Study group reports on clinical trials in children with MS

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

In 19 March 2013 the journal Neurology published a report from the IPMSSG Therapeutic Summit Workshop called Towards Therapeutic Trials in Pediatric Multiple Sclerosis.

 Background to childhood MS

MS can be observed as early as two years of age and becomes more frequent after the age of ten. 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 is 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 pediatric clinical trials is challenging for several key reasons:

• MS onset in childhood and adolescence is rare, relative to adult-onset disease, and so the pediatric MS population available to take part in trials is limited
• There is an absence of prior randomised clinical trials experience and data in children
• Pediatric clinical trials require sensitivity to ethical considerations in their design, and an acknowledgement of the importance of meaningful paediatric-relevant outcome measures.
  

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Goals and outcomes of the workshop

The primary goal of the workshop, held in 2012, was to analyse the feasibility, methodology, and priorities for paediatric clinical

trials for new medications in MS. To address these issues, seven groups of academic experts met in advance to discuss the current state of knowledge and best practice in the following areas as they relate to pediatric MS:

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

The meeting was attended by 69 delegates, including 35 academic experts and 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.

 Next steps

After the Summit Workshop, the IPMSSG’s steering committee set up a clinical trials task force to further develop consensus around clinical trial design, outcome measures and feasibility. The task force will continue to meet 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.