International Pediatric MS Study Group publishes important report on clinical trials in children with MS

On 19 March 2013 the journal Neurology published a meeting report from the International Pediatric MS Study Group (IPMSSG) Therapeutic Summit Workshop entitled "Towards Therapeutic Trials in Pediatric Multiple Sclerosis". Read the full article.

The International Pediatric MS Study Group (IPMSSG), which the Multiple Sclerosis International Federation (MSIF) coordinates, is a voluntary group of more than 150 academic physicians and researchers treating and/or studying multiple sclerosis (MS) in children.

Background to childhood MS

MS can be observed as early as two years of age and becomes more frequent after the age of ten years. Several new medications are currently (or soon to be) approved as new options in the treatment of MS. So far, all of the medications approved for treating MS have only been tested in an adult population. But in the last decade, regulatory authorities in the US and Europe have mandated that new product development should include paediatric studies when paediatric use is anticipated.

The prospect of new MS treatments are welcomed by patients, their families and physicians, but careful thought must be given to methodologies of testing the emerging MS therapies in children with MS. Designing paediatric clinical trials is challenging for several key reasons: MS onset in childhood and adolescence is rare, relative to adult-onset disease, and thus the paediatric MS population available for participation in trials is limited. There is also an absence of prior randomised clinical trials experience and data in children. And finally, paediatric clinical trials require sensitivity to ethical considerations in their design, and an acknowledgement of the importance of meaningful paediatric-relevant outcome measures.

Goals and outcomes of the Summit

The primary goal of the Summit Workshop, held in 2012, was to analyse the feasibility, methodology, and priorities for paediatric clinical trials for new medications in MS. To address these major issues, seven workgroups consisting of academic experts met in advance to discuss and present the current state of knowledge and best practice in the following areas as they relate to paediatric MS:

  • Clinical outcome measures
  • Clinical trial design
  • MRI outcome measures
  • Cognitive outcome measures
  • Paediatric pharmacology
  • Safety and biological mechanisms
  • Long-term outcome registries

The meeting was well attended, with 69 delegates, including 35 academic experts as well as regulatory and pharmaceutical representatives.

The meeting attendees agreed that clinical trials were necessary in paediatric MS to obtain pharmacokinetic, safety and efficacy data, and regulatory approval allowing for greater access to medications. They also agreed that a child having a relapse is an appropriate primary outcome measure for phase III paediatric trials. An international standardised cognitive battery was identified. The pros and cons of various trial designs were discussed. Guidelines surrounding MRI studies, pharmacokinetics, pharmacodynamics and registries were developed.

To read the full report as published in Neurology please click here.

Next steps

After the Summit Workshop, the Steering Committee of the IPMSSG established a Clinical Trials Task Force to further develop consensus around clinical trial design, outcome measures and feasibility. The task force will continue to meet with the key stakeholders of the academic, clinical, regulatory agency, pharmaceutical industry and patient organisation communities to ensure advances in the treatment of MS in children are achieved in the shortest possible time with the highest degree of safety.

For more information about the International Pediatric MS Study Group please visit its website at www.ipmssg.org.