Open Label Extension Study for GWP42003-P in Rett Sydrome patients
Research type
Research Study
Full title
An open-label extension trial to investigate the long-term safety of cannabidiol oral solution (GWP42003-P, CBD-OS) in patients with Rett Syndrome
IRAS ID
273329
Contact name
Ram Kumar
Contact email
Sponsor organisation
GW Research Ltd
Eudract number
2019-001605-24
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Duration of Study in the UK
0 years, 9 months, 1 days
Research summary
Rett Syndrome (RTT) is a rare, non-inherited (not passed from parent to child), X-linked neurodevelopmental disorder that affects approximately 1 in 10,000 live female births. It is the leading cause of intellectual disability in females and is rarely seen in males. RTT affects the way the brain develops causing a progressive loss of motor skills and speech.RTT is a progressive disorder, with early onset at 6 to 18 months. It is characterised by a regression in development. Infants aged 1 to 4 will rapidly lose the motor skills in their hands, loss of language as well as breathing difficulties and autistic-like behaviours. Over time, motor function will continue to deteriorate.
This open label extension study will evaluate the long-term safety of GWP42003-P in patients with Rett Syndrome that carry the MeCP2 gene mutation. Patients will be recruited to this study from a previous study, IRAS ID 247457.
The purpose of this study is to evaluate the safety of GWP42003-P, a cannibidiol liquid solution that has the potential to treat and reduce the core symptoms of RTT. Cannibidiol is made from the extracts prepared from cannabis plants.
Participants will be required to take GWP42003-P twice a day at the same time each day for 28 weeks. Approximately 35 patients from the UK will be invited to take part in this extension study. Participants will be required to have 10 visits. Participants will be required to undergo physical examinations, height measurement, vital sign measurement, blood and urine tests as well as an electrocardiogram (ECG). Parents and/or caregivers will be required to complete dosing diaries and questionnaires. At the end of the study, all participants will return to their standard of care treatment plan.
The study will take place at secondary care sites that are well experienced in working with children with Rett syndrome.
REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
19/NW/0711
Date of REC Opinion
15 Jan 2020
REC opinion
Favourable Opinion