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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: 15 September 2020
Main ID:  EUCTR2019-001605-24-GB
Date of registration: 23/10/2019
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: 15/01/2020
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: no
Placebo: no
Other: no
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 France 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 the patient’s parent(s)/legal representative is willing and able to give informed consent/assent for participation in the trial.
• Patient and the patient’s 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 the patient has one) and consultant (if the patient has one) to be notified of participation in the trial if the primary care practitioner/consultant is different from the investigator.

Per Protocol Annex 1, with the exception of the inclusion criterion that requires patients must have completed all scheduled visits of the treatment phase of the RCT, GWND18064, and transitioned to OLE by the point of RCT follow-up (Visit 11), all inclusion and exclusion criteria defined in the GWND19002 protocol must be met. In addition, the following inclusion criteria apply:
Inclusion criteria
• Patient has completed all scheduled visits of the treatment phase of the RCT, either in clinic or completed as many scheduled assessments as feasible remotely, or has withdrawn from GWND18064 after discussion with the medical monitor due to inability to adequately monitor safety and benefit risk.
• Visit 1 is taking place no later than 3 months after screening of new subjects for study GWND18064 has reopened at their trial site.
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 0

Exclusion criteria:
The patient may not enter the trial if ANY of the following apply:
• 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).
• Females 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 contraception associated with inhibition of ovulation [oral, intravaginal or transdermal], progestogen-only hormonal contraception associated with inhibition of ovulation [oral, injectable or implantable ] intrauterine devices/hormone-releasing systems , bilateral tubal occlusion, vasectomized partner, sexual abstinence during the trial and for 3 months after the last dose.
• Patient has been previously enrolled and dosed in this trial.
• Patient is unwilling to abstain from donation of blood during the trial.
• Male participants who are fertile (i.e., after puberty unless permanently sterile by bilateral orchidectomy) and with a partner of childbearing potential unless agree to ensure that they use male contraception (e.g., condom) or remain sexually abstinent during the trial and for 3?months after the last dose.

Per Protocol Annex 1, with the exception of the inclusion criterion that requires patients must have completed all scheduled visits of the treatment phase of the RCT, GWND18064, and transitioned to OLE by the point of RCT follow-up (Visit 11), all inclusion and exclusion criteria defined in the GWND19002 protocol must be met. In addition, the following exclusion criteria apply:
• Any history of suicidal behavior or any suicidal ideation in the last month or at Visit 1.
• Patient has clinically relevant abnormalities in the electrocardiogram measured at Visit 1 (including QT interval, corrected by Bazett's correction formula [QTcB] > 450 msec, average of 3 measurements).
• Patient has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the IMP (active and placebo), such as sesame oil.
• Patient has moderately impaired hepatic function at screening, defined as serum ALT or AST > 3 × ULN or total bilirubin [TBL] > 2 × ULN.
This criterion can only be confirmed once the Visit 1 laboratory results are available. If Visit 1 laboratory results indicate the patient is not eligible, the patient must be withdrawn. Prior to withdrawal for the transaminase elevations noted above, the investigator may choose to confirm the transaminase elevations by repeating the following laboratory tests within 24 to 48 hours: ALT, AST, TBL, international normalized ratio, % eosinophils, gamma-glutamyl transferase, alkaline phosphatase, and eosinophils. Should the above transaminase elevation criteria be confirmed, the patient must be withdrawn from the trial.
• Patient has received an IMP (other than GWND18064 IMP) since participation in GWND18064.
• Patient has been taking felbamate for less than 1 year prior to Visit 1
• Patient is currently using or has used recreational or medicinal cannabis, cannabinoid-based medications (including Sativex
® [nabiximols]) or cannabidiol oral solutions (excluding GWND18064 IMP) within the 3


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
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: CBD Oral solution, is known as 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)
Main Objective: To evaluate the long-term safety of GWP42003-P in patients with RTT
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
Timepoint(s) of evaluation of this end point: Please refer to the schedule of the assessment within the protocol (Appendix 1).
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)
GWND19002
Source(s) of Monetary Support
GW Research Ltd
Secondary Sponsor(s)
Ethics review
Status: Approved
Approval date: 15/01/2020
Contact:
Results
Results available:
Date Posted:
Date Completed:
URL:
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