Ongoing Clinical Trials for Acute Myeloid Leukaemia Recurrent
There are currently 7 clinical trials underway across Europe investigating new treatments for acute myeloid leukaemia that has returned or not responded to previous treatment. These trials are testing innovative therapies including targeted medications, immunotherapies, and combination treatments in countries including Belgium, Denmark, Finland, France, Germany, Italy, Netherlands, Poland, Spain, and Sweden.
Clinical trial locations
- Belgium
- Denmark
- Finland
- France
- Study of Ziftomenib (KO-539) in Patients with Relapsed or Refractory Acute Myeloid Leukemia
- Study on CCTx-001, Cyclophosphamide Monohydrate, and Fludarabine Phosphate for Patients with Relapsed or Refractory Acute Myeloid Leukemia
- Study on the Safety of CD19 CAR-T Cells, Cyclophosphamide, and Fludarabine in Adults with Refractory or Relapsed Acute Myeloid Leukemia Expressing CD19
- Study of S227928 Alone and with Venetoclax for Patients with Relapsed or Refractory Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelomonocytic Leukemia
- Study on the Safety and Effectiveness of Quizartinib for Children and Young Adults with Acute Myeloid Leukemia (AML)
- Germany
- Study of Ziftomenib (KO-539) in Patients with Relapsed or Refractory Acute Myeloid Leukemia
- Study on CCTx-001, Cyclophosphamide Monohydrate, and Fludarabine Phosphate for Patients with Relapsed or Refractory Acute Myeloid Leukemia
- Study of S227928 Alone and with Venetoclax for Patients with Relapsed or Refractory Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelomonocytic Leukemia
- Italy
- Study of Ziftomenib (KO-539) in Patients with Relapsed or Refractory Acute Myeloid Leukemia
- Study on the Safety and Effectiveness of Eltanexor for Patients with Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia
- Study on the Safety and Effectiveness of Quizartinib for Children and Young Adults with Acute Myeloid Leukemia (AML)
- Netherlands
- Poland
- Spain
- Study of Ziftomenib (KO-539) in Patients with Relapsed or Refractory Acute Myeloid Leukemia
- Study on the Safety of Eganelisib Alone and with Cytarabine for Patients with Relapsed or Refractory Acute Myeloid Leukemia or Higher-Risk Myelodysplastic Syndromes
- Study on CCTx-001, Cyclophosphamide Monohydrate, and Fludarabine Phosphate for Patients with Relapsed or Refractory Acute Myeloid Leukemia
- Study on the Safety and Effectiveness of Quizartinib for Children and Young Adults with Acute Myeloid Leukemia (AML)
- Sweden
Study of Ziftomenib (KO-539) in Patients with Relapsed or Refractory Acute Myeloid Leukemia
This trial is evaluating ziftomenib, a new oral medication, for patients whose disease has returned or is not responding to previous treatments. The study is being conducted across multiple European countries including Poland, Italy, France, Spain, Germany, and Belgium.
Main inclusion criteria: Patients must be at least 18 years old with confirmed relapsed or refractory disease showing at least 5% cancer cells in the bone marrow. White blood cell count should be 30,000/μL or less, and participants must have a performance status of 2 or better with a life expectancy of at least 2 months.
Main exclusion criteria: The trial excludes patients under 18 years, those with severe allergic reactions to similar medications, active uncontrolled infections, cancer cells in the brain or spinal fluid, pregnant or breastfeeding women, and those with severe heart, liver, or kidney problems. Patients with other active cancers requiring treatment or those who have had major surgery within 4 weeks are also excluded.
Focus and goals: The study is evaluating ziftomenib, which works by blocking a protein called menin that plays a role in leukemia development. The medication is particularly targeted at patients with specific genetic changes in their leukemia cells, such as NPM1 mutations or KMT2A rearrangements. The research aims to find the most appropriate dose, check safety, and evaluate how well the treatment works in different patient groups.
Investigational drug: Ziftomenib (KO-539) is taken as hard capsules by mouth. It is a menin-MLL inhibitor that targets specific genetic changes in leukemia cells.
Study on the Safety and Effectiveness of Eltanexor for Patients with Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia
This trial is testing eltanexor in patients with a specific genetic mutation called NPM1 whose disease has returned or not responded to treatment. The study is taking place in Italy.
Main inclusion criteria: Patients must be at least 60 years old with documented relapsed or refractory NPM1-mutated disease. They must be able to swallow oral medication and have adequate liver and kidney function. Participants should have received at least one line of standard chemotherapy and one venetoclax-based regimen unless unsuitable. White blood cell count must be no more than 10 x 10⁹/L, and patients must have a life expectancy of at least 3 months.
Main exclusion criteria: The trial excludes patients without the NPM1 mutation, those outside the specified age range, and patients who are eligible for bone marrow transplantation at screening. Vulnerable populations unable to give consent are also excluded.
Focus and goals: The study aims to evaluate how well eltanexor works in reducing leukemia cells and to assess its safety and tolerability. Participants take eltanexor in tablet form, with monitoring throughout the study to check for effectiveness and side effects. Response assessment is conducted by the end of cycle 2.
Investigational drug: Eltanexor (KPT-8602) is taken orally as tablets. It works by inhibiting the export of certain proteins from the cell nucleus, which can lead to cancer cell death.
Study on the Safety of Eganelisib Alone and with Cytarabine for Patients with Relapsed or Refractory Acute Myeloid Leukemia or Higher-Risk Myelodysplastic Syndromes
This Spanish trial is exploring eganelisib both as a standalone treatment and in combination with cytarabine for patients with relapsed or refractory disease or higher-risk myelodysplastic syndromes.
Main inclusion criteria: Patients must be at least 18 years old with a confirmed diagnosis of disease showing at least 10% abnormal cells in the bone marrow. They must have disease that has returned or not responded to treatment with no other effective treatments available. Performance status should be 0, 1, or 2 on the ECOG scale, with adequate liver and kidney function. Participants must agree to use effective birth control and sun protection during the study.
Main exclusion criteria: The trial excludes patients with other types of cancer not being studied, those who have had a heart attack or stroke in the past 6 months, patients with uncontrolled high blood pressure, active infections, pregnant or breastfeeding women, and those with severe liver or kidney disease. Patients who have had an organ transplant or are currently participating in another clinical trial are also excluded.
Focus and goals: The study is conducted in two parts. The first part tests different doses of eganelisib to find the most suitable dose for further research. The second part evaluates how well the treatment controls the disease. Participants receive either eganelisib alone or in combination with cytarabine through intravenous infusion.
Investigational drugs: Eganelisib (IPI-549) is available as 5mg or 30mg capsules taken orally. It is a PI3K inhibitor that works by inhibiting a specific enzyme involved in cancer cell survival. Cytarabine is administered as an intravenous infusion and is commonly used in treating blood cancers.
Study on CCTx-001, Cyclophosphamide Monohydrate, and Fludarabine Phosphate for Patients with Relapsed or Refractory Acute Myeloid Leukemia
This multi-country trial across France, Germany, Sweden, and Spain is testing a new cell therapy called CCTx-001, which uses modified immune cells known as CAR-T cells to attack cancer cells.
Main inclusion criteria: Patients must be at least 18 years old with active relapsed or refractory disease. They must have a circulating blast count of less than 20,000/mm³, which can be controlled with medication. An absolute lymphocyte count of more than 200/mm³ is required, along with a performance status of 0 or 1 and life expectancy of more than 3 months. Women who can have children must have a negative pregnancy test, and all participants must agree to use effective birth control. Patients must be eligible for leukapheresis and have adequate organ function.
Main exclusion criteria: The trial excludes patients under 18 years old and those who cannot safely undergo leukapheresis or receive the CAR-T cell treatment.
Focus and goals: The study is conducted in two phases. The first focuses on determining the safest dose and understanding side effects, while the second evaluates how well the treatment controls the leukemia. Patients undergo leukapheresis to collect white blood cells, followed by treatment with cyclophosphamide and fludarabine to prepare the body, and then receive CCTx-001 through intravenous infusion.
Investigational drugs: CCTx-001 is a CAR-T cell therapy given intravenously. Cyclophosphamide monohydrate and fludarabine phosphate are administered through intravenous infusion to prepare the body for the main treatment.
Study on the Safety of CD19 CAR-T Cells, Cyclophosphamide, and Fludarabine in Adults with Refractory or Relapsed Acute Myeloid Leukemia Expressing CD19
This French trial is investigating CD19 CAR-T cell therapy in patients whose disease expresses the CD19 protein and has returned or not responded to treatment.
Main inclusion criteria: Patients must be at least 18 years old with disease that shows CD19 through flow cytometry testing. They must have relapsed or refractory disease with a performance status of less than 2 and estimated life expectancy of more than 2 months. Brain MRI should show no signs of cancer affecting the central nervous system. Adequate organ function is required, including kidney function with creatinine clearance of at least 40 mL/min, liver function, and heart function with cardiac ejection fraction of 45% or higher. Platelet count should be at least 30,000 per microliter and lymphocyte count at least 200 per microliter.
Main exclusion criteria: The trial excludes patients under 18 years old, those without CD19-expressing disease, patients unable to undergo flow cytometry testing, and those who cannot safely receive the CAR-T cell treatment.
Focus and goals: The study assesses the safety and tolerability of CD19 CAR-T cell therapy. Patients receive preparatory treatment with cyclophosphamide and fludarabine, followed by infusion of their own modified T-cells. Regular monitoring and follow-up assessments are conducted at one, three, six, and twelve months after treatment.
Investigational drugs: CD19 CAR-T cells are modified immune cells administered through intravenous infusion. Cyclophosphamide and fludarabine are given as injections or infusions to prepare the body for the CAR-T cell therapy.
Study of S227928 Alone and with Venetoclax for Patients with Relapsed or Refractory Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelomonocytic Leukemia
This trial taking place in France, Finland, and Germany is testing S227928, an antibody-drug conjugate, both alone and in combination with venetoclax.
Main inclusion criteria: Patients must be at least 18 years old with adequate kidney and liver function within 7 days before joining the study. They must have a confirmed diagnosis of relapsed or refractory disease, myelodysplastic syndrome, or chronic myelomonocytic leukemia. Participants should have received at least one standard treatment and not be candidates for further standard therapy. Performance status should be 2 or less, and circulating white blood cell count should be less than 10 x 10⁹/L, which can be managed with hydroxycarbamide if needed. Female patients must be postmenopausal, and male patients must use effective contraception.
Main exclusion criteria: The trial excludes patients without the specified diagnoses, those outside the age range, and members of vulnerable populations who cannot safely participate.
Focus and goals: The study is conducted in two phases. Phase 1 evaluates safety and tolerability of S227928 as a single agent and in combination with venetoclax, focusing on finding the maximum tolerated dose. Phase 2 assesses the anti-leukemic activity of the combination treatment. S227928 is given through intravenous infusion, while venetoclax is taken as 100 mg tablets by mouth.
Investigational drugs: S227928 is an anti-CD74 antibody-drug conjugate delivered intravenously that targets the CD74 protein on cancer cells. Venetoclax is taken orally and works by blocking the BCL-2 protein that helps cancer cells survive.
Study on the Safety and Effectiveness of Quizartinib for Children and Young Adults with Acute Myeloid Leukemia (AML)
This trial spanning France, Sweden, Belgium, Italy, Denmark, Spain, and the Netherlands is specifically designed for children and young adults with a genetic mutation called FLT3-ITD.
Main inclusion criteria: Patients must be between 1 month and 21 years old with a diagnosis showing at least 5% abnormal cells in the bone marrow. They must have a specific genetic mutation called FLT3-ITD in their bone marrow or blood and be in their first relapse or not responding to the first round of high-dose chemotherapy. Previous stem cell transplant is allowed. Participants must have adequate kidney, liver, and heart function as defined by the study, with a performance status that meets study requirements. Patients who can have children must use highly effective birth control during the study and for 6 months after the last dose.
Main exclusion criteria: The trial excludes patients outside the age range of 1 month to 21 years, those unable to safely receive the study medication or chemotherapy, pregnant or breastfeeding patients, and those with certain infections or serious health issues that could interfere with the study. Patients unable to follow study procedures or attend required visits are also excluded.
Focus and goals: The study aims to determine the best dose of quizartinib for different age groups and assess how well it works in achieving remission. Participants receive quizartinib along with chemotherapy for up to two cycles, with optional additional consolidation therapy. Following this, quizartinib may be given alone for up to twelve cycles to help prevent the cancer from returning.
Investigational drug: Quizartinib (AC220) is an oral solution taken by mouth. It is a tyrosine kinase inhibitor that works by blocking the FLT3 receptor involved in cancer cell growth and survival.
Summary
Seven clinical trials are currently recruiting patients across Europe for recurrent acute myeloid leukaemia. France is notably active in this research area, hosting five of the seven trials, followed by Spain and Germany with four trials each. Several countries including Belgium, Italy, and Sweden are participating in multiple studies.
The trials represent diverse therapeutic approaches, including targeted oral medications like ziftomenib and eltanexor that focus on specific genetic mutations, innovative CAR-T cell immunotherapies that modify patients’ own immune cells to attack cancer, and combination treatments pairing investigational drugs with established therapies such as venetoclax and cytarabine. Several studies specifically target patients with particular genetic mutations, including NPM1, FLT3-ITD, and CD19 expression.
Notably, one trial is dedicated to paediatric and young adult patients aged 1 month to 21 years, while most others focus on adult populations, with some specifically recruiting patients aged 60 and older. The studies range from early phase 1 dose-finding research to more advanced phase 2 efficacy evaluations, reflecting the various stages of development for these new treatments.
