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

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What is this study about?

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.

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.

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.

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.

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.

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.

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.

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 Study?

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 Study?

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.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name	City	Country
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Virgen del Rocío University Hospital	Sevilla	Spain
Fkadslgna Pxrt Lk Imteqmxozxhnd Bgysrwowt Doj Hodfyuax Ucnkobznoupfe Lu Pnh	Madrid	Spain
Hmsypomu Vlhl dpdvodzz	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Spain	Not recruiting	23.03.2023

Trial locations

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.

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.

Trial ID:

2024-515467-66-00

Protocol code:

CART19-BE-03Ped

Trial Phase:

Therapeutic exploratory (Phase II)

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

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