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Ongoing Clinical Trials for Myeloid Leukaemia

Currently, there are 3 ongoing clinical trials investigating new treatments for myeloid leukaemia. These studies are testing different medication combinations in specific patient groups, including children with Down syndrome, young patients with recurrent or refractory disease, and adults unable to undergo intensive chemotherapy. The trials are being conducted across multiple European countries.

Clinical trial locations

Study of Cytarabine and Daunorubicin for Treating Myeloid Leukemia in Children with Down Syndrome

This trial focuses on children with Down syndrome who have been diagnosed with myeloid leukaemia. The study is evaluating a medication called Vyxeos Liposomal, which contains two active substances: cytarabine and daunorubicin. These chemotherapy drugs are packaged together in tiny fat-like particles called liposomes, which help them stay in the body longer and target cancer cells more effectively.

Main inclusion criteria: Children must have Down syndrome or mosaic Down syndrome and be between 6 months and 4 years old. Children aged 4 to 6 years can participate if they have a specific genetic change called GATA1 mutation. They must have a confirmed diagnosis of myeloid leukaemia or myelodysplastic syndrome and be able to perform daily activities at a reasonable level. Parents or legal guardians must provide written permission for the child to participate.

Main exclusion criteria: Children who do not meet the age requirements or do not have the specified type of leukaemia cannot participate. Those outside the study’s defined vulnerable population are also excluded.

Focus and goal: The primary goal is to evaluate event-free survival, which measures the time from diagnosis until significant events occur, such as disease relapse or failure to achieve remission. The study will monitor how well the treatment works, assess any side effects, and gather data on overall survival and disease-free survival rates. The aim is to determine if this treatment can achieve similar or better success rates compared to previous studies.

Investigational drug: CPX-351 is administered as an intravenous infusion. This liposomal formulation delivers cytarabine and daunorubicin in a specific ratio designed to enhance their effectiveness against cancer cells while potentially reducing side effects.

Study on Ponatinib for Treating Recurrent or Refractory Leukemias and Solid Tumors in Children

This study investigates the safety and effectiveness of ponatinib in children with cancers that have returned after treatment or have not responded to standard therapies. The trial includes patients with recurrent or refractory leukaemias, lymphomas, and solid tumours.

Main inclusion criteria: Participants must be between 1 and 18 years old with a confirmed diagnosis of leukaemias, lymphomas, or solid tumours that have not responded to standard treatments or are not suitable for such treatments. They must have sufficient physical ability to perform some daily activities, as measured by performance status scales. Participants must have recovered from previous treatment side effects to an acceptable level. Parents or legal guardians must provide consent.

Main exclusion criteria: Patients with cancer types not covered by the study, those outside the specified age range, or those unable to take oral medication cannot participate. Pregnant or breastfeeding individuals are excluded, as are those with serious health conditions that could make participation unsafe, active infections requiring treatment, known allergies to the study medication, or those participating in other clinical trials simultaneously.

Focus and goal: The study is divided into two phases. Phase 1 aims to find the best dose of ponatinib for children with advanced blood cancers or solid tumours. Phase 2 evaluates how well the medication works in children with chronic myeloid leukaemia and other selected cancers. Throughout the trial, researchers will monitor participants’ health and any side effects using medical tests and imaging studies.

Investigational drug: Ponatinib is a kinase inhibitor taken orally once a day in tablet form. It works by blocking certain proteins that help cancer cells grow, particularly the BCR-ABL protein involved in the growth and survival of cancer cells.

Study Comparing Pevonedistat, Venetoclax, and Azacitidine with Venetoclax and Azacitidine in Adults with Acute Myeloid Leukemia Unfit for Intensive Chemotherapy

This trial compares two treatment combinations for adults with newly diagnosed acute myeloid leukaemia who cannot undergo intensive chemotherapy. One group receives pevonedistat, venetoclax, and azacitidine, while the other receives only venetoclax and azacitidine.

Main inclusion criteria: Patients must be 18 years or older with newly diagnosed acute myeloid leukaemia confirmed by a doctor. They must be considered unfit for intensive chemotherapy due to age (75 years or older) or other health issues such as poor physical health status, severe heart or lung problems, reduced kidney function, or liver problems. Laboratory test results must meet specific criteria, including appropriate levels of liver enzymes, kidney function, and albumin. White blood cell count must be below a certain threshold.

Main exclusion criteria: Patients who are fit for intensive chemotherapy, have previously received treatment with the study drugs, or have uncontrolled severe heart disease cannot participate. Those with active uncontrolled infections, pregnant or breastfeeding women, individuals with severe allergic reactions to study drugs, those who recently received other investigational drugs, or patients with serious medical conditions that could interfere with the study are excluded.

Focus and goal: The study aims to determine whether the three-drug combination is more effective than the two-drug combination in treating patients who cannot undergo intensive chemotherapy. Researchers will monitor participants over time to assess treatment effectiveness, safety, disease progression, overall survival, and how the leukaemia responds to treatment. The trial will provide valuable information that could improve treatment options for this patient group.

Investigational drugs: Pevonedistat is an experimental drug administered intravenously that blocks a specific protein in cancer cells, potentially stopping their growth and spread. Venetoclax is taken orally as a tablet and works by targeting and blocking a protein that helps cancer cells survive. Azacitidine is administered either intravenously or subcutaneously and works by interfering with cancer cell growth, incorporating into their DNA and disrupting their ability to multiply.

Summary

The three ongoing clinical trials for myeloid leukaemia address different patient populations and treatment approaches. Two trials focus on paediatric patients, including one specifically for children with Down syndrome and another for young patients with recurrent or treatment-resistant disease. The third trial targets adults unable to undergo intensive chemotherapy.

Italy participates in all three trials, making it the most represented country, followed by Belgium, the Netherlands, and Poland, which participate in two trials each. Other participating countries include Germany, Austria, Czechia, France, Spain, and Sweden. The trials investigate various drug combinations, including liposomal formulations, kinase inhibitors, and combinations of targeted therapies with hypomethylating agents.

These studies reflect ongoing efforts to develop more effective and better-tolerated treatments for different forms of myeloid leukaemia, particularly for vulnerable populations who cannot receive standard intensive chemotherapy.

Ongoing Clinical Trials on Myeloid leukaemia

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