A Study of CG-806 in Patients With Relapsed or Refractory AML or Higher-Risk MDS

March 5, 2025 updated by: Aptose Biosciences Inc.

A Phase 1a/b Trial of CG-806 in Patients With Relapsed/Refractory Acute Myeloid Leukemia or Higher-Risk Myelodysplastic Syndromes

This study is being done to evaluate the safety, tolerability and antitumor activity of oral CG-806 (luxeptinib) for the treatment of patients with Acute Myeloid Leukemia (except APML), secondary AML, therapy-related AML, or higher-risk MDS, whose disease has relapsed, is refractory or who are ineligible for or intolerant of intensive chemotherapy or transplantation.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

This is a multicenter, open-label, Phase 1 a/b dose escalation study of safety, pharmacodynamics, and pharmacokinetics of CG-806 in ascending cohorts (3+3 design) to determine the MTD or recommended dose in patients with relapsed or refractory Acute Myeloid Leukemia (except APML), secondary AML, therapy-related AML, or higher-risk MDS whose disease has relapsed, is refractory or who are ineligible for or intolerant of intensive chemotherapy or transplantation. This is to be followed by a cohort expansion phase.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
45

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

  • Name: Nawazish Khan, MD, MS
  • Phone Number: 858-275-6359
  • Email: nkhan@aptose.com

Study Locations

  • United States
    • California
      • Duarte, California, United States, 91010
        • City of Hope National Medical Center
      • Los Angeles, California, United States, 90033
        • University of Southern California
    • Florida
      • Miami, Florida, United States, 33136
        • University of Miami
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Northwestern University
    • Louisiana
      • New Orleans, Louisiana, United States, 70121
        • Ochsner Healthcare
    • New Jersey
      • Morristown, New Jersey, United States, 07962
        • Atlantic Hematological Oncology Center
    • New York
      • Buffalo, New York, United States, 14263
        • Roswell Park Comprehensive Cancer Center
      • New York, New York, United States, 10065
        • Memorial Sloan Kettering Cancer Center
    • Ohio
      • Cleveland, Ohio, United States, 44106
        • University Hospital of Cleveland
    • Texas
      • Houston, Texas, United States, 77030
        • University of Texas MD Anderson Cancer Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  • Age ≥18 years
  • Life expectancy of at least 3 months
  • ECOG Performance Status ≤ 2
  • Patients must be able to swallow capsules
  • Adequate hematologic parameters, unless cytopenias are disease caused
  • Adequate renal, liver and cardiac functions

Key Exclusion Criteria:

  • Patients with GVHD requiring systemic immunosuppressive therapy
  • Uncontrolled leptomeningeal disease, auto-immune hemolytic anemia and uncontrolled and clinically significant disease related metabolic disorder
  • Clinically significant leukostasis
  • Treatment with other investigational drugs or receipt of cytotoxic therapy within 14 days prior to first study treatment administration
  • Receipt of cellular immunotherapeutic agents within 4 weeks prior to first study treatment administration

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Dose Escalation and Expansion
Dose Escalation and Expansion; CG-806 will be given orally in ascending doses in patients with relapsed or refractory AML or higher-risk MDS (escalation cohort), until the maximum tolerated dose or candidate recommended Phase 2 dose is reached. Followed up by up to 50 patients enrolled in the expansion cohort at the recommended dose.
Drug: CG-806
CG-806 will be given orally in ascending doses starting at 450 mg PO BID until the maximum tolerated dose or candidate recommended Phase 2 dose is reached.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Incidence of treatment-emergent adverse events of CG-806
Time Frame: At the end of Cycle 1 (each cycle is 28 days)
Patients will be assessed for adverse events during all cycles of treatment and for dose limiting toxicities in Cycle 1 (28-days). Dose escalation to a higher dose level will be considered if none of the first three patients who complete Cycle 1 (28-days) at a given dose level experience a dose limiting toxicity or if only 1 of 6 patients at a given dose level experience a dose-limiting toxicity.
At the end of Cycle 1 (each cycle is 28 days)
Establish a CG-806 dose that maintains a biologically active plasma concentration
Time Frame: At the end of Cycle 1 (each cycle is 28 days)
To determine the dose of CG-806 given orally every 12 hours daily that maintains a biologically active plasma concentration during 28-day cycles.
At the end of Cycle 1 (each cycle is 28 days)
Establish a recommended dose for future development of CG-806
Time Frame: At the end of Cycle 1 (each cycle is 28 days)
To establish the maximum tolerated dose and/or recommended Phase 2 dose (RP2D) of CG-806 for future clinical trials in patients with AML and other advanced myeloid malignancies.
At the end of Cycle 1 (each cycle is 28 days)

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Pharmacokinetics variables including maximum plasma concentration (Cmax).
Time Frame: At the end of Cycle 1 (each cycle is 28 days)
Pharmacokinetics variables including maximum plasma concentration at various timepoints.
At the end of Cycle 1 (each cycle is 28 days)
Pharmacokinetics variables including minimum plasma concentration (Cmin)
Time Frame: At the end of Cycle 1 (each cycle is 28 days)
Pharmacokinetics variables including minimum plasma concentration at various timepoints.
At the end of Cycle 1 (each cycle is 28 days)
Pharmacokinetics variables including area under the curve (AUC)
Time Frame: At the end of Cycle 1 (each cycle is 28 days)
Pharmacokinetics variables including plasma concentration at various timepoints.
At the end of Cycle 1 (each cycle is 28 days)
Pharmacokinetics variables including volume of distribution
Time Frame: At the end of Cycle 1 (each cycle is 28 days)
Pharmacokinetics variables including plasma concentration at various timepoints.
At the end of Cycle 1 (each cycle is 28 days)
Pharmacokinetics variables including clearance
Time Frame: At the end of Cycle 1 (each cycle is 28 days)
Pharmacokinetics variables including plasma concentration at various timepoints.
At the end of Cycle 1 (each cycle is 28 days)
Pharmacokinetics variables including plasma half-life.
Time Frame: At the end of Cycle 1 (each cycle is 28 days)
Pharmacokinetics variables including plasma concentration at various timepoints.
At the end of Cycle 1 (each cycle is 28 days)
To determine the ability of CG-806 to modulate the expression or activity of pharmacodynamic biomarkers of drug effect.
Time Frame: At the end of Cycle 1 (each cycle is 28 days)
To determine the ability of CG-806 to modulate the expression or activity of pharmacodynamic biomarkers of drug effect.
At the end of Cycle 1 (each cycle is 28 days)
To assess patients for evidence of anti-tumor activity of CG-806 based on hematologic, bone marrow, physical examination, evaluations
Time Frame: At the end of Cycle 1 (each cycle is 28 days)
To assess patients for evidence of anti-tumor activity of CG-806 based on hematologic, bone marrow, physical examination, and FDG PET-CT imaging evaluations.
At the end of Cycle 1 (each cycle is 28 days)
To determine the Relative Bioavailability of Generation 3 formulation given to up to 18 patients on Cycle 1 Day -3 compared to Generation 1 formulation of study drug given to patients during Cycle 1.
Time Frame: At the end of Cycle 1 (each cycle is 28 days)
Compare G1 to G3 pharmacokinetics variables including maximum plasma concentration (Cmax)
At the end of Cycle 1 (each cycle is 28 days)
To determine the Relative Bioavailability of Generation 3 formulation given to up to 18 patients on Cycle 1 Day -3 compared to Generation 1 formulation of study drug given to patients during Cycle 1.
Time Frame: At the end of Cycle 1 (each cycle is 28 days)
Compare G1 to G3 Pharmacokinetics variables including minimum plasma concentration (Cmin)
At the end of Cycle 1 (each cycle is 28 days)
To determine the Relative Bioavailability of Generation 3 formulation given to up to 18 patients on Cycle 1 Day -3 compared to Generation 1 formulation of study drug given to patients during Cycle 1.
Time Frame: At the end of Cycle 1 (each cycle is 28 days)
Compare G1 to G3 Pharmacokinetics variables including area under the curve (AUC)
At the end of Cycle 1 (each cycle is 28 days)
To determine the Relative Bioavailability of Generation 3 formulation given to up to 18
Time Frame: At the end of Cycle 1 (each cycle is 28 days)
Compare G1 to G3 Pharmacokinetics variables including volume of distribution
At the end of Cycle 1 (each cycle is 28 days)
To determine the Relative Bioavailability of Generation 3 formulation given to up to 18
Time Frame: At the end of Cycle 1 (each cycle is 28 days)
Compare G1 to G3 Pharmacokinetics variables including clearance
At the end of Cycle 1 (each cycle is 28 days)
Compare G1 to G3 Pharmacokinetics variables including clearance
Time Frame: At the end of Cycle 1 (each cycle is 28 days)
Compare G1 to G3 Pharmacokinetics variables including plasma half-life.
At the end of Cycle 1 (each cycle is 28 days)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Aptose Biosciences Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Rafael Bejar, MD, PhD, Aptose Biosciences Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 6, 2020

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 15, 2024

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
April 15, 2024

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 13, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 15, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 20, 2020

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 25, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 5, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • APTO-CG-806-03

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

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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