Active not recruitingPHASE2INTERVENTIONAL

Irinotecan Hydrochloride, Temozolomide, and Dinutuximab With or Without Eflornithine in Treating Patients With Relapsed or Refractory Neuroblastoma

This important study is for children and young people with neuroblastoma that has returned after treatment (relapsed) or isn't getting better with current medicines (refractory). Researchers are testing if adding a drug called eflornithine makes existing treatments more effective. The main treatments being studied are a combination of chemotherapy drugs (irinotecan and temozolomide) and an immunotherapy drug (dinutuximab). Eflornithine works by blocking chemicals that help cancer cells grow. The study aims to find out if this new combination leads to better results for patients and fewer side effects. We hope this research will help improve future treatments for neuroblastoma.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Children's Oncology Group

Enrolment target

Start

08 Jul 2019

Estimated completion

31 Mar 2029

High Risk Neuroblastoma Recurrent Neuroblastoma Refractory Neuroblastoma

Results

Results from this study

Posted January 2026

Results have been published for this study.

Primary outcome

Response Rate

Responders are defined as patients who achieve a \>= minor response (MR) per the International Neuroblastoma Response Criteria (INRC) as their best overall response by the end of 6 cycles. The response rate to treatment will be calculated among all eligible patients, including placement of a 95% confidence interval on the response rate.

Full results on the registry

What is this study about?

This clinical trial is designed for children and young people who have a type of cancer called neuroblastoma. Specifically, it's for those where the cancer has either returned after being treated (this is called relapsed neuroblastoma) or hasn't responded well to previous treatments (known as refractory neuroblastoma). Finding effective ways to treat these situations is really important, and this study aims to explore new possibilities.

The trial is looking at a combination of medicines. There are two main chemotherapy drugs called irinotecan and temozolomide, which work by stopping cancer cells from growing and spreading. There's also an immunotherapy drug called dinutuximab, which helps your body's own immune system fight the cancer. On top of these, the study is investigating a drug called eflornithine. Eflornithine works by blocking certain chemicals that cancer cells need to grow, so the hope is that adding it to the other medicines will make the treatment more powerful.

The main goal is to see if combining eflornithine with the other medicines leads to a better response from the cancer compared to just using the chemotherapy and immunotherapy drugs alone. Researchers will also be carefully watching for any side effects and how long patients stay well. The results from this study could help doctors decide on the best treatments for neuroblastoma in the future, giving hope to many families.

Key takeaways

This study investigates new treatments for neuroblastoma that has returned or is difficult to treat.
It tests if adding eflornithine to current medicines (chemo and immunotherapy) makes them more effective.
The study aims to improve treatment outcomes and manage side effects.
Participants will be randomly assigned to receive eflornithine or not.
Regular hospital visits, treatments, and tests will be part of taking part.
The results could help shape future treatments for neuroblastoma.

Who may be eligible?

To join this study, you would need to have been diagnosed with neuroblastoma, and it must be either a high-risk type that has come back after treatment, or hasn't responded to previous treatments.

Doctors would need to confirm your diagnosis using special tests, and your cancer must show up on scans like MRI, CT, or MIBG. It's important that you are generally well enough to take part and that your body's organs (like your heart, liver, and kidneys) are working properly. There are also specific rules about how much time must have passed since your last cancer treatment.

This study is for children and young people from age one upwards. The doctors on the study will carefully check all your medical information to make sure this trial is the right and safest option 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.

Do I have a confirmed diagnosis of high-risk neuroblastoma?
Has my neuroblastoma returned after treatment, or is it not responding?
Am I generally well enough to take part in a clinical trial?
Are my scans (like MRI, CT, or MIBG) showing the cancer?
Is my age between 1 year old and any age older?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would be randomly assigned to one of two groups: one group will receive the standard treatment combination (irinotecan, temozolomide, and dinutuximab) and the other group will receive the same combination plus the added drug eflornithine. This happens over treatment cycles, with each cycle lasting 21 days.

Throughout the study, you'll need to take some medicines by mouth, and others will be given through a drip (IV) into your vein. You'll have regular hospital visits for these treatments and check-ups. These visits will involve blood tests, scans (like MRI, CT, or PET), and sometimes bone marrow samples to see how the treatment is working. You might also be asked about any pain you experience. The treatment takes place over up to 6 cycles, which means it could last for several months. After the treatment period, there will be follow-up appointments to monitor your health. The medical team will explain all the details, including how long each visit might take and the total duration you'll be involved in the study.

Potential risks and benefits

Taking part in this study could potentially offer benefits, such as receiving a new treatment combination that might be more effective for your neuroblastoma than current options, with close medical supervision. However, like all medical treatments, there are potential risks, including side effects from the chemotherapy, immunotherapy, and eflornithine. Doctors will inform you about the known side effects, which can vary. Your safety and well-being are a top priority, and you'll be monitored closely for any issues. It's 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 (145)

Children's Hospital of Alabama
Verified postcode
Birmingham, United States
Arkansas Children's Hospital
Verified postcode
Little Rock, United States
Kaiser Permanente Downey Medical Center
Verified postcode
Downey, United States
Miller Children's and Women's Hospital Long Beach
Verified postcode
Long Beach, United States
Children's Hospital Los Angeles
Verified postcode
Los Angeles, United States
Cedars Sinai Medical Center
Verified postcode
Los Angeles, United States
Valley Children's Hospital
Verified postcode
Madera, United States
Kaiser Permanente-Oakland
Verified postcode
Oakland, United States
Children's Hospital of Orange County
Verified postcode
Orange, United States
Lucile Packard Children's Hospital Stanford University
Verified postcode
Palo Alto, United States
University of California Davis Comprehensive Cancer Center
Verified postcode
Sacramento, United States
Rady Children's Hospital - San Diego
Verified postcode
San Diego, United States

Common questions

What is neuroblastoma?

Neuroblastoma is a rare type of cancer that mostly affects young children. It develops from special nerve cells called neuroblasts.

Why is this study important?

This study aims to find better treatments for neuroblastoma that has come back or is hard to treat, with the hope of improving patient outcomes.

What is a 'relapsed' or 'refractory' cancer?

Relapsed means the cancer has returned after treatment. Refractory means the cancer isn't responding well to current treatments.

Will I definitely get the new drug?

Patients are randomly put into one of two groups, so you might or might not receive the eflornithine in addition to the standard drugs.

What are the main treatments involved?

The main treatments are chemotherapy (irinotecan, temozolomide) and immunotherapy (dinutuximab), with some patients also getting eflornithine.