Beyond Seizure Impacts in TSC
Research type
Research Study
Full title
Mixed Methods Study on the Beyond Seizure Impacts of Epidyolex in Tuberous Sclerosis Complex (TSC)
IRAS ID
336984
Contact name
Nicola Barnes
Contact email
Sponsor organisation
Jazz Pharmaceuticals
Duration of Study in the UK
0 years, 7 months, 3 days
Research summary
Tuberous sclerosis complex (TSC) is a rare genetic condition, characterised by the growth of benign tumours in different bodily organs—most commonly the skin, brain, kidneys, lungs and heart. Epileptic seizures are a common clinical manifestation of TSC. In addition, TSC is associated with neurodevelopmental and neuropsychiatric disorders, including learning difficulties, autism, challenging behaviour, anxiety and depression.
Epidyolex is derived from the Cannabis sativa plant, and is a highly purified, oil-based, liquid oral formulation of cannabidiol. In August 2021, the United Kingdom (UK) Medicines and Healthcare Products Regulatory Agency approved Epidyolex as an adjunctive treatment of seizures associated with TSC for patients two years or older. There is also some evidence from open-label studies of Epidyolex that the treatment has positive impacts on HRQoL, independent of reduced seizure frequency effects.
The study plans to explore the non-seizure-related treatment effects of Epidyolex in TSC in a real-world setting, using both surveys and qualitative interviews. This research is a non-interventional, longitudinal, mixed methods international study with caregivers of individuals with TSC who experience epileptic seizures and have recently started taking Epidyolex.
To describe short- and longer-term non-seizure treatment effects with Epidyolex, caregivers (up to n=125) will be asked to complete two online surveys. The first (Survey 1) will be completed at baseline and the second (Survey 2) will be completed 3 months (± 2 weeks) after completion of Survey 1. A random selection of participants will be invited to participate in qualitative, one-to-one interviews (approximately n = 36) lasting up to 60 minutes following completion of Survey 2.
Participants will be recruited via three potential routes —vendor recruitment, patient association groups, clinician referrals. Approval has already been obtained for UK based participant identification through patient advocacy groups and vendor panels by an independent research ethics board that reviews international studies. We seek IRAS approval for recruitment in the UK via clinician referrals. Individual country ethics applications are being submitted as required. The estimated duration of the study is 74 weeks.REC name
London - Camden & Kings Cross Research Ethics Committee
REC reference
24/PR/0382
Date of REC Opinion
24 Apr 2024
REC opinion
Favourable Opinion