Long-Term CBD Treatment for TSC-Associated Seizures Effective Across Patient Ages

Long-term use of cannabidiol (CBD) is effective and safe for the treatment of tuberous sclerosis complex (TSC)-associated seizures among both pediatric and adult patients, according to the results of a study presented at the American Epilepsy Society (AES) 2023 annual meeting, held from December 1 to 5 in Orlando, Florida.

An international team of researchers conducted a randomized controlled trial (GWPCARE6; ClinicalTrials.gov Identifier: NCT02544763) of 224 pediatric (n=166; median age, 8) and adult (n=58; median age, 25) patients with TSC-related seizures to assess the efficacy and safety of adjunctive Epidiolex^®, a 100 mg/mL oral solution of CBD, in this patient population.

Of the 166 pediatric patients, 55 received 25 mg/kg/d of CBD, 55 received 50 mg/kg/d of CBD, and 56 received placebo. Of the 58 adults, 20 received 25 mg/kg/d of CBD, 18 received 50 mg/kg/d of CBD, and 20 received placebo. The study timeline consisted of a 4-week titration period followed by a 12-week maintenance period.

At baseline, pediatric patients who took between 1 and 5 antiseizure medications experienced a median of 62 TSC-associated seizures over a 28-day period. At baseline, adult patients who took between 0 and 5 antiseizure medications experienced 49 TSC-associated seizures over 28 days.

During the maintenance period of the randomized controlled trial, a 50% or greater reduction in TSC-associated seizure frequency occurred in:

• 47% of children and 50% of adults receiving 25 mg/kg of CBD daily,
• 52% of children and 50% of adults receiving 50 mg/kg of CBD daily, and
• 21% of children and 25% of adults taking placebo.

Notably, a 75% or greater reduction in seizure frequency occurred in 24% of children and 20% of adults receiving 25 mg/kg of CBD daily and 28% of children and 31% of adults receiving 50 mg/kg of CBD daily, compared to only 5% of both children and adults taking placebo.

Following the completion of the randomized controlled trial, 199 of these patients continued to receive treatment in the open-label extension (OLE) study (GWP42003-P, CBD; ClinicalTrials.gov Identifier: NCT02544750). Approximately 53% of children and 54% of adults who continued in the OLE study achieved a 50% or greater responder rate, while 33% of children and 33% of adults achieved a 75% or greater responder rate.

During the randomized controlled trial, 96% of children and 85% of adults taking 25 mg/kg of CBD reported adverse events, while 100% of children and adults reported adverse events at the higher dose of CBD compared to 95% each of children and adults in the placebo groups.

Adverse events occurred at high rates in the OLE study in 97% of children and 91% of adults. Although adverse events occurred at high rates in the treatment groups, these rates remained consistent with the overall population as evidenced by similarly high rates of adverse events in the placebo groups.

“These results support long-term use of CBD for seizures associated with TSC in both pediatric and adult patients,” the researchers concluded.

Disclosures: Several study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see original source for full list of disclosures.