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Study on WT1-loaded CBDC Vaccine for Children and Young Adults with Acute Myeloid Leukemia

InvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying Acute Myeloid Leukemia (AML), a type of cancer that affects the blood and bone marrow. The trial is testing a new treatment called the WT1-loaded CBDC Vaccine. This vaccine is made from a patient's own cells, specifically from cord blood-derived dendritic cells, which are loaded with a protein called WT1. The aim is to see if this vaccine can help prevent the return of AML after a patient has received a cord blood transplant.

The purpose of the study is to find a safe dose of the vaccine and to see if it can improve the chances of staying free from AML for a year after the first vaccination. The study is divided into two parts. In the first part, the focus is on determining the safest dose of the vaccine. In the second part, the goal is to see if the vaccine can increase the number of patients who remain free from AML by 20% compared to past patients who did not receive the vaccine. Participants will receive the vaccine through an injection into a vein, and the study will monitor them for any side effects and the effectiveness of the treatment over a period of time.

Throughout the study, researchers will also look at other important factors, such as the overall survival rate of participants and any changes in the immune system's response to the WT1 protein. The study aims to provide valuable information on whether this new vaccine can be a beneficial treatment option for young patients with AML.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the trial

    Upon joining the trial, you will be required to provide a signed informed consent. This is a document that confirms your understanding and agreement to participate in the study.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to ensure eligibility. This includes confirming a diagnosis of acute myeloid leukemia (AML) and checking specific medical criteria related to your condition.

  3. Step 3

    Vaccination schedule

    You will receive the WT1-loaded CBDC Vaccine, which is a suspension for injection. The vaccine is administered intravenously, meaning it is given through a vein.

    The vaccination process involves multiple doses. The first vaccination marks the beginning of the trial period.

  4. Step 4

    Monitoring period

    After each vaccination, there will be a monitoring period to observe any potential side effects or dose limiting toxicities (DLTs). This period lasts for 84 days after the third vaccination.

    Regular check-ups and assessments will be conducted to ensure your safety and to monitor the effects of the vaccine.

  5. Step 5

    Follow-up assessments

    Follow-up assessments will be conducted to evaluate the effectiveness of the vaccine. This includes checking the relapse-free survival rate of AML one year after the first vaccination.

    Additional tests may be performed to measure your immune response to the vaccine.

  6. Step 6

    Completion of trial

    The trial concludes after the one-year follow-up period. Final assessments will be conducted to gather data on your overall health and the long-term effects of the vaccine.

Who can join the trial?

9 criteria

  • Must have Acute Myeloid Leukemia (AML), which is a type of cancer affecting the blood and bone marrow.
  • Eligible for a specific type of stem cell transplant called allo-HCT based on standard medical guidelines.
  • Must have high levels of a specific genetic marker called WT1 mRNA in their AML sample, which is checked at diagnosis or after treatment.
  • Need to meet the criteria for a type of transplant called CB-HCT according to the guidelines of the Transplant Center.
  • The selected cord blood unit for transplant should have a certain number of cells, specifically a minimum of 3x107 nucleated cells per kilogram of body weight.
  • The cord blood unit should also have more than 7.5x106 total CD34+ cells before being frozen.
  • Must have a Karnofsky/Lansky score of 70 or higher, which measures the ability to perform daily activities.
  • For Part A of the study, must be between 12 and 30 years old (first three patients must be 16 years or older). For Part B, must be between 0 and 30 years old.
  • Must provide signed informed consent, which means agreeing to participate after understanding the study details.

Who cannot join the trial?

8 criteria

  • Patients with any other type of cancer besides Acute Myeloid Leukemia (AML) cannot participate.
  • Patients who have not received a CBT (Cord Blood Transplant) are excluded.
  • Patients who have experienced severe allergic reactions to vaccines in the past are not eligible.
  • Patients with uncontrolled infections or diseases that affect the immune system cannot join the study.
  • Pregnant or breastfeeding women are not allowed to participate.
  • Patients who are currently participating in another clinical trial are excluded.
  • Patients with a history of drug or alcohol abuse that could interfere with the study are not eligible.
  • Patients who are unable to follow the study procedures or attend follow-up visits are excluded.
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Investigated drugs

CBDC Vaccine: This is a special type of vaccine made from dendritic cells, which are a part of the immune system. In this trial, the vaccine is designed to help the body recognize and fight against a protein called WT1, which is often found in acute myeloid leukemia (AML) cells. The goal of the vaccine is to train the immune system to target and destroy these cancer cells, potentially reducing the chance of the cancer coming back after a cord blood transplant.

What is already known about the treatment

WT1-loaded CBDC vaccine – This medication is administered as a vaccine, specifically designed for patients who have undergone cord blood transplantation (CBT) to treat Acute Myeloid Leukemia (AML), a type of cancer affecting the blood and bone marrow. Currently, this vaccine is being studied in clinical trials to determine its safety and effectiveness in preventing the relapse of AML. The main therapeutic goal is to increase the survival rate of patients without the disease returning. At the molecular level, the vaccine works by targeting a specific protein called WT1, which is often found in high amounts in leukemia cells, helping the immune system recognize and attack these cancerous cells. This vaccine falls under the pharmacological classification of cancer immunotherapy, which aims to boost the body's natural defenses to fight cancer.

Investigated diseases

Acute Myeloid Leukemia – Acute Myeloid Leukemia is a type of cancer that affects the blood and bone marrow. It begins in the bone marrow, where blood cells are produced, and leads to the rapid growth of abnormal white blood cells. These abnormal cells can accumulate and interfere with the production of normal blood cells. As the disease progresses, it can spread to other parts of the body, including the lymph nodes, liver, and spleen. The progression of the disease can lead to symptoms such as fatigue, frequent infections, and easy bruising or bleeding. The disease is characterized by its fast progression and requires medical attention to manage its effects on the body.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-517922-24-00Protocol codeSP_PT18UDAEstimated enrolment54 patientsSponsorPrincess Maxima Center For Pediatric Oncology

sourced from the EU Clinical Trials Register and site verification

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