GWND18064 study of CBD-OS in Patients with Rett Syndrome
Research type
Research Study
Full title
A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED,TRIAL TO INVESTIGATE THE EFFICACY AND SAFETY OF CANNABIDIOL ORAL SOLUTION (GWP42003-P, CBD-OS) IN PATIENTS WITH RETT SYNDROME.
IRAS ID
247457
Contact name
Justyna Rooney
Contact email
Sponsor organisation
GW Research Ltd
Eudract number
2018-003370-27
Clinicaltrials.gov Identifier
141153, IND
Duration of Study in the UK
2 years, 4 months, 31 days
Research summary
Rett Syndrome (RTT) is a rare, non-inherited (not passed from parent to child), neurodevelopmental disorder affecting approximately 1 in 10,000 girls. The RTT 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. Most babies with RTT develop normally for the first 6 to 18 months of age, and then lose skills they previously had, such as the ability to crawl, walk, communicate or use their hands.
Currently there are no effective treatments for RTT and therefore there is a critical need for treatments.
Existing treatment options focus on managing symptoms and providing support for patients and their families. Additional therapies may include physiotherapy, occupational therapy, speech therapy and feeding assistance.
The purpose of this study is to evaluate the safety and efficacy of canabidiol (GWP42003-P) liquid solution compared to a placebo (dummy medicine).
Canabidiol is made from extracts prepared from cannabis plants. The medicine is aimed to reduce the severity of symptoms experienced in children with RTT.
Approximately 252 females with RTT, aged 4 to 18 years, are invited to take part in this study.
Patients’ participation is expected to last about 33 weeks and includes the following phases: screening, randomisation, treatment and end of treatment.
At the beginning of the study patients will be randomly assigned to 1 of 2 treatment groups (GWP42003-P oral solution or Placebo). For every 3 children who take part in the study, 2 will be given GWP42003-P and 1 will be given the placebo. The study is blinded which means neither caregiver/patient nor the study team will know which medicine patient is receiving.
The study medication will be given in the form of oral solution for 24 weeks. After the treatment period patients will start 10-day taper period to gradually reduce the dose of study medication, followed by the 4-week follow-up period. Patients will be required to attend 7 clinic visits and 3 telephone visit.
During the study patients will undergo physical examinations, genetic mutation test of MECP2 gene, have blood and urine tests and ECG (electrocardiogram). Caregiver will be required to complete questionnaires and dosing diaries throughout the study.
At the end of the study patients may be offered to enter 6-month open label extension trial to continue on active treatment. Patients who do not take part in the extension study will return to their standard of care treatment.REC name
London - Brighton & Sussex Research Ethics Committee
REC reference
19/LO/0160
Date of REC Opinion
9 Apr 2019
REC opinion
Further Information Favourable Opinion