Study of Tisagenlecleucel for Treating Adult Patients with Resistant or Relapsed B-cell 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 B-cell acute lymphoblastic leukemia (B-ALL) in adults. This disease is characterized by the presence of cancerous white blood cells in the bone marrow. The study is specifically for patients whose disease has shown resistance to treatment, has relapsed, or has measurable residual disease, meaning that small amounts of cancer cells remain after treatment.

The treatment being tested in this study is called tisagenlecleucel, also known by its code name FCTX-CL19-1 or Tarcidomgen Kimleucel. This is a type of therapy known as CAR T-cell therapy, which involves modifying a patient’s own immune cells to better recognize and attack cancer cells. The purpose of the study is to evaluate the safety and effectiveness of this treatment in managing B-ALL.

Participants in the study will receive the treatment through an infusion, which is a method of delivering medication directly into the bloodstream. The study will monitor participants over a period of time to observe how well the treatment works and to identify any side effects. The goal is to determine if this therapy can help achieve remission, which means the cancer is no longer detectable, and to understand how long the benefits of the treatment last. The study will also track the overall health and survival of participants following the treatment.

1 initial assessment

The initial assessment involves confirming the diagnosis of B-cell acute lymphoblastic leukemia with resistance, relapse, or measurable residual disease.

Eligibility criteria include being over 18 years old, having undergone at least two cycles of chemotherapy, and having a performance status of 2 or less on the ECOG scale.

Organ function is assessed, including liver enzymes, heart function, and respiratory capacity.

2 informed consent

Understanding and providing informed consent is required to participate in the study.

For women of childbearing age, a negative pregnancy test is necessary, and a commitment to use contraception for 12 months after therapy is required.

3 preparation for treatment

Preparation includes ensuring satisfactory organ function and confirming the absence of significant health issues that could interfere with the treatment.

4 CAR-T cell infusion

The treatment involves the infusion of anti-CD19 CAR T cells, specifically the product named FCTX-CL19-1.

The infusion is administered as a single dose.

5 post-infusion monitoring

Monitoring for treatment-emergent adverse events is conducted for 12 weeks post-infusion.

Adverse events of interest include cytokine release syndrome, neurological events, infections, and febrile neutropenia.

6 follow-up assessments

Follow-up assessments occur at 1, 3, 6, 12, 18, and 24 months post-infusion to evaluate disease status and treatment response.

Key measures include overall response rate, progression-free survival, and overall survival.

7 long-term observation

Long-term observation continues for up to 24 months to assess the duration of response and any persistent effects of the treatment.

Cellular kinetics of the CAR-T cells are monitored to understand their behavior in the body over time.

Who Can Join the Study?

The patient must have a diagnosis of B-cell acute lymphoblastic leukemia (B-ALL) at the time of resistance, relapse, or when there is measurable residual disease (MRD). MRD means that a small number of cancer cells are still present after treatment.
The patient must have experienced resistance, relapse, or have MRD, defined as at least 0.01% of leukemic lymphoblasts (cancer cells) among bone marrow cells.
The patient must have previously undergone at least two cycles of chemotherapy.
The patient must be over 18 years old at the time of screening.
The patient must have a performance status of 2 or less on the ECOG scale, which is a way to measure how well a patient can perform daily activities.
The patient must have satisfactory organ function during screening, which includes:
- ALT/AST activity (liver enzymes) less than 4 times the upper limit of normal and total bilirubin less than 2 mg/dL (or less than 4 mg/dL for patients with Gilbert’s syndrome).
- Ejection fraction (EF) greater than 40% confirmed by an ECHO (a heart ultrasound), with no significant fluid around the heart within 6 weeks prior to screening.
- Respiratory capacity with arterial blood saturation greater than 92% without oxygen therapy and no significant fluid around the lungs.
- Creatinine clearance greater than 30 ml/min, which measures kidney function.
The patient must understand and provide informed consent to participate in the study.
Women of childbearing age must have a negative pregnancy test (serum).
Women capable of having children must agree to refrain from heterosexual sexual intercourse or use an appropriate method of contraception for 12 months after CAR-T therapy. If using hormonal contraception, it must start at least one month before CAR-T therapy.
Men capable of having children must agree to refrain from heterosexual sexual intercourse or use an appropriate method of contraception for 12 months after CAR-T therapy.

Who Cannot Join the Study?

Patients who do not have B-cell acute lymphoblastic leukemia with resistance, relapse, or detectable measurable residual disease cannot participate. This means the study is only for those with this specific type of leukemia that has not responded to treatment, has come back, or still shows signs of disease after treatment.
Individuals who are not adults are excluded. The study is only for adult participants.
Patients who are not able to understand or follow the study procedures will not be able to participate.
Individuals who are pregnant or breastfeeding are not allowed to join the study.
Patients with other serious health conditions that could interfere with the study or increase risk are excluded.
Anyone who has participated in another clinical trial recently may not be eligible.
Patients who have had certain treatments recently that could affect the study results are not allowed to participate.

Where you can join this trial?

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Other Sites

Site Name	City	Country
Instytut Hematologii I Transfuzjologii	Warsaw	Poland
Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego	Warsaw	Poland
Nvqjugpm Iemewurg Ovkwjjkym Irp Mbduw Ssrruytsgzgjobfyrrobwebqnkwj Iqvmrcov Bawhdptx	Cracow	Poland

Trial status

Country	Status	Recruitment Start
Poland	Not yet recruiting	12.01.2024

Trial locations

Investigated drugs:

Tisagenlecleucel

Anti-CD19 CAR T Cells are a type of therapy used in this clinical trial. These are specially modified immune cells designed to target and destroy cancer cells in patients with B-cell acute lymphoblastic leukemia. The therapy involves taking a patient’s own T cells, a type of white blood cell, and genetically modifying them to better recognize and attack cancer cells that express the CD19 protein on their surface. This treatment is being tested for its safety and effectiveness in patients whose leukemia has not responded to other treatments, has returned after treatment, or still shows signs of disease.

B-cell 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 primarily involves B-cells, which are a type of white blood cell responsible for producing antibodies. The disease progresses rapidly, leading to symptoms such as fatigue, fever, frequent infections, and easy bruising or bleeding. In cases of resistance, relapse, or detectable measurable residual disease, the leukemia cells persist despite treatment, making the condition more challenging to manage. The presence of measurable residual disease indicates that a small number of cancer cells remain after treatment, which can lead to a recurrence of the disease.

Trial ID:

2024-519808-27-00

Protocol code:

MERMAID1

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

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Study of Tisagenlecleucel for Treating Adult Patients with Resistant or Relapsed B-cell Acute Lymphoblastic Leukemia

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?