- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02204085
A Phase I/II Trial of the MUC1 Inhibitor, GO-203-2C in Patients With Relapsed or Refractory Acute Myeloid Leukemia
Study Overview
Status
Intervention / Treatment
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
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Locations
-
-
Massachusetts
-
Boston, Massachusetts, United States, 02215
- Beth Israel Deaconess Medical Center
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Boston, Massachusetts, United States, 02115
- Dana-Farber Cancer Institute
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: GO-203-2c
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 |
|
Experimental: GO-203-2c + Decitabine
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. |
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Maximum Tolerated Dose of GO-203-2c
Time Frame: 28 days
|
Phase I
|
28 days
|
Maximum Tolerated Dose of GO-203-2c in combination with decitabine
Time Frame: 28 days
|
Phase 1
|
28 days
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Time Frame: 2 years
|
2 years
|
|
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
|
2 Years
|
|
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
|
2 Years
|
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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
|
2 Years
|
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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
|
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).
|
2 years
|
Collaborators and Investigators
Collaborators
Investigators
- Principal Investigator: David Avigan, MD, Beth Israel Deaconess Medical Center
Study record dates
Study Major Dates
Study Start
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimated)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 14-222
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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