Acute Myeloid Leukemia, in RelapsePhase 1/2NCT02204085
A Phase I/II Trial of the MUC1 Inhibitor, GO-203-2C in Patients With Relapsed or Refractory Acute Myeloid Leukemia
A Phase 1/2 study testing GO-203-2c and GO-203-2c + Decitabine in people with Acute Myeloid Leukemia, in Relapse and Recurrent Adult Acute Myeloid Leukemia, run by Beth Israel Deaconess Medical Center at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia, in Relapse, Recurrent Adult Acute Myeloid Leukemia
- What is being tested?
- GO-203-2c and GO-203-2c + Decitabine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 33 participants
- Where
- 2 sitesUnited States
- Study dates
- Sep 2014to Jun 2026
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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)
Who cannot join
- 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
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- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
About this study
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.
Read the 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
- Being tested
- GO-203-2c, GO-203-2c + Decitabine
- Keywords
- Refractory acute myeloid leukemia, Relapsed acute myeloid leukemia
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GO-203-2cDrug
GO-203-2c + DecitabineDrug
| Study group | Role | What they receive |
|---|---|---|
| GO-203-2c | Experimental | 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 Receives: Drug: GO-203-2c |
| GO-203-2c + Decitabine | Experimental | 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. Receives: Drug: GO-203-2c; Drug: GO-203-2c + Decitabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose of GO-203-2c
Phase I: Maximum Tolerated Dose of GO-203-2c
Measured: 28 days
Maximum Tolerated Dose of GO-203-2c in combination with decitabine
Phase 1: Maximum Tolerated Dose of GO-203-2c in combination with decitabine
Measured: 28 days
Secondary outcomes (2)
Safety and Tolerability
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Measured: 2 years
Determine if therapy 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).
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jul 2014 (estimated)
- Study startSep 2014
- Primary completionSep 2025
- Study completionJun 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia, in RelapseStudies of the same condition, open and under way.
- More trials run by Beth Israel Deaconess Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 33 participants (actual)
Sponsor and investigators
- Lead sponsor
- Beth Israel Deaconess Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Dana-Farber Cancer Institute
- Responsible party
- Principal investigator, David Avigan, Beth Israel Deaconess Medical Center
- Investigators
- David Avigan, MD, Principal investigator, Beth Israel Deaconess Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02204085
- Sponsor's protocol ID
- 14-222
- Status last verified
- Nov 2026
- First submitted
- 9 Jul 2014
- Last updated
- 6 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Trial of the MUC1 Inhibitor, GO-203-2C in Patients With Relapsed or Refractory Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT02204085
- Acronym / sponsor ID
- 14-222
- Keywords
- Refractory acute myeloid leukemia, Relapsed acute myeloid leukemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 33 participants (actual)
- Lead sponsor
- Beth Israel Deaconess Medical CenterUniversity, hospital or other
- Collaborators
- Dana-Farber Cancer Institute
- Study start
- Sep 2014 (actual)
- Primary completion
- Sep 2025 (actual)
- Study completion
- Jun 2026 (actual)
- First posted
- 30 Jul 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| GO-203-2c | Experimental | Drug: GO-203-2cDose 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 + Decitabine | Experimental | Drug: GO-203-2c, Drug: GO-203-2c + DecitabineDose 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- 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
Key exclusion criteria (11 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GO-203-2c | Drug | GO-203-2c, GO-203-2c + Decitabine |
| GO-203-2c + Decitabine | Drug | GO-203-2c + Decitabine |
Primary outcome
Maximum Tolerated Dose of GO-203-2c
Time frame: 28 days
Phase I: Maximum Tolerated Dose of GO-203-2c
Maximum Tolerated Dose of GO-203-2c in combination with decitabine
Time frame: 28 days
Phase 1: Maximum Tolerated Dose of GO-203-2c in combination with decitabine
2 secondary outcomes
Safety and Tolerability
Time frame: 2 years
Determine if therapy results in at least 20% of patients achieving a clinical response
Time frame: 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Nov 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
David Avigan, MD principal investigator
Beth Israel Deaconess Medical Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing GO-203-2c and GO-203-2c + Decitabine in people with Acute Myeloid Leukemia, in Relapse and Recurrent Adult Acute Myeloid Leukemia, run by Beth Israel Deaconess Medical Center at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum Tolerated Dose of GO-203-2c (time frame: 28 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Nov 2026.