Active not recruitingPHASE2INTERVENTIONAL

Study of Efficacy and Safety of Tisagenlecleucel in HR B-ALL EOC MRD Positive Patients

This study is testing a special cell therapy called tisagenlecleucel for children and young adults (aged 1 to 25) who have a type of blood cancer called B-cell acute lymphoblastic leukemia (B-ALL). These patients have already had their first round of standard treatment but still show some signs of the cancer. The study wants to find out if this new therapy is effective at fighting the cancer and if it's safe to use. Patients will be carefully monitored with regular check-ups and tests for several years to track their progress and any potential side effects. This is a Phase 2 study, meaning it's still relatively early in testing new treatments.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Novartis Pharmaceuticals

Enrolment target

121

Start

24 Jun 2019

Estimated completion

19 Oct 2027

B-Cell Acute Lymphoblastic Leukemia

What is this study about?

This research study is looking into a new treatment for a type of blood cancer affecting children and young adults, called B-cell acute lymphoblastic leukemia (B-ALL). This cancer affects white blood cells, and the study focuses on patients aged 1 to 25. These young people have already received their first standard course of treatment for B-ALL, but simple tests still show some evidence of the cancer cells remaining in their body. This study wants to see if a special treatment, called tisagenlecleucel, can help these patients.

Tisagenlecleucel is a type of treatment that uses your own immune cells, called T-cells, to fight cancer. These cells are taken from your blood, specially trained in a lab to recognise and attack cancer cells, and then given back to you. The main goals of this study are to find out how well this treatment works in reducing the remaining cancer cells and to carefully monitor its safety. While this sounds complex, the team will explain everything simply if you or your child consider taking part.

This is a Phase 2 study, which means it’s still early in understanding the effects of this new treatment. The study will follow patients for several years, initially with more frequent checks, to see the long-term results of the treatment. The findings from this study will help doctors understand if tisagenlecleucel could be a helpful new option for young people with B-ALL who still have signs of cancer after their first treatment.

Key takeaways

This study evaluates a new cell therapy (tisagenlecleucel) for B-ALL.
It's for children and young adults (1-25) who still have signs of cancer after first treatment.
The treatment involves using your own immune cells to fight cancer.
Participation includes regular check-ups for several years to monitor safety and effectiveness.
This is a Phase 2 study, so it's still an investigational treatment.

Who may be eligible?

To be considered for this study, patients need to be generally aged between 1 and 25 years old. They must have a specific type of B-cell acute lymphoblastic leukemia (B-ALL) where the cancer cells have a particular marker (called CD19). Importantly, they must have already completed their first standard treatment for B-ALL but still have a level of cancer cells detected in their bone marrow.

Patients also need to be well enough to take part, with good general health. This means their kidneys, liver, and heart should be working well, and they should be able to breathe comfortably. Doctors will check these things to make sure it's safe for someone to join the study.

There are also some reasons why a patient might not be able to join. For example, if their cancer has progressed in certain ways, if they have certain genetic conditions, or if they have previously had specific cancer treatments like tyrosine kinase inhibitors. The study team will review all medical history to determine if a patient can safely participate.

Quick self-check

Are you/is your child between 1 and 25 years old?
Do you/does your child have B-cell acute lymphoblastic leukemia (B-ALL) with a CD19 marker?
Have you/has your child completed the first standard treatment for B-ALL, but still have some cancer cells (MRD positive)?
Are your/your child's kidneys, liver, and heart generally working well?
Do you/does your child have good breathing and general physical health?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you or your child decide to take part in this study, the first step is a 'screening' phase. This involves various tests to make sure that the treatment is suitable and safe for you. After screening, you would enter the 'pre-treatment' phase where preparations are made for the special cell therapy.

The main part is the 'treatment' phase, where you would receive the tisagenlecleucel cells. After the treatment, you will enter the 'follow-up' phase. During this time, you'll have regular appointments to see how you're doing. These visits will be quite frequent at first, especially in the first month and at around Day 29. After that, they will become less frequent: every 3 months for the first year, then every 6 months during the second year, and then once a year until the study finishes. The study is planned to last for about 8 years from when the first patient started treatment. During these visits, doctors will perform various assessments and tests to check how well the treatment is working and to monitor for any side effects. Even after this study formally ends, there will be further long-term monitoring as part of a separate safety follow-up plan.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. The potential benefit of this study is that tisagenlecleucel might be an effective new treatment for B-ALL, especially for patients who still have signs of cancer after initial treatment. However, it's a new treatment, so there might be side effects or risks that we don't fully know about yet. The study team will carefully monitor participants for any problems, and you'll be told about any known risks before you decide to join. It's very important to remember that participating is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (43)

Children s Hospital of Alabama
Birmingham, United States
Phoenix Childrens Hospital
Phoenix, United States
City of Hope National Medical
Duarte, United States
Childrens Hospital Los Angeles
Los Angeles, United States
Mattel Childrens Hospital UCLA
Los Angeles, United States
Childrens Hospital of Orange County
Orange, United States
Rady Children s Hospital
San Diego, United States
UCSF Medical Center
San Francisco, United States
Stanford University Medical Center
Stanford, United States
Childrens Hospital Colorado
Aurora, United States
Childrens National Hospital
Washington D.C., United States
Johns Hopkins All Childrens
St. Petersburg, United States

+31 more sites — see the official record for the full list.

Common questions

What is B-cell acute lymphoblastic leukemia (B-ALL)?

It's a fast-growing cancer of the white blood cells that usually affects children and young adults.

What is tisagenlecleucel?

It's a special type of cell therapy where your own immune cells are trained to fight cancer and given back to you.

Who can join this study?

Children and young adults aged 1-25 with B-ALL who still have some cancer cells after their first standard treatment, and meet other health requirements.

How long will the study last if I join?

The study itself is expected to last about 8 years, with regular check-ups that become less frequent over time. There will also be long-term safety follow-up afterwards.

Can I stop participating if I want to?

Yes, joining is voluntary, and you can withdraw from the study at any time without it affecting your medical care.