RecruitingPHASE3INTERVENTIONAL

Study of Out of Specification for Tisagenlecleucel

This study is investigating a special type of cell therapy, known as tisagenlecleucel (also called CTL019), for children, young adults, and adults who have certain types of blood cancer that haven't responded to other treatments. These cancers include B-cell acute lymphoblastic leukemia and diffuse large B-cell lymphoma. What's unique about this study is that it's looking at the safety of this treatment even when the manufactured cell product doesn't perfectly match all the usual quality checks. The aim is to see if these cell products are still safe and effective for patients who desperately need treatment. Researchers will closely monitor patients for a short time after they receive the treatment.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Novartis Pharmaceuticals

Enrolment target

200

Start

21 Nov 2019

Estimated completion

31 Mar 2027

B-cell Acute Lymphoblastic Leukemia Diffuse Large B-cell Lymphoma

What is this study about?

Imagine your body has special cells that fight off illness. This treatment, called tisagenlecleucel, is a type of cell therapy where doctors take some of your own immune cells, called T-cells, and genetically change them in a lab to make them better at finding and killing cancer cells. These changed cells are then given back to you.

This study is focusing on patients with serious types of blood cancer like B-cell acute lymphoblastic leukemia (a type of blood cancer common in children and young adults) and diffuse large B-cell lymphoma (a type of lymphoma often found in adults). These cancers can be very difficult to treat with standard methods, so new options are very important. The main goal of this study is to check the safety of this cell therapy.

What's particularly interesting about this study is that it includes patients whose manufactured cell product didn't quite meet all the usual quality control standards. This doesn't necessarily mean it's unsafe, but rather that it deviated slightly from the exact manufacturing recipe. Doctors and experts will carefully review each case to ensure that using this product isn't expected to cause any extra safety concerns for the patient. The study wants to understand if these slightly different batches are still safe and can help patients who have run out of other treatment options.

Key takeaways

This study evaluates a cell therapy (CTL019) for severe blood cancers.
It specifically looks at the safety of treatments that are 'out of specification' but deemed safe for use.
The therapy uses your own immune cells modified to fight cancer.
Participation involves a single infusion and careful monitoring for a short period.
It's for patients with limited other treatment options.
Your safety and careful assessment are top priorities.

Who may be eligible?

To be considered for this study, you or your child would need to meet several important criteria. Firstly, your doctors will need to confirm that your type of tisagenlecleucel cell product, which has been made specifically for you, didn't perfectly match all the standard manufacturing checks. However, importantly, experts would have carefully reviewed this product to ensure that it's not thought to be a significant extra risk to your safety.

You also need to be suffering from a serious or life-threatening blood cancer that your doctor believes could benefit from this treatment. Your doctor would likely consider you for this study if it's not possible to perform a second procedure to collect your cells again (called leukapheresis).

There are also reasons why someone might not be able to join the study. For example, if you have HIV, active Hepatitis B or C, certain types of brain lymphoma, or a history of severe allergic reactions to similar medications, you might not be eligible for one part of the study. Women who are pregnant or breastfeeding also cannot take part. Your study doctor will discuss all these points with you in detail.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

My cancer is one of the specific types being studied (e.g., B-cell ALL, DLBCL).
My manufactured cell treatment didn't meet all standard quality checks, but has been approved by experts.
I have signed an informed consent form.
I am not pregnant or breastfeeding.
I do not have HIV or active Hepatitis B or C (for certain parts of the study).
My doctor believes I cannot undergo another cell collection procedure.

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, the first step is to carefully read and sign a consent form, which explains everything involved. You would receive a single infusion of the special cell therapy (CTL019). After receiving this treatment, doctors would closely monitor you. For some patients, this monitoring period would last about 3 months, while for others, it might be just one day, depending on which part of the study you are in. During this time, you would have regular check-ups and tests to see how you are responding to the treatment and to keep an eye on any side effects. The total duration of your participation in the study, including follow-up, would depend on which group you are in, but the main intense monitoring is for a shorter period.

Potential risks and benefits

Participating in this study might offer a potential benefit if your cancer has not responded to other treatments, as it provides access to a specialized cell therapy that could help. However, like all medical treatments, there are potential risks. The cell therapy itself can cause side effects, and because the product in this study didn't meet all usual standards, there could theoretically be unforeseen risks, though each case is carefully assessed. Your safety will be closely monitored throughout the study. It's very important to remember that you have the right to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (53)

Novartis Investigative Site
Verified postcode
Hamilton, Canada· Completed
Novartis Investigative Site
Verified postcode
Ottawa, Canada· Withdrawn
Novartis Investigative Site
Verified postcode
Toronto, Canada· Completed
Novartis Investigative Site
Verified postcode
Toronto, Canada· Completed
Novartis Investigative Site
Verified postcode
Montreal, Canada· Withdrawn
Novartis Investigative Site
Verified postcode
Québec, Canada· Withdrawn
Novartis Investigative Site
Verified postcode
Nagoya, Japan· Recruiting
Novartis Investigative Site
Verified postcode
Nagoya, Japan· Recruiting
Novartis Investigative Site
Verified postcode
Tōon, Japan· Recruiting
Novartis Investigative Site
Verified postcode
Gifu, Japan· Recruiting
Novartis Investigative Site
Verified postcode
Sapporo, Japan· Recruiting
Novartis Investigative Site
Verified postcode
Sapporo, Japan· Recruiting

Common questions

What does 'out of specification' mean for my treatment?

It means the manufactured cell product for your treatment didn't perfectly match all the usual quality checks, but experts have reviewed it and believe it's still safe for you to receive.

What is CTL019?

CTL019 is a special type of cell therapy, also known as tisagenlecleucel, where your own immune cells are changed in a lab to fight your cancer.

What types of cancer is this study for?

This study is for certain types of difficult-to-treat blood cancers in children and adults, specifically B-cell acute lymphoblastic leukemia and diffuse large B-cell lymphoma.

How long will I be followed after the treatment?

Monitoring after treatment will be for either 3 months or just 1 day, depending on which part of the study you are in.

Can I leave the study if I change my mind?

Yes, you can choose to leave the study at any time, and this will not affect your future medical care.

How to find out more

Novartis Pharmaceuticals

novartis.email@novartis.com +41613241111 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.