Clinical trials in children and adolescents – an updated global consensus

Updated recommendations for standards in pediatric clinical trials have recently been published by the International Pediatric MS Study Group (IPMSSG).

The IPMSSG is a group of care providers and researchers looking to optimize worldwide care, education and research in pediatric MS. The group met in January 2018, supported by the National MS Society (US) and the MS Society of Canada. Subsequent discussion and review has led to an updated consensus on recommendations for future trials of experimental MS treatments in children and adolescents.

Whilst MS occurs more frequently in adults, there are many children and adolescents around the world with MS. Most clinical trials of new treatments are only conducted in adults, which means there is uncertainty around whether they would also be safe and effective in younger people with MS. Conducting clinical trials in children and adolescents is essential in order to widen the number of treatments available to them, but it is not easy. There are not as many of them to take part in trials; approval from the young person and his/her parents is required; and there may be unexpected side effects of the treatment in younger people.

Some of the recommendations resulting from the group include:

• Testing how a treatment is absorbed in the body and how it affects people should occur for all new treatments first, to identify the appropriate dose to use in children.
• Phase 3 trials in adults should consider enrolling teenagers.
• No more than one Phase 3 trial should be performed for a new experimental treatment in children and adolescents.
• In some cases, open-label studies (ones that don’t require a comparison with another treatment) are acceptable.

The group also pointed to areas for future research, including refining the criteria that children need to meet to be included in a clinical trial; and determining novel ways to measure treatment effectiveness.

The full report from the IPMSSG can be accessed in the journal Neurology.

With thanks to MS Research Australia – the lead provider of research summaries on our website.