A trial looking at two different types of intensive treatment for neuroblastoma that has spread (VERITAS)

Cancer type:

Children's cancers

Neuroblastoma

Status:

Results

Phase:

Phase 2

This trial compared high dose chemotherapy with 131-I-mIBG targeted radiotherapy and chemotherapy.

It was for children and young people with neuroblastoma that had:

spread to other parts of the body and
the first course of treatment hadn’t worked well

The trial was open for people to join between 2018 and 2022. The team reported the results in 2024.

Cancer Research UK supported this trial.

More about this trial

When this trial was done one of the first treatments (induction Open a glossary item ) for neuroblastoma that had spread was chemotherapy. This is to either get rid of, or reduce, the cancer spread. This doesn’t work so well in a small number of children and young people. We know from research that this means their neuroblastoma is more difficult to treat.

At the time of this trial, there was no one standard treatment Open a glossary item option for children and young people in this situation. In this trial, doctors compared two different types of intensive treatment to see how useful they were. These treatments were:

high dose thiotepa chemotherapy
topotecan chemotherapy with a type of molecular radiotherapy called 131-I-mIBG

Molecular radiotherapy is when a substance called mIBG is added to radioactive iodine (131-I). It is also called targeted radiotherapy. It’s used to deliver targeted radiation therapy. The mIBG is picked up by the cancer cells. The dose of radioactive iodine attached to the mIBG is high enough to kill the neuroblastoma cells. But generally, the treatment does not cause serious side effects to surrounding tissues.

In this trial, everyone had chemotherapy and a stem cell transplant Open a glossary item . They all had treatment in stages as usual. The treatment plan was:

induction chemotherapy (stage 1)
collecting stem cells (stage 2)
first intensive consolidation treatment (stage 3)
second consolidation treatment and stem cells back (stage 4)

The first intensive treatment part was randomised. This was stage 3 of the plan:

half the people taking part had 131-I-mIBG and topotecan
half the people taking part had high dose thiotepa

The main aims of this phase 2 trial were to find out:

how safe treatment is
if 131-I-mIBG and topotecan or high dose thiotepa works better as an intensive treatment
more about the side effects of intensive treatment

Summary of results

34 people took part. For the intensive randomised part of the trial:

18 had 131-I-mIBG and topotecan
16 had high dose thiotepa

The team had planned to recruit more people but the trial closed early. This was due to a number of factors including:

delays to the trial because of the COVID-19 pandemic
not finding enough people to join the trial
supply issues with 131-I-mIBG

The team had the results for 21 people. They looked at how well treatment worked.

For the 131-I-mIBG and topotecan group they found that:

no one’s cancer got worse
10 people had cancer that either went away completely, got a bit better or stayed the same

For the high dose thiotepa group they found that:

1 person’s cancer had come back or got worse
10 people had cancer that either went away completely, got a bit better or stayed the same

The team say the early results showed that it was possible to have the intensive treatments. Some early analyses of the results seemed to show that it might work better than treatment available in the past.

Side effects
Most people who took part in this trial had at least 1 side effect. Some side effects were mild, but others were more serious.

Serious side effects
Researchers can class a side effect as serious for a number of reasons, including if:

the person has to go to hospital because of it
it is particularly important for the specific treatment in the trial

The most common serious side effects for both groups were:

a drop in the number of white blood cells and a high temperature
blocked veins in the liver

Non serious side effects
The most common side effects for both groups were:

a drop in the number of white blood cells that could lead to an increased risk of infection
a drop in the number of white blood cells called neutrophils and a high temperature
tummy pain
blocked veins in the liver

The team say there weren’t enough people in the trial to work out which intensive treatment caused more side effects. Although they do say that most people were able to cope with the side effects that the intensive treatments caused.

Conclusion
This was a very small trial. The team found it was possible for people to have the intensive trial treatments and the side effects weren’t too bad. But it wasn’t possible to say for sure which intensive treatment worked better. This was because there weren’t enough people in the trial.

All clinical trial results add to our knowledge of cancer and how to treat it. And help to shape future research.

Further studies
When this summary was written, there was an intensive chemotherapy option being looked at in another clinical trial. This is the HR-NBL2 trial for high risk neuroblastoma.

The team also think that the results of a trial called MiNivAN could produce more information about future trials that include mIBG.

Where this information comes from
We have based this summary on information from the research team. As far as we are aware, the information they sent us has not been reviewed independently (peer reviewed Open a glossary item ) or published in a medical journal yet. The figures we quote above were provided by the research team. We have not analysed the data ourselves.