Ongoing Clinical Trials for Myelodysplastic Syndrome With Excess Blasts

Currently, there is 1 ongoing clinical trial for patients with Myelodysplastic Syndrome With Excess Blasts. This trial is investigating targeted treatments combined with chemotherapy for patients who have a specific genetic mutation (FLT3). The study is being conducted across multiple European countries and compares two different oral medications to find the most effective treatment approach.

Clinical trial locations

• Austria
  • Study Comparing Gilteritinib and Midostaurin with Chemotherapy for Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome with FLT3 Mutation
• Belgium
  • Study Comparing Gilteritinib and Midostaurin with Chemotherapy for Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome with FLT3 Mutation
• Finland
  • Study Comparing Gilteritinib and Midostaurin with Chemotherapy for Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome with FLT3 Mutation
• France
  • Study Comparing Gilteritinib and Midostaurin with Chemotherapy for Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome with FLT3 Mutation
• Germany
  • Study Comparing Gilteritinib and Midostaurin with Chemotherapy for Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome with FLT3 Mutation
• Ireland
  • Study Comparing Gilteritinib and Midostaurin with Chemotherapy for Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome with FLT3 Mutation
• Lithuania
  • Study Comparing Gilteritinib and Midostaurin with Chemotherapy for Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome with FLT3 Mutation
• Netherlands
  • Study Comparing Gilteritinib and Midostaurin with Chemotherapy for Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome with FLT3 Mutation
• Norway
  • Study Comparing Gilteritinib and Midostaurin with Chemotherapy for Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome with FLT3 Mutation
• Spain
  • Study Comparing Gilteritinib and Midostaurin with Chemotherapy for Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome with FLT3 Mutation
• Sweden
  • Study Comparing Gilteritinib and Midostaurin with Chemotherapy for Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome with FLT3 Mutation

Study Comparing Gilteritinib and Midostaurin with Chemotherapy for Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome with FLT3 Mutation

This clinical trial is investigating two types of blood cancers: Acute Myeloid Leukemia and Myelodysplastic Syndrome with excess blasts-2 (MDS-EB2). Both conditions affect the bone marrow and blood cells, disrupting the production of healthy blood cells.

Main goal of the trial: The study aims to compare the effectiveness of two oral medications, gilteritinib and midostaurin, when used alongside chemotherapy in patients with a specific genetic change called the FLT3 mutation. This mutation affects how these blood cancers progress. The trial will evaluate which treatment is better at prolonging the time patients remain free from disease progression or relapse.

Investigational drugs: The trial is testing two medications:

• Gilteritinib (also known as ASP2215 or Xospata): This medication is a tyrosine kinase inhibitor that works by blocking the FLT3 protein, which is often overactive in these types of cancer. It is taken orally in tablet form.
• Midostaurin (also known as Rydapt): This is a multi-kinase inhibitor that blocks multiple proteins, including FLT3, to prevent cancer cell growth. It is taken orally in capsule form.

How the trial works: The study follows a structured treatment plan with several phases:

1. Induction therapy: Participants receive either gilteritinib or midostaurin combined with chemotherapy to reduce cancer cells and achieve remission.
2. Consolidation therapy: The same medication continues with chemotherapy to eliminate any remaining cancer cells.
3. Maintenance therapy: For one year, participants continue taking their assigned medication to prevent the cancer from returning.

Who can participate – Inclusion criteria:

• You must be at least 18 years old
• You must have newly diagnosed Acute Myeloid Leukemia or MDS with excess blasts-2 (MDS-EB2)
• Your condition must have a confirmed FLT3 gene mutation
• You must be considered eligible for intensive chemotherapy
• You must be able to take medication by mouth
• You must have a WHO/ECOG performance status of 2 or less, meaning you can perform most daily activities
• Your liver and kidney function must be adequate, with creatinine clearance greater than 40 mL/min
• You must provide written informed consent
• Female participants must agree to use effective birth control during the study and for a specified time afterward, and must not breastfeed or donate eggs
• Male participants must use effective birth control methods with their partners and must not donate sperm during the study

Who cannot participate – Exclusion criteria:

• Patients who do not have newly diagnosed AML with a FLT3 mutation or MDS-EB2
• Patients who are not eligible for intensive chemotherapy
• Patients who fall outside the specified age range
• Patients who belong to vulnerable populations that the study is not designed to include

The trial is expected to continue until 2031 and will monitor participants for their overall survival, how quickly their blood cell counts recover after chemotherapy, and any side effects they experience. This long-term study will provide important information about the benefits and safety of these treatments for patients with FLT3-positive blood cancers.

Summary

There is currently one clinical trial available for patients with Myelodysplastic Syndrome With Excess Blasts who have the FLT3 mutation. This trial offers access to patients across 11 European countries, including France, Germany, Finland, Belgium, Netherlands, Spain, Austria, Norway, Sweden, Ireland, and Lithuania, making it widely accessible across Europe.

The study is particularly focused on comparing two targeted therapies, gilteritinib and midostaurin, both of which work by blocking the FLT3 protein that drives cancer cell growth in patients with this specific mutation. Both medications are taken orally, which may be more convenient for patients compared to intravenous treatments.

The trial uses a comprehensive treatment approach with three distinct phases: induction to achieve remission, consolidation to strengthen it, and a full year of maintenance therapy to help prevent relapse. This structured approach aims to provide both immediate and long-term disease control. Patients interested in participating should discuss with their healthcare provider whether they meet the eligibility criteria, particularly the requirement for FLT3 mutation testing and suitability for intensive chemotherapy.