Fenfluramine in CDKL5 Deficiency Disorder: Primary Efficacy and Safety Results from a Phase 3, Randomized, Double-blind, Placebo-controlled Study (S5.010)

Brian Moseley; Nicola Specchio; Eric Marsh; Orrin Devinsky; Ángel Aledo‐Serrano; Domenica Battaglia; Rajsekar Rajaraman; Karen Keough; Ryoko Honda; Renzo Guerrini; Gia Melikishivili; Sam Amin; Amanda Jaksha; Peter St. Wecker; Brian Kilgallen; Najla Dickson; Joseph Sullivan

doi:10.1212/wnl.0000000000216367

June 9, 2026Neurology

Fenfluramine in CDKL5 Deficiency Disorder: Primary Efficacy and Safety Results from a Phase 3, Randomized, Double-blind, Placebo-controlled Study (S5.010)

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Key Points

To evaluate the primary efficacy and safety of fenfluramine in patients with CDKL5 deficiency disorder.
Conducted a phase 3 randomized double-blind placebo-controlled trial.
Included patients diagnosed with CDKL5 deficiency disorder.
Trial registered under NCT05064878.
Primary efficacy results demonstrated significant improvement with fenfluramine compared to placebo.
Safety profile showed manageable adverse events in the treated cohort.

Implication

Randomized trial reports efficacy and safety of fenfluramine in patients with CDKL5 deficiency disorder, indicating its potential therapeutic value.

Abstract

To report primary results from a randomized placebo-controlled trial (RCT; NCT05064878) of fenfluramine in patients with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD).

Discussion

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Cite This Study

Moseley et al. (2026) studied this question.

synapsesocial.com/papers/6a2cf393faef96ed7f05612c https://doi.org/10.1212/wnl.0000000000216367

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Fenfluramine in CDKL5 Deficiency Disorder: Primary Efficacy and Safety Results from a Phase 3, Randomized, Double-blind, Placebo-controlled Study (S5.010)

Key Points

Implication

Abstract

Discussion

Cite This Study

Fenfluramine in CDKL5 Deficiency Disorder: Primary Efficacy and Safety Results from a Phase 3, Randomized, Double-blind, Placebo-controlled Study (S5.010)

Key Points

Implication

Abstract

Discussion

Cite This Study

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