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Study on the Safety and Effects of AUTO1, Cyclophosphamide, and Fludarabine Phosphate in Children with Relapsed or Refractory B-cell Leukemia and Non-Hodgkin Lymphoma

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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 two types of blood cancers: B-cell Acute Lymphoblastic Leukemia (B-ALL) and Aggressive Mature B-cell Non-Hodgkin Lymphoma (B-NHL). These are serious conditions where certain white blood cells grow uncontrollably. The study is testing a new treatment called AUTO1, which involves using a patient's own immune cells that are specially modified to target and fight the cancer cells. This treatment is given through an infusion, which means it is delivered directly into the bloodstream.

The purpose of the study is to evaluate the safety and initial effectiveness of AUTO1 in children who have these types of cancers that have either returned after treatment or have not responded to previous treatments. Participants in the study will receive the AUTO1 treatment and will be monitored closely by healthcare professionals. The study will look at how well the treatment is tolerated and any side effects that may occur. It will also assess how the cancer responds to the treatment over time.

In addition to AUTO1, the study involves two other medications, Cyclophosphamide and Fludara (fludarabine phosphate), which are used to prepare the body for the AUTO1 treatment. These medications are also given through an infusion. Some participants may receive a placebo, which is a substance with no active medication, to help compare the effects of the treatment. The study is expected to continue until 2027, with participants being followed up regularly to gather information on the treatment's long-term effects and benefits.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment
Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a review of medical history and a physical examination.
Blood tests and imaging studies may be performed to assess the current state of the condition.
Step 2
Pre-treatment phase
Before starting the main treatment, a preparation phase involves the administration of two medications: cyclophosphamide and fludarabine phosphate.
Cyclophosphamide is given as a 500 mg solution for injection or infusion, administered intravenously.
Fludarabine phosphate is provided as a 50 mg solution for injection or infusion, also administered intravenously.
These medications are given to prepare the body for the main treatment and are typically administered over a few days.
Step 3
Main treatment
The main treatment involves the infusion of AUTO1, which consists of specially modified T cells designed to target specific cancer cells.
This is administered as an intravenous infusion. The duration and frequency of this infusion are determined by the study protocol and the patient's response.
Step 4
Monitoring and follow-up
After the main treatment, regular monitoring is conducted to assess the patient's response and to identify any side effects.
This includes frequent medical check-ups, blood tests, and imaging studies.
The frequency and duration of follow-up visits are determined by the study protocol and the patient's condition.
Step 5
End of study participation
Upon completion of the study, a final assessment is conducted to evaluate the overall response to the treatment.
Patients may be provided with a summary of their participation and any relevant findings related to their health.

Who can join the trial?

12 criteria

Participants must be boys or girls under 18 years old at the time of screening.
Participants must weigh at least 6 kilograms at the time of screening.
Participants and/or their parent or legal guardian must be willing and able to provide written, informed consent to join the study.
If applicable, participants must agree to have a pregnancy test and use adequate contraception.
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Participants must have a diagnosis of either:
- B-cell Acute Lymphoblastic Leukemia (B ALL) that is relapsed or refractory, meaning the disease has returned or not responded to treatment. This includes specific conditions such as:
  - Primary refractory disease with certain risk levels and measurable disease after initial treatments.
  - Relapsed or refractory disease after two or more lines of systemic therapy.
  - Relapsed or refractory disease after a stem cell transplant, with the study treatment given at least 3 months after the transplant.
  - Philadelphia chromosome positive disease where the patient cannot tolerate or has not responded to at least one specific type of medication called a tyrosine kinase inhibitor.
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    Aggressive Mature B-cell Non-Hodgkin Lymphoma (B NHL) that is relapsed or refractory, meaning the disease has returned or not responded to treatment. This includes:
    
    Relapsed after one or more prior therapies, which can include stem cell transplants.
    Primary refractory disease with measurable disease by imaging tests at screening.
    Participants must have a performance status score of at least 50% on a scale that measures their ability to perform daily activities. This is called the Karnofsky score for those aged 10 years or older, and the Lansky score for those under 10 years old.
    For participants with B ALL, there must be local documentation showing the presence of a protein called CD19 on the cancer cells, tested within 30 days before giving consent. For those treated with a drug called blinatumomab, this test should be done after stopping the drug. For participants with mature B NHL, CD19 expression must be confirmed in a biopsy after any CD19-targeted therapies.

Who cannot join the trial?

3 criteria

Patients who have a different type of cancer than the ones being studied, which are B-cell Acute Lymphoblastic Leukemia (B ALL) and Aggressive Mature B-cell Non-Hodgkin Lymphoma (B NHL).
Patients who are not within the specified age range for the study.
Patients who are part of a vulnerable population, which means they might need special protection or care.

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Hospital Vall d’Hebron
Barcelona, Spain

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Investigated drugs

AUTO1 is a type of therapy being studied for its safety and effectiveness in treating children with certain types of blood cancers. These cancers include B cell Acute Lymphoblastic Leukemia and Aggressive Mature B cell Non-Hodgkin Lymphoma. AUTO1 is a special treatment that uses the patient's own immune cells, which are modified to better recognize and attack cancer cells. This study aims to see how well patients tolerate this treatment and how safe it is for them.

What is already known about the treatment

AUTO1 – AUTO1 is administered through an intravenous infusion, specifically designed for pediatric patients. It is currently being studied in clinical trials to assess its safety and effectiveness in treating relapsed or refractory B-cell Acute Lymphoblastic Leukemia (B ALL) and Aggressive Mature B-cell Non-Hodgkin Lymphoma (B NHL). The medication works by targeting CD19-positive cancer cells, using genetically modified T-cells to recognize and destroy these cells. AUTO1 is classified as a type of CAR T-cell therapy, which is a form of immunotherapy that harnesses the body's immune system to fight cancer.

Investigated diseases

B-cell Acute Lymphoblastic Leukemia (B-ALL)
This is a type of cancer that affects the blood and bone marrow, characterized by the overproduction of immature white blood cells known as lymphoblasts. It progresses rapidly, leading to a decrease in the number of healthy blood cells, which can cause symptoms like fatigue, frequent infections, and easy bruising or bleeding. The disease primarily affects children but can also occur in adults. As the disease advances, it can spread to other parts of the body, including the lymph nodes, liver, and spleen.
Aggressive Mature B-cell Non-Hodgkin Lymphoma (B-NHL)
This is a group of blood cancers that originate in the lymphatic system, specifically affecting mature B-cells. It is characterized by the rapid growth of malignant cells, leading to swollen lymph nodes, fever, night sweats, and weight loss. The disease can spread quickly to other organs and tissues, including the bone marrow and spleen. B-NHL is more common in adults and can vary in its specific subtypes, each with distinct characteristics and progression patterns.