Phase l/II Study of Ruxolitinib for Acute Leukemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01251965
Recruitment Status : Completed
First Posted : December 2, 2010
Last Update Posted : October 16, 2015
Incyte Corporation
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Brief Summary:
The goal of this clinical research study is to find the highest tolerable dose of ruxolitinib that can be given to patients with acute leukemia and to learn if the study drug can help control the disease. The safety of the drug will also be studied.

Condition or disease Intervention/treatment Phase
Leukemia Drug: Ruxolitinib Phase 1 Phase 2

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 30 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I/II Study to Determine the Safety and Efficacy of Ruxolitinib, a JAK1/JAK2 Inhibitor, in Subjects With Relapsed or Refractory Acute Leukemia
Study Start Date : December 2010
Actual Primary Completion Date : October 2015

Arm Intervention/treatment
Experimental: Ruxolitinib

Phase I - Starting dose of Ruxolitinib 50 mg by mouth twice a day for 28 day cycle.

Phase II - MTD reached in Phase I.

Drug: Ruxolitinib

Phase I - Starting dose of 50 mg by mouth twice a day for 28 day cycle.

Phase II - MTD reached in Phase I.

Other Names:
  • Jakafi
  • INCBO18424
  • INC424

Primary Outcome Measures :
  1. Maximum Tolerated Dose (MTD) of Ruxolitinib [ Time Frame: 28 days ]
    MTD defined as highest dose level at which no more than one out of six subject experiences dose limiting toxicity (DLT) during first cycle (28 days) of therapy. A non-hematologic DLT defined as a clinically significant grade 3 or 4 adverse event or abnormal laboratory value (according to Common Toxicity Criteria for Adverse Effects (CTCAE) criteria) assessed as related to study drug (and unrelated to disease progression, intercurrent illness, or concomitant medications) occurring during first 28 days on study.

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Ages Eligible for Study:   14 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Must be >14 years of age
  2. Must be diagnosed with refractory or relapsed AML or ALL.
  3. Must have adequate organ function as demonstrated by the following: o ALT (SGOT) and/or AST (SGPT) equal to or less than 1.5x upper limit of normal o Serum creatinine equal to or less than 2.5 mg/dL
  4. Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0, 1, or 2
  5. At least 2 weeks from prior leukemia-directed treatment to starting treatment drug (except for hydroxyurea, which is allowed if clinically indicated but should be stopped after 2 weeks of receiving study drug, and glucocorticoids, which are allowed but should be stopped upon starting treatment drug).
  6. Treatment-related toxicities from prior therapies must have resolved to Grade equal to or less than 1 (except for peripheral neuropathy, which should resolve to grade equal to or less than 2)
  7. No active malignancies with the exception of basal cell or squamous cell carcinoma of the skin, or carcinoma "in situ" of the cervix or breast.
  8. Females of childbearing potential (FCBP)(A female of childbearing potential is a sexually mature woman who: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months) must have negative pregnancy test. FCBP and males participating in the study must agree to use a reliable form of contraception or to practice complete abstinence from heterosexual intercourse while participating in the study and for at least 28 days after discontinuation from the study. If pregnancy or a positive pregnancy test does occur in a study subject, treatment with the study drug must be immediately discontinued.

Exclusion Criteria:

  1. Known positive status for HIV, or known active hepatitis A, B, or C infection.
  2. Any serious medical condition or psychiatric illness that would prevent, (as judged by the treating physician) the subject from signing the informed consent form or any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
  3. Pregnant or lactating females.
  4. Acute promyelocytic leukemia
  5. Concurrent use of strong inducers or strong inhibitors of cytochrome P450 3A4 (CYP3A4). Strong inducers are rifampin and St. John's Worth. Strong inhibitors are HIV-antivirals, clarythromycin, itraconazole, ketoconazole, nefazodone, and telithromycin.
  6. Participating in any other research trial.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01251965

United States, Texas
University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Incyte Corporation
Principal Investigator: Srdan Verstovsek, MD M.D. Anderson Cancer Center

Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: M.D. Anderson Cancer Center Identifier: NCT01251965     History of Changes
Other Study ID Numbers: 2010-0450
NCI-2011-02439 ( Registry Identifier: NCI CTRP )
First Posted: December 2, 2010    Key Record Dates
Last Update Posted: October 16, 2015
Last Verified: October 2015

Keywords provided by M.D. Anderson Cancer Center:
Relapsed Acute Leukemia
Acute myeloid leukemia
Acute lymphocytic leukemia

Additional relevant MeSH terms:
Neoplasms by Histologic Type