The phase 3 PARADIGMS clinical trial is the first-ever study designed for children and adolescents aged 10 to 17 years old with relapsing forms of multiple sclerosis.
Treatment with fingolimod (Gilenya, Novartis) reduced rates of relapse in children and adolescents with relapsing forms of multiple sclerosis (MS) over a 2-year period compared with interferon beta-1a (Avonex), according to data from a phase 3 study.
Fingolimod, an oral disease-modifying therapy, was initially approved by the FDA in 2010 to treat adults with relapsing MS. In May 2018, the FDA expanded its indication for use in pediatric patients 10 years of age and older.
The phase 3 clinical trial PARADIGMS is the first-ever controlled study specifically designed for children and adolescents aged 10 to 17 years old with relapsing forms of MS. According to Novartis, relapsing forms of MS account for 98% of all MS cases in children and adolescents, who often experience more frequent and severe relapses than adults with the disease. Fingolimod is the only FDA-approved treatment for this patient population.
The trial, which included 215 children and adolescents with MS with an Expanded Disability Status Scale score of 0 and 5.5, evaluated the safety and efficacy of fingolimod compared with interferon beta-1a. Patients either received once-daily oral fingolimod or intramuscular interferon beta-1a once weekly.
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According to the findings, treatment with fingolimod significantly reduced relapse rates by 82% and delayed the time to first relapse. An estimated 85.7% of fingolimod-treated patients were relapse-free at 24 weeks versus 38.8% of patients treated with interferon beta-1a.
Fingolimod also significantly reduced the number of new or newly enlarged T2 lesions up to 24 months by 53% and significantly reduced the average number of gadolinium enhancing T1 (Gd+) lesions per scan at 24 months by 66%, according to the study. Additional analyses also found that fingolimod significantly reduced the annualized rate of brain volume loss by 40%.
Adverse events, excluding relapses, occurred in 88.8% of patients treated with fingolimod and 95.3% of patients treated with interferon beta-1a.
The safety profile of fingolimod in the study was overall consistent with the findings from previous clinical trials in adults, according to Novartis.
The results of the PARADIGMS clinical trial were published in the New England Journal of Medicine.
Chitnis T, Arnold DL, Banwell B, et al. Trial of fingolimod versus interferon beta-1a in pediatric multiple sclerosis. NEJM. 2018. Doi: 10.1056/NEJMoa1800149
Novartis announces NEJM publication of landmark PARADIGMS study demonstrating significant benefit of Gilenya in children and adolescents with MS [news release]. Novartis’ website. https://www.novartis.com/news/media-releases/novartis-announces-nejm-publication-landmark-paradigms-study-demonstrating-significant-benefit-gilenya-children-and-adolescents-ms. Accessed September 17, 2018.