Phase III Marigold trial of Ztalmy in CDKL5 deficiency disorder published in The Lancet Neurology.- Marinus Pharma

The paper presents the results of the Phase III Marigold trial, a double-blind placebo-controlled trial in which 101 patients were randomized and individuals treated with Ztalmy showed a median 30.7% reduction in 28-day major motor seizure frequency, compared to a median 6.9% reduction for those receiving placebo, achieving the trial’s primary endpoint (p=0.0036). Ztalmy was generally well-tolerated and showed a safety profile consistent with previous clinical trials, with the most frequent adverse event being somnolence.

See: "Safety and efficacy of ganaxolone in patients with CDKL5 deficiency disorder: results from the double-blind phase of a randomised, placebo-controlled, phase 3 trial" Elia M Pestana Knight May, The Lancet Neurology 2022 DOI:https://doi.org/10.1016/S1474-4422(22)00077-1