Erydex trial FAQs

Dr William Whitehouse is Clinical Associate Professor and Consultant Paediatric Neurologist and part of the A-T specialist team in Nottingham. Here he answers some questions about the planned clinical trial of the Erydex System.

 

Is the trial going to take place and if so, when will it start?

The short answer is yes, it is going to happen and we are getting closer, but are not quite there yet. The trial, which is now being called ‘ATTesT’ is currently being “set up” in centres several countries across the world, including here at Nottingham.

The company running the trial, EryDel, have twice visited our facilities for the trial at the Queen’s Medical Centre, Nottingham and were very impressed with them.  However, a clinical trial like this is a very complex project and there are many issues to be organised.

The Medicines and Healthcare Products Regulatory Authority (MHRA), and the Research Ethics Committee have approved the study for the UK and we are currently finalising the costings for all the components needed including nurse and doctor time, tests, etc. The equipment is already on site in Nottingham and ready to go.

As soon as the costings are agreed we will advertise for a clinical research fellow to run the study in Nottingham. They will be supported by research nurses who will help with the patient assessments and paperwork, paediatric doctors (myself and Dr Katherine Martin in particular), our paediatric physiotherapists, and our haemodialysis nurses who will work the EryDex machine. As the appointment of the research fellow will take a few months, it is still not certain exactly when we will be ready for the 1st participant!

 

What will the trial involve?

This is a very intense and complicated trial. The treatment involves taking 50 ml of blood, putting it through the EryDel machine and then re-infusing it back into the participant. The EryDel machine loads the red cells in the blood with dexamethasone, a steroid, which once back in the body slowly leaks out at a constant low dose. This constant low dose of dexamethasone is what we hope will slow the disease progression, without causing side effects.  

These transfusions will happen once a month, in the Children’s Dialysis Unit at Queen’s Medical Centre Nottingham. Participants can stay in the trial for 6 months or 12 months.

In order to monitor the effects of the treatment there will also need to be detailed assessments, involving videoed neurological and general examinations, questionnaires, blood tests, ECGs, bone scans, and hormone tests. These will occur at an initial ”screening”, before the transfusions start and then on some transfusion days, about every third month.

This process will be quite tiring for the young people and their families, and some families who live further away may need to stay 1 or 2 nights in a local hotel, for these monthly visits.

 

Will everybody get the dexamethasone treatment?

No, not everyone will get the actual drug during the course of the trial. During the first six months, about 2/3 participants will get the dexamethasone, while 1/3 will get placebo (an inactive control treatment). Over the second 6 months, some of those on the placebo will move to the active treatment, so that by the end of the 12 months only one in nine participants is on placebo. We cannot choose who gets what, and neither the child or young person, their family, the research nurses, nor the doctors involved will know.

 

What happens after the trial?

If the treatment seems to be working, when the trial finishes, EryDel will continue to supply the active dexamethasone treatment to any participants that wish to continue, including those that were on placebo right through the trial. However, eventually they will expect the NHS to fund the treatment long term and that cannot been agreed until we know the results.

If the treatment does not work, then of course we will continue to support the search for effective treatments and hope to run other international trials in the UK in future.

 

Who will be able to participate?

We are aiming for a minimum of 12 children and young people to complete the first 6 months, but hope to be able to include up to 16. We won’t be able to have more than 16 patients in the trial at any one time.

All the participants will have A-T, with a clinical diagnosis confirmed by DNA analysis (this has already been done for everyone with A-T in the UK). We think one way that the dexamethasone might work will be to allow the body to make “mini-ATM”, so converting someone with “classic A-T” to a milder type, like “mild variant A-T”.

To be safe: all children enrolled in the trial must weigh at least 15 kg, and be at least 6 years old. All the participants must be in general good health at the time. The screening blood test results have to be normal or near normal.  

Walking is an important component of the scale used to measure the effectiveness of the treatment. So all the participants must be able to walk a bit with either no support or with just an arm held for balance.

Finally they and their families must be able to manage the tiresome assessments and frequent visits for the monthly transfusions.

 

What if we want to volunteer for the trial?

We are not open for recruitment yet, but if you are interested and would like more information about the trial, please contact Kay or Ann at the A-T Society. They will make a list so we can send you more information when we start.

If we have too many patients wanting to volunteer, we will have to choose those who are fittest first, as they are more likely to get some benefit, and less likely to get side effects. It also makes sense to pick those who might benefit from the “mini-ATM”.

Remember, we do not know if the treatment works, which is why we need this intensive randomised controlled trial. While we all hope that it will slow the disease, we cannot be sure at this stage what will happen. And it is possible there might be unwanted or unexpected side effects.

 

Picture of Dr William Whitehouse

Dr William Whitehouse

 

 

 

 

Queen's Medical Centre Nottingham

 

Queen's Medical Centre where the trial will take place