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Study of ARI-0001 Cells for Children and Adolescents with Resistant or Refractory Acute Lymphoblastic Leukemia

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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 a type of blood cancer called acute lymphoblastic leukemia (ALL) in children and adolescents aged 0-18 years. The study is specifically for those whose leukemia is resistant or does not respond to standard treatments. The main treatment being tested is called Varnimcabtagene autoleucel, also known by its code name ARI-0001. This treatment involves using a patient's own immune cells, which are modified in a laboratory to better recognize and attack cancer cells. These modified cells are then infused back into the patient's body.

In addition to Varnimcabtagene autoleucel, the study involves several other medications that may be used to support the treatment process. These include cytarabine, dexchlorpheniramine maleate, human immunoglobulin G, allopurinol, methylprednisolone, etoposide, fludarabine, tocilizumab, paracetamol, and cyclophosphamide. These medications serve various roles, such as chemotherapy, antihistamines, and other supportive therapies.

The purpose of the study is to evaluate how effective the ARI-0001 cells are in treating patients with this type of leukemia. Participants will receive the treatment and be monitored over a period of time to assess their response. The study will track the complete response rate, which includes remission and recovery of blood cell counts, as well as other outcomes like survival rates and any side effects. The study aims to provide valuable information on the potential benefits and risks of this new treatment approach for children and adolescents with difficult-to-treat acute lymphoblastic leukemia.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment and preparation
Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a review of medical history and current health status.
Blood samples are collected to evaluate the presence of CD19+ acute lymphoblastic leukemia and to ensure adequate venous access for future procedures.
Step 2
Lymphoapheresis
A procedure called lymphoapheresis is performed to collect T-cells from the blood. These cells are essential for the creation of ARI-0001 cells.
Step 3
Cell modification and expansion
The collected T-cells are modified in a laboratory to express a chimeric antigen receptor (CAR) with anti-CD19 specificity. This process involves the use of a lentivirus.
The modified T-cells, now called ARI-0001 cells, are expanded to increase their number.
Step 4
Pre-treatment medication
Before the infusion of ARI-0001 cells, medications are administered to prepare the body. These include fludarabine and cyclophosphamide, given through intravenous infusion to reduce the number of existing immune cells and make space for the new cells.
Step 5
Infusion of ARI-0001 cells
The ARI-0001 cells are infused back into the bloodstream through an intravenous infusion. This is the main treatment phase aimed at targeting and eliminating leukemia cells.
Step 6
Post-infusion monitoring
After the infusion, regular monitoring is conducted to assess the response to treatment. This includes blood tests and imaging tests to evaluate the presence of leukemia cells.
Monitoring for side effects is also crucial. Medications such as tocilizumab and paracetamol may be administered to manage potential side effects like fever or inflammation.
Step 7
Follow-up assessments
Follow-up assessments are scheduled to track the effectiveness of the treatment and the patient's overall health. These assessments occur weekly for the first month, monthly for the first six months, and then at 12 months.
The primary goal is to achieve a complete response with undetectable leukemia cells by day 100 after infusion.

Who can join the trial?

10 criteria

Age between 0 to 18 years.
Diagnosis of **relapsed/refractory CD19+ ALL**. This means the leukemia has returned or is not responding to treatment.
**High-risk first relapse** includes any relapse before 6 months after finishing chemotherapy or specific genetic changes in the leukemia cells.
**Disease burden** is defined as having 5% or more leukemia cells in the bone marrow or leukemia cells in other parts of the body.
**MRD positivity** means a small number of leukemia cells are still present, detected by special tests.
Patients with only leukemia in other parts of the body, Down syndrome, or certain other conditions are not excluded.
**Performance status** should be 50% or higher, which means the patient can do daily activities with some help.
Life expectancy should be more than 3 months.
Must have good veins for a procedure called **lymphoapheresis** and no reasons to avoid it.
Patient and/or legal guardian must sign an **informed consent**, agreeing to participate in the study.

Who cannot join the trial?

5 criteria

Patients who have a different type of cancer other than acute lymphoblastic leukemia cannot participate. Acute lymphoblastic leukemia is a type of blood cancer that affects white blood cells.
Patients who do not have the specific marker called CD19+ on their cancer cells are excluded. CD19+ is a protein found on the surface of certain cancer cells.
Patients who are not resistant or refractory to therapy are excluded. Resistant or refractory means that the cancer does not respond to standard treatments.
Patients who are outside the age range specified for the trial cannot participate. The trial is for a specific age group.
Patients who are not able to follow the trial procedures or do not meet other specific health requirements may be excluded.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Responsive
Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Paz
Madrid, Spain
Verified
Hospital Sant Joan De Deu Barcelona
Esplugues De Llobregat, Spain
Verified
Hospital Vall d’Hebron
Barcelona, Spain

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Investigated drugs

ARI-0001 cells are a type of therapy used in this clinical trial. These are specially modified T-cells taken from the patient's own blood. The T-cells are expanded and changed in the lab using a lentivirus to help them recognize and attack cancer cells. They are designed to target a specific protein called CD19 found on the surface of certain leukemia cells. The ARI-0001 cells are equipped with additional components, known as co-stimulatory regions, which help them to better fight the cancer. This therapy is being tested in children and adolescents with a type of leukemia that has not responded to other treatments.

What is already known about the treatment

ARI-0001 cells – This medication is administered through an infusion of specially modified T-cells derived from the patient's own blood. These T-cells are expanded and genetically altered using a lentivirus to express a chimeric antigen receptor (CAR) targeting the CD19 protein, which is commonly found on the surface of certain leukemia cells. The current status of ARI-0001 cells in medical literature is within the clinical trial phase, specifically a Phase 2 study, focusing on children and adolescents with CD19+ acute lymphoblastic leukemia that is resistant or refractory to standard treatments. The main therapeutic indication is for treating this specific type of leukemia. At the molecular level, the mechanism of action involves the modified T-cells recognizing and binding to the CD19 protein on cancer cells, leading to their destruction. ARI-0001 cells fall under the pharmacological classification of CAR T-cell therapies, which are a form of immunotherapy designed to enhance the body's immune response against cancer cells.

Investigated diseases

Acute Lymphoblastic Leukemia – 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 an accumulation of these cells in the bone marrow, which interferes with the production of normal blood cells. As the disease advances, it can spread to other parts of the body, including the lymph nodes, liver, spleen, and central nervous system. Symptoms may include fatigue, fever, frequent infections, and easy bruising or bleeding. The disease is most common in children but can also occur in adults. It requires prompt medical attention to manage its progression.