GO-203-2C for Acute Myeloid Leukemia

Not currently recruiting at 1 trial location

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Beth Israel Deaconess Medical Center

Must not be taking: Investigational agents, Anti-leukemic therapy

Disqualifiers: Leukemic meningitis, Uncontrolled hypertension, Cardiac disease, Active infection, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 28

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test a new treatment, GO-203-2C, for people with acute myeloid leukemia (AML). Researchers seek to determine if this treatment, alone or with the chemotherapy drug decitabine, can target cancer cells while sparing healthy ones. Participants should have AML that hasn’t responded to previous chemotherapy, has relapsed, or are unsuitable for standard treatments due to other health factors.

As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial requires that you stop any other anti-leukemic therapy (except hydroxyurea) at least 2 weeks before starting the study treatment. The protocol does not specify about other medications, so it's best to discuss with the study team.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

In earlier studies, researchers tested GO-203-2C in patients with acute myeloid leukemia (AML) and found it to be generally well-tolerated. Research indicates that this treatment targets cancer cells while sparing healthy ones, potentially reducing side effects.

The combination of GO-203-2C with decitabine has also been studied. Decitabine is already known to be effective and generally well-tolerated in older patients with AML.

Although detailed safety data from the current trial is not yet available, the approval of decitabine for AML provides some confidence in its safety.

In summary, both GO-203-2C and its combination with decitabine have shown promise in terms of safety in earlier studies. However, since this is an early phase trial, the main goal is to confirm safety and determine the right dosage. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about GO-203-2c for treating Acute Myeloid Leukemia (AML) because it targets cancer cells uniquely by inhibiting a protein called MUC1-C, which is not typically addressed by standard chemotherapy. This approach could potentially disrupt the cancer cells' ability to survive and multiply. The treatment is also being tested in combination with Decitabine, a drug that interferes with DNA replication in cancer cells, which might enhance the effectiveness of the treatment. Unlike traditional chemotherapy, which often affects both healthy and cancerous cells, GO-203-2c aims to specifically target cancer cells, offering a promising new angle in AML treatment.

What evidence suggests that GO-203-2c might be an effective treatment for acute myeloid leukemia?

Studies have shown that GO-203-2c targets and blocks a protein called MUC1-C, found in the cancer cells of acute myeloid leukemia (AML). Early research indicates that it can stop the growth of AML cells in some patients. In this trial, some participants will receive GO-203-2c alone, while others will receive it in combination with decitabine, a drug that helps older AML patients. When used with decitabine, GO-203-2c may lead to a positive response in at least 20% of patients, effectively reducing cancer cells. These findings highlight the promise of GO-203-2c, both on its own and with decitabine, in treating AML. ^{1 3 6 7 8}

Who Is on the Research Team?

David Avigan, MD

Principal Investigator

Beth Israel Deaconess Medical Center

Are You a Good Fit for This Trial?

This trial is for adults over 18 with Acute Myeloid Leukemia (AML) that's come back or didn't respond to initial treatment. They should be in decent health otherwise, not pregnant, and willing to use birth control. People with severe heart issues, active infections, recent major surgery, or HIV can't join.

Inclusion Criteria

corrected calcium level ≥ institutional LLN

Negative pregnancy test in women of child-bearing potential

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

Exclusion Criteria

Unwilling or unable to comply with the requirements of the study protocol

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Phase I Treatment

Determination of the maximum tolerated dose (MTD) of GO-203-2c, with dose escalation using a 3+3 approach

28 days

Daily visits for drug administration

Phase II Treatment

Evaluation of clinical response to the combination of GO-203-2c and decitabine

2 years

28-day treatment cycles with decitabine administered on days 8-12

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Decitabine
• GO-203-2c

Trial Overview The study tests GO-203-2C alone and combined with Decitabine as treatments for AML. It aims to find the safest dose of GO-203-2C and see how well it works against leukemia cells without harming normal cells.

How Is the Trial Designed?

Group I: GO-203-2c + Decitabine Experimental Treatment 2 Interventions

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.

Group II: GO-203-2c Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

Beth Israel Deaconess Medical Center

Lead Sponsor

Trials

872

Recruited

12,930,000+

Dana-Farber Cancer Institute

Collaborator

Trials

1,128

Recruited

382,000+

Citations

1. clinicaltrial.be clinicaltrial.be/en/details/11716?per_page=20&only_recruiting=0&only_eligible=0&only_active=0

A Phase I/II Trial of the MUC1 Inhibitor, GO-203-2C in Pa... To determine if therapy GO-203-2c in combination with decitabine results in at least 20% of patients achieving a clinical response (blast ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC3100696/

MUC1-C oncoprotein suppresses reactive oxygen species ... In addition, growth of AML patient 1 blasts was inhibited by GO-203 and not CP-2 (Figure 2D). AML blasts from 2 other patients (2 and 3) were also sensitive to ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT02204085

A Phase I/II Trial of the MUC1 Inhibitor, GO-203-2C in ... 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 ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S2950328024000177

Real-world outcomes of newly diagnosed AML treated with ... A total of 272 (47%) achieved CR, and a further 113 (20%) achieved CRi, for a CR/CRi rate of 67% (Table 2). The best response was morphological leukemia-free ...

5. dana-farber.org dana-farber.org/clinical-trials/14-222

A Phase I/II Trial of the MUC1 inhibitor, GO-203-2c alone ... 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 ...

6. sciencedirect.com sciencedirect.com/science/article/pii/S0006497119831750

Phase I/Ib Trial of the MUC1 Inhibitor GO-203-2C Alone ... MUC1 inhibition also resulted in decreased PD-L1 expression in leukemia cells and reduction in myeloid derived suppressor cells as demonstrated ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11966364/

Acute Myeloid Leukemia: 2025 Update on Diagnosis, Risk ... Acute myeloid leukemia (AML) is a bone marrow stem cell cancer that is often fatal despite available treatments. Diagnosis, risk assessment, monitoring, ...

8. acsjournals.onlinelibrary.wiley.com acsjournals.onlinelibrary.wiley.com/doi/full/10.1002/cncr.21326

Final report of the efficacy and safety of gemtuzumab ... The current results indicate that GO is an effective monotherapy for patients with AML in recurrence and confirm the Phase II data from the ...