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

Aptose Biosciences

Status and phase: Terminated; Phase 1

Conditions: Myelodysplastic Syndromes; Acute Myeloid Leukemia

Treatments: Drug: CG-806

Study type: Interventional

Funder types: Industry

Identifiers

NCT04477291

APTO-CG-806-03

Details and patient eligibility

About

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.

Full 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.

Enrollment: 45 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Sequential Assignment

Masking: None (Open label)

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45 participants in 1 patient group

Dose Escalation and Expansion

Experimental group

Description:

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.

Treatment:

Drug: CG-806