Epidiolex Treatment of Nodule Sclerosis (TSC) Related Epilepsy Phase III Clinical Success
Last Update: 2020-06-17
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This is a randomized, double-blind, placebo-controlled trial conducted in 46 clinical centres in 6 countries, with a total of 224 cases (aged 1-65 years) diagnosed with Treatment Resistance (difficult to treat) TSC patientsIn the study, these patients were randomly assigned to receive Epidiolex at 25 mg/kg/day (n?75), Epidiolex 50mg/kg/day (n?73), placebo (n-76), and treatment for 16 weeks (4-week drops regularly, 12-week maintenance period)The main endpoint was the percentage change in the frequency of Epidiolex and placebo compared to placebo during treatmentKey secondary endpoints include: a reduction in the proportion of patients with seizures of more than 50%, a reduction in the total frequency of seizures (including onset sensations and epilepsy spasms) by a total of 50%, and an overall impression of changes in the overall condition of the subject/caregiver (S/CGIC)results showed that the study reached the primary endpoint, with a significant reduction in the frequency of TSC-related seizures in the Epidiolex treatment group compared to the placebo group: 49% and 48% of the relative baseline sized in the Epidiolex 25mg/kg/day treatment group, 48% in the Epidiolex 50mg/kg/day treatment group, and 29% lower in the placebo group (p.0009, p.00118)Secondary endpoint data showed a 50 percent or a much greater reduction in seizures in patients with a higher proportion of patients in the Epidiolex treatment group than in the placebo group (36 percent for 25 mg/kg/day group, 40 percent for 50 mg/kg/day group, 22 percent for the placebo group, and p.0.0692 and p.0245)In addition, the total seizure frequency (including pontomy sensation and epilepsy spasms) in both doses of Epidiolex was significantly reduced in both doses of Epidiolex compared to the placebo group, with a 27% reduction in the placebo group (p-0.0013 and p-0.0018)Based on the results of the patient/caregiver's overall impression (S/CGIC) questionnaire, the percentages of Epidiolex 25mg/kg/day group and Epidiolex 50mg/kg/day group reported improvements in overall condition were 69%, 62%, and placebo groups were 39% (p.0074 and p-0.0580)Additional analysis showed that patients treated with Epidiolex experienced a significantdecrease in combined episode epilepsy compared to placebo patients (52 percent, 50 percent, 50 percent, and 32 percent in the placebo group, p-0.0076 and p.0116 in the 25 mg/kg/day treatment group, 50 mg/kg/day treatment group, respectivelythe safety profile observed in the study is broadly consistent with previous researchThe incidence of adverse events (AE) was 93% in the 25 mg/kg/day group, 100% in the 50mg/kg/day group and 95% in the placebo groupBoth doses have acceptable safety, with adverse events of 25 mg/kg/day less than 50 mg/kg/dayThe most common adverse reactions are diarrhea, loss of appetite and drowsiness25 mg/kg/day group, 50 mg/kg/day group, placebo group, 5 cases, 10 cases, 2 cases due to adverse events and discontinued treatmentIn addition, 13% and 25% of the 25mg/kg/day group and 50mg/kg/day group showed elevated liver enzymes each day, and 79% of these patients were also taking the anti-epileptic drug sodium valproateAll patients with alT/AST increases were alleviatedNo cases of Hy's law were observed in the trial, and no deaths were reported "Epilepsy is the most common neurological characteristic of TSC," said Dr Elizabeth Thiele, study and professor of neurology at Harvard Medical School, professor of neurology at Harvard Medical School, director of pediatric epilepsy at Massachusetts General Hospital, Carol and James Herscot, center for nodule sclerosis syndrome TSC is a rare, severe, childhood disease in which up to two-thirds of patients experience drug-incurable seizures There is a significant need for new treatments to address TSC-related seizures These positive results suggest that patients may benefit from the treatment of epidiolex, a high-purity CBD formulation "
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