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Study on the Safety of Anti-GD2 CAR T Cells, Cyclophosphamide, and Fludarabine in Children and Young Adults with Relapsed or Refractory Brain Tumors

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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 treating children and young adults with relapsed or refractory central nervous system tumors, which are brain tumors that have returned or are not responding to standard treatments. The study is testing a new treatment involving anti-GD2 Chimeric Antigen Receptor-Expressing T cells. These are special immune cells that have been modified in a lab to better recognize and attack cancer cells. The trial also uses other medications, including Cyclophosphamide, Fludarabine, and Rimiducid. Cyclophosphamide and Fludarabine are chemotherapy drugs that help prepare the body for the T cell treatment, while Rimiducid is used to control the activity of the modified T cells.

The purpose of the study is to evaluate the safety and determine the best dose of the modified T cells for patients with these challenging brain tumors. Participants will receive the treatment through an intravenous method, which means the medication is given directly into a vein. The study will monitor how the body responds to the treatment and how long the modified T cells stay active in the body. This will help researchers understand the potential benefits and any side effects of the treatment.

Throughout the study, participants will undergo regular MRI scans to check the size and growth of the tumors. The trial aims to find out if the new treatment can help control the tumors and improve the quality of life for patients. The study is expected to continue for several years to gather enough information about the treatment's effectiveness and safety.

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 imaging assessments and evaluation of disease status through MRI scans.
Eligibility is determined based on age, disease characteristics, and previous treatment effects. A negative pregnancy test is required for females of childbearing potential.
Step 2
Preparation for treatment
Before treatment begins, a device is placed to allow for the administration of medications directly into the brain.
Patients must have adequate venous access for apheresis, a process to collect blood cells.
Step 3
Medication administration
The treatment involves the administration of several medications through intravenous use.
Cyclophosphamide and Fludarabine are given to prepare the body for the main treatment.
The main treatment consists of autologous T cells that have been modified to target cancer cells. These are infused into the patient.
Step 4
Monitoring and follow-up
After the infusion, patients are closely monitored for any side effects or reactions to the treatment.
Regular follow-up visits are scheduled to assess the persistence and effectiveness of the treatment using blood tests and imaging.
Step 5
End of trial
The trial is expected to continue until 2038, with ongoing assessments to determine the long-term effects and safety of the treatment.
Patients are encouraged to maintain regular communication with their healthcare providers throughout the duration of the trial.

Who can join the trial?

12 criteria

The patient must have a confirmed diagnosis of a relapsed or difficult-to-treat **central nervous system (CNS) tumor**. This includes specific types like **medulloblastoma** or other similar tumors, **high-grade glioma (HGG)** in the brain, or other rare CNS tumors.
The tumor must show **GD2 positivity**, meaning it has a specific marker that makes the patient eligible for the study. If the tumor is **GD2 negative**, the patient cannot participate. If it's not possible to get a tumor sample, the patient can still be eligible based on existing scientific data.
The patient must be between **6 months and 30 years old**.
The patient must have suitable **vein access** for a procedure called **apheresis**, or be eligible for a catheter placement, which is a small tube inserted into a vein.
A signed **informed consent** is required from the patient or their legal guardians. For those under 18, a legal guardian must provide consent, and children 7 years or older should also give their agreement in a way they understand.
The patient must have a **Karnofsky/Lansky score** of at least 60, which is a way to measure their ability to perform daily activities.
Recent **imaging tests** must be done within 14 days before starting treatment to assess the tumor.
The patient must have a **measurable or evaluable disease** on an MRI scan at the time of joining the study.
The patient must have recovered from severe side effects of previous treatments, like radiation or chemotherapy. Any remaining side effects should be mild or stable.
The patient must have an **implantable intraventricular access device**, which is a device placed in the brain to deliver treatment.
Patients who can have children must agree to use **birth control** during the study and for six months after the treatment to prevent pregnancy.
Females who can become pregnant must have a **negative pregnancy test** before joining the study to ensure safety for any potential pregnancy.

Who cannot join the trial?

7 criteria

Patients who have a different type of tumor that is not a **relapsed/refractory malignant central nervous system tumor** cannot participate. This means the study is only for those whose brain or spinal cord tumors have returned or are not responding to treatment.
Patients who are not within the specified age range for the study cannot participate. The study is for **pediatric and young adult patients**, which typically means children and young adults.
Patients who are not able to follow the study procedures or who have conditions that make it unsafe for them to participate cannot join the study.
Patients who are pregnant or breastfeeding cannot participate, as the study may pose risks to the baby.
Patients who have other serious health conditions that could interfere with the study or put them at risk cannot participate.
Patients who are currently participating in another clinical trial or have recently participated in one may not be eligible.
Patients who have allergies or reactions to the study treatment or its components cannot participate.

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Ospedale Pediatrico Bambino Gesu'
Rome, Italy

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Investigated drugs

iC9-GD2-CAR T cells are a type of therapy used in this clinical trial. These are specially modified immune cells designed to recognize and attack cancer cells in the brain and spinal cord. The therapy involves taking a patient's own T cells, a type of white blood cell, and altering them in the lab to express a receptor called GD2. This receptor helps the T cells identify and target cancer cells more effectively. The modified T cells are then infused back into the patient to help fight the tumor. The study aims to determine the safety and appropriate dosage of these modified T cells in young patients with difficult-to-treat brain tumors.

What is already known about the treatment

iC9-GD2-CAR T cells – This medication is administered through infusion, specifically designed for pediatric and young adult patients with relapsed or refractory malignant central nervous system tumors. Currently, it is in the clinical trial phase, with ongoing research to determine its safety, feasibility, and optimal dosing. The main therapeutic indication is for treating these specific types of brain tumors. At the molecular level, the mechanism of action involves genetically modified T cells that express a chimeric antigen receptor (CAR) targeting the GD2 antigen, which is commonly found on the surface of certain tumor cells. This medication falls under the pharmacological classification of cell-based immunotherapy.

Investigated diseases

Relapsed/Refractory Malignant Central Nervous System Tumors – These are aggressive tumors located in the brain or spinal cord that have either returned after treatment or have not responded to initial therapies. They can affect various functions depending on their location, such as movement, sensation, or cognition. As the disease progresses, symptoms may worsen, leading to increased neurological deficits. The tumors can vary in type, including gliomas or medulloblastomas, and are often challenging to manage due to their resistance to standard treatments. The progression of these tumors can lead to increased pressure within the skull, causing headaches, nausea, or changes in consciousness.