Guadecitabine With or Without Idarubicin or Cladribine in Treating Older Patients With Previously Untreated Acute Myeloid Leukemia

Sponsor
M.D. Anderson Cancer Center (Other)
Overall Status
Completed
CT.gov ID
NCT02096055
Collaborator
National Cancer Institute (NCI) (NIH)
44
1
4
79.7
0.6

Study Details

Study Description

Brief Summary

This randomized phase II trial studies how well guadecitabine with or without idarubicin or cladribine works in treating older patients with previously untreated acute myeloid leukemia. Guadecitabine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as idarubicin and cladribine, 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. It is not yet known whether guadecitabine with or without idarubicin or cladribine is more effective in treating older patients with previously untreated acute myeloid leukemia.

Condition or Disease Intervention/Treatment Phase
Phase 2

Detailed Description

PRIMARY OBJECTIVES:
  1. To determine the complete remission (CR) rate, remission duration, leukemia-free survival, and survival in patients >= 70 years with previously untreated acute myeloid leukemia (AML) with 4 different guadecitabine (SGI-110) single agent and SGI-110 based combination regimens.

  2. To determine the safety profile and tolerability of the 4 SGI-110 single agent and SGI-110 based combination regimens in patients >= 70 years of age with previously untreated AML.

OUTLINE: Patients are randomized to 1 of 2 arms.

ARM I:

INDUCTION THERAPY: Patients receive guadecitabine subcutaneously (SC) on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CR with incomplete platelet recovery (CRp) continue on to Maintenance therapy; patients not achieving CR or complete remission with incomplete hematologic recovery (CRi) but deriving clinical benefit may continue to Maintenance therapy at the discretion of the Principal Investigator (PI).

MAINTENANCE THERAPY: Patients receive guadecitabine as in Induction therapy. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity.

ARM II (CLOSED):

INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-10. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI.

MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 (days 1-10 of courses 1 and 2). Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity.

ARM III:

INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin intravenously (IV) over up to 1 hour on days 1-2. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI.

MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin IV over up to 1 hour on day 1. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity.

ARM IV (CLOSED):

INDUCTION THERAPY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI.

MAINTENANCE THERAPY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every month.

Study Design

Study Type:
Interventional
Actual Enrollment :
44 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Four-Arm Randomized Phase II Study of SGI-110: 5 Days, Versus 10 Days, Versus 5 Days + Idarubicin, Versus 5 Days + Cladribine, in Previously Untreated Patients >/= 70 Years With Acute Myeloid Leukemia
Actual Study Start Date :
Apr 4, 2014
Actual Primary Completion Date :
Nov 24, 2020
Actual Study Completion Date :
Nov 24, 2020

Arms and Interventions

Arm Intervention/Treatment
Experimental: Arm I (guadecitabine)

INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine as in Induction therapy. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity.

Drug: Guadecitabine
Given SC
Other Names:
  • DNMT inhibitor SGI-110
  • S110
  • SGI-110
  • Experimental: Arm II (CLOSED) (guadecitabine)

    INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-10. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 (days 1-10 of courses 1 and 2). Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity.

    Drug: Guadecitabine
    Given SC
    Other Names:
  • DNMT inhibitor SGI-110
  • S110
  • SGI-110
  • Experimental: Arm III (guadecitabine, idarubicin)

    INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin IV over up to 1 hour on days 1-2. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin IV over up to 1 hour on day 1. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity.

    Drug: Guadecitabine
    Given SC
    Other Names:
  • DNMT inhibitor SGI-110
  • S110
  • SGI-110
  • Drug: Idarubicin
    Given IV
    Other Names:
  • 4-Demethoxydaunomycin
  • 4-demethoxydaunorubicin
  • 4-DMDR
  • Experimental: Arm IV (CLOSED) (guadecitabine, cladribine)

    INDUCTION THERPAY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity.

    Drug: Cladribine
    Given IV
    Other Names:
  • 2-CdA
  • 2CDA
  • CdA
  • Cladribina
  • Leustat
  • Leustatin
  • Leustatine
  • RWJ-26251
  • Drug: Guadecitabine
    Given SC
    Other Names:
  • DNMT inhibitor SGI-110
  • S110
  • SGI-110
  • Outcome Measures

    Primary Outcome Measures

    1. Number of Participants With a Complete Response [Up to 4 years, 3 months]

      Complete Response = Complete Remission (CR) + Complete Remission without blood count recovery (CRi) - CR: Neutrophil count >/= 1.0 x 10^9/L and platelet count >/= 100 x 10^9/L, and normal bone marrow differential (</+ 5% blasts). (CRi): Peripheral blood and bonemarrow results as for CR, but with platelet counts of < 100 x 109/L or ANC < 1.0 x 109/L

    2. Remission Duration [Up to 4 years, 3 months]

      The date of Complete Response to the date of loss of response or last follow-up.

    3. Leukemia-free Survival [Up to 4 years, 3 months]

      Survival time will be estimated using the Kaplan-Meier method. The two-sided log-rank test will be used to assess the differences of time to events between groups. Time from date of treatment start until the date of first objective documentation of disease-relapse.

    4. Survival [Up to 4 years, 3 months]

      Survival time will be estimated using the Kaplan-Meier method. The two-sided log-rank test will be used to assess the differences of time to events between groups.

    5. Number of Participants With the Most Frequently Reports Grade 3 or 4 Adverse Event. [Up to 4 years, 3 months]

      The most frequently reported adverse events will be determined by the Principal Investigator. The number of participants who experienced the most frequent grade 3 or 4 adverse events will be reported.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    70 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:
    • Previously untreated AML patients, except those who have received prior therapy with hydroxyurea, single agent chemotherapy (e.g. decitabine), hematopoietic growth factors, biological or targeted therapies are allowed

    • Eastern Cooperative Oncology Group (ECOG) performance status =< 2

    • Sign a written informed consent form

    • Total bilirubin =< 2 mg/dL

    • Serum glutamate pyruvate transaminase (SGPT) or serum glutamic oxaloacetic transaminase (SGOT) =< 4 x upper limit of normal (ULN)

    • Creatinine clearance of >= 50 mL/min (estimated by the Cockcroft-Gault [C-G] formula)

    • Male patients must use an effective contraceptive method during the study and for a minimum of 8 weeks after study treatment

    • Baseline left ventricular ejection fraction (LVEF) >= 40%

    Exclusion Criteria:
    • Patients with >= New York Heart Association (NYHA) grade 3 heart disease as assessed by history and/or physical examination

    • Patients who received more than one full course of prior hypomethylating agents azacitidine or decitabine

    • Have any other severe concurrent disease, or have a history of serious organ dysfunction or disease involving the heart, kidney, liver, or other organ system that may place the patient at undue risk to undergo treatment

    • Patients with a systemic fungal, bacterial, viral, or other infection not controlled (defined as exhibiting ongoing signs/symptoms related to the infection and without improvement, despite appropriate antibiotics or other treatment)

    • Pregnant or lactating patients

    • Any significant concurrent disease, illness, or psychiatric disorder that would compromise patient safety or compliance, interfere with consent, study participation, follow up, or interpretation of study results

    • Any concurrent malignancy with the exception of the following: a) patients with treated non-melanoma skin cancer, in situ carcinoma, or cervical intraepithelial neoplasia, regardless of the disease-free duration, are eligible for this study if definitive treatment for the condition has been completed; b) patients with organ-confined prostate cancer with no evidence of recurrent or progressive disease based on prostate-specific antigen (PSA) values are also eligible for this study if hormonal therapy has been initiated or a radical prostatectomy has been performed

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 M D Anderson Cancer Center Houston Texas United States 77030

    Sponsors and Collaborators

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

    Investigators

    • Principal Investigator: Hagop M Kantarjian, M.D. Anderson Cancer Center

    Study Documents (Full-Text)

    More Information

    Additional Information:

    Publications

    None provided.
    Responsible Party:
    M.D. Anderson Cancer Center
    ClinicalTrials.gov Identifier:
    NCT02096055
    Other Study ID Numbers:
    • 2013-0843
    • NCI-2014-00981
    • 2013-0843
    First Posted:
    Mar 26, 2014
    Last Update Posted:
    May 24, 2022
    Last Verified:
    Apr 1, 2022
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:

    Study Results

    Participant Flow

    Recruitment Details Recruitment Period: April 2014 to July 2018
    Pre-assignment Detail
    Arm/Group Title Arm I (Guadecitabine) x 5 Days Arm II (CLOSED) (Guadecitabine) x 10 Days Arm III (Guadecitabine, Idarubicin) Arm IV (CLOSED) (Guadecitabine, Cladribine)
    Arm/Group Description INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine as in Induction therapy. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-10. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 (days 1-10 of courses 1 and 2). Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin IV over up to 1 hour on days 1-2. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin IV over up to 1 hour on day 1. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC Idarubicin: Given IV INDUCTION THERPAY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Cladribine: Given IV Guadecitabine: Given SC
    Period Title: Overall Study
    STARTED 13 9 13 9
    COMPLETED 13 9 13 9
    NOT COMPLETED 0 0 0 0

    Baseline Characteristics

    Arm/Group Title Arm I (Guadecitabine) x 5 Days Arm II (CLOSED) (Guadecitabine) x 10 Days Arm III (Guadecitabine, Idarubicin) Arm IV (CLOSED) (Guadecitabine, Cladribine) Total
    Arm/Group Description INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine as in Induction therapy. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-10. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 (days 1-10 of courses 1 and 2). Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin IV over up to 1 hour on days 1-2. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin IV over up to 1 hour on day 1. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC Idarubicin: Given IV INDUCTION THERPAY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Cladribine: Given IV Guadecitabine: Given SC Total of all reporting groups
    Overall Participants 13 9 13 9 44
    Age (Count of Participants)
    <=18 years
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    Between 18 and 65 years
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    >=65 years
    13
    100%
    9
    100%
    13
    100%
    9
    100%
    44
    100%
    Age (years) [Median (Full Range) ]
    Median (Full Range) [years]
    73
    76
    75
    76
    75
    Sex: Female, Male (Count of Participants)
    Female
    2
    15.4%
    4
    44.4%
    4
    30.8%
    2
    22.2%
    12
    27.3%
    Male
    11
    84.6%
    5
    55.6%
    9
    69.2%
    7
    77.8%
    32
    72.7%
    Race (NIH/OMB) (Count of Participants)
    American Indian or Alaska Native
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    Asian
    1
    7.7%
    0
    0%
    0
    0%
    0
    0%
    1
    2.3%
    Native Hawaiian or Other Pacific Islander
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    Black or African American
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    White
    11
    84.6%
    8
    88.9%
    13
    100%
    9
    100%
    41
    93.2%
    More than one race
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    Unknown or Not Reported
    1
    7.7%
    1
    11.1%
    0
    0%
    0
    0%
    2
    4.5%
    Region of Enrollment (participants) [Number]
    United States
    13
    100%
    9
    100%
    13
    100%
    9
    100%
    44
    100%

    Outcome Measures

    1. Primary Outcome
    Title Number of Participants With a Complete Response
    Description Complete Response = Complete Remission (CR) + Complete Remission without blood count recovery (CRi) - CR: Neutrophil count >/= 1.0 x 10^9/L and platelet count >/= 100 x 10^9/L, and normal bone marrow differential (</+ 5% blasts). (CRi): Peripheral blood and bonemarrow results as for CR, but with platelet counts of < 100 x 109/L or ANC < 1.0 x 109/L
    Time Frame Up to 4 years, 3 months

    Outcome Measure Data

    Analysis Population Description
    [Not Specified]
    Arm/Group Title Arm I (Guadecitabine) x 5 Days Arm II (CLOSED) (Guadecitabine) x 10 Days Arm III (Guadecitabine, Idarubicin) Arm IV (CLOSED) (Guadecitabine, Cladribine)
    Arm/Group Description INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine as in Induction therapy. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-10. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 (days 1-10 of courses 1 and 2). Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin IV over up to 1 hour on days 1-2. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin IV over up to 1 hour on day 1. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC Idarubicin: Given IV INDUCTION THERPAY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Cladribine: Given IV Guadecitabine: Given SC
    Measure Participants 13 9 13 9
    Count of Participants [Participants]
    5
    38.5%
    5
    55.6%
    10
    76.9%
    0
    0%
    2. Primary Outcome
    Title Remission Duration
    Description The date of Complete Response to the date of loss of response or last follow-up.
    Time Frame Up to 4 years, 3 months

    Outcome Measure Data

    Analysis Population Description
    Zero participants in the guadecitabine + Cladribine arm had a Complete Response.
    Arm/Group Title Arm I (Guadecitabine) x 5 Days Arm II (CLOSED) (Guadecitabine) x 10 Days Arm III (Guadecitabine, Idarubicin) Arm IV (CLOSED) (Guadecitabine, Cladribine)
    Arm/Group Description INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine as in Induction therapy. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-10. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 (days 1-10 of courses 1 and 2). Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin IV over up to 1 hour on days 1-2. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin IV over up to 1 hour on day 1. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC Idarubicin: Given IV INDUCTION THERPAY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Cladribine: Given IV Guadecitabine: Given SC
    Measure Participants 5 5 10 0
    Median (Full Range) [Months]
    7.4
    14.2
    5.3
    3. Primary Outcome
    Title Leukemia-free Survival
    Description Survival time will be estimated using the Kaplan-Meier method. The two-sided log-rank test will be used to assess the differences of time to events between groups. Time from date of treatment start until the date of first objective documentation of disease-relapse.
    Time Frame Up to 4 years, 3 months

    Outcome Measure Data

    Analysis Population Description
    [Not Specified]
    Arm/Group Title Arm I (Guadecitabine) x 5 Days Arm II (CLOSED) (Guadecitabine) x 10 Days Arm III (Guadecitabine, Idarubicin) Arm IV (CLOSED) (Guadecitabine, Cladribine)
    Arm/Group Description INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine as in Induction therapy. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-10. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 (days 1-10 of courses 1 and 2). Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin IV over up to 1 hour on days 1-2. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin IV over up to 1 hour on day 1. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC Idarubicin: Given IV INDUCTION THERPAY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Cladribine: Given IV Guadecitabine: Given SC
    Measure Participants 13 9 13 9
    Median (Full Range) [Months]
    4.2
    10.0
    7.4
    4.3
    4. Primary Outcome
    Title Survival
    Description Survival time will be estimated using the Kaplan-Meier method. The two-sided log-rank test will be used to assess the differences of time to events between groups.
    Time Frame Up to 4 years, 3 months

    Outcome Measure Data

    Analysis Population Description
    [Not Specified]
    Arm/Group Title Arm I (Guadecitabine) x 5 Days Arm II (CLOSED) (Guadecitabine) x 10 Days Arm III (Guadecitabine, Idarubicin) Arm IV (CLOSED) (Guadecitabine, Cladribine)
    Arm/Group Description INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine as in Induction therapy. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-10. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 (days 1-10 of courses 1 and 2). Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin IV over up to 1 hour on days 1-2. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin IV over up to 1 hour on day 1. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC Idarubicin: Given IV INDUCTION THERPAY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Cladribine: Given IV Guadecitabine: Given SC
    Measure Participants 13 9 13 9
    Median (Full Range) [Months]
    13.1
    13.0
    15.4
    11.9
    5. Primary Outcome
    Title Number of Participants With the Most Frequently Reports Grade 3 or 4 Adverse Event.
    Description The most frequently reported adverse events will be determined by the Principal Investigator. The number of participants who experienced the most frequent grade 3 or 4 adverse events will be reported.
    Time Frame Up to 4 years, 3 months

    Outcome Measure Data

    Analysis Population Description
    [Not Specified]
    Arm/Group Title Arm I (Guadecitabine) x 5 Days Arm II (CLOSED) (Guadecitabine) x 10 Days Arm III (Guadecitabine, Idarubicin) Arm IV (CLOSED) (Guadecitabine, Cladribine)
    Arm/Group Description INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine as in Induction therapy. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-10. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 (days 1-10 of courses 1 and 2). Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin IV over up to 1 hour on days 1-2. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin IV over up to 1 hour on day 1. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC Idarubicin: Given IV INDUCTION THERPAY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Cladribine: Given IV Guadecitabine: Given SC
    Measure Participants 13 9 13 9
    Infection/Sepsis
    4
    30.8%
    8
    88.9%
    8
    61.5%
    9
    100%
    Febrile Neutropenia
    5
    38.5%
    2
    22.2%
    4
    30.8%
    9
    100%
    Fatigue
    0
    0%
    0
    0%
    3
    23.1%
    0
    0%

    Adverse Events

    Time Frame Up to 4 years, 3 months
    Adverse Event Reporting Description
    Arm/Group Title Arm I (Guadecitabine) x 5 Days Arm II (CLOSED) (Guadecitabine) x 10 Days Arm III (Guadecitabine, Idarubicin) Arm IV (CLOSED) (Guadecitabine, Cladribine)
    Arm/Group Description INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine as in Induction therapy. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-10. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 (days 1-10 of courses 1 and 2). Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC INDUCTION THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin IV over up to 1 hour on days 1-2. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC on days 1-5 and idarubicin IV over up to 1 hour on day 1. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Guadecitabine: Given SC Idarubicin: Given IV INDUCTION THERPAY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-6 weeks for 2-3 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a CR or CRp continue on to Maintenance therapy; patients not achieving CR or CRi but deriving clinical benefit may continue to Maintenance therapy at the discretion of the PI. MAINTENANCE THERAPY: Patients receive guadecitabine SC and cladribine IV over up to 1 hour on days 1-5. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Cladribine: Given IV Guadecitabine: Given SC
    All Cause Mortality
    Arm I (Guadecitabine) x 5 Days Arm II (CLOSED) (Guadecitabine) x 10 Days Arm III (Guadecitabine, Idarubicin) Arm IV (CLOSED) (Guadecitabine, Cladribine)
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total 2/13 (15.4%) 1/9 (11.1%) 1/13 (7.7%) 2/9 (22.2%)
    Serious Adverse Events
    Arm I (Guadecitabine) x 5 Days Arm II (CLOSED) (Guadecitabine) x 10 Days Arm III (Guadecitabine, Idarubicin) Arm IV (CLOSED) (Guadecitabine, Cladribine)
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total 9/13 (69.2%) 7/9 (77.8%) 10/13 (76.9%) 6/9 (66.7%)
    Blood and lymphatic system disorders
    Anemia 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Neutropenic Fever 4/13 (30.8%) 7 2/9 (22.2%) 3 4/13 (30.8%) 5 6/9 (66.7%) 9
    Cardiac disorders
    Acute Coronary Syndrome 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Atrial Fibrillation 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Chest Pain 0/13 (0%) 0 1/9 (11.1%) 1 1/13 (7.7%) 1 0/9 (0%) 0
    Heart Failure 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 3 0/9 (0%) 0
    Myocardial Infarction 0/13 (0%) 0 0/9 (0%) 0 0/13 (0%) 0 1/9 (11.1%) 1
    Sinus bradycardia 0/13 (0%) 0 1/9 (11.1%) 1 0/13 (0%) 0 0/9 (0%) 0
    Supraventricular tachycardia 0/13 (0%) 0 1/9 (11.1%) 1 0/13 (0%) 0 0/9 (0%) 0
    Gastrointestinal disorders
    Dehydration 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Diarrhea 0/13 (0%) 0 1/9 (11.1%) 1 0/13 (0%) 0 0/9 (0%) 0
    Ileus 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Nausea 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    General disorders
    Back Pain 0/13 (0%) 0 1/9 (11.1%) 2 0/13 (0%) 0 0/9 (0%) 0
    Death 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Fatigue 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Fever 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Non-Cardiac Chest Pain 1/13 (7.7%) 1 1/9 (11.1%) 1 1/13 (7.7%) 1 0/9 (0%) 0
    Pain Extremity 0/13 (0%) 0 0/9 (0%) 0 0/13 (0%) 0 1/9 (11.1%) 1
    Hepatobiliary disorders
    Gallbladder infection 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Infections and infestations
    Breast Infection 0/13 (0%) 0 1/9 (11.1%) 2 0/13 (0%) 0 0/9 (0%) 0
    Catheter related infection 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Infection 0/13 (0%) 0 1/9 (11.1%) 1 1/13 (7.7%) 1 0/9 (0%) 0
    Lung infection 4/13 (30.8%) 4 3/9 (33.3%) 3 3/13 (23.1%) 3 0/9 (0%) 0
    Sepsis 1/13 (7.7%) 1 1/9 (11.1%) 1 0/13 (0%) 0 0/9 (0%) 0
    Sinusitis 0/13 (0%) 0 1/9 (11.1%) 1 0/13 (0%) 0 0/9 (0%) 0
    Skin Infection 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 1/9 (11.1%) 1
    Soft tissue infection 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Urinary tract infeciton 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Injury, poisoning and procedural complications
    Fall 0/13 (0%) 0 1/9 (11.1%) 1 0/13 (0%) 0 0/9 (0%) 0
    Musculoskeletal and connective tissue disorders
    Muscle Weakness 0/13 (0%) 0 1/9 (11.1%) 1 0/13 (0%) 0 0/9 (0%) 0
    Myositis 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Neoplasms benign, malignant and unspecified 0/13 (0%) 0 1/9 (11.1%) 1 0/13 (0%) 0 0/9 (0%) 0
    Nervous system disorders
    Dizziness 0/13 (0%) 0 1/9 (11.1%) 1 0/13 (0%) 0 0/9 (0%) 0
    Seizure 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Renal and urinary disorders
    Acute Kidney Injury 0/13 (0%) 0 0/9 (0%) 0 0/13 (0%) 0 1/9 (11.1%) 1
    Urinary Retention 0/13 (0%) 0 1/9 (11.1%) 1 0/13 (0%) 0 0/9 (0%) 0
    Respiratory, thoracic and mediastinal disorders
    Bronchopulmonary Hemorrhage 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Dyspnea 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 4 0/9 (0%) 0
    Pleural Effusion 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Respiratory failure 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Vascular disorders
    Hypotension 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Thromboembolic Event 0/13 (0%) 0 1/9 (11.1%) 2 0/13 (0%) 0 0/9 (0%) 0
    Other (Not Including Serious) Adverse Events
    Arm I (Guadecitabine) x 5 Days Arm II (CLOSED) (Guadecitabine) x 10 Days Arm III (Guadecitabine, Idarubicin) Arm IV (CLOSED) (Guadecitabine, Cladribine)
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total 13/13 (100%) 7/9 (77.8%) 13/13 (100%) 9/9 (100%)
    Blood and lymphatic system disorders
    Anemia 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 1/9 (11.1%) 1
    Neutropenic Fever 4/13 (30.8%) 6 1/9 (11.1%) 2 4/13 (30.8%) 4 7/9 (77.8%) 10
    Cardiac disorders
    Atrial Fibrillation 0/13 (0%) 0 0/9 (0%) 0 2/13 (15.4%) 2 0/9 (0%) 0
    Cardiac Ischemia/Infarction 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 1/9 (11.1%) 1
    Congestive Heart Failure 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Sinus Bradycardia 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Ventricular arrhythmia--Select 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Gastrointestinal disorders
    Anorexia 0/13 (0%) 0 0/9 (0%) 0 2/13 (15.4%) 2 1/9 (11.1%) 1
    Bloating 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Cholecystitis 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Colitis 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 1/9 (11.1%) 1
    Constipation 2/13 (15.4%) 2 1/9 (11.1%) 1 6/13 (46.2%) 6 4/9 (44.4%) 4
    Diarrhea 4/13 (30.8%) 4 0/9 (0%) 0 6/13 (46.2%) 6 1/9 (11.1%) 1
    Flatulence 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Heartburn 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Mucositis 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 5/9 (55.6%) 5
    Nausea 2/13 (15.4%) 2 2/9 (22.2%) 2 3/13 (23.1%) 3 5/9 (55.6%) 5
    Obstruction GI 0/13 (0%) 0 1/9 (11.1%) 1 0/13 (0%) 0 0/9 (0%) 0
    Vomiting 0/13 (0%) 0 1/9 (11.1%) 1 0/13 (0%) 0 1/9 (11.1%) 1
    General disorders
    Chest Pain 0/13 (0%) 0 1/9 (11.1%) 1 0/13 (0%) 0 0/9 (0%) 0
    Constitutional Symptoms 2/13 (15.4%) 2 3/9 (33.3%) 4 1/13 (7.7%) 1 0/9 (0%) 0
    Edema Limb 3/13 (23.1%) 3 4/9 (44.4%) 4 7/13 (53.8%) 7 1/9 (11.1%) 1
    Fatigue 2/13 (15.4%) 2 1/9 (11.1%) 1 4/13 (30.8%) 4 2/9 (22.2%) 2
    Fever 1/13 (7.7%) 2 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Flu-like syndrome 0/13 (0%) 0 2/9 (22.2%) 2 0/13 (0%) 0 0/9 (0%) 0
    Hemorrhage 0/13 (0%) 0 0/9 (0%) 0 2/13 (15.4%) 3 1/9 (11.1%) 1
    Pain 2/13 (15.4%) 2 2/9 (22.2%) 2 4/13 (30.8%) 6 1/9 (11.1%) 1
    Sweating 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Immune system disorders
    Allergy 1/13 (7.7%) 1 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Infections and infestations
    Infection 3/13 (23.1%) 5 7/9 (77.8%) 14 8/13 (61.5%) 15 6/9 (66.7%) 11
    Injury, poisoning and procedural complications
    Bruising 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Investigations
    Elevated Alanine Aminotransferase 0/13 (0%) 0 1/9 (11.1%) 1 2/13 (15.4%) 2 1/9 (11.1%) 1
    Elevated Aspartate Aminotransferase 0/13 (0%) 0 1/9 (11.1%) 1 0/13 (0%) 0 0/9 (0%) 0
    Hyperbillirubinemia 0/13 (0%) 0 1/9 (11.1%) 1 1/13 (7.7%) 1 2/9 (22.2%) 2
    Neutropenia 2/13 (15.4%) 3 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    thrombocytopenia 2/13 (15.4%) 3 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Weight Loss 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Metabolism and nutrition disorders
    Dehydration 1/13 (7.7%) 1 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Hypomagnesemia 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Musculoskeletal and connective tissue disorders
    Muscle weakness 0/13 (0%) 0 1/9 (11.1%) 1 0/13 (0%) 0 0/9 (0%) 0
    Musculoskeletal/Soft Tissue-Other (Specify) 0/13 (0%) 0 2/9 (22.2%) 2 1/13 (7.7%) 1 0/9 (0%) 0
    Myositis (inflammation/damage of muscle) 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Nervous system disorders
    Dizziness 0/13 (0%) 0 2/9 (22.2%) 2 3/13 (23.1%) 3 1/9 (11.1%) 1
    Memory Impairment 0/13 (0%) 0 1/9 (11.1%) 1 1/13 (7.7%) 1 0/9 (0%) 0
    Neurology-Other (Specify) 0/13 (0%) 0 1/9 (11.1%) 1 0/13 (0%) 0 0/9 (0%) 0
    Neuropathy 0/13 (0%) 0 1/9 (11.1%) 2 1/13 (7.7%) 1 0/9 (0%) 0
    Seizure 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Syncope 0/13 (0%) 0 0/9 (0%) 0 0/13 (0%) 0 1/9 (11.1%) 1
    Tremor 0/13 (0%) 0 0/9 (0%) 0 0/13 (0%) 0 1/9 (11.1%) 1
    Psychiatric disorders
    Confusion 0/13 (0%) 0 0/9 (0%) 0 0/13 (0%) 0 1/9 (11.1%) 1
    Insomnia 4/13 (30.8%) 4 0/9 (0%) 0 4/13 (30.8%) 4 0/9 (0%) 0
    Renal and urinary disorders
    Bladder Spasms 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Dysuria 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Urinary frequency/urgency 1/13 (7.7%) 1 1/9 (11.1%) 1 1/13 (7.7%) 1 0/9 (0%) 0
    Urinary retention (including neurogenic bladder) 0/13 (0%) 0 1/9 (11.1%) 1 0/13 (0%) 0 0/9 (0%) 0
    Respiratory, thoracic and mediastinal disorders
    Cough 0/13 (0%) 0 1/9 (11.1%) 1 1/13 (7.7%) 1 0/9 (0%) 0
    Dyspnea 0/13 (0%) 0 2/9 (22.2%) 2 1/13 (7.7%) 1 0/9 (0%) 0
    Pleural effusion 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Respiratory Failure 0/13 (0%) 0 0/9 (0%) 0 0/13 (0%) 0 1/9 (11.1%) 1
    Skin and subcutaneous tissue disorders
    Alopecia 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Dermatology Skin/Other 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 1/9 (11.1%) 2
    Pruritus/itching 2/13 (15.4%) 2 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Rash/desquamation 5/13 (38.5%) 5 1/9 (11.1%) 1 2/13 (15.4%) 2 2/9 (22.2%) 2
    Rash: dermatitis associated with radiation--Select 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Ulceration 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Vascular disorders
    Flushing 1/13 (7.7%) 1 0/9 (0%) 0 0/13 (0%) 0 0/9 (0%) 0
    Hypotension 0/13 (0%) 0 0/9 (0%) 0 1/13 (7.7%) 1 0/9 (0%) 0
    Phlebitis 0/13 (0%) 0 1/9 (11.1%) 1 0/13 (0%) 0 0/9 (0%) 0
    Thrombosis/embolism (vascular access-related) 1/13 (7.7%) 1 2/9 (22.2%) 3 4/13 (30.8%) 4 0/9 (0%) 0

    Limitations/Caveats

    [Not Specified]

    More Information

    Certain Agreements

    All Principal Investigators ARE employed by the organization sponsoring the study.

    There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

    Results Point of Contact

    Name/Title Hagop Kantarjian, MD/Chair
    Organization The University of Texas MD Anderson Cancer Center
    Phone 713-792-7026
    Email hkantarjian@mdanderson.org
    Responsible Party:
    M.D. Anderson Cancer Center
    ClinicalTrials.gov Identifier:
    NCT02096055
    Other Study ID Numbers:
    • 2013-0843
    • NCI-2014-00981
    • 2013-0843
    First Posted:
    Mar 26, 2014
    Last Update Posted:
    May 24, 2022
    Last Verified:
    Apr 1, 2022