Clinical Trials /

A Study of APTO-253 in Patients With Relapsed or Refractory AML or MDS

NCT02267863

Description:

This study is being done to evaluate the safety and effectiveness of APTO-253 for the treatment of patients with the condition of acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS) for which either the standard treatment has failed, is no longer effective, or can no longer be administered safely or poses a risk for your general well being.

Related Conditions:
  • Acute Myeloid Leukemia
  • Myelodysplastic Syndromes
Recruiting Status:

Recruiting

Phase:

Phase 1

Trial Eligibility

Document

Title

  • Brief Title: A Study of APTO-253 in Patients With Relapsed or Refractory AML or MDS
  • Official Title: A Phase Ib Dose Escalation and Expansion, Multicenter, Open-label, Safety, Pharmacokinetic and Pharmacodynamic Study of APTO-253 in Patients With Relapsed or Refractory Acute Myelogenous Leukemia or High-Risk Myelodysplasia

Clinical Trial IDs

  • ORG STUDY ID: 253-HEM1-01
  • NCT ID: NCT02267863

Conditions

  • Acute Myelogenous Leukemia in Relapse
  • Acute Myelogenous Leukemia, Relapsed, Adult
  • Acute Myelogenous Leukemia, Adult
  • Acute Myelogenous Leukemia
  • High Risk Myelodysplasia

Interventions

DrugSynonymsArms
APTO-253Dose Escalation and Expansion

Purpose

This study is being done to evaluate the safety and effectiveness of APTO-253 for the treatment of patients with the condition of acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS) for which either the standard treatment has failed, is no longer effective, or can no longer be administered safely or poses a risk for your general well being.

Detailed Description

      This is a multicenter, open-label, Phase Ib dose escalation study of safety,
      pharmacodynamics, and pharmacokinetics of APTO-253 in ascending cohorts (3+3 design) to
      determine the MTD or recommended dose in patients with relapsed or refractory acute
      myelogenous leukemia (AML) or high-risk MDS patients. This is to be followed by a cohort
      expansion phase at the MTD or recommended dose.
    

Trial Arms

NameTypeDescriptionInterventions
Dose Escalation and ExpansionExperimentalAPTO-253 will be given in ascending doses in patients with relapsed or refractory AML or high risk MDS (escalation cohort), until the maximum tolerated dose or recommended dose is reached. Followed by up to 30 patients enrolled in the expansion cohort at the recommended dose.
  • APTO-253

Eligibility Criteria

        Inclusion Criteria:

          -  Patients ≥18 years old

          -  Life expectancy of at least 2 months

          -  Off previous cancer therapy for at least 14 days, or 5 half-lives for noncytotoxic
             agents prior to first study treatment administration

          -  Patients must have a calculated creatinine clearance >60 mL/min

          -  Acceptable hematologic, renal and liver functions and coagulation status parameters

        Exclusion Criteria:

          -  Patients with GVHD requiring systemic immunosuppressive therapy

          -  Uncontrolled leptomeningeal disease, auto-immune hemolytic anemia and uncontrolled and
             clinical significant disease related metabolic disorder

          -  Clinically significant intravascular coagulation

          -  Treatment with other investigational drugs within 14 days prior to first study
             treatment administration
      
Maximum Eligible Age:N/A
Minimum Eligible Age:18 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Incidence of treatment-emergent adverse events of APTO-253
Time Frame:Cycle 1 (28 days)
Safety Issue:
Description:To determine the safety and tolerability of APTO-253 by assessing treatment-related adverse events as assessed by CTCAE v4.0.

Secondary Outcome Measures

Measure:Pharmacokinetic variables including maximum plasma concentration (Cmax)
Time Frame:Cycle 1 (28 days)
Safety Issue:
Description:Pharmacokinetic variables including maximum plasma concentration (Cmax)
Measure:Pharmacokinetic variables including minimum plasma concentration (Cmin)
Time Frame:Cycle 1 (28 days)
Safety Issue:
Description:Pharmacokinetic variables including minimum plasma concentration (Cmin)
Measure:Pharmacokinetic variables including Area Under the Curve (AUC)
Time Frame:Cycle 1 (28 days)
Safety Issue:
Description:Pharmacokinetic variables including Area Under the Curve (AUC)
Measure:Pharmacokinetic variables including volume of distribution
Time Frame:Cycle 1 (28 days)
Safety Issue:
Description:Pharmacokinetic variables including volume of distribution
Measure:Pharmacokinetic variables including clearance
Time Frame:Cycle 1 (28 days)
Safety Issue:
Description:Pharmacokinetic variables including clearance
Measure:Pharmacokinetic variables including serum half-life
Time Frame:Cycle 1 (28 days)
Safety Issue:
Description:Pharmacokinetic variables including serum half-life
Measure:Assess for any evidence of antitumor activity of APTO-253 by hematologic and bone marrow evaluations in acute leukemia and MDS.
Time Frame:Average 2 Cycles (8 weeks)
Safety Issue:
Description:To observe patients for any evidence of antitumor activity of APTO-253 by hematologic and bone marrow evaluations in acute leukemia and MDS.
Measure:Determine the ability of APTO-253 to alter the expression of pharmacodynamic biomarkers of drug effect.
Time Frame:Average 2 Cycles (8 weeks)
Safety Issue:
Description:To determine the ability of APTO-253 to alter the expression of pharmacodynamic biomarkers of drug effect.

Details

Phase:Phase 1
Primary Purpose:Interventional
Overall Status:Recruiting
Lead Sponsor:Aptose Biosciences Inc.

Trial Keywords

  • AML
  • MDS
  • Leukemia
  • Acute Myeloid Leukemia
  • Aptose
  • APTO-253
  • Kinase Inhibitor

Last Updated

December 16, 2019