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Activo, no reclutandoFASE1INTERVENCIONISTA

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

Neuroblastoma is a childhood cancer that forms in nerve cells. While treatments exist, it can be very difficult to cure if it comes back (relapses) or stops responding to treatment (refractory). This trial is testing a new approach by combining three treatments: a targeted radiotherapy called 131-I mIBG and two immunotherapy medicines, Nivolumab and Dinutuximab beta. Immunotherapies help the child's own immune system recognise and attack cancer cells. Because this is an early Phase 1 study, the main goal is to find out if giving these medicines together is safe and what doses are best tolerated. Doctors will carefully monitor how children cope with the treatment in small groups. The study takes place across specialist centres in the UK, Germany, and the USA, aiming to open up better, more effective treatment options for high-risk childhood cancer in the future.

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Estado
Activo, no reclutando
Fase
FASE1
Patrocinador
Fundación del Hospital Universitario de Southampton NHS
Objetivo de inscripción
44
Inicio
24 de mayo de 2018
Estimación de finalización
30 Sep 2026

¿De qué trata este estudio?

Neuroblastoma is a rare type of cancer that mostly affects babies and young children, developing from specialised nerve cells left behind from development. When neuroblastoma returns after treatment, or when standard therapies stop working, it can be very hard to treat successfully. Doctors and scientists are looking for smarter, more powerful ways to help the body fight back against this stubborn illness.

In this study, researchers want to see if combining three different cancer treatments is safe and helpful. The first is 131-I mIBG, a special type of radiation therapy that travels straight to neuroblastoma cells. The other two are immunotherapy drugs called Nivolumab and Dinutuximab beta. Rather than attacking cells directly with harsh chemicals, immunotherapy works by training and encouraging the child's own immune system to spot and destroy cancer cells.

Because this combination has not been used in this exact way before, this is a Phase 1 trial. This means the chief focus is on child safety, checking for side effects, and finding the best dose. Small groups of children will try the treatments step-by-step. If this new approach proves safe, it could pave the way for stronger, longer-lasting treatments for children facing difficult forms of neuroblastoma.

Puntos clave

  • Phase 1 trial testing a three-drug combination for children with hard-to-treat neuroblastoma.
  • Combines targeted radiation (131-I mIBG) with two immunotherapy drugs (Nivolumab and Dinutuximab beta).
  • Open to children aged 1 year and older whose cancer has relapsed or not responded to standard care.
  • Primary focus is checking the safety, side effects, and best doses of the combined medicines.
  • Taking part is completely voluntary and requires close monitoring in a specialist children's hospital.

¿Quién puede ser elegible?

This study is open to children aged 1 year and older who have high-risk neuroblastoma that has come back or has not improved with standard treatments. The child's cancer must also show up clearly on a specific scan called an mIBG scan, which shows that the targeted radiation therapy will be able to reach the tumours.

To safely take part, children must be well enough to carry out everyday activities and have vital organs that are working reasonably well. Doctors will check blood counts, kidney, liver, heart, and lung function. Children must also have had a break from previous intense treatments, such as stem cell transplants or other immunotherapies, to allow their bodies time to recover.

Children cannot join if the cancer has spread into the main tissue of the brain and is active on scans, or if their organs are not strong enough to handle the trial medicines safely. A doctor will carefully review all previous medical records to confirm whether a child is eligible.

¿Podría este estudio ser adecuado para ti?

Responde estas preguntas rápidas para ver si puedes ser elegible. Esto es solo una guía: el equipo de investigación toma la decisión final.

  1. The child is at least 1 year old.
  2. The child has high-risk neuroblastoma that has returned or did not respond to standard treatment.
  3. The tumour shows up clearly on recent mIBG scans.
  4. It has been at least 3 months since any high-dose chemotherapy or stem cell transplant.
  5. Heart, kidneys, liver, and lungs are working well enough for treatment.
  6. The child is well enough to be up and active for a good part of the day.
Responde cada pregunta para ver tu resultado.

¿En qué consiste la participación?

Taking part involves receiving treatment in a specialist hospital under close medical care. Before beginning, children will undergo baseline checks, including blood tests, heart scans, breathing tests, and imaging scans. A central line (a soft tube placed in a vein) will be needed to give the medicines smoothly and take blood samples without repeated needle sticks.

Children will receive the 131-I mIBG radiation therapy first, followed by the immunotherapy drugs given through a drip. Because the trial uses an adaptive approach, treatment is divided into small groups to check safety at each stage. Families can expect frequent hospital visits, regular blood tests, and routine scans over at least 12 weeks of active treatment. The research team will track each child's health closely throughout the process, and total follow-up will continue for several months to monitor recovery and long-term wellbeing.

Riesgos y beneficios potenciales

The trial drugs can cause side effects, including low blood cell counts, tiredness, fever, allergic reactions, inflammation, or pain. The care team will watch for these closely and treat them promptly. While there is no guarantee that this treatment will shrink the cancer, it may help, and taking part helps doctors discover better therapies for future children. Joining the study is entirely voluntary; you can withdraw your child at any time without affecting their regular NHS care.

Locations (3)

  • University of Wisconsin Carbone Cancer Center; UW Hospital and Clinics; American Family Children's Hospital
    Código postal verificado
    Madison, United States
  • Fundación del Hospital Universitario de Southampton NHS
    Código postal verificado
    Southampton, Reino Unido
  • Hospital Universitario College de Londres
    Código postal verificado
    Londres, Reino Unido

Preguntas comunes

What is the main goal of this trial?

The main goal is to find out if giving 131-I mIBG together with Nivolumab and Dinutuximab beta is safe, and to discover the most suitable doses for children.

Does my child have to stay in hospital?

Yes, some parts of the treatment—particularly the 131-I mIBG radiation therapy and early drug doses—will require stays in a specialist hospital ward.

Is this a cure for neuroblastoma?

Because this is an early Phase 1 trial, doctors do not yet know how well it works. The aim is to test safety and see if it can help control the disease.

What happens if the side effects are too strong?

The medical team will treat side effects straight away. If needed, doses can be lowered, paused, or stopped completely to protect your child's health.

Can we change our minds after starting?

Yes. You can leave the study at any point. Your child will continue to receive the best available supportive care from their medical team.

Cómo obtener más información

Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.

Discusión

Discusión comunitaria

Impulsado por nuestro foro en community.patient.info. Por favor, sea respetuoso — esto no es un consejo médico.