CG-806 for Acute Myeloid Leukemia and High-Risk MDS

No longer recruiting at 9 trial locations

Overseen By Nawazish Khan, MD, MS

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Aptose Biosciences Inc.

Must not be taking: Immunosuppressive, Cytotoxic, Investigational drugs

Disqualifiers: GVHD, Leukostasis, Metabolic disorder, others

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

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new oral medication, CG-806, to determine its safety and effectiveness for individuals with certain blood cancers, such as Acute Myeloid Leukemia (AML) and high-risk Myelodysplastic Syndromes (MDS). The trial targets patients whose disease has returned, does not respond to treatment, or who cannot tolerate strong chemotherapy. Participants will take increasing doses of CG-806 to identify the optimal dose that works without causing excessive side effects. Suitable candidates have AML or high-risk MDS that has recurred or resisted other treatments and can swallow pills. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Do I have to stop taking my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. However, you cannot have received cytotoxic therapy (a type of cancer treatment) within 14 days before starting the study treatment.

Is there any evidence suggesting that CG-806 is likely to be safe for humans?

Research has shown that CG-806, also known as luxeptinib, has been tested for safety in treating blood cancers like acute myeloid leukemia (AML). Luxeptinib targets specific cancer pathways, which helps stop cancer cells from growing.

Studies have found that CG-806 is generally well-tolerated, with most participants not experiencing severe side effects. However, some side effects have been reported, which is common with cancer treatments. Researchers continue to study these side effects to ensure the drug's safety for a broader population.

As this study is in an early phase, its main goal is to determine the right dose that balances effectiveness with safety, minimizing side effects. While the data is promising, research is ongoing to ensure its safety for more people. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS), which often include chemotherapy and stem cell transplants, CG-806 offers a novel approach by being administered orally. Researchers are excited about CG-806 because it targets multiple pathways associated with cancer cell growth, potentially offering a more comprehensive attack on cancer cells. This dual-targeting mechanism sets it apart by not only inhibiting one but several key proteins that contribute to the disease, which could lead to improved outcomes for patients with relapsed or refractory AML or higher-risk MDS.

What evidence suggests that CG-806 might be an effective treatment for Acute Myeloid Leukemia and high-risk MDS?

Research has shown that CG-806, also known as luxeptinib, may help treat acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS). Studies have found that CG-806 effectively blocks FLT3, a protein that promotes cancer cell growth. In lab tests, CG-806 proved more effective against AML samples than other similar treatments. It demonstrated a strong ability to kill cancer cells in various AML groups, including those that have returned after treatment or have genetic issues. This trial will evaluate CG-806 in a dose escalation and expansion study to determine its effectiveness for patients whose cancer hasn't responded to previous treatments. ^{1 4 6 7 8}

Who Is on the Research Team?

Rafael Bejar, MD, PhD

Principal Investigator

Aptose Biosciences Inc.

Are You a Good Fit for This Trial?

This trial is for adults over 18 with relapsed or stubborn Acute Myeloid Leukemia (AML) or high-risk Myelodysplastic Syndrome (MDS), who can't have intense chemo or a transplant. They should be fairly active, able to swallow pills, and not on other experimental drugs recently.

Inclusion Criteria

My blood counts are within normal ranges, unless caused by my condition.

You are expected to live for at least 3 more months.

My kidney, liver, and heart are functioning well.

Exclusion Criteria

I haven't received any cell-based immune therapy in the last 4 weeks.

I haven't taken experimental drugs or had chemotherapy in the last 14 days.

I have GVHD and need drugs to suppress my immune system.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Escalation

CG-806 is given orally in ascending doses to determine the maximum tolerated dose or recommended Phase 2 dose

28 days per cycle

Multiple visits per cycle for dose escalation and monitoring

Cohort Expansion

Up to 50 patients are enrolled at the recommended dose to further evaluate safety and efficacy

28 days per cycle

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• CG-806

Trial Overview The study tests CG-806 (luxeptinib), an oral medication, to see if it's safe and effective against certain types of AML/MDS that haven't responded well to other treatments or when patients can't handle stronger therapies.

How Is the Trial Designed?

Group I: Dose Escalation and Expansion Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Aptose Biosciences Inc.

Lead Sponsor

Trials

8

Recruited

850+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04477291

A Study of CG-806 in Patients With Relapsed or Refractory ... 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 ...

2. aptose.com aptose.com/news-media/press-releases/detail/133/aptose-presents-new-preclinical-data-on-cg-806-and-apto-253

Aptose Presents New Preclinical Data on CG-806 and ... CG-806 demonstrated significant potency across sub-groups of AML cells, including relapsed/refractory AML and those with genetic abnormalities ...

3. researchgate.net researchgate.net/publication/327087928_Abstract_791_CG'806_a_first-in-class_pan-FLT3pan-BTK_inhibitor_demonstrates_superiority_to_other_FLT3_and_BTK_inhibitors_against_primary_patient_samples

Abstract 791: CG'806, a first-in-class pan-FLT3/pan-BTK ... We profiled CG'806 against primary samples from patients with AML, CLL and other hematologic malignancies to determine its activity and potency relative to ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9256809/

Luxeptinib (CG-806) Targets FLT3 and Clusters of Kinases ... In vitro profiling of luxeptinib against 186 AML fresh patient samples demonstrated greater potency relative to other FLT3 inhibitors, including ...

5. clin.larvol.com clin.larvol.com/trial-detail/NCT03893682

A Study of CG-806 in Patients With Relapsed or Refractory ... The event will include an up-to-date review of clinical data available for Aptose's clinical programs: luxeptinib (CG-806), Aptose's oral, first-in-class FLT3 ...

6. aptose.com aptose.com/clinical-trials/luxeptinib-for-aml

Luxeptinib for AML 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 ...

7. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0006497123125523

A Phase 1a/b Trial of Luxeptinib (CG-806) in Patients with ... LUX has been shown to suppress aberrant proliferative signaling in B cell malignancies and acute myeloid leukemia (AML) via regulation of BTK, LYN, SYK, AKT, ...

8. clin.larvol.com clin.larvol.com/trial-detail/NCT04477291

A Study of CG-806 in Patients With Relapsed or Refractory ... The event will include an up-to-date review of clinical data available for Aptose's clinical programs: luxeptinib (CG-806), Aptose's oral, first-in-class FLT3 ...