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

Aptose Biosciences

Status and phase: Terminated; Phase 1

Conditions: High Risk Myelodysplasia; Acute Myelogenous Leukemia; Acute Myelogenous Leukemia in Relapse; Acute Myelogenous Leukemia, Relapsed, Adult; Acute Myelogenous Leukemia, Adult

Treatments: Drug: APTO-253

Study type: Interventional

Funder types: Industry

Identifiers

NCT02267863

253-HEM1-01

Details and patient eligibility

About

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.

Full 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.

Enrollment: 21 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Sequential Assignment

Masking: None (Open label)

---

21 participants in 1 patient group

Dose Escalation and Expansion

Experimental group

Description:

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.

Treatment:

Drug: APTO-253