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

NCT02204085 | Active Not Recruiting Phase 1 DMC

• 1 other identifier: 14-222
• Study Type: interventional
• Target: 33
• Locations: 1 country
• Sites: 2

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.

Conditions

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

Keywords

Refractory acute myeloid leukemia; Relapsed acute myeloid leukemia

Outcome Measures

Primary Outcomes (2)

• Maximum Tolerated Dose of GO-203-2c

  Phase I

  28 days

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

  Phase 1

  28 days

Secondary Outcomes (5)

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

  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

  2 Years

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

  2 Years

• 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

  2 Years

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

  2 years

Study Arms (2)

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

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.

Drug: GO-203-2c; Drug: GO-203-2c + Decitabine

Interventions

GO-203-2c DRUG

GO-203-2c; GO-203-2c + Decitabine

GO-203-2c + Decitabine DRUG

GO-203-2c + Decitabine

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Beth Israel Deaconess Medical Center: lead
• Dana-Farber Cancer Institute: collaborator

Study Sites (2)

Dana-Farber Cancer Institute

Boston, Massachusetts, 02115, United States

Location

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

Decitabine

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Azacitidine; Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Officials

• David Avigan, MD

  Beth Israel Deaconess Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: July 9, 2014
• First Posted: July 30, 2014
• Study Start: September 1, 2014
• Primary Completion: September 1, 2025
• Study Completion: December 1, 2025
• Last Updated: January 24, 2025

Record last verified: 2025-01

Locations

US (2)