Understanding the KD-CAAP Trial
Clinical trials and the information shared about them often use a lot of jargon and technical information which can be difficult to understand. We have tried to explain our KD-CAAP trial in a way that is helpful and doesn’t use too much jargon. We hope we’ve been successful! But get in touch if there’s anything we need to do to explain things better.
For doctors and those with medical knowledge, you can find more information on trial details including information on types of participants, interventions, hypothesis, outcome measures and more – here
Explaining the technical jargon
For families considering taking part in the trial, here’s some jargon busters:
Types of participants: That’s the range of people (in our trial, children) who will be invited to take part in the trial. For KD-CAAP that’s infants and children affected by Kawasaki Disease when they are first ill, being treated at one of the hospitals taking part in the trial and who are aged between 30 days (infants from one month old) and 15 years old. We know that a lot of children, young people and adults live with the consequences of Kawasaki Disease having had it as a child. This trial isn’t for those people, but for children who are first being affected right now.
There are other criteria too which might affect whether a child with Kawasaki Disease can be invited to join the trial. One example of a criteria which would exclude someone from being able to join the trial would be if they already have a heart condition. This is because it would be hard to decide whether that existing condition affected their outcome (see below) or if that was as a result of the treatment they had. If you have been invited to join the trial, your doctors will have discussed this with you in more detail.
Interventions: These are the things we plan to do, to try and make changes happen. In our KD-CAAP clinical trial, the interventions are the treatments we give the children who take part in the trial.
Our KD-CAAP trial will select children at random to receive two different treatments (interventions):
Half will receive the normal treatment:
IVIG + aspirin
Half will receive standard treatment and one additional medicine, a steroid – so they will have:
IVIG + aspirin + corticosteroid
Hypothesis: This is the point for which we gather evidence to prove, or disprove. Doctors have a hypothesis – or idea – that by including an additional medicine in the initial treatment for Kawasaki Disease we might (but we might not!) reduce the number of children who have serious heart damage. The “hypothesis” is the central idea around which the trial “interventions” are designed.
Outcome measures: These are linked directly to the things we think might be achieved as a result of the trial – and how we measure them. For our trial, our “hypothesis” or idea is that we might see a reduction in serious heart damage (coronary artery aneurysms) in the group of children who receive the extra medicine (steroids). The purpose of the trial is to see if this idea is correct. Our outcome measures will therefore include looking at any difference between those children given the standard treatment, and those having the additional medicine – and of those, how many are affected with serious heart damage.
There will be a number of other outcome measures too. But this is the main one.
Why don’t all children get the treatment with Steroids if you think this might help?
That’s a great question. As parents and carers – and doctors – we want to be able to give our children the best possible treatment. Our trial is investigating whether adding steroids to the usual treatment for Kawasaki Disease will make a difference and reduce the serious heart damage. At the moment, we don’t know. We think it might but we don’t have the evidence we need to show this. That is why this trial is so important. Children getting the standard treatment (which you’d get if you weren’t taking part in the trial) who are part of our trial will help us understand more – and compare whether adding in the new treatment makes a difference. Without this group, we couldn’t properly understand whether the treatment including steroids was better than the standard treatment.
Why is this trial important?
Kawasaki Disease is the leading cause of acquired heart disease in U.K. children – and in children in the western world.
Treatments haven’t changed for several decades – and the rate of heart damage in children, even with the standard treatment, is just too high.
A number of studies conducted in European countries, Russia, and the United States showed consistently high rates of heart complications, despite treatment. Coronary artery aneurysm rates ranged from 16% of children – to as high as 42%. In the UK and Ireland, a recent survey by the British Paediatrics Surveillance Unit (2013-2015) found that 19% of children with Kawasaki Disease still developed coronary artery aneurysms despite treatment; and, even more worryingly in that survey, 39% of patients under the age of 1 year developed serious heart damage. These high complication rates really show why there’s an urgent need to find better treatments for Kawasaki Disease, which help reduce the number of children who have life long heart disease because of Kawasaki Disease.
Corticosteroids (steroids) are an effective treatment for virtually all forms of vasculitis (inflammatory diseases), but they have not been used regularly as a treatment for Kawasaki Disease. Children at known risk of developing complications from Kawasaki Disease are sometimes treated with steroids as well as standard treatment, but this doesn’t happen often.
There have been studies and trials in a number of countries elsewhere in the world which suggest significant reduction in heart damage is possible for children who receive steroids as part of their initial treatment for Kawasaki Disease. But in the UK and Europe their use hasn’t been adopted by doctors – because there hasn’t been a UK or European study on whether or not this is an effective treatment for our populations.
So, there’s has been an ongoing debate amongst doctors about whether or not children should be treated with steroids at the onset of Kawasaki Disease, together with the standard treatment.
Our trial has been designed to provide the missing evidence about the effectiveness of adding in steroids alongside standard treatment. The trial will show whether or not steroids make a difference to outcomes and whether they can help reduce serious heart damage in children affected by Kawasaki Disease.
Who is managing the trial?
KD-CAAP is managed by the Medical Research Council (MRC) Clinical Trials Unit.
What is Societi’s role?
We are a co-investigator in the trial, and have responsibility for patient and public involvement throughout the trial period. We were involved in the trial planning and design – and involved families in the trial design process to help us shape it.
We’ll be raising awareness of the trial (and Kawasaki Disease!) throughout the project and we’ll be working to provide the information families need as they consider joining the trial – and if they do join, provide the information they need before, during and after their active involvement in the trial.
Who is funding and sponsoring KD-CAAP?
Innovative Medicines Initiative 2 Joint Undertaking (JU) that supports the conect4children (c4c) research consortium have provided funding for the trial.
The trial is sponsored by University College London (UCL).