Ongoing Clinical Trials for Chronic Myelomonocytic Leukaemia
Eight clinical trials are currently investigating new treatments for chronic myelomonocytic leukaemia, testing various medication combinations and oral alternatives to traditional injectable therapies. These trials are taking place across multiple European countries and offer options for patients at different stages of the disease.
Clinical trial locations
- Austria
- Bulgaria
- Czechia
- Study Comparing Oral Azacitidine and Cedazuridine with Subcutaneous Azacitidine for Patients with Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Acute Myeloid Leukemia
- Study on Continued Treatment with Sabatolimab for Adults with High-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Acute Myeloid Leukemia Unfit for Chemotherapy
- Denmark
- Finland
- France
- Study on the Safety and Effectiveness of Azacitidine and Venetoclax for Patients with Higher-Risk Chronic Myelomonocytic Leukemia (CMML)
- Study of S227928 Alone and with Venetoclax for Patients with Relapsed or Refractory Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelomonocytic Leukemia
- Study on Continued Treatment with Sabatolimab for Adults with High-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Acute Myeloid Leukemia Unfit for Chemotherapy
- Germany
- Study of Azacitidine, Venetoclax, and Tagraxofusp for Patients with Higher-Risk Chronic Myelomonocytic Leukemia
- Long-term treatment study of oral decitabine and cedazuridine combination for patients with AML, MDS, CMML, or solid tumors who previously received ASTX727
- Study of S227928 Alone and with Venetoclax for Patients with Relapsed or Refractory Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelomonocytic Leukemia
- Study on Continued Treatment with Sabatolimab for Adults with High-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Acute Myeloid Leukemia Unfit for Chemotherapy
- Greece
- Hungary
- Study Comparing Oral Azacitidine and Cedazuridine with Subcutaneous Azacitidine for Patients with Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Acute Myeloid Leukemia
- Long-term treatment study of oral decitabine and cedazuridine combination for patients with AML, MDS, CMML, or solid tumors who previously received ASTX727
- Italy
- Poland
- Romania
- Slovakia
- Spain
- Study of Azacitidine, Venetoclax, and Tagraxofusp for Patients with Higher-Risk Chronic Myelomonocytic Leukemia
- Long-term treatment study of oral decitabine and cedazuridine combination for patients with AML, MDS, CMML, or solid tumors who previously received ASTX727
- Study of AZD2962 tablets alone and with other medications in patients with myelodysplastic syndromes and dysplastic chronic myelomonocytic leukemia
- Study on Continued Treatment with Sabatolimab for Adults with High-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Acute Myeloid Leukemia Unfit for Chemotherapy
Study Comparing Oral Azacitidine and Cedazuridine with Subcutaneous Azacitidine for Patients with Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Acute Myeloid Leukemia
This trial is testing an oral medication called ASTX030, which combines azacitidine and cedazuridine in capsule form. The study compares this oral version with the traditional subcutaneous injection of azacitidine to determine if they work equally well. This crossover study allows participants to receive both forms of treatment, making it easier to compare their effectiveness directly.
Main inclusion criteria: Participants must be 18 years or older with a confirmed diagnosis of the condition and be candidates for azacitidine treatment. They should have an ECOG performance status of 0 to 1, meaning they can carry out daily activities with minimal restrictions. Adequate organ function is required, including proper liver and kidney function, with specific measurements needed to qualify. Those who have had an allogeneic stem cell transplant must be off immunosuppressive therapy for at least three weeks.
Main exclusion criteria: Patients with serious medical conditions that could affect participation, pregnant or breastfeeding women, those who participated in another trial within 30 days, or people with known allergies to the medication cannot join. Patients who have had recent major surgery, received cytotoxic chemotherapy within four weeks, or have uncontrolled infections are also excluded.
Trial focus: The study aims to provide a more convenient treatment option by offering oral medication instead of injections. Researchers will assess whether the oral form is as effective as the injection in controlling disease progression while monitoring for side effects and measuring treatment response.
Investigational drugs: ASTX030 combines cedazuridine with azacitidine in an oral form. Cedazuridine helps prevent the breakdown of azacitidine in the body, allowing it to work longer and more effectively.
Study of Azacitidine, Venetoclax, and Tagraxofusp for Patients with Higher-Risk Chronic Myelomonocytic Leukemia
This trial evaluates a combination of three treatments: azacitidine, venetoclax, and tagraxofusp for patients with higher-risk disease. The study aims to understand how well these medications work together and what side effects may occur over a treatment period of up to 12 months.
Main inclusion criteria: Participants must be 18 years or older with a CPSS risk score of intermediate-2 or high, confirming higher-risk disease. They must be eligible for and willing to receive azacitidine as standard treatment. An ECOG performance status of 0-2 is required, along with adequate organ function including liver, kidney, heart, and sufficient albumin levels. Blood count and transfusion records from the previous eight weeks must be available.
Main exclusion criteria: Patients with other types of cancer, those outside the specified age range, and vulnerable populations who cannot make decisions for themselves cannot participate. Those unable to follow study procedures or take medications as required are also excluded.
Trial focus: The primary outcome measure is the response rate after three treatment cycles, looking at complete response, marrow complete response, or partial response. Secondary outcomes include overall survival, time to leukemia transformation, and changes in quality of life.
Investigational drugs: The trial uses azacitidine as standard care, combined with venetoclax (an oral tablet that blocks a protein helping cancer cells survive) and tagraxofusp (an intravenous infusion that targets specific cancer cells).
Study on Metformin for Preventing Leukemia in Patients with Clonal Cytopenia and Low-Risk Myelodysplastic Syndromes
This trial explores whether metformin hydrochloride, a medication commonly used for diabetes, can help prevent the progression of blood disorders into leukemia. The study focuses on patients with very low and low-risk conditions and will last up to 52 weeks.
Main inclusion criteria: Participants must be diagnosed with either very low or low-risk myelodysplastic syndromes or clonal cytopenia of undetermined significance with specific genetic changes. Women must be in menopause, defined as being over 45 years old without a menstrual period for at least 12 months. Participants must be at least 18 years old, provide written informed consent, and be able to swallow pills.
Main exclusion criteria: Patients with the specified conditions who do not meet the age requirements or belong to vulnerable populations cannot participate.
Trial focus: The study assesses the safety and feasibility of using metformin to control disease progression. Researchers will monitor changes in blood cell counts and other health markers throughout the treatment period.
Investigational drugs: Metformin is being studied for its potential to prevent leukemia by activating an enzyme called AMP-activated protein kinase, which helps regulate energy balance and can inhibit cell growth.
Study on the Safety and Effectiveness of Azacitidine and Venetoclax for Patients with Higher-Risk Chronic Myelomonocytic Leukemia (CMML)
This trial investigates the safety and effectiveness of combining venetoclax tablets with azacitidine injections for patients with higher-risk disease. The study includes a safety run-in phase followed by a main phase to assess treatment response.
Main inclusion criteria: Participants must be 18 years or older with a diagnosis according to WHO 2016 criteria and an intermediate-2 or high-risk score according to the CPSS system. They should not have had prior treatment with hypomethylating agents, though prior treatment with certain other medications is allowed under specific conditions. An ECOG performance status of 0-2 is required, along with adequate organ function and signed informed consent.
Main exclusion criteria: Patients without the specified diagnoses, those outside the eligible age range, and those not meeting the study’s specific criteria cannot participate.
Trial focus: The study evaluates the overall response rate after three and six treatment cycles, including complete remission, partial remission, marrow response, and clinical benefit. Additional endpoints include overall survival, progression-free survival, and event-free survival.
Investigational drugs: Azacitidine works by interfering with cancer cell growth, while venetoclax blocks a specific protein in cancer cells, leading to their death. The combination aims to improve overall treatment response.
Long-term Treatment Study of Oral Decitabine and Cedazuridine Combination for Patients with AML, MDS, CMML, or Solid Tumors Who Previously Received ASTX727
This long-term safety study continues treatment with ASTX727 for patients who previously participated in clinical trials and benefited from the medication. The study monitors health status and side effects during continued treatment.
Main inclusion criteria: Participants must have previously received ASTX727 in an earlier clinical trial and still be benefiting from treatment. They must be able to understand study procedures and provide informed consent. Women who can become pregnant must use effective birth control during the study and for six months after the last dose, along with regular pregnancy testing. Men must use condoms and ensure their partners use birth control during treatment and for three months afterward.
Main exclusion criteria: Patients who did not previously participate in ASTX727 trials, those who did not benefit from previous treatment, patients under 18 years, pregnant or breastfeeding women, and those unable to take oral medications are excluded. Patients with serious side effects during previous treatment or participating in other trials also cannot join.
Trial focus: The study gathers long-term safety information about ASTX727 in patients who have shown benefit, with regular health assessments conducted throughout participation.
Investigational drugs: ASTX727 combines decitabine with cedazuridine in an oral form. Cedazuridine helps the body better absorb decitabine when taken by mouth, making it more convenient than injectable treatments.
Study of AZD2962 Tablets Alone and with Other Medications in Patients with Myelodysplastic Syndromes and Dysplastic Chronic Myelomonocytic Leukemia
This trial tests a new medication called AZD2962, which blocks a protein called IRAK4 that may contribute to blood disorders. The study evaluates the medication both alone and combined with other treatments to determine safety and optimal dosing.
Main inclusion criteria: Participants must be at least 18 years old with relapsed or refractory disease, having received at least one previous treatment. They must have an ECOG performance status of 2 or better and symptoms requiring measurable treatment. A bone marrow sample is required before starting treatment. Specific blood test requirements include white blood cell counts within certain limits and adequate liver and kidney function.
Main exclusion criteria: Patients outside the eligible age range, pregnant or breastfeeding women, those with severe allergies to medications, active infections, serious illness, or participation in other trials within 30 days cannot join. Those with severe organ problems, recent bone marrow transplant, or life expectancy less than three months are also excluded.
Trial focus: The study determines the safest dose and how well patients tolerate AZD2962. Researchers monitor treatment response through regular blood and bone marrow examinations, tracking how the medication affects the disease over time.
Investigational drugs: AZD2962 is an IRAK4 inhibitor that blocks a key enzyme involved in inflammatory signaling pathways important in blood cell disorders. It represents a novel therapeutic approach currently in Phase I/II clinical trials.
Study of S227928 Alone and with Venetoclax for Patients with Relapsed or Refractory Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelomonocytic Leukemia
This trial investigates S227928, an anti-CD74 antibody-drug conjugate designed to target and attack cancer cells. The study evaluates the medication alone and combined with venetoclax for patients whose disease has returned or not responded to previous treatments.
Main inclusion criteria: Participants must be adults at least 18 years old with a performance status of 2 or less. They need confirmed diagnosis of the specified conditions and should have received at least one standard treatment with disease that has relapsed or not responded to therapy. White blood cell counts must be below specified levels, and adequate kidney and liver function is required within seven days of joining.
Main exclusion criteria: Patients without the specified diagnoses, those outside the eligible age range, and those not meeting the study’s criteria cannot participate.
Trial focus: The first phase determines the safest dose of S227928 and tolerability, both alone and with venetoclax. The second phase evaluates the combination’s effectiveness in fighting cancer. Researchers monitor for side effects and how the body processes the medication.
Investigational drugs: S227928 targets the CD74 protein, delivering a cytotoxic agent directly to cancer cells. Venetoclax inhibits the BCL-2 protein, helping cancer cells survive, thereby promoting their death when used in combination.
Study on Continued Treatment with Sabatolimab for Adults with High-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Acute Myeloid Leukemia Unfit for Chemotherapy
This trial evaluates the safety of continuing sabatolimab treatment for adult patients who are not suitable for chemotherapy and have already participated in a previous study with this medication. The study monitors side effects and treatment benefits over an extended period.
Main inclusion criteria: Participants must already be enrolled in a previous sabatolimab study sponsored by Novartis and currently receiving treatment while meeting all original study requirements. They must be benefiting from the treatment as determined by study guidelines and the doctor’s judgment. Participants should demonstrate compliance with the original study rules and be willing to attend scheduled visits and follow the treatment plan. Written informed consent is required before joining.
Main exclusion criteria: Patients with different types of cancer than those being studied, those receiving interfering treatments, or those with serious medical conditions making participation unsafe cannot join. Pregnant or breastfeeding women, those with recent or planned surgery, history of allergic reactions to similar treatments, or inability to follow procedures are also excluded.
Trial focus: The study assesses the long-term safety of sabatolimab, including monitoring for serious and non-serious side effects during continued treatment. Regular evaluations track treatment effectiveness and health status over time.
Investigational drugs: Sabatolimab is an intravenous immunotherapy agent that targets specific proteins on immune cells to enhance the body’s immune response against cancer cells. It is being studied for patients with intermediate, high, or very high-risk disease who cannot undergo chemotherapy.
Summary
The eight ongoing clinical trials for chronic myelomonocytic leukaemia demonstrate diverse therapeutic approaches across Europe. A notable concentration of research is occurring in Germany and Spain, each hosting four trials, followed by France with three trials. Several trials focus on developing oral alternatives to injectable treatments, such as the ASTX030 and ASTX727 studies, potentially offering more convenient treatment options for patients.
The trials predominantly target higher-risk patients, with multiple studies investigating combination therapies involving azacitidine and venetoclax. Particularly interesting is the metformin trial in Denmark, which explores repurposing a diabetes medication for preventing disease progression. The research also includes novel approaches such as antibody-drug conjugates like S227928 and immunotherapy agents like sabatolimab, representing innovative treatment strategies for patients who cannot undergo traditional chemotherapy.
Most trials require participants to be at least 18 years old with adequate organ function and specific performance status criteria. The studies generally exclude pregnant women, those with serious medical conditions, and patients participating in other trials simultaneously. These trials collectively offer multiple options for patients at different disease stages and treatment histories across various European countries.
