Active not recruitingPHASE1INTERVENTIONAL

Phase I Study of 131-I mIBG Followed by Nivolumab & Dinutuximab Beta Antibodies in Children With Relapsed/Refractory Neuroblastoma

This research is investigating a new treatment approach for children and young people with neuroblastoma, a type of cancer that starts in nerve cells and is the most common solid tumour outside the brain in children. It focuses on patients whose cancer has returned or hasn't responded well to previous treatments. The study combines existing treatments with two newer medications, Nivolumab and Dinutuximab Beta, which are types of immunotherapy that help the body's immune system fight cancer. Researchers want to check if this new combination is safe and how well it works. This study runs in hospitals in the UK, Germany, and the USA, aiming to find more effective options for children with high-risk neuroblastoma.

At a glance

Status

Active not recruiting

Phase

PHASE1

Sponsor

University Hospital Southampton NHS Foundation Trust

Enrolment target

Start

24 May 2018

Estimated completion

30 Sep 2026

Neuroblastoma

What is this study about?

Neuroblastoma is a type of cancer that mostly affects young children. While treatments have improved over the years, it can still be very challenging to treat, especially if it returns or doesn't respond well to initial therapies. This study is an important step in trying to find better ways to help children facing these difficulties.

The research combines a standard treatment for neuroblastoma, an antibody called Dinutuximab Beta (also known as Ch14.18/CHO), with two newer medicines: 131-I mIBG and Nivolumab, which is an anti-PD1 antibody. 131-I mIBG uses a radioactive substance to target and kill cancer cells, while Nivolumab is an immunotherapy that works by 'unblocking' the immune system so it can better recognise and attack cancer. The main goal is to see if this new combination of treatments is safe for children and to gather early information on whether it helps in fighting neuroblastoma.

This study uses a clever design called an 'adaptive study'. This means researchers can adjust parts of the study as they go along, based on new information they gather, without affecting the study's reliability. They will start by testing the safety of Nivolumab with 131-I mIBG, then add Dinutuximab Beta, and finally test all three at full doses. This careful step-by-step approach helps ensure patient safety while finding the most effective treatment levels.

Key takeaways

This study is testing new treatments for children with neuroblastoma.
It aims to find safer and more effective ways to treat difficult cases.
It combines existing treatments with newer immunotherapy drugs.
Safety is the main focus in the early stages of the study.
The study involves hospitals in the UK, Germany, and USA.
It's an important step in finding better care for children with neuroblastoma.

Who may be eligible?

This study is for children and young people aged 1 year and older who have specific types of neuroblastoma that have either come back or have not responded well to previous treatments. Importantly, their cancer must show up on a special type of scan called an MIBG scan.

To be considered, patients must have recovered well from any previous strong chemotherapy or stem cell treatments, with at least 3 months passed. For other treatments like immunotherapy or certain antibody therapies, there are also specific waiting periods. Patients also need to be generally well enough to take part, meaning they have a reasonable level of activity and energy.

There are also specific medical checks for the heart, kidneys, liver, lungs, and pancreas to make sure these organs are working well. Patients with brain involvement previously treated are sometimes eligible, but there are certain brain abnormalities that would prevent participation. Essentially, the medical team needs to ensure that taking part in the study would be as safe as possible for the child.

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.

Is your child aged 1 year or older?
Has your child been diagnosed with neuroblastoma that has relapsed or is difficult to treat?
Does their neuroblastoma show up on an MIBG scan?
Have at least 3 months passed since their last strong chemotherapy or stem cell treatment?
Is your child generally well enough to participate, with good organ function?
Do you understand that there are other specific medical requirements that need to be met?

Answer every question to see your result.

What does participation involve?

If your child joins this study, they will receive a combination of medicines as part of their treatment plan. The study is divided into three groups, with patients moving through them as the researchers gather safety information. The full duration of treatment and exact number of visits will depend on which group your child is in and how they respond, but the study itself is planned to last for about 2 years in total. During this time, there will be regular hospital appointments, blood tests, scans, and other medical checks to monitor your child's health and how they are responding to the treatment. These assessments are crucial for understanding the safety and effectiveness of the new drug combination. You will be kept fully informed about the schedule and what to expect at each stage.

Potential risks and benefits

Participating in a clinical trial offers potential benefits, such as access to promising new treatments before they are widely available. While the specific benefits for your child cannot be guaranteed, the hope is that this new combination of medicines may be more effective against their neuroblastoma. However, like all medical treatments, there are potential risks and side effects associated with the medicines used in this study, including those from the existing standard treatments and the newer investigational drugs. The research team will carefully monitor your child for any side effects and manage them as best they can. It is very important to remember that joining a clinical trial is always voluntary, and you have the right to withdraw your child from the study at any time, for any reason, without it affecting their ongoing medical care.

Locations (3)

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

University of Wisconsin Carbone Cancer Center; UW Hospital and Clinics; American Family Children's Hospital
Verified postcode
Madison, United States
University Hospital Southampton NHS Foundation Trust
Verified postcode
Southampton, United Kingdom
University College London Hospital
Verified postcode
London, United Kingdom

Common questions

What is neuroblastoma?

Neuroblastoma is a type of cancer that starts in nerve cells, usually in young children. It's the most common solid tumour found outside the brain in children.

What does 'relapsed/refractory' mean?

This means the cancer has come back after previous treatment (relapsed) or hasn't responded well to previous treatments (refractory).

What is immunotherapy?

Immunotherapy is a type of treatment that uses the body's own immune system to fight cancer. It helps the immune system to better recognise and attack cancer cells.

Where is this study taking place?

This study is being run in hospitals in the UK, Germany, and the USA.

Can my child definitely join this study?

Not necessarily. There are strict rules about who can take part to ensure safety and that the study results are clear. Your child's doctor will need to check if they meet all the requirements.