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

Primary Purpose

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

Status

Active

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

GO-203-2c

GO-203-2c + Decitabine

About this trial

This is an interventional treatment trial for Acute Myeloid Leukemia, in Relapse focused on measuring Refractory acute myeloid leukemia, Relapsed acute myeloid leukemia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Dana-Farber Cancer Institute
Beth Israel Deaconess Medical Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

GO-203-2c

GO-203-2c + Decitabine

Arm Description

Dose 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

Dose 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.

Outcomes

Primary Outcome Measures

Maximum Tolerated Dose of GO-203-2c

Phase I

Maximum Tolerated Dose of GO-203-2c in combination with decitabine

Phase 1

Secondary Outcome Measures

Number of Participants with Adverse Events as a Measure of Safety and Tolerability

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

To assess whether in vitro response to GO-203-2c alone and in combination with decitabine is associated with clinical response

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

To determine if therapy GO-203-2c in combination with decitabine results in at least 20% of patients achieving a clinical response

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).

Full Information

NCT ID

NCT02204085

First Posted

July 9, 2014

Last Updated

June 16, 2023

Sponsor

Beth Israel Deaconess Medical Center

Collaborators

Dana-Farber Cancer Institute

1. Study Identification

Unique Protocol Identification Number

NCT02204085

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

Study Type

Interventional

2. Study Status

Record Verification Date

June 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

September 2014 (undefined)

Primary Completion Date

July 2023 (Anticipated)

Study Completion Date

December 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Beth Israel Deaconess Medical Center

Collaborators

Dana-Farber Cancer Institute

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

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

Keywords

Refractory acute myeloid leukemia, Relapsed acute myeloid leukemia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

33 (Actual)

8. Arms, Groups, and Interventions

Arm Title

GO-203-2c

Arm Type

Experimental

Arm Description

Arm Title

GO-203-2c + Decitabine

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

GO-203-2c

Intervention Type

Drug

Intervention Name(s)

GO-203-2c + Decitabine

Primary Outcome Measure Information:

Title

Maximum Tolerated Dose of GO-203-2c

Description

Phase I

Time Frame

28 days

Title

Maximum Tolerated Dose of GO-203-2c in combination with decitabine

Description

Phase 1

Time Frame

28 days

Secondary Outcome Measure Information:

Title

Number of Participants with Adverse Events as a Measure of Safety and Tolerability

Time Frame

2 years

Title

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

Title

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

Title

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

Title

To determine if therapy GO-203-2c in combination with decitabine results in at least 20% of patients achieving a clinical response

Description

Time Frame

2 years

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

David Avigan, MD

Organizational Affiliation

Beth Israel Deaconess Medical Center

Official's Role

Principal Investigator

Facility Information:

Facility Name

Dana-Farber Cancer Institute

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02115

Country

United States

Facility Name

Beth Israel Deaconess Medical Center

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02215

Country

United States

12. IPD Sharing Statement

Learn more about this trial

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

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