Active not recruitingPHASE1, PHASE2INTERVENTIONAL

Safety and Dose Finding Study of Neratinib in Children and Young Adults With Cancer That Has Returned or Not Responded to Treatment

This study is for children and young adults aged 3 to 21 who have cancer that has returned or hasn't responded to previous treatments, including solid tumours, brain tumours, lymphomas, and leukaemias. The research is testing a drug called neratinib. The main aims are to find out the safest dose of neratinib for these patients and to understand its effects – both good and bad – on their health and the cancer itself. Participants will be closely monitored. This is an early-stage study, meaning it's one of the first times this drug is being tested in this specific group of patients to learn more about how it works and whether it could be a helpful treatment option in the future.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Memorial Sloan Kettering Cancer Center

Enrolment target

Start

01 Oct 2016

Estimated completion

01 Oct 2026

Solid Tumor Central Nervous System Tumor Lymphoma Leukemia

What is this study about?

This study is looking at a new medicine called neratinib for children and young adults aged 3 to 21 who are dealing with cancer. Specifically, it's for those whose cancer has either come back after treatment or hasn't responded to the treatments they've tried so far. This includes different types of cancer like solid tumours (growths in organs), brain tumours, lymphomas (cancers of the immune system), and leukaemias (cancers of the blood). The purpose of this research is to carefully check how safe neratinib is and to find the best dose to give. It’s important to understand both the helpful effects the drug might have on the cancer and any side effects it could cause.

Because this is an early-stage study (called Phase 1 and Phase 2), it means that researchers are still learning a lot about neratinib in this specific group of patients. They want to see how the body handles the medicine, if it can shrink or stop the cancer from growing, and generally how patients feel while taking it. The information gathered from this study will help doctors decide if neratinib could be a useful treatment option for more children and young adults with these types of cancer in the future.

Finding new treatments is a vital part of improving care, especially when current treatments are no longer working. By taking part, patients help scientists learn more about new drugs and contribute to medical knowledge that could benefit many others. Participants will be looked after very carefully throughout the study, with regular check-ups and monitoring.

Key takeaways

Tests a new drug (neratinib) for children and young adults with specific cancers.
Aims to find a safe dose and understand its effects.
For cancers that have returned or not responded to previous treatments.
Participants are closely monitored throughout the study.
Participation is voluntary, and you can withdraw at any time.
Could help find new treatment options for cancer in young people.

Who may be eligible?

To be considered for this study, you must be a child or young adult between 3 and 21 years old. Your cancer must have been confirmed by doctors and include types such as solid tumours (which can be in many parts of the body), brain tumours, or lymphomas.

It's important that your cancer has either come back after previous treatments or hasn't responded well to them, and there are no other standard treatments that are known to be effective for you at this point. You must have already tried at least one other treatment. Doctors need to be able to measure your cancer to see if the treatment is working, either through scans for solid tumours or specific checks for brain tumours. You also need to be well enough to take part, based on certain health scores.

There are also some specific health checks, such as your heart function, that need to be within a healthy range. If you are a female who could become pregnant, you would need a negative pregnancy test and agree to use effective contraception throughout the study. Male participants must also use contraception. You would also need to be able to swallow tablets or have a feeding tube. All participants must have recovered from any serious side effects of previous treatments.

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 between 3 and 21 years old?
Has your cancer come back or not responded to past treatments?
Have you already tried at least one other cancer treatment?
Are your doctors able to measure your cancer (e.g., with scans)?
Are you generally well enough to take part and able to swallow tablets?
Do you understand that previous treatment effects must have mostly cleared up?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be taking a tablet called neratinib. The dose might be adjusted to find the safest and most effective level. You'll have regular appointments with the study team, which will involve check-ups, blood tests, and scans to see how your body is reacting to the medicine and if the cancer is changing. These checks are important to monitor your health and the drug's effects.

Before you start, and throughout the study, doctors will collect tissue samples from your cancer (if available) to learn more about it. You'll need to be able to swallow tablets, or if you have a feeding tube, the medicine can be given through that. The study will continue as long as the treatment is helping you and you're not experiencing difficult side effects. The total length of your participation will depend on how you respond to the treatment, but you will be monitored closely throughout.

Potential risks and benefits

Taking part in a study like this might offer the chance to try a new medicine when other treatments haven't worked, which could potentially help control your cancer. Researchers will learn valuable information that could benefit future patients. However, like all medicines, neratinib can have side effects, which could be mild or more serious. The study team will carefully monitor you for these and provide support. It's important to remember that there's no guarantee the treatment will work for you personally. You are entirely free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (9)

Phoenix Children'S Hospital
Verified postcode
Phoenix, United States
Arkansas Children's Hospital
Verified postcode
Little Rock, United States
Stanford University School of Medicine and Stanford Cancer Institute
Verified postcode
Palo Alto, United States
Arnold Palmer Hospital for Children
Verified postcode
Orlando, United States
Memorial Sloan Kettering Cancer Center
Verified postcode
New York, United States
Pennsylvania State Hershey Children's Hospital
Verified postcode
Hershey, United States
University of Texas
Verified postcode
San Antonio, United States
Huntsman Cancer Institue
Verified postcode
Salt Lake City, United States
Alberta Children'S Hospital
Verified postcode
Calgary, Canada

Common questions

What is neratinib?

Neratinib is a new drug being tested in this study to see if it can help children and young adults with certain types of cancer.

What types of cancer is this study for?

This study is for solid tumours, brain tumours, lymphomas, and leukaemias that have returned or haven't responded to previous treatments.

How old do I need to be to join?

You need to be between 3 and 21 years old to take part in this study.

What if I decide I don't want to be in the study anymore?

You can leave the study at any time, for any reason, without it affecting your medical care.

Will I have to swallow pills?

Yes, you'll need to be able to swallow tablets, or if you have a feeding tube, the medicine can be given that way.