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.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

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

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)

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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.

United States1 site

M D Anderson Cancer Center
Houston, Texas, 77030

Other trials in United States

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 groups in NCT02257138
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.

12 Feb 201519 Mar 2021

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)

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

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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