(New York, NY) - The International Pediatric MS Study Group (IPMSSG) has released updated standards for clinical trials involving children and teens with multiple sclerosis (MS). The new recommendations were published in the May 1, 2019, online issue of Neurology®, the medical journal of the American Academy of Neurology.
The updated recommendations include:
Pharmacokinetic/pharmacodynamic studies (how a therapy is absorbed and how it affects the patient) should be completed for all new agents to identify the appropriate dose in children.
If an immune-modulating agent has been proven to be effective in adult MS, it is inappropriate to test that agent in pediatric MS using a placebo (inactive) control.
Phase 3 trials in adults should consider enrolling teenagers.
Open-label extension studies should be conducted.
In some cases, when therapies have been well studied in adults, an open-label study should be considered as sufficient for approval in pediatric MS.
"Thankfully, the number of pediatric MS cases is low compared to adult cases - but these are children, facing a chronic illness and we need to do everything we can to help them," said lead author Emmanuelle Waubant, MD, Professor of Neurology at the University of California, San Francisco. "We are confident these consensus recommendations will help address the need for high-quality evidence to inform the optimal treatment of children and teens living with multiple sclerosis."
Conducting clinical trials in children can be complicated; some issues include the low numbers of children with MS at any one study site, the necessity to obtain parental approval in addition to the child agreeing to participate, and the fact that treatments can have unanticipated side effects in immature individuals. The IPMSSG is a group of 165 care providers representing 44 countries, dedicated to optimizing worldwide care, education and research in pediatric MS. In 2012, they held an international meeting to develop consensus on how to conduct clinical trials in children with MS. In 2018, they recognized the need to update recommendations, and convened a meeting in New York City in January, which was sponsored by the National MS Society (USA) and the MS Society of Canada.