Phase 2 Study of GWP42003-P in Autism Spectrum Disorder
Research type
Research Study
Full title
An exploratory, Phase 2, randomized, double-blind, placebo-controlled trial to investigate the safety and efficacy of cannabidiol oral solution (GWP42003-P; CBD-OS) in children and adolescents with Autism Spectrum Disorder
IRAS ID
294263
Contact name
Andre Strydom
Contact email
Sponsor organisation
GW Research Ltd
Eudract number
2020-002819-21
Duration of Study in the UK
2 years, 0 months, 0 days
Research summary
Autism Spectrum Disorder (ASD) is a lifelong condition that affects how people communicate and interact with the world. People with autism see, hear, and feel the world differently to other people and symptoms include problems with communication, social skills, and patterns of behaviour. Autism is a spectrum condition, which means it affects people in different ways. Current therapies have limited effect, there is no single standard treatment for ASD.
This study will look at whether the investigational drug GWP42003-P, improves symptom severity in people with ASD. GWP42003-P is a liquid that contains extracts from cannabis plants. Clinical literature on cannabinoids extracts, containing cannabidiol (CBD) and tetrahydrocannabinol (THC) show some positive effects on behaviour in ASD, however there are known potential safety issues administering THC to children, while administration of GWP42003-P has been well tolerated in other paediatric populations. The study will also look at the safety of the study drug; what the body does to the study drug, and the quality of life of the participant’s parent/caregiver. This is a Phase 2 study, the study drug has already been tested in other people, but it has not been tested in people with ASD. The study drug will be compared to a placebo (no active ingredients). The study is “double-blind”, so neither the patient or the study doctor will know which “study medication” (study drug or placebo) the patient receives. Approximately, 160 patients aged 6-17 years with ASD will take part globally, for up to 17 weeks. The study has a screening period (2weeks), treatment period (13weeks) and follow up period (2weeks); there will be a number of assessments (Medical history, physical exams, vital signs, ECGs, blood/urine samples, drug test), and questionnaires to be completed (patients and caregivers).REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
21/EM/0126
Date of REC Opinion
26 Jul 2021
REC opinion
Further Information Favourable Opinion