Clinical Trials /

A Phase I/II Trial of the MUC1 Inhibitor, GO-203-2C in Patients With Relapsed or Refractory Acute Myeloid Leukemia

NCT02204085

Description:

This research study is studying a targeted therapy known as GO-203-2C as a possible treatment for with acute myeloid leukemia (AML) both alone and in combination with decitabine. GO-203-2c targets cancer cells, while leaving healthy cells unaffected.This is a Phase I/II clinical trial. A Phase I clinical trial tests the safety of an investigational intervention and also tries to define the appropriate dose of the investigational intervention to use for further studies.

Related Conditions:
  • Acute Myeloid Leukemia
Recruiting Status:

Active, not recruiting

Phase:

Phase 1/Phase 2

Trial Eligibility

Document

Title

  • Brief Title: A Phase I/II Trial of the MUC1 Inhibitor, GO-203-2C in Patients With Relapsed or Refractory Acute Myeloid Leukemia
  • Official Title: A Phase I/II Trial of the MUC1 Inhibitor, GO-203-2C in Patients With Relapsed or Refractory Acute Myeloid Leukemia

Clinical Trial IDs

  • ORG STUDY ID: 14-222
  • NCT ID: NCT02204085

Conditions

  • Acute Myeloid Leukemia, in Relapse
  • Recurrent Adult Acute Myeloid Leukemia

Interventions

DrugSynonymsArms
GO-203-2cGO-203-2c
GO-203-2c + DecitabineGO-203-2c + Decitabine

Purpose

This research study is studying a targeted therapy known as GO-203-2C as a possible treatment for with acute myeloid leukemia (AML) both alone and in combination with decitabine. GO-203-2c targets cancer cells, while leaving healthy cells unaffected.This is a Phase I/II clinical trial. A Phase I clinical trial tests the safety of an investigational intervention and also tries to define the appropriate dose of the investigational intervention to use for further studies.

Detailed Description

      -  Phase I

             -  The maximum tolerated dose (MTD) will be determined in the phase I section of the
                trial.

             -  Patients who fulfill eligibility criteria will be entered into the trial to
                GO-203-2c.

             -  After the screening procedures confirm participation in the research study. The
                investigators are looking for the highest dose of the combination of study drugs
                that can be administered safely without severe or unmanageable side effects in
                participants that have acute myeloid leukemia (AML) not everyone who participates
                in this research study will receive the same dose of the study drug. The dose given
                will depend on the number of participants who have been enrolled in the study prior
                and how well the dose was tolerated.

             -  A subsequent dose escalation will evaluate the combination of GO-203-2c and
                decitabine.

        -  Phase II

             -  The primary goal is to determine if the combination of the two drugs results in
                clinical response
    

Trial Arms

NameTypeDescriptionInterventions
GO-203-2cExperimentalDose escalation will occur using a standard 3+3 dose escalation approach, beginning in dose level I with dose cohorts and rules for escalation and de-escalation. GO-203-2c given daily on predetermined schedule of a 28-day treatment cycle
  • GO-203-2c
GO-203-2c + DecitabineExperimentalDose escalation will occur for GO-203-2c using a standard 3+3 dose escalation approach, beginning in dose level I with dose cohorts and rules for escalation and de-escalation. GO-203-2c given daily on predetermined schedule of a 28-day treatment cycle. Decitabine will be administered at a dose of 20mg/m2 on days 8-12 of a 28-day treatment cycle.
  • GO-203-2c
  • GO-203-2c + Decitabine

Eligibility Criteria

        Inclusion Criteria:

          -  To be considered eligible for enrollment into this study, all of the following
             inclusion criteria must be met during the screening period:

          -  Documented AML by peripheral blood and bone marrow analyses meeting WHO criteria,
             excluding patients with acute promyelocytic leukemia (APL)

          -  Patients with AML refractory to primary induction chemotherapy, relapsed disease, or
             age ≥ 60 and not appropriate for standard cytotoxic therapy due to age, performance
             status, and/or adverse risk factors according to the treating physician

          -  Age ≥ 18 years

          -  Karnofsky performance status ≥ 50% or ECOG performance status 0-2

          -  Life expectancy ≥ 6 weeks

          -  Able to understand the investigational nature of this study and to provide written
             consent to participate in it

          -  Signed written IRB-approved Informed Consent document

          -  Adequate hepatic and renal function:

               -  serum bilirubin ≤ 1.5 X institutional ULN OR serum direct bilirubin ≤ 2 X
                  institutional ULN

               -  serum ALT and AST ≤ 2.5 X institutional ULN

               -  serum alkaline phosphatase < 5 X institutional ULN

               -  serum creatinine ≤ 2.0 mg/dL

               -  corrected calcium level ≥ institutional LLN

          -  Negative pregnancy test in women of child-bearing potential

          -  Women and men of child-producing potential must agree to use effective contraceptive
             methods during the study period (including post-treatment observation period)

        Exclusion Criteria:

          -  A patient will be considered not eligible for enrollment into this study if any of the
             following criteria are met during the screening period:

          -  Evidence of leukemic meningitis or other CNS involvement by leukemia

          -  Uncontrolled or poorly controlled hypertension (systolic BP ≥ 160 mmHg or diastolic BP
             ≥ 100 mmHg) Note: an isolated reading that is not sustained will be permitted.

          -  Evidence of NYHA Class III or IV cardiac disease, or presence of unstable
             life-threatening arrhythmia, or history of myocardial infarction during the past 6
             months

          -  Active bacterial, fungal, or viral infection requiring systemic treatment

          -  Known infection with HIV

          -  History or major surgery within 4 weeks before the first dose of study treatment, or
             not recovered from prior surgery

          -  Exposure to any other investigational agent at any time within 4 weeks before the
             first dose of study treatment

          -  Exposure to any other anti-leukemic therapy (except hydroxyurea, see Section 5.5.1)
             within 2 weeks before the first dose of study treatment

          -  Pregnant or lactating female

          -  Unwilling or unable to comply with the requirements of the study protocol
      
Maximum Eligible Age:N/A
Minimum Eligible Age:18 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Maximum Tolerated Dose of GO-203-2c
Time Frame:28 days
Safety Issue:
Description:Phase I

Secondary Outcome Measures

Measure:Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Time Frame:2 years
Safety Issue:
Description:
Measure:To investigate whether GO-203-2c alone and in combination with decitabine is effective in targeting MUC1-C overexpressing AML progenitor cells in the lab
Time Frame:2 Years
Safety Issue:
Description:
Measure:To assess whether in vitro response to GO-203-2c alone and in combination with decitabine is associated with clinical response
Time Frame:2 Years
Safety Issue:
Description:
Measure:To determine if therapy with GO-203-2c alone and in combination with decitabine results in decreased engraftment potential of AML progenitor cells in an NSG mouse model
Time Frame:2 Years
Safety Issue:
Description:
Measure:To determine if therapy GO-203-2c in combination with decitabine results in at least 20% of patients achieving a clinical response
Time Frame:2 years
Safety Issue:
Description:To determine if therapy GO-203-2c in combination with decitabine results in at least 20% of patients achieving a clinical response (blast response, minor response, partial response, or complete response).

Details

Phase:Phase 1/Phase 2
Primary Purpose:Interventional
Overall Status:Recruiting
Lead Sponsor:Dana-Farber Cancer Institute

Trial Keywords

  • Refractory acute myeloid leukemia
  • Relapsed acute myeloid leukemia

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