The safety, tolerability, and effectiveness of PTL-101, an oral cannabidiol formulation, in pediatric intractable epilepsy: A phase II, open-label, single-center study

Epilepsy & Behavior : E&B
Alexis MitelpunktHagit Sacks

Abstract

Several works have reported on the antiepileptic impact of cannabis-based preparations in patients with treatment-resistant epilepsy (TRE). However, current formulations suffer from low bioavailability and side effects. PTL-101, an oral formulation containing highly purified cannabidiol (CBD) embedded in seamless gelatin matrix beadlets was designed to enhance bioavailability and maintain a constant gastrointestinal transit time. This phase II, prospective study was open to pediatric patients with TRE on stable antiepileptic drugs' (AEDs) doses, who experienced ≥4 seizures within four weeks of enrolment and with a history of ≥4 AEDs failing to provide seizure control. Following a 4-week observation period, patients began a 2-week dose-titration phase (up to ≤25mg/kg or 450mg, the lower of the two), followed by a 10-week maintenance treatment period. Caregivers recorded seizure frequency, type, and severity and ranked their global impressions after 7 and 12weeks of treatment. Responders were those showing a ≥50% reduction from baseline monthly seizure frequency. Safety assessments monitored vital signs, adverse effects, physical and neurological exams, and laboratory tests. Sixteen patients (age: 9.1±3.4) enrolled in the study; ...Continue Reading

Citations

Sep 3, 2020·Pharmaceuticals·Sophie Anne MillarSaoirse Elizabeth O'Sullivan
Dec 30, 2020·Journal of Korean Medical Science·Chung Mo KooHoon Chul Kang
Jul 12, 2020·Current Opinion in Psychology·Irene BraithwaiteGiles Newton-Howes
Jul 14, 2021·Expert Opinion on Drug Delivery·Ízola Morais de Medeiros RamalhoEryvaldo Socrates Tabosa do Egito
Sep 10, 2021·Developmental Medicine and Child Neurology·Martin Kirkpatrick, Finbar O'callaghan

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