Summary results

What was the purpose of the study?

The purpose of the study was to look at how effective the use of Tocilizumab in combination with methotrexate is for the treatment of JIA- associated uveitis.

Why was I/my child invited?

You/your child were asked to participate because you/your child had persistently active JIA- associated uveitis. You/your child had also been chosen as you/they were taking methotrexate without an improvement in your/their uveitis since starting it and had also not responded to treatments with other medicines. A total of 22 participants aged between 2 - 18 years with JIA associated uveitis throughout the UK participated in the study.

Which drug/ drugs were being tested?

The study involved the use of Tocilizumab in combination with Methotrexate.

Methotrexate is a powerful treatment used to treat young people and children who suffer from JIA- associated uveitis along with other rheumatic conditions. It helps to reduce the activity of the cells which cause inflammation and is used when anti- inflammatory drugs are not enough or in those with moderate or more severe disease. Even when treated with methotrexate, a number of children still have inflammation of the eye and therefore methotrexate may not work on its own.

In children and young people with inflammatory diseases a protein called Tumour Necrosis factor (TNF) is overproduced in the body, causing inflammation and damage. Anti-TNF drugs block its action and so can reduce this inflammation. Sometimes a person’s uveitis may not respond to the anti-TNF drug and so a different type of medicine is being investigated for the APTITUDE study to see if it can help reduce the uveitis, this drug is called Tocilizumab.

Tocilizumab, like anti-TNF drugs, is a type of drug known as a biologic. However it works differently to anti-TNF drugs because it binds to interleukin-6 receptors in the body which helps to reduce the inflammatory effects caused by the interleukin-6 protein.

What happens to me/my child now the study has stopped?

After you/your child finished taking part in the study, your doctor will have explained what happens next. Your doctor will have treated you/your child according to normal clinical practice.

Are there any side effects that we should report now the study has ended?

As Tocilizumab is a relatively new drug, the long-term side effects are not fully understood. During the study we closely monitored any side effects that may be experienced by you/your child and now that the study has finished your doctor has been asked to continue to report any side effects they feel that may be related to Tocilizumab to the LCTC study team. If you/your child are concerned at any time please contact the doctor or nurse who looked after you during the study.

Will my taking part in the study be kept confidential?

Yes. We will follow ethical and regulatory requirements and all information which is collected about you/your child during the course of the research will be kept strictly confidential. All data collected during the study will be kept for a maximum of 15 years after the study has ended and during this time all of you/your child’s information will be kept confidential.

What were the results of the study and how will they be made available?

In the APTITUDE study participants received Tocilizumab injections every 2 or 3 weeks in combination with Methotrexate for up to 6 months. After 3 months of treatment participants were reviewed to see if they were getting better with treatment and if they were then they would continue taking Tocilizumab for another 3 months. If participants were not getting better after 3 months then they would not continue taking Tocilizumab and their Doctor would start other treatments. We said that a Participant was getting better if they had an improvement in their SUN score. The Standardization of Uveitis Nomenclature (SUN) score shows how much the eye is inflamed. The score goes from 0 to 4+ with 0 being no inflammation and 4+ being severe inflammation. If a participant went down the scale 2 points after 3 months, then we would say this Participant is getting better with treatment. For example, if a participant had a score of 4+ when they started taking part in the study and then had a score of 2+ at 3 months then we would say this participant is getting better with treatment and should continue to take Tocilizumab for another 3 months. If the participant went down the scale by 1 point, then we would say this participant has partially gotten better but should stop taking Tocilizumab and go on to other treatments. If a participant did not go down the scale or went up the scale then we would say this participant is not getting better and should stop taking Tocilizumab and go on to other treatments. All participants were followed up at an appointment 3 months after they stopped taking Tocilizumab.

In this study we wanted to see 8 participants out of 22 get better after 3 months. If this happened, then we would recommend that further studies should be done. We saw 7 participants out of 22 get better after 3 months and 3 participants get partially better. So we didn’t quite make it to our target of 8 participants but we still feel this result tells us a lot. Participants entering this study had not got better with other drugs and we have shown that almost half of the participants got better with Tocilizumab and Methotrexate. We do not know yet if new studies in Tocilizumab will take place but we know this study will help other people decide if other studies should take place and also help Doctors make treatment choices when dealing with participants with JIA-Uveitis.

We found that any side effects that participants had during the study were expected in participants taking Tocilizumab. This means that we have added to the knowledge about how safe Tocilizumab is. None of the participants had any serious side effects from taking Tocilizumab.

Have the results of the study been seen by anyone else

Roche, a pharmaceutical company, provided the Tocilizumab that was used in this study and has seen the results from the study.

We have also shown the results to Rheumatology Doctors at a conference meeting.

Will I be given any results about me as an individual?

We will not publish any individual results, if you would like to know more about your results during the study please speak to your study nurse or doctor.

Invitation to take part in participant involvement

When research studies like this one are being set up we like to get input from participants into the design of the study and what impact the study may have on participants. We also like to have participant involvement throughout the study.

Examples of participant involvement include: - help make sure researchers ask the right questions - help make sure the research design is appropriate - help make sure information about the research is easy to understand - help make sure reported results are clear and easy to understand

If this is something you might be interested in doing, please visit the following websites which provide general information about participant and public involvement in research including how you can get involved:

The National Institute for Health Research website: https://www.nihr.ac.uk/patients-carers-and-the-public/

INVOLVE: http://www.invo.org.uk/

The Liverpool Clinical Trial Centre, University of Liverpool is keen to ensure participants and the public have the opportunity to be actively involved in research

 




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