolitinib phosphate orally (PO) twice daily (BID) on days 1-28 and decitabine intravenously (IV) over 1-2 hours on days 1-5. Treatment repeats every 4-6 weeks for up to 24 courses in the absence of disease progression or unacceptable toxicity.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Treatment (ruxolitinib phosphate, decitabine) Ph1 | Experimental | Drug: Decitabine, Other: Laboratory Biomarker Analysis, Drug: Ruxolitinib PhosphatePatients receive ruxolitinib phosphate PO BID on days 1-28 and decitabine IV on days 1-5. Treatment repeats every 4-6 weeks for up to 24 courses in the absence of disease progression or unacceptable toxicity. |
| Treatment (ruxolitinib phosphate, decitabine) Ph2 | Experimental | Drug: Decitabine, Other: Laboratory Biomarker Analysis, Drug: Ruxolitinib PhosphatePatients receive 50mg ruxolitinib phosphate PO BID on days 1-28 and decitabine IV on days 1-5. Treatment repeats every 4-6 weeks for up to 24 courses in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Diagnosis of AML (World Health Organization [WHO] classification definition of >= to 20% blasts)
- In the phase I portion of the study all patients with relapsed or refractory AML are eligible; for the Phase II portion of the study, patients must have AML progressing from prior MPN (MPN-BP) or have myelodysplastic syndrome (MDS)/MPN with more than 20% blasts; temporary prior measures to control blood counts, such as apheresis or Hydrea are allowed; patients with newly diagnosed or previously treated disease are eligible as long as prior therapy does not include hypomethylating agents; prior therapy for ruxolitinib for MPN is allowed
- Serum biochemical values with the following limits unless considered due to leukemia:
- Creatinine =< 1.5 mg/dl
- Total bilirubin =< 1.5 mg/dL, unless increase is due to hemolysis or congenital disorder
Key exclusion criteria (4 listed)
- Any coexisting medical condition that in the judgment of the treating physician is likely to interfere with study procedures or results including uncontrolled severe infections, as well as uncontrolled cardiac disease, or other organ dysfunction; patients with history of tuberculosis, human immunodeficiency virus (HIV) or hepatitis B and C are excluded
- Nursing women, women of childbearing potential with positive blood pregnancy test within 30 days of study start, or women of childbearing potential who are not willing to maintain adequate contraception (such as birth control pills, intrauterine device [IUD], diaphragm, abstinence, or condoms by their partner) over the entire course of the study
- Incomplete recovery from any prior surgical procedures or had surgery within 4 weeks prior to study entry, excluding the placement of vascular access
- Active clinically serious and uncontrolled infection
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 |
|---|---|---|
| DecitabineAlso: 5-Aza-2'-deoxycytidine, Aza-TdC, Dacogen, Decitabine for InjectionGiven IV | Drug | Treatment (ruxolitinib phosphate, decitabine) Ph1, Treatment (ruxolitinib phosphate, decitabine) Ph2 |
| Laboratory Biomarker AnalysisCorrelative studies | Other | Treatment (ruxolitinib phosphate, decitabine) Ph1, Treatment (ruxolitinib phosphate, decitabine) Ph2 |
| Ruxolitinib PhosphateAlso: INCB-18424 Phosphate, JakafiGiven PO | Drug | Treatment (ruxolitinib phosphate, decitabine) Ph1, Treatment (ruxolitinib phosphate, decitabine) Ph2 |
Primary outcome
Maximum Tolerated Dose of Ruxolitinib Phosphate (Phase I)
Time frame: Up to 6 weeks
Maximum tolerated dose (MTD) is defined as the highest dose studied for which the incidence of dose-limiting toxicity (DLT) is less than or equal to 17% (1 out of 6).
Number of Participants With a Response (Complete Response [CR] + CR With Incomplete Blood Count Recovery) (Phase 2)
Time frame: Up to 6 years
Complete Response (CR) is defined as - The participant must be free of all symptoms related to leukemia and have an absolute neutrophil count >/= 1 z 10^9/L, no need for red blood cell transfusion, platelet count>/+ 100 x 10^9/L, and normal marrow differential (</= 5 % blasts) in a normo- or hypercellular marrow. Complete Response with incomplete blood count recovery (CRi) is defined as - Same as CR but incomplete count recovery.
Number of Participants With Post-MPN Acute Myeloid Leukemia (AML) With JAK2 Mutations (Phase 2)
Time frame: Baseline
JAK2 mutations were assessed with the baseline Bone Marrow or Peripheral Blood.
1 secondary outcome
Number of JAK2 Positive+ and JAK2 Negative- Participants With a Response (Phase 2)
Time frame: up to 6 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jun 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- MD Anderson Cancer Center WebsiteLink provided by the sponsor.
Related trials for Blasts More Than 20 Percent of Bone Marrow Nucleated Cells
Active, not recruitingPhase 2
Ruxolitinib in Treating Patients With Hypereosinophilic Syndrome or Primary Eosinophilic Disorders
BCR-JAK2 Fusion Protein Expression, Blasts 20 Percent or Less of Peripheral Blood White Cells
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. This study is not recruiting at the moment.
Farhad Ravandi-Kashani 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 1/2 study testing Decitabine, Laboratory Biomarker Analysis and Ruxolitinib Phosphate in people with Blasts More Than 20 Percent of Bone Marrow Nucleated Cells and Blasts More Than 20 Percent of Peripheral Blood White Cells, 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 9 inclusion and 4 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: finished, as of the sponsor's last update on 8 Jun 2025. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Yes. Results are posted in the registry (first posted 15 Aug 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Maximum Tolerated Dose of Ruxolitinib Phosphate (Phase I) (time frame: Up to 6 weeks). The study lists 3 primary outcomes in all. 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 8 Jun 2025.