Study on ONC201 for Treating Patients with Relapsed or Hard-to-Treat Acute Leukemia or High-Risk Myelodysplastic Syndrome

Sponsor: M.D. Anderson Cancer Center

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT02392572

Provider View

This clinical trial is testing the safety and best dose of a new drug, ONC201, to see if it can help treat patients with acute leukemia or high-risk myelodysplastic syndrome that has come back or is not responding to treatment.

Patient Parameters

Program Overview

This study is exploring the effects of a new drug called ONC201 on patients with acute leukemia or high-risk myelodysplastic syndrome that has either returned after treatment or is not responding to current treatments. The drug works by blocking certain enzymes that cancer cells need to grow. The trial aims to find the best dose and understand the side effects of ONC201, as well as how well it works in shrinking cancer cells.

Description

The study is divided into two phases: Phase I and Phase II.

Primary Objectives:

  • Phase I: Determine the best dose of ONC201 for patients with relapsed or refractory acute myelogenous leukemia (AML), myelodysplastic syndrome (MDS), or acute lymphoblastic leukemia (ALL).
  • Phase I: Identify any side effects of ONC201 in these patients.
  • Phase II: Measure how well ONC201 works in treating these conditions.

Secondary Objectives:

  • Phase I: Study how the body processes ONC201.
  • Phase I: Observe the effects of ONC201 on tumors.
  • Phase II: Confirm the tolerability of the recommended dose.
  • Phase II: Assess the clinical outcomes of ONC201 treatment.
  • Phase II: Link clinical outcomes with specific tumor and blood markers.

Study Design:

  • This is a dose-escalation study followed by a Phase II study.
  • Patients are divided into five groups, each receiving ONC201 in different schedules:
    • Arm A: ONC201 once every 3 weeks.
    • Arm B: ONC201 once every week.
    • Arm C: ONC201 on the first two consecutive days of every week.
    • Arm D: ONC201 once daily.
    • Arm E: ONC201 twice weekly.
  • Treatment cycles repeat every 21 or 28 days unless the disease progresses or side effects become unacceptable.
  • After completing the study treatment, patients are followed up after 28 days.

Eligibility Criteria

Inclusion Criteria

  • Must have relapsed or refractory acute leukemia or high-risk myelodysplastic syndrome with no expected effective standard treatments.
  • Age 18 years or older.
  • Good overall health with an ECOG performance status of 0-2.
  • Women of childbearing potential must use effective birth control during the study and for 16 weeks after the last dose. Men must also use birth control during the study and for 16 weeks after.
  • Must be able to provide written informed consent.
  • Must have stopped previous treatments for at least 2 weeks for cytotoxic agents or 5 half-lives for noncytotoxic agents.
  • Must have adequate kidney and liver function as shown by specific blood tests.
  • If previously treated with stem cell transplantation, must be more than 6 months since the procedure and meet specific health criteria.

Exclusion Criteria

  • Have uncontrolled illnesses such as infections, heart failure, or psychiatric conditions that would interfere with the study.
  • Have active heart disease or recent heart attack.
  • Have received other treatments for their blood cancer within the past 2 weeks for cytotoxic agents or 5 half-lives for noncytotoxic agents.
  • Have been treated for another cancer within the past 3 years, except for certain low-risk cancers.
  • Have a history of HIV, hepatitis B, or hepatitis C.
  • Have active drug or alcohol use.
  • Have known or active leukemia in the central nervous system.
  • Are using strong CYP3A inhibitors.

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