Active not recruitingPHASE1, PHASE2INTERVENTIONAL

Durvalumab and Tremelimumab for Pediatric Malignancies

This research study is about finding the right dose of two new anticancer medicines, durvalumab and tremelimumab, for children and teenagers with advanced solid tumours or blood cancers that haven't responded to previous treatments. These medicines work by using the body's own immune system to fight cancer. In the first part of the study, we'll carefully increase the dose to find one that is both safe and well-tolerated. Once we find the best dose, the second part of the study will expand to see how well these drugs work against the cancer. This study is for patients for whom there are currently no other treatment options available.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

AstraZeneca

Enrolment target

Start

07 Mar 2019

Estimated completion

31 Dec 2026

Pediatric Cancer Solid Tumor Pediatric Hematological Malignancies

Results

Results from this study

Posted March 2024

Results have been published for this study.

Primary outcome

Dose-Finding Phase: Maximum Serum Concentration (Cmax) of Durvalumab

Serum samples were collected from the participants at the defined timepoints. Cmax was determined using standard non-compartmental methods.

Full results on the registry

What is this study about?

This study is a pioneering effort to explore new treatment options for children and teenagers facing advanced cancer. The main goal is to find a safe and effective starting dose for two special drugs called durvalumab and tremelimumab. These are immunotherapy drugs, which means they work differently from traditional chemotherapy. Instead of directly killing cancer cells, they help your body's own immune system find and fight the cancer more effectively.

Because this is the first time these particular combinations of drugs are being used in children, the study starts very carefully. We'll begin with very small doses and gradually increase them, all while closely monitoring the young patients for any side effects. This careful approach helps us understand how the drugs behave in children and find a dose that is not only safe but also shows promise in tackling the cancer.

While the primary focus of this early stage study is on safety and finding the right dose, we will also be looking to see if the drugs have any helpful effects on the cancer. For children joining this study, standard treatments have unfortunately not worked, or there are no standard treatments available. Therefore, this study offers a chance to try a different approach that might offer some benefit.

Key takeaways

This study is testing new immune-boosting drugs for children with advanced cancer.
It's an early-stage study focused on finding safe doses and understanding how the drugs work.
It's for children and teenagers (up to 17) whose cancer hasn't responded to other treatments.
Close monitoring for side effects and treatment response will be a key part of taking part.
Participation offers a chance at new treatment options where standard ones are not available.

Who may be eligible?

This study is designed for children and teenagers up to 17 years old. You might be able to join if you have certain types of advanced solid cancer (not including brain cancer) or specific kinds of blood cancer called non-Hodgkin's lymphoma (specifically primary mediastinal B-cell lymphoma and anaplastic large cell lymphoma). In all cases, your cancer must have come back or not responded to previous standard treatments, and there are no other standard treatment options available for your specific condition.

To be considered, we will need a sample of your cancer tissue if one is available. You must also be generally well enough to take part, meaning your major organs like your heart and kidneys are working properly. It's important that you haven't received any immune-based therapies before joining this study, and you should not have any serious ongoing medical conditions or infections.

There are also some reasons why you might not be able to join. For example, if you've had an organ transplant (though in some cases for a specific type of lymphoma, this might be discussed), or if you have certain autoimmune conditions or uncontrolled ongoing illnesses. We'll also need to make sure any side effects from previous cancer treatments have mostly cleared up. Your medical team will carefully review all these details to see if this study is suitable for you.

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.

Are you 17 years old or younger?
Do you have an advanced solid cancer (not brain cancer) or a specific type of non-Hodgkin's lymphoma?
Has your cancer come back or not responded to standard treatments, and are there no other options?
Have you NOT had any immune-boosting cancer treatments before?
Are you generally well enough, with important organs working properly?
Do you not have any active serious infections or uncontrolled long-term illnesses?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, the exact details will depend on which phase of the study you are in. Initially, the focus will be on carefully giving you the study drugs, durvalumab and tremelimumab, likely through an IV drip. You will have regular hospital visits for these treatments and for close monitoring including blood tests, scans, and physical examinations. These visits will help your doctors track how you are responding to the medication and manage any side effects.

During the study, doctors will collect blood samples to understand how the drugs move through your body and how your immune system reacts to them. They will also assess your cancer regularly to see if it’s responding to the treatment. The total length of your participation can vary, but it will involve ongoing assessments and follow-up care as long as you are benefiting from the treatment and tolerating it well.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. For patients in this study, a potential benefit is the chance to receive new medication combinations that could help fight your cancer, especially if standard treatments haven't worked or aren't available. While it's not guaranteed, there's always a hope for improvement. However, there are also risks. The study drugs may cause side effects, some of which could be serious, as your immune system is being changed. Your medical team will closely monitor you for any side effects and will do their best to manage them. You have the right to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (19)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Research Site
Verified postcode
Baltimore, United States
Research Site
Verified postcode
Boston, United States
Research Site
Verified postcode
New Hyde Park, United States
Research Site
Verified postcode
Oklahoma City, United States
Research Site
Verified postcode
Charleston, United States
Research Site
Verified postcode
Lille, France
Research Site
Verified postcode
Marseille, France
Research Site
Verified postcode
Paris, France
Research Site
Verified postcode
Cologne, Germany
Research Site
Verified postcode
Genova, Italy
Research Site
Verified postcode
Milan, Italy
Research Site
Verified postcode
Rome, Italy

Common questions

What are durvalumab and tremelimumab?

These are special drugs called immunotherapies. They work by boosting your body's own defence system (immune system) to find and attack cancer cells.

Is this a new treatment?

Yes, this is an early-stage study, meaning it's the first time these drugs are being used together in children and teenagers for these types of cancer.

Will I definitely get better on this study?

We can't guarantee that. The main goal is to find a safe dose and see how the drugs affect the body. There's a chance it might help, but it's not certain.

What kind of cancers are being studied?

This study is for certain advanced solid tumours and some types of blood cancer (specific non-Hodgkin's lymphomas) in children and teenagers.

Can I stop being part of the study if I want to?

Yes, absolutely. You can decide to leave the study at any time, and it won't change your medical care in the future.