Study on Azacitidine and Quizartinib for Treating Myelodysplastic Syndrome or Myelodysplastic/Myeloproliferative Neoplasm with FLT3 or CBL Mutations

Sponsor: M.D. Anderson Cancer Center

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT04493138

Provider View

This clinical trial is testing a combination of two drugs, azacitidine and quizartinib, to see if they can effectively treat patients with certain blood disorders that have specific genetic mutations. The study aims to find the best dose and understand the side effects of this treatment.

Patient Parameters

Program Overview

This study is exploring whether a combination of two drugs, azacitidine and quizartinib, can help control myelodysplastic syndrome (MDS) or myelodysplastic/myeloproliferative neoplasm (MDS/MPN) in patients with specific genetic mutations (FLT3 or CBL). Azacitidine is a chemotherapy drug that works by stopping cancer cells from growing, while quizartinib may block enzymes needed for cancer cell growth. The trial will determine the safest and most effective dose of quizartinib when used with azacitidine and will assess how well this combination works in treating these conditions.

Description

The main goals of this study are to determine the safety and best dose of quizartinib when combined with azacitidine, and to see how well this combination works in treating patients with certain blood disorders. The study will also look at how long patients live without the disease getting worse and how long they respond to the treatment.

In the first phase, different doses of quizartinib will be tested to find the safest and most effective dose. In the second phase, the study will focus on how well the treatment works.

Participants will receive azacitidine either as an injection under the skin or through an IV over about 30 minutes on days 1-5 of each 28-day cycle. They will also take quizartinib by mouth once a day for 28 days. This cycle will repeat as long as the treatment is effective and side effects are manageable.

After finishing the treatment, participants will have a follow-up visit 30 days later to check on their health and any side effects.

Eligibility Criteria

Inclusion Criteria

  • Must be 18 years or older, as MDS is rare in children.
  • Diagnosed with myelodysplastic syndrome (MDS) or myelodysplastic/myeloproliferative neoplasm (MDS/MPN) according to specific medical criteria.
  • For patients who have not received hypomethylating agents before: must have a certain level of disease severity or specific genetic features.
  • For patients who have received hypomethylating agents before: must not have responded to previous treatments or have experienced a relapse.
  • Must have specific genetic mutations (FLT3-ITD or CBL mutations) detectable in bone marrow or blood.
  • Must have adequate kidney and liver function based on blood tests.
  • Must have a performance status score of 0-2, indicating good overall health.
  • Must provide informed consent to participate in the study.
  • Previous use of certain medications for blood cell growth or leukocytosis control is allowed.

Exclusion Criteria

  • Have an uncontrolled infection not responding to antibiotics.
  • Have a heart condition that affects the heart's electrical activity or rhythm.
  • Have a history of severe heart problems, such as heart attacks or heart failure.
  • Are taking medications that affect the heart's rhythm or interact with the study drugs.
  • Are pregnant or breastfeeding.
  • Are not willing to use effective birth control during the study.
  • Have a positive pregnancy test at screening.
  • Are participating in another clinical trial or receiving other cancer treatments.
  • Have active hepatitis B or C infections or a history of HIV.

Get This Program In Your Inbox

Receive detailed information about this treatment opportunity to review with your healthcare provider.