Ruxolitinib Phosphate and Decitabine in Treating Patients With Relapsed or Refractory or Post Myeloproliferative Acute Myeloid Leukemia

NCT02257138 | Completed Phase 1 Results FDA Drug

• 2 other identifiers: 2014-0344NCI-2014-02299
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief 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 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.

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

Outcome Measures

Primary Outcomes (3)

• 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).

  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.

  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.

  Baseline

Secondary Outcomes (1)

• Number of JAK2 Positive+ and JAK2 Negative- Participants With a Response (Phase 2)

  up to 6 years

Study Arms (2)

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.

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.

Drug: Decitabine; Other: Laboratory Biomarker Analysis; Drug: Ruxolitinib Phosphate

Interventions

Decitabine DRUG

Given IV

Also known as: 5-Aza-2'-deoxycytidine, Aza-TdC, Dacogen, Decitabine for Injection, Deoxyazacytidine, Dezocitidine

Treatment (ruxolitinib phosphate, decitabine) Ph1; Treatment (ruxolitinib phosphate, decitabine) Ph2

Laboratory Biomarker Analysis OTHER

Correlative studies

Treatment (ruxolitinib phosphate, decitabine) Ph1; Treatment (ruxolitinib phosphate, decitabine) Ph2

Ruxolitinib Phosphate DRUG

Given PO

Also known as: INCB-18424 Phosphate, Jakafi

Treatment (ruxolitinib phosphate, decitabine) Ph1; Treatment (ruxolitinib phosphate, decitabine) Ph2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

M D Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

• MD Anderson Cancer Center Website

MeSH Terms

Conditions

Myelodysplastic-Myeloproliferative Diseases; Leukemia, Myeloid, Acute

Interventions

Decitabine; Injections; ruxolitinib

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms

Intervention Hierarchy (Ancestors)

Azacitidine; Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides; Drug Administration Routes; Drug Therapy; Therapeutics

Results Point of Contact

• Title: Dr. Farhad Ravandi-Kashani
• Organization: The University of Texas MD Anderson Cancer Center

fravandi@mdanderson.org

713-745-0394

Study Officials

• Farhad Ravandi-Kashani

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 26, 2014
• First Posted: October 6, 2014
• Study Start: February 12, 2015
• Primary Completion: March 19, 2021
• Study Completion: March 19, 2021
• Last Updated: June 8, 2025
• Results First Posted: August 15, 2022

Record last verified: 2025-05

Locations

US (1)