| Title: |
Fenfluramine in CDKL5 Deficiency Disorder: Primary Efficacy and Safety Results from a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study. |
| Authors: |
Specchio, N.; Marsh, E.; Devinsky, O.; Aledo-Serrano, Á.; Battaglia, D. I.; Rajaraman, R.; Keough, K.; Honda, R.; Guerrini, R.; Melikishvili, G.; Amin, S.; Jaksha, A.; Haas, S.; Moseley, B.; Wecker, P. St.; Kilgallen, B.; Dickson, N.; Sullivan, J. |
| Source: |
Neuropediatrics; 2026 Supplement 1, Vol. 57, pS1-S24, 24p |
| Subject Terms: |
SEIZURES (Medicine); GENETIC mutation; RANDOMIZED controlled trials; ANTICONVULSANTS; MEDICATION safety; BRAIN diseases; PEDIATRIC neurology |
| Abstract: |
This article reports on a randomized placebo-controlled trial evaluating fenfluramine (FFA) for treating seizures in patients with CDKL5 deficiency disorder (CDD), an ultra-rare, drug-resistant developmental and epileptic encephalopathy caused by CDKL5 gene mutations. The study included patients aged 1 to 35 years experiencing frequent motor seizures and found that FFA significantly reduced countable motor seizure frequency compared to placebo, with 45.2% of FFA-treated patients achieving at least a 50% reduction. Clinically meaningful improvements in global functioning were also observed, and adverse events were consistent with the known safety profile of FFA, with no new safety concerns or serious cardiac issues reported. These results suggest that FFA may be a promising therapeutic option for managing seizures associated with CDD. [Extracted from the article] |
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| Database: |
Complementary Index |