Creating Clinical Trials for Children

: Affecting around 200 children in the UK, Juvenile Localised Scleroderma is an incredibly rare condition. Developing new treatments is challenging due to small numbers of patients. Find out how SRUK and Alder Hey are tackling this!

Scleroderma itself is a rare autoimmune condition: and in children, juvenile localised scleroderma (JLS) is even rarer. Typically it affects just 221 children in the UK, with very serious implications for them and their families. SRUK has funded two projects based at Alder Hey Children’s Hospital, led by Dr Clare Pain which have potential to help with facilitate new treatments for these children.

Dr Clare Pain, Consultant Paediatric Rheumatologist at Alder Hey Children’s Hospital is a leading clinician working to research scleroderma in children. This incredibly rare autoimmune condition has a significant impact for both the children who have it, and their families. As part of her role in championing for research in this area, Dr Pain is chair of the UK paediatric scleroderma Topic Specific Group. This is an open group of doctors, allied health professionals and parents who are continually aiming to facilitate research into juvenile scleroderma both in the UK and internationally through links with other groups in Europe and North America. Research into scleroderma in adults has shown just how key collaboration can be, therefore ensuring the same connectivity in researching juvenile scleroderma is crucial. SRUK have long recognised this, and to that end in 2016,we brought together the paediatric rheumatology team from Alder Hey and Great Ormond Street, with the families of children with juvenile scleroderma, for a Family Day, to facilitate the sharing of information and experiences.

The Family Day involved a session where children and their families were asked about what areas of research they thought was the most important to juvenile scleroderma. The main priorities identified were:

  • Better treatment options for children (including drugs with fewer side effects)
  • The need for a better understanding about the long-term outcomes of treatment
  • An increased understanding how to assess juvenile localised scleroderma.

These findings have also been identified by the aforementioned UK paediatric scleroderma Topic Specific Group who are working towards the development of clinical trials which will facilitate the development of better treatments for juvenile scleroderma.

In response to the clear need highlighted through both the Family day and the Topic Specific Group,  SRUK funded a research project led by Dr Clare Pain, examining an alternative treatment for juvenile localised scleroderma, which was initiated in 2017.

Currently, the only drug available to treat JLS is Methotrexate; typically used in chemotherapy for treatment of cancer. It acts by suppressing the immune system which can result in toxic side effects that impair quality of life. In addition to this, when tested in a clinical trial 1 in 3 children did not response to the treatment – highlighting just how important an alternative is. One such alternative treatment, Mycophenolate Mofetil works in a similar way, however supresses the immune system to a lesser extent which reduces how sever the side effects can be whilst potentially having the same efficacy as methotrexate in treating JLS.

As the condition is so rare, a traditional clinical trial to test the effects of these two drugs in comparison to each other will not be feasible, as these require large numbers of participants. In order to speed up progress and bring a potentially beneficial treatment to children with JLS sooner, SRUK  funded the setup of a critical expert meeting and panel, were responsible for  designing a novel clinical trial that does not rely on large numbers. A consensus amongst clinical experts and leaders in the design of this novel clinical trial is essential, and this meeting will consolidate expert opinion, thus forming the basis of a future trial.

Clinicians from across the globe participated in the meeting to offer their expertise on how a future clinical trial should be shaped and who should be included within the trial. This brings a new treatment for juvenile localised scleroderma one step closer, however there is still a way to go! One aspect of clinical trials that is constantly undergoing innovation, is how to effectively and accurately measure changes in a patient’s condition to evaluate how effective a new treatment is.  

On this theme, SRUK awarded a second grant in 2017 to Dr Pain for a project exploring new methods of measuring disease activity in children with localised scleroderma. Monitoring disease activity more effectively will allow clinicians to better assess the efficacy of different treatments both in standard clinical practice, with the potential for integration into clinical trials. Various imaging techniques will be tested to examine whether differences between healthy and affected skin in juvenile localised scleroderma (JLS) can be detected, and whether these techniques can detect changes over time.

These two projects take the children with juvenile scleroderma a little closer to new treatments, which can better manage the symptoms of this rare condition. Progress like this cannot happen without the dedicated work of researchers and scientists, vital to which is funding from organisations such as SRUK.

SRUK Webinars

SRUK host a series of webinars each year, engaging expert speakers on a range of topics linked to Raynaud’s and scleroderma. On the 10th November Dr Pain and her colleagues Tavi Aragon (rheumatology pharmacist), Katie Dobson (rheumatology occupational therapist) and Lindsey Clarke (rheumatology occupational therapist) will be speaking on the topic of ‘Understanding and Managing Scleroderma and Raynaud’s in Children & Families.’ The talk will run from 4:30-5:30pm and is free to attend, keep an eye on our website for further details of how to get involved!

SRUK Virtual Conference – 2021

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Lastly, we’d like to let you know about SRUK’s virtual conference, which took place last month – featuring an appearance from Dr Clare Pain, who has been instrumental in benefiting the lives of the children with juvenile scleroderma. To find out more about current progress in treatment regimens and what care you should expect your child with scleroderma to receive, you can catch up on Dr Pain’s talk by signing up here to watch the talks ‘on demand.’

Where: Online! All you need is a laptop, desktop, tablet or mobile, and a WiFi connection.

How: Register online

Price: Free (The conference is free to everyone to participate, however if you would like to make a donation to SRUK then you can do that by clicking here)

The full schedule for the day can be found here, with the on demand line-up including sessions from clinicians offering advice, and expertise on both Raynaud’s and Scleroderma. Within the virtual conference hall there is also an exhibition space, with materials such as videos and information leaflets from the stalls still available! We hope you take this opportunity to learn about more of the research that SRUK are supporting. If you’d like a taster of each talk, check out SRUK’s latest article on the topic.