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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 11 March 2020
Main ID:  EUCTR2019-001605-24-ES
Date of registration: 21/02/2020
Prospective Registration: Yes
Primary sponsor: GW Research Ltd
Public title: An open label extension study for long-term safety of GWP42003-P in patients with Rett Syndrome
Scientific 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
Date of first enrolment: 04/03/2020
Target sample size: 252
Recruitment status: Authorised-recruitment may be ongoing or finished
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2019-001605-24
Study type:  Interventional clinical trial of medicinal product
Study design: 
Controlled: no
Randomised: no
Open: yes
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product:
Placebo:
Other:
Number of treatment arms in the trial: 1
 
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no
Countries of recruitment
Australia Canada Italy Spain United Kingdom United States
Contacts
Name: GW Research Ltd Switchboard   
Address:  Sovereign House, Vision Park, Chivers Way CB24 9BZ Histon, Cambridge United Kingdom
Telephone: +441223266800
Email: info@gwpharm.com
Affiliation:  GW Research Ltd
Name: GW Research Ltd Switchboard   
Address:  Sovereign House, Vision Park, Chivers Way CB24 9BZ Histon, Cambridge United Kingdom
Telephone: +441223266800
Email: info@gwpharm.com
Affiliation:  GW Research Ltd
Key inclusion & exclusion criteria
Inclusion criteria:
For inclusion in the trial, patients must fulfil all of the following criteria:
• Patient has completed all scheduled visits of the treatment phase of the RCT, GWND18064, and has transitioned to OLE by the point of RCT follow-up (Visit 11).
• Patient (if possessing adequate understanding, in the investigator’s opinion) and/or her parent(s)/legal representative is willing and able to give informed consent/assent for participation in the trial.
• Patient and her caregiver are willing and able (in the investigator’s opinion) to comply with all trial requirements (including the completion of all caregiver assessments by the same caregiver throughout the trial).
• Patient must have the ability to swallow the investigational medicinal product (IMP) provided as a liquid solution or the ability for the IMP to be delivered via gastrostomy (G) or nasogastric (NG) feeding tube (only G- or NG-tubes made from polyurethane or silicon are allowed).
• Patient and/or parent(s)/legal representative is willing to allow the responsible authorities to be notified of participation in the trial, if mandated by local law.
• Patient and/or parent(s)/legal representative is willing to allow the patient’s primary care practitioner (if she has one) and consultant (if she has one) to be notified of participation in the trial if the primary care practitioner/consultant is different from the investigator.
Are the trial subjects under 18? yes
Number of subjects for this age range: 252
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 12
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
• Patient meets the withdrawal criteria (including clinically significant abnormal laboratory values), in the investigator’s opinion.
• Patient met during the RCT the criteria for permanent IMP discontinuation (unless in case of an AE, if AE was not considered related with the IMP; patients that met alanine aminotransferase (ALT)/aspartate aminotransferase (AST) elevations discontinuation criteria must be excluded).
• Patient is of childbearing potential, unless willing to ensure that they or their partner use a highly effective method of birth control (e.g., combined [estrogen and progestogen containing] hormonal contraceptiona associated with inhibition of ovulation [oral, intravaginal or transdermal], progestogen-only hormonal contraceptiona associated with inhibition of ovulation [oral, injectable or implantableb] intrauterine devices/hormone-releasing systemsc, bilateral tubal occlusiona, vasectomized partnera, a, sexual abstinenceb during the trial and for 3 months thereafter.
• Patient has been previously enrolled and dosed in this trial.
• Patient is unwilling to abstain from donation of blood during the trial.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: no
Health Condition(s) or Problem(s) studied
Rett syndrome (RTT) [typical or atypical]
MedDRA version: 20.0 Level: PT Classification code 10077709 Term: Rett syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Intervention(s)

Trade Name: Epidyolex and is the approved name in the EU
Product Name: Cannabidiol (CBD)
Product Code: GWP42003-P
Pharmaceutical Form: Oral solution
INN or Proposed INN: Cannabidiol
CAS Number: 13956-29-1
Current Sponsor code: GWP42003-P
Other descriptive name: CANNABIDIOL
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-

Primary Outcome(s)

Primary end point(s): Safety:
The long-term safety profile of GWP42003-P will be assessed by evaluating changes in the following, relative to the prerandomization baseline of the RCT:
• AEs
• Clinical laboratory parameters
• Vital signs
• Physical examination procedures
• 12-lead ECG
• Effects on menstruation cycles
• Suicidality
• Change in growth and development by measurement of height, weight, IGF-1 levels, and Tanner Staging (for patients aged = 7 years or earlier, if clinically indicated by the onset of menarche or other signs of precocious puberty)

Secondary Objective: •To evaluate the effect of GWP42003-P in measures of disease severity
o Rett Syndrome Behaviour Questionnaire (RSBQ)
o Clinical Global Impressions - Improvement (CGI-I)
o Clinical Global Impressions - Severity (CGI-S)
o 9-items Motor Behavioral Assessment (MBA-9)
o Children’s Sleep Habits Questionnaire (CSHQ)

Exploratory objectives:
•To evaluate the effect of GWP42003-P on caregiver and patient quality of life (QoL)
o 36-item Short Form [SF-36] and Child Health Questionnaire Parent Form 50 [CHQ-PF50], respectively
• To evaluate the effect of GWP42003-P on health utilization
o Hospital Services Use Questionnaire
o Caregiver Assessment of Rett Symptoms
Main Objective: To evaluate the long-term safety of GWP42003-P in patients with RTT
Timepoint(s) of evaluation of this end point: at all visits
Secondary Outcome(s)

Secondary end point(s): The following will be assessed by evaluating changes relative to the prerandomization baseline of the RCT:
• RSBQ
• CGI-I
• CGI-S
• MBA-9
• CSHQ
Timepoint(s) of evaluation of this end point: Please refer to Protocol schedule of events table for detailed information
Secondary ID(s)
2019-001605-24-GB
GWND19002
Source(s) of Monetary Support
GW Research Ltd
Secondary Sponsor(s)
Ethics review
Status: Approved
Approval date: 26/02/2020
Contact:
Results
Results available:
Date Posted:
Date Completed:
URL:
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