Acute Myeloid LeukemiaPhase 1NCT04477291
A Study of CG-806 in Patients With Relapsed or Refractory AML or Higher-Risk MDS
A Phase 1 study testing CG-806 in people with Acute Myeloid Leukemia and Myelodysplastic Syndromes, run by Aptose Biosciences Inc. at 10 sites in United States.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia, Myelodysplastic Syndromes
- What is being tested?
- CG-806
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 45 participants
- Where
- 10 sitesUnited States
- Study dates
- 6 Oct 2020to 15 Apr 2024
- Phase
- Phase 1
- 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
- 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
Who cannot join
- 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
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Roswell Park Comprehensive Cancer Center
Buffalo, New York, 14263
Northwestern University
Chicago, Illinois, 60611
University Hospital of Cleveland
Cleveland, Ohio, 44106
City of Hope National Medical Center
Duarte, California, 91010
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
University of Southern California
Los Angeles, California, 90033
University of Miami
Miami, Florida, 33136
Atlantic Hematological Oncology Center
Morristown, New Jersey, 07962
Ochsner Healthcare
New Orleans, Louisiana, 70121
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
About this study
Official title: 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.
Read the 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.
- Conditions
- Acute Myeloid Leukemia, Myelodysplastic Syndromes
- Being tested
- CG-806
- Keywords
- CG-806, Aptose, FLT3, FLT3-ITD, D835Y, F691L, BTK, C481S, TP53, NRAS, IDH1, BCL2, Gilteritinib, Quizartinib, Midostaurin, Crenolanib, Venetoclax, Ibrutinib, Acalabrutinib, Zanubrutinib
- Medical subject headings
- Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Leukemia, Lymphocytic, Chronic, B-Cell, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CG-806Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| Dose Escalation and Expansion | Experimental | 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. Receives: Drug: CG-806 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events of CG-806
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.
Measured: At the end of Cycle 1 (each cycle is 28 days)
Establish a CG-806 dose that maintains a biologically active plasma concentration
To determine the dose of CG-806 given orally every 12 hours daily that maintains a biologically active plasma concentration during 28-day cycles.
Measured: At the end of Cycle 1 (each cycle is 28 days)
Establish a recommended dose for future development of CG-806
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.
Measured: At the end of Cycle 1 (each cycle is 28 days)
Secondary outcomes (14)
Pharmacokinetics variables including maximum plasma concentration (Cmax).
Pharmacokinetics variables including maximum plasma concentration at various timepoints.
Measured: At the end of Cycle 1 (each cycle is 28 days)
Pharmacokinetics variables including minimum plasma concentration (Cmin)
Pharmacokinetics variables including minimum plasma concentration at various timepoints.
Measured: At the end of Cycle 1 (each cycle is 28 days)
Pharmacokinetics variables including area under the curve (AUC)
Pharmacokinetics variables including plasma concentration at various timepoints.
Measured: At the end of Cycle 1 (each cycle is 28 days)
Pharmacokinetics variables including volume of distribution
Pharmacokinetics variables including plasma concentration at various timepoints.
Measured: At the end of Cycle 1 (each cycle is 28 days)
Pharmacokinetics variables including clearance
Pharmacokinetics variables including plasma concentration at various timepoints.
Measured: At the end of Cycle 1 (each cycle is 28 days)
Pharmacokinetics variables including plasma half-life.
Pharmacokinetics variables including plasma concentration at various timepoints.
Measured: 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.
To determine the ability of CG-806 to modulate the expression or activity of pharmacodynamic biomarkers of drug effect.
Measured: 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
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.
Measured: 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.
Compare G1 to G3 pharmacokinetics variables including maximum plasma concentration (Cmax)
Measured: 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.
Compare G1 to G3 Pharmacokinetics variables including minimum plasma concentration (Cmin)
Measured: 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.
Compare G1 to G3 Pharmacokinetics variables including area under the curve (AUC)
Measured: 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
Compare G1 to G3 Pharmacokinetics variables including volume of distribution
Measured: 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
Compare G1 to G3 Pharmacokinetics variables including clearance
Measured: At the end of Cycle 1 (each cycle is 28 days)
Compare G1 to G3 Pharmacokinetics variables including clearance
Compare G1 to G3 Pharmacokinetics variables including plasma half-life.
Measured: At the end of Cycle 1 (each cycle is 28 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2020
- Study start6 Oct 2020
- Primary completion15 Apr 2024
- Study completion15 Apr 2024
Why it stopped: Change in corporate strategy
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 45 participants (actual)
Sponsor and investigators
- Lead sponsor
- Aptose Biosciences Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Rafael Bejar, MD, PhD, Study director, Aptose Biosciences Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04477291
- Sponsor's protocol ID
- APTO-CG-806-03
- Status last verified
- Aug 2024
- First submitted
- 13 Jul 2020
- Last updated
- 7 Mar 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Yu G, Zhang W, Basyal M, Nishida Y, Mizumo H, Ly C, Zhang H, Rice WG, Andreeff M. The multi-kinase inhibitor CG-806 exerts anti-cancer activity against acute myeloid leukemia by co-targeting FLT3, BTK, and aurora kinases. Leuk Lymphoma. 2024 Nov;65(11):1659-1674. doi: 10.1080/10428194.2024.2364839. Epub 2024 Jun 13.
PubMed 38871487Yu G, Zhang W, Zhang H, Ly C, Basyal M, Rice WG, Andreeff M. The multi-kinase inhibitor CG-806 exerts anti-cancer activity against acute myeloid leukemia by co-targeting FLT3, BTK, and Aurora kinases. Res Sq [Preprint]. 2023 Feb 22:rs.3.rs-2570204. doi: 10.21203/rs.3.rs-2570204/v1.
PubMed 36865133
Study at a glance
From the registry record, last updated by the sponsor on 7 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1a/b Trial of CG-806 in Patients With Relapsed/Refractory Acute Myeloid Leukemia or Higher-Risk Myelodysplastic Syndromes
- ClinicalTrials.gov ID
- NCT04477291
- Acronym / sponsor ID
- APTO-CG-806-03
- Conditions
- Acute Myeloid Leukemia, Myelodysplastic Syndromes
- Keywords
- CG-806, Aptose, FLT3, FLT3-ITD, D835Y, F691L, BTK, C481S
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Change in corporate strategy
- Enrolment
- 45 participants (actual)
- Lead sponsor
- Aptose Biosciences Inc.Industry (pharma, biotech, devices)
- Study start
- 6 Oct 2020 (actual)
- Primary completion
- 15 Apr 2024 (actual)
- Study completion
- 15 Apr 2024 (actual)
- First posted
- 20 Jul 2020 (actual)
- 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 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.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dose Escalation and Expansion | Experimental | Drug: CG-806Dose 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (5 listed)
- 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
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 |
|---|---|---|
| CG-806CG-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. | Drug | Dose Escalation and Expansion |
Primary outcome
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.
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.
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.
14 secondary outcomes
Pharmacokinetics variables including maximum plasma concentration (Cmax).
Time frame: 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 area under the curve (AUC)
Time frame: 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 clearance
Time frame: 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)
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 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 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)
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)
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)
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)
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
Time frame: At the end of Cycle 1 (each cycle is 28 days)
Results and safety
The study was stopped early (reason given: Change in corporate strategy). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Mar 2025.
- Derived from this study: PubMed 38871487Yu G, Zhang W, Basyal M, Nishida Y, Mizumo H, Ly C, Zhang H, Rice WG, Andreeff M. The multi-kinase inhibitor CG-806 exerts anti-cancer activity against acute myeloid leukemia by co-targeting FLT3, BTK, and aurora kinases. Leuk Lymphoma. 2024 Nov;65(11):1659-1674. doi: 10.1080/10428194.2024.2364839. Epub 2024 Jun 13.
- Derived from this study: PubMed 36865133Yu G, Zhang W, Zhang H, Ly C, Basyal M, Rice WG, Andreeff M. The multi-kinase inhibitor CG-806 exerts anti-cancer activity against acute myeloid leukemia by co-targeting FLT3, BTK, and Aurora kinases. Res Sq [Preprint]. 2023 Feb 22:rs.3.rs-2570204. doi: 10.21203/rs.3.rs-2570204/v1.
- Individual participant dataSharing planned: No
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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.
Rafael Bejar, MD, PhD study director
Aptose Biosciences Inc.
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CG-806 in people with Acute Myeloid Leukemia and Myelodysplastic Syndromes, run by Aptose Biosciences Inc. at 10 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 6 inclusion and 5 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: stopped early, as of the sponsor's last update on 7 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 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 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.
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 was stopped early (reason given: Change in corporate strategy). Results are not available in the source record.
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?
Incidence of treatment-emergent adverse events of CG-806 (time frame: At the end of Cycle 1 (each cycle is 28 days)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04477291, which also lists 2 linked publications.
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 7 Mar 2025.