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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: ClinicalTrials.gov
Last refreshed on: 12 December 2020
Main ID:  NCT04252586
Date of registration: 30/01/2020
Prospective Registration: Yes
Primary sponsor: GW Research Ltd
Public title: An Open-label Extension Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome ARCH
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: February 28, 2020
Target sample size: 252
Recruitment status: Enrolling by invitation
URL:  https://clinicaltrials.gov/show/NCT04252586
Study type:  Interventional
Study design:  Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 3
Countries of recruitment
Australia Canada Italy Spain United Kingdom United States
Contacts
Key inclusion & exclusion criteria

Inclusion Criteria:

- Participant has completed all scheduled visits of the treatment phase of the
randomized controlled trial (RCT), GWND18064 (NCT03848832), and has transitioned to
open-label extension (OLE) by the point of RCT follow-up

- Participant (if possessing adequate understanding, in the investigator's opinion)
and/or the participant(s)/legal representative is willing and able to give informed
consent/assent for participation in the trial.

- Participant and the participant'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).

- 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).

- Participant 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.

- Participant and/or parent(s)/legal representative is willing to allow the
participant's primary care practitioner (if the participant has one) and consultant
(if the participant has one) to be notified of participation in the trial, if the
primary care practitioner/consultant is different from the investigator.

Exclusion Criteria:

- Participant meets the withdrawal criteria (including clinically significant abnormal
laboratory values), in the investigator's opinion.

- Participant met during the RCT the criteria for permanent IMP discontinuation (unless
in the case of an adverse event [AE], if the AE was not considered related with the
IMP; participants 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

- Participant has been previously enrolled and dosed in this trial.

- Participant 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



Age minimum: 2 Years
Age maximum: 18 Years
Gender: All
Health Condition(s) or Problem(s) studied
Rett Syndrome
RTT
Intervention(s)
Drug: GWP42003-P
Primary Outcome(s)
Number of participants with any adverse event [Time Frame: up to Week 110]
Number of participants with any change in growth and development by measurement of height, weight, serum insulin-like growth factor-1 (IGF-1) levels, and Tanner Staging [Time Frame: up to Week 106]
Number of participants with any clinically significant 12-lead electrocardiogram (ECG) value [Time Frame: up to Week 106]
Number of participants with any clinically significant clinical laboratory parameter value [Time Frame: up to Week 106]
Number of participants with any clinically significant effects on their menstruation cycle [Time Frame: up to Week 105]
Number of participants with any clinically significant physical examination procedure value [Time Frame: up to Week 106]
Number of participants with any clinically significant vital sign value [Time Frame: up to Week 106]
Number of participants with suicidality, as assessed by the investigator via a clinical interview with the caregiver [Time Frame: up to Week 106]
Secondary Outcome(s)
Change from prerandomization Baseline of the Randomized Controlled Trial (RCT) to Week 105 in Rett Syndrome Behaviour Questionnaire (RSBQ) scores [Time Frame: from Baseline up to Week 105]
Change from prerandomization Baseline of the RCT to Week 105 in 9-item Motor Behavioral Assessment (MBA-9) scores [Time Frame: from Baseline up to Week 105]
Change from prerandomization Baseline of the RCT to Week 105 in Children's Sleep Habits Questionnaire (CSHQ) scores [Time Frame: from Baseline up to Week 105]
Change from prerandomization Baseline of the RCT to Week 105 in Clinical Global Impressions - Improvement (CGI-I) scores [Time Frame: from Baseline up to Week 105]
Change from prerandomization Baseline of the RCT to Week 105 in Clinician Global Impressions - Severity Scale (CGI-S) scores [Time Frame: from Baseline up to Week 105]
Secondary ID(s)
2019-001605-24
GWND19002
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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