Ongoing Clinical Trials for Acute Myeloid Leukaemia Refractory
There are currently 7 ongoing clinical trials investigating new treatments for patients with acute myeloid leukaemia that has not responded to previous treatments or has returned after therapy. These studies are testing innovative approaches including cell therapies, targeted medications, and combination treatments across multiple European countries.
Clinical trial locations
- Finland
- France
- 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
- Study of SAR443579 Infusion for Adults and Children with Relapsed or Refractory Acute Myeloid Leukemia, B-Cell Acute Lymphoblastic Leukemia, HR-MDS, or BPDCN
- Germany
- 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
- Study on the Safety and Effectiveness of Mebendazole and Low-Dose Cytarabine for Elderly Patients with Relapsed or Refractory Acute Myeloid Leukemia
- Italy
- Netherlands
- Spain
- Study of CART84 Treatment for Adult Patients with Relapsed or Refractory Acute Myeloid Leukemia and T-Cell Acute Lymphoblastic 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
- Sweden
Study of CART84 Treatment for Adult Patients with Relapsed or Refractory Acute Myeloid Leukemia and T-Cell Acute Lymphoblastic Leukemia
This trial is testing an innovative cell therapy called CART84 for patients whose blood cancer has not responded to previous treatments or has returned. CART84 is made from the patient’s own immune cells, which are collected, modified in a laboratory to better recognise and attack cancer cells, and then returned to the patient through an infusion into a vein.
Inclusion criteria: Participants must be at least 18 years old with confirmed diagnosis of the disease showing at least 5% cancer cells in bone marrow or blood. The cancer must either have not responded to initial treatment, returned after treatment, or relapsed despite salvage therapy. Patients need to have adequate organ function including good kidney, liver, heart, and lung function. They must have a blood oxygen level above 92% when breathing room air and be able to perform light work (ECOG score of 0 or 1). The cancer cells must have a specific protein called CD84. Importantly, patients must have an available stem cell donor for a possible transplant 30-90 days after treatment. Both men and women of childbearing potential must use effective contraception during treatment and for 12 months afterwards.
Exclusion criteria: Patients cannot participate if they have active or uncontrolled infections including hepatitis B, hepatitis C, or HIV, or if they have had other cancers in the past 3 years. Pregnant or breastfeeding women are excluded. Those with severe heart, lung, kidney, or liver problems, or active disease in the brain or spinal cord cannot join. Patients who have previously received CAR-T cell therapy or who have participated in another clinical trial within 30 days are not eligible.
Trial focus: The study aims to determine if CART84 is safe to use and to find the most effective dose for treating these types of blood cancers. After receiving CART84, some patients may proceed to receive a stem cell transplant from a matched donor. Throughout the study, participants will be monitored for their response to therapy and any side effects through regular blood tests and bone marrow examinations.
Investigational drug: CART84 is a personalised therapy consisting of genetically modified T-cells designed to target cancer cells expressing CD84. The modified cells are administered as a single intravenous infusion.
Study on the Safety and Effectiveness of Eltanexor for Patients with Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia
This trial is investigating eltanexor, a medication taken as a tablet, specifically for patients whose disease has a particular genetic mutation called NPM1. The study aims to understand how well eltanexor works in treating this specific type of disease and how safe it is for patients.
Inclusion criteria: Participants must be at least 60 years old and have documented relapsed or refractory NPM1-mutated disease with more than 1000 cancer cells per microlitre of blood. Patients must have received at least one line of standard chemotherapy and one venetoclax-based treatment (unless not suitable). Those with FLT3 mutations must have received at least one FLT3 inhibitor unless not suitable. Patients not eligible for standard treatment at diagnosis must have received at least one venetoclax-based regimen. Participants must not be eligible for bone marrow transplantation at screening, must be able to swallow oral medication, and have adequate liver and kidney function. They must have a life expectancy of at least 3 months and a white blood cell count of no more than 10×10⁹/L.
Exclusion criteria: The trial excludes patients without NPM1 mutation or those whose disease has not relapsed or proven refractory to treatment. Patients outside the specified age range or who are unable to give informed consent cannot participate.
Trial focus: The study is designed in two phases. The primary goal is to evaluate the response rate of the disease to eltanexor treatment and to monitor for any significant side effects. By the end of cycle 2, researchers will assess whether patients have achieved complete or partial remission.
Investigational drug: Eltanexor (KPT-8602) is a selective inhibitor of nuclear export that works by blocking the movement of certain proteins out of the cell nucleus, which can lead to cancer cell death. It is administered orally in tablet form.
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 trial explores a medication called eganelisib (IPI-549), testing it both on its own and in combination with cytarabine, a commonly used chemotherapy medication. The study is conducted in two parts, with the first focusing on finding the most suitable dose and the second evaluating how well the treatment controls the disease.
Inclusion criteria: Participants must be at least 18 years old with confirmed diagnosis of either acute myeloid leukaemia with at least 10% abnormal cells in the bone marrow (excluding acute promyelocytic leukaemia) or higher-risk myelodysplastic syndromes with at least 10% abnormal cells. The disease must have returned or not responded to treatment with no other effective treatments available. Patients need a performance status of 0, 1, or 2 on the ECOG scale, adequate liver function shown by specific blood tests, adequate kidney function with creatinine clearance of at least 30 L/min, and an acceptable blood clotting profile. Both men and women of childbearing potential must agree to use effective birth control during the study and afterwards. Participants must agree to use sun protection throughout the study.
Exclusion criteria: Patients with other types of cancer, those who have had a heart attack or stroke in the past 6 months, those with uncontrolled high blood pressure, or active infections requiring treatment cannot participate. Pregnant or breastfeeding women, patients who have had an organ transplant, those with severe liver or kidney disease, and those currently in another clinical trial are excluded. Patients with a history of allergic reactions to similar medications or drug/alcohol abuse in the past year are not eligible.
Trial focus: The study will evaluate the safety and tolerability of eganelisib both as a standalone treatment and when combined with cytarabine. Participants will be closely monitored for any adverse effects, and the dosage will be optimised based on response and tolerance.
Investigational drugs: Eganelisib is a PI3K inhibitor administered orally in capsule form (5mg or 30mg). Cytarabine is given as an intravenous infusion and is a standard chemotherapy agent commonly used for blood cancers.
Study on CCTx-001, Cyclophosphamide Monohydrate, and Fludarabine Phosphate for Patients with Relapsed or Refractory Acute Myeloid Leukemia
This trial tests CCTx-001, a type of CAR-T cell therapy where immune cells are modified to better recognise and attack cancer cells. The study also uses two preparatory medications, cyclophosphamide monohydrate and fludarabine phosphate, given through intravenous infusion to prepare the body for the CAR-T cell treatment.
Inclusion criteria: Participants must be 18 years or older with active relapsed or refractory disease. Patients need a circulating blast count of less than 20,000/mm³ (which can be controlled with hydroxyurea), an absolute lymphocyte count of more than 200/mm³, and an ECOG performance status of 0 or 1. They must have a life expectancy of more than 3 months and be eligible for leukapheresis (a procedure to collect white blood cells). Adequate organ function is required. Women who can become pregnant must have a negative pregnancy test, and all participants of childbearing potential must agree to use highly effective contraception during treatment and for 12 months afterwards.
Exclusion criteria: Specific exclusion criteria are not fully detailed in the source, but generally patients who do not meet the inclusion requirements or have conditions that would make participation unsafe would be excluded.
Trial focus: The study is divided into two phases. Phase 1 focuses on determining the safest dose and understanding side effects. Phase 2 looks at how well the treatment works in controlling the disease. Participants undergo leukapheresis to collect their white blood cells, receive preparatory chemotherapy, and then receive CCTx-001 infusion. Regular monitoring assesses response and side effects.
Investigational drugs: CCTx-001 is a CAR-T cell therapy administered via intravenous infusion. Cyclophosphamide monohydrate and fludarabine phosphate are given intravenously as preparatory chemotherapy to help the body accept the CAR-T cells.
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, both on its own and in combination with venetoclax. The study includes patients with acute myeloid leukaemia, myelodysplastic syndrome, or chronic myelomonocytic leukaemia whose disease has returned or not responded to previous treatments.
Inclusion criteria: Participants must be at least 18 years old with a performance status of 2 or less (in some European countries, 0 or 1). They must provide written informed consent and have confirmed diagnosis of one of the target conditions with disease that has returned or not responded to treatment after at least one standard therapy. Patients must have circulating white blood cells less than 10×10⁹/L (hydroxycarbamide can be used to meet this requirement) and adequate kidney and liver function within 7 days before starting.
Exclusion criteria: Patients without the specified diagnoses, those outside the age range, or those not meeting the gender criteria (both males and females are eligible) cannot participate. Members of vulnerable populations not selected for the study are excluded.
Trial focus: The study is conducted in two phases. Phase 1 focuses on evaluating safety and tolerability of S227928 alone and in combination with venetoclax, determining the maximum tolerated dose. Phase 2 assesses the anti-leukaemic activity of the combination therapy. Treatment involves intravenous infusion of S227928 and oral tablets of venetoclax.
Investigational drugs: S227928 is an antibody-drug conjugate that targets the CD74 protein on cancer cells, delivering a cytotoxic agent directly to them. It is given via intravenous infusion. Venetoclax is a BCL-2 inhibitor that helps kill cancer cells by blocking a protein that helps them survive. It is taken orally as 100mg film-coated tablets.
Study of SAR443579 Infusion for Adults and Children with Relapsed or Refractory Acute Myeloid Leukemia, B-Cell Acute Lymphoblastic Leukemia, HR-MDS, or BPDCN
This trial tests SAR443579, administered through intravenous infusion, in both adults and children with various blood cancers including acute myeloid leukaemia. The study aims to find the most suitable dose and evaluate effectiveness across different age groups.
Inclusion criteria: Participants must be at least 1 year old (adults 12 years and older, children 1-17 years). For the escalation phase, patients need confirmed diagnosis of primary or secondary disease (excluding specific subtypes) that either did not respond to initial treatment, relapsed early (within 6 months), or is in first or higher relapse. Adults may also have high-risk myelodysplastic syndrome with no available effective therapy. Patients must weigh at least 10kg. For children and escalation phase, those with blastic plasmacytoid dendritic cell neoplasm with relapsed or refractory disease are included.
Exclusion criteria: Patients with other types of cancer not part of the study, those who have had different treatment recently, those with serious uncontrolled infections, significant heart problems, severe liver or kidney issues, pregnant or breastfeeding women, those unable to follow study procedures, those with history of allergic reactions to similar treatments, recent participants in other clinical trials, or certain unrelated blood disorders cannot participate.
Trial focus: The study is divided into dose escalation and dose expansion phases. The first part determines the maximum tolerated dose for both adults and children. The second part evaluates the medication’s effectiveness at the recommended dose. Regular monitoring tracks response and side effects.
Investigational drug: SAR443579 is an investigational anti-cancer agent administered as a single agent through intravenous infusion. It targets specific pathways involved in cancer cell growth and survival.
Study on the Safety and Effectiveness of Mebendazole and Low-Dose Cytarabine for Elderly Patients with Relapsed or Refractory Acute Myeloid Leukemia
This trial specifically targets elderly patients aged 70 and older, testing a combination of mebendazole (commonly used to treat parasitic infections) and low-dose cytarabine (Ara-C), a chemotherapy drug. The study explores whether this combination can help patients who are not eligible for more intensive treatments.
Inclusion criteria: Participants must be older than 70 years and not able to undergo intensive treatment. They must have a diagnosis of the disease (excluding promyelocytic leukaemia type) that has returned or not responded to standard treatment, with more than 1000 cancer cells (blasts) per microlitre of blood. Patients must be able to swallow and keep down oral medication, have no known nutrient absorption issues, and have organs working well enough. They must be able to understand the study and provide informed consent.
Exclusion criteria: Patients under age 70, those not diagnosed with the specific condition, those unable to safely take the study medications, and those part of vulnerable populations who cannot give consent or understand the study cannot participate.
Trial focus: The study is conducted in two phases. Phase 1 focuses on determining a safe dose of mebendazole when combined with low-dose Ara-C. Phase 2 assesses effectiveness in achieving remission within three months. Throughout treatment, participants are monitored for side effects, changes in health, effects on cancer cells, and overall survival. Treatment is administered in cycles with mebendazole given orally in tablet form and Ara-C injected under the skin.
Investigational drugs: Mebendazole is administered orally in tablet form (either 500mg or 100mg doses). It works by disrupting microtubules essential for cell division, thereby inhibiting cancer cell growth. Low-dose Ara-C (cytarabine) is administered subcutaneously (injected under the skin) as chemotherapy to help fight the disease.
Summary
The seven ongoing trials for refractory acute myeloid leukaemia represent diverse therapeutic approaches, from innovative cell therapies to targeted medications and combination treatments. Several trials are testing CAR-T cell therapies (CART84, CCTx-001) that harness the patient’s own immune system to fight cancer, whilst others investigate novel targeted agents like eltanexor for specific genetic mutations (NPM1) and antibody-drug conjugates such as S227928.
Germany and France appear as common trial locations, with Spain hosting multiple studies. Several trials specifically address the needs of elderly patients who cannot tolerate intensive chemotherapy, such as the mebendazole study for patients over 70. Many studies are exploring combination approaches, pairing new investigational drugs with established treatments like venetoclax and cytarabine.
A notable feature across these trials is the focus on personalised medicine, with some requiring specific genetic markers (NPM1 mutation, CD84 expression) for eligibility. The trials span early-phase research (Phase 1/2) aimed at establishing safe doses and preliminary effectiveness data. Most studies include rigorous monitoring protocols to track both safety and treatment response through regular blood tests and bone marrow examinations.
