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FC02.04 - Teriflunomide efficacy and safety in pediatric patients with relapsing forms of MS: Interim analysis of open-label TERIKIDS trial extension
Treatment options for pediatric patients with relapsing forms of multiple sclerosis (RMS) are limited. Teriflunomide, approved for adults with RMS in >80 countries, was investigated in pediatric RMS in TERIKIDS (NCT02201108), a 2-year, multicenter, multinational, randomized, double-blind (DB), placebo-controlled, parallel-group phase 3 study.
To report the interim results in pediatric patients from the open-label (OL) period of the TERIKIDS study as of 27 November 2019.
Patients who either completed 96-week DB treatment or qualified for early switch from DB treatment to OL teriflunomide could continue in the OL period until 192 weeks after initial randomization. All patients in the OL period received teriflunomide at a dose based on body weight, equivalent to 14 mg in adults.
In the DB period, teriflunomide reduced risk of relapse (−34%); however, the difference was not statistically significant versus placebo (P=0.29) so TERIKIDS did not meet its primary endpoint. Teriflunomide significantly reduced risk of relapse or high MRI activity (−43%; P=0.041; prespecified sensitivity analysis), number of new/enlarging T2 lesions (−55%; P=0.0006), and number of gadolinium-enhancing lesions (−75%; P<0.0001) relative to placebo. At the cut-off date, 100 (91.7%) patients from the teriflunomide and 52 (91.2%) from the placebo group enrolled in the OL period; 34 patients discontinued, 30 completed, and 88 were ongoing. From DB randomization to week 192, risk of relapse was numerically lower with continuous teriflunomide versus placebo/teriflunomide (hazard ratio [95% CI]: 0.61 [0.38 to 0.98]; P=0.098), as was risk of disability progression sustained for 24 weeks (hazard ratio [95% CI]: 0.552 [0.245 to 1.242]). Number of new/enlarging T2 lesions per MRI scan was reduced with continuous teriflunomide versus placebo/teriflunomide (6.3 vs 13.0; P=0.0006). Incidence of adverse events during the OL period was lower with continuous teriflunomide versus placebo/teriflunomide (68.0% vs 82.7%). Adverse events led to treatment discontinuation during the OL period in 8 patients (increased alanine aminotransferase [n=5], peripheral neuropathy [n=1], pancreatitis [n=2]).
Interim analysis showed that continuous teriflunomide numerically lowered the risk of clinical relapses and 24-week sustained disability progression in pediatric patients compared with delayed initiation of teriflunomide after placebo. Teriflunomide was well tolerated and had a manageable safety profile.
STUDY SUPPORT: Sanofi.