NCT02267863

Brief Summary

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.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
21

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Oct 2014

Longer than P75 for phase_1

Geographic Reach
1 country

13 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2014

Completed
2 days until next milestone

First Submitted

Initial submission to the registry

October 3, 2014

Completed
17 days until next milestone

First Posted

Study publicly available on registry

October 20, 2014

Completed
6.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2021

Completed
Last Updated

August 22, 2022

Status Verified

August 1, 2022

Enrollment Period

6.9 years

First QC Date

October 3, 2014

Last Update Submit

August 18, 2022

Conditions

Keywords

AMLMDSLeukemiaAcute Myeloid LeukemiaAptoseAPTO-253Kinase Inhibitor

Outcome Measures

Primary Outcomes (3)

  • Incidence of treatment-emergent adverse events of APTO-253

    To determine the safety and tolerability of APTO-253 by assessing treatment-related adverse events as assessed by CTCAE v4.0.

    Cycle 1 (28 days)

  • Maximum tolerated dose and dose limiting toxicities

    To determine the maximum tolerated dose (MTD) and the dose limiting toxicities (DLT) of APTO-253 when given on days 1, 8, 15, and 22 of each 28-day cycle.

    Cycle 1 (28 days)

  • Establish recommended dose for future development of APTO-253

    To establish the dose of APTO-253 recommended for future development of APTO-253 for patients with specific types of hematologic malignancies.

    Up to 7 months

Secondary Outcomes (8)

  • Pharmacokinetic variables including maximum plasma concentration (Cmax)

    Cycle 1 (28 days)

  • Pharmacokinetic variables including minimum plasma concentration (Cmin)

    Cycle 1 (28 days)

  • Pharmacokinetic variables including Area Under the Curve (AUC)

    Cycle 1 (28 days)

  • Pharmacokinetic variables including volume of distribution

    Cycle 1 (28 days)

  • Pharmacokinetic variables including clearance

    Cycle 1 (28 days)

  • +3 more secondary outcomes

Study Arms (1)

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.

Drug: APTO-253

Interventions

APTO-253 will be given in ascending doses starting at 20 mg/m2 until the maximum tolerated dose or recommended dose is reached.

Dose Escalation and Expansion

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (13)

University of Arizona Cancer Center

Tucson, Arizona, 85724, United States

Location

UC San Diego Moores Cancer Center

La Jolla, California, 92093-0698, United States

Location

University of California, Irvine

Orange, California, 92868, United States

Location

Emory University; Winship Cancer Institute

Atlanta, Georgia, 30322, United States

Location

Ochsner Cancer Institute

New Orleans, Louisiana, 70121, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

St. Vincent Frontier Cancer Center

Billings, Montana, 59102, United States

Location

University of Rochester; Wilmot Cancer Institute Clinical Trials Office

Rochester, New York, 14643, United States

Location

University Hospital

Cleveland, Ohio, 44106, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Prisma Health, Institute for Translational Oncology Research

Greenville, South Carolina, 29605, United States

Location

Baylor Research Institute

Dallas, Texas, 75246, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

MeSH Terms

Conditions

Leukemia, Myeloid, AcuteRecurrenceLeukemia

Interventions

APTO-253

Condition Hierarchy (Ancestors)

Leukemia, MyeloidNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • Rafael Bejar, MD., PhD.

    Aptose Biosciences Inc.

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 3, 2014

First Posted

October 20, 2014

Study Start

October 1, 2014

Primary Completion

September 1, 2021

Study Completion

September 1, 2021

Last Updated

August 22, 2022

Record last verified: 2022-08

Locations