RecruitingRare disease

Study of GD2-CAR T Cells, Cyclophosphamide, and Fludarabine for Children with High-Risk or Relapsed Neuroblastoma and Other GD2+ 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 studying treatments for children and young adults with certain types of cancer, specifically Neuroblastoma and other tumors that have a protein called GD2 on their surface. These cancers are either high-risk, have come back after treatment, or have not responded to previous treatments. The study is testing a new treatment involving special immune cells called T cells that are modified in the lab to better recognize and attack cancer cells. These modified cells are known as iC9-GD2-CAR T cells. The trial also involves the use of other medications, including Cyclophosphamide and Fludarabine Phosphate, which are used to prepare the body for the T cell treatment, and Rimiducid, which can help control the activity of the T cells if needed.

The purpose of the study is to evaluate the safety and effectiveness of the iC9-GD2-CAR T cells in treating these cancers. Participants will receive the treatment through an intravenous infusion, which means the medicine is given directly into a vein. The study is divided into two phases. In the first phase, the focus is on determining the safest dose of the T cells. In the second phase, the study will look at how well the treatment works at the best dose found in the first phase. The study will monitor how the T cells behave in the body, including how long they stay active and how well they reach and affect the tumors.

This trial is an important step in exploring new ways to treat difficult-to-treat cancers in young patients. By participating, researchers hope to gather valuable information that could lead to better treatments in the future. The study is expected to continue for several years to gather comprehensive data on the treatment's safety and effectiveness.

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 reviewing medical history and current health status.
Tests such as bone marrow biopsy, ultrasound, CT/MRI scan, or MIBG scan are performed to evaluate the disease.
Step 2
Pre-treatment phase
Before starting the main treatment, patients receive cyclophosphamide and fludarabine intravenously. These medications help prepare the body for the main treatment.
The dosage and frequency of these medications are determined by the medical team based on individual patient needs.
Step 3
Main treatment
The main treatment involves the infusion of autologous T cells that have been modified to target specific cancer cells. This is done intravenously.
The goal is to evaluate the safety and effectiveness of these modified T cells in treating the disease.
Step 4
Monitoring and follow-up
After the infusion, regular monitoring is conducted to assess the body's response to the treatment. This includes blood tests and imaging studies.
The persistence and expansion of the infused T cells are evaluated using various tests, including blood and cerebrospinal fluid analysis.
Step 5
End of study
The study is expected to conclude by January 1, 2037. Patients will continue to be monitored for any long-term effects or outcomes related to the treatment.
Final assessments will be conducted to determine the overall effectiveness and safety of the treatment.

Who can join the trial?

8 criteria

The patient must have a diagnosis of Neuroblastoma (NBL) that has been treated with initial therapy and is considered incurable. This can be due to a relapse after the first treatment or the disease not improving after starting treatment.
The patient must have a measurable or visible disease at the time of treatment enrollment, which can be shown by tests like a bone marrow biopsy, ultrasound, CT/MRI scan, or a special scan called an MIBG scan.
The patient must have recovered from the side effects of previous chemotherapy treatments. If some side effects have become long-term, the patient must be stable according to the doctor and meet all other criteria.
The patient must be between 12 months and 18 years old for Phase I, and between 12 months and 35 years old for Phase II.
The patient or their legal guardian must give voluntary informed consent to participate in the study. For children 12 years and older, they will be included in discussions and asked for their agreement to participate.
The patient must have a certain level of physical ability. For those older than 16, a Karnofsky score of 60% or higher is needed. For those 16 and younger, a Lansky scale score of 60% or higher is required. These scores measure how well a person can perform daily activities.
Patients who can have children must agree to use birth control during the study and for four months after the treatment to prevent pregnancy.
Females who can have children must have a negative pregnancy test before starting the study because the treatment could harm a developing baby.

Who cannot join the trial?

5 criteria

Patients who have a different type of cancer that is not related to Neuroblastoma or GD2+ tumors. Neuroblastoma is a type of cancer that usually affects young children, and GD2+ tumors are a specific kind of tumor that has a certain marker on their cells.
Patients who have not experienced a return of their cancer after treatment or whose cancer is not resistant to treatment. "Relapsed" means the cancer has come back after treatment, and "refractory" means the cancer does not respond to treatment.
Patients who are not considered at high risk for their cancer returning or worsening. "High risk" refers to patients who have a greater chance of their cancer coming back or getting worse.
Patients who are not within the age range specified for the study. The study is for children, so adults cannot participate.
Patients who are not able to understand or agree to the study requirements. This means that the patient or their guardian must be able to understand what the study involves and agree to participate.

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Verified sites

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Verified
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, known as T cells, that have been engineered to recognize and attack cancer cells. The modification involves adding a receptor that targets a specific protein called GD2, which is found on the surface of certain cancer cells, including those in neuroblastoma. This therapy is being tested to see if it is safe and effective for treating children with high-risk or relapsed neuroblastoma and other tumors that express the GD2 protein.

What is already known about the treatment

iC9-GD2-CAR T cells – This medication is administered through infusion and is currently being studied in clinical trials for its safety and effectiveness. It is primarily used for treating high-risk or relapsed/refractory neuroblastoma and other GD2-positive tumors in pediatric patients. The mechanism of action involves genetically modifying T cells to express a chimeric antigen receptor (CAR) that targets the GD2 antigen on tumor cells, enhancing the immune system's ability to attack cancer cells. It falls under the pharmacological classification of CAR T-cell therapies, which are a type of immunotherapy.

Investigated diseases

Neuroblastoma
Neuroblastoma is a type of cancer that most commonly affects children and develops from immature nerve cells found in several areas of the body. It often begins in the adrenal glands but can also develop in the neck, chest, abdomen, or spine. The disease can spread to other parts of the body, including lymph nodes, bones, bone marrow, liver, and skin. Symptoms may vary depending on the location of the tumor and can include abdominal pain, a lump in the abdomen, or changes in bowel habits. As the disease progresses, it may cause fatigue, loss of appetite, and weight loss. Neuroblastoma is considered high-risk when it is likely to recur or has not responded to initial treatments.
GD2+ Tumors
GD2+ tumors are characterized by the presence of a specific molecule called GD2 on the surface of cancer cells. This molecule is commonly found in certain types of cancers, including neuroblastoma and some soft tissue sarcomas. The presence of GD2 can make these tumors more aggressive and challenging to treat. These tumors can grow and spread to other parts of the body, leading to various symptoms depending on their location. As they progress, they may cause pain, swelling, or dysfunction of affected organs. The identification of GD2 on tumor cells is important for targeted therapies and research into new treatment options.