Abstract Details

Title Delayed-release Dimethyl Fumarate Reduces the Formation of T2 Lesions in Pediatric Patients with Relapsing-Remitting MS: Results From FOCUS, the First Clinical Trial to Evaluate the Neuroradiological Efficacy of an MS Disease-Modifying Therapy in Pediatric Patients

Topic Multiple Sclerosis

Presentation(s) P4 - (-)

Poster/Presentation
Number 406

Background No disease-modifying therapy (DMT) has been demonstrated safe and effective in an interventional trial in pediatric MS patients.

Objective

Design/Methods FOCUS (NCT02410200) was a single arm, open-label study in RRMS patients aged 10–17 years. The study comprised an 8-week, off-treatment baseline period, followed by a 24-week treatment period during which patients received oral DMF 240 mg twice daily. Brain MRI scans were obtained at the beginning and at the end of the baseline period, and at weeks 16 and 24 of the treatment period. The primary endpoint was the change in the number of new/newly enlarging T2 hyperintense lesions during the 8-week baseline versus the final 8-week treatment period.

Results Twenty-two patients (14 [64%] female; mean age 15.8 [range: 13–17] years; mean weight 66 kg) were enrolled (12 sites, 10 countries). The mean change in the number of new/newly enlarging T2 hyperintense lesions was -7.9 (90% confidence interval [-8.0, -1.5], p=0.009). The geometric mean C_max and AUC_0-12of DMF following the morning dose were 1.6 μg/mL and 3.3 h*μg/mL, respectively. The most common adverse events were gastrointestinal events, flushing, and MS relapse, occurring in 55%, 45%, and 32% of patients, respectively. Lymphopenia, alanine aminotransferase elevation, and aspartate aminotransferase elevation occurred in 23%, 15%, and 10% of patients, respectively. There were no serious adverse events considered by the investigators to be related to DMF treatment. All 20 patients completing the study enrolled in a 2-year extension study (NCT02555215).

Conclusions In this study, the first clinical trial to examine the neuroradiological efficacy of a DMT in pediatric MS patients, the efficacy, safety, and pharmacokinetics of DMF were consistent with those observed in adult RRMS patients.

Authors/Disclosures
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