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.
Title
- Brief Title: A Study of APTO-253 in Patients With Relapsed or Refractory AML or MDS
- Official Title: A Phase Ia/b 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
| Drug | Synonyms | Arms |
|---|
| APTO-253 | | Dose 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 Ia/b 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
| Name | Type | Description | Interventions |
|---|
| Dose Escalation and Expansion | Experimental | APTO-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. | |
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
March 25, 2021
