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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT04252586




Registration number
NCT04252586
Ethics application status
Date submitted
30/01/2020
Date registered
5/02/2020
Date last updated
28/04/2020

Titles & IDs
Public title
An Open-label Extension Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) 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
Secondary ID [1] 0 0
2019-001605-24
Secondary ID [2] 0 0
GWND19002
Universal Trial Number (UTN)
Trial acronym
ARCH
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Rett Syndrome 0 0
RTT 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - GWP42003-P

Experimental: GWP42003-P - 100 milligrams per milliliter (mg/mL) GWP42003-P oral solution, taken twice daily (morning and evening).


Treatment: Drugs: GWP42003-P
GWP42003-P presented as an oral solution containing cannabidiol in the excipients sesame oil with anhydrous ethanol, sweetener (sucralose), and strawberry flavoring

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of participants with any adverse event
Timepoint [1] 0 0
up to Week 110
Primary outcome [2] 0 0
Number of participants with any clinically significant clinical laboratory parameter value
Timepoint [2] 0 0
up to Week 106
Primary outcome [3] 0 0
Number of participants with any clinically significant vital sign value
Timepoint [3] 0 0
up to Week 106
Primary outcome [4] 0 0
Number of participants with any clinically significant physical examination procedure value
Timepoint [4] 0 0
up to Week 106
Primary outcome [5] 0 0
Number of participants with any clinically significant 12-lead electrocardiogram (ECG) value
Timepoint [5] 0 0
up to Week 106
Primary outcome [6] 0 0
Number of participants with any clinically significant effects on their menstruation cycle
Timepoint [6] 0 0
up to Week 105
Primary outcome [7] 0 0
Number of participants with suicidality, as assessed by the investigator via a clinical interview with the caregiver
Timepoint [7] 0 0
up to Week 106
Primary outcome [8] 0 0
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
Timepoint [8] 0 0
up to Week 106
Secondary outcome [1] 0 0
Change from prerandomization Baseline of the Randomized Controlled Trial (RCT) to Week 105 in Rett Syndrome Behaviour Questionnaire (RSBQ) scores
Timepoint [1] 0 0
from Baseline up to Week 105
Secondary outcome [2] 0 0
Change from prerandomization Baseline of the RCT to Week 105 in Clinical Global Impressions - Improvement (CGI-I) scores
Timepoint [2] 0 0
from Baseline up to Week 105
Secondary outcome [3] 0 0
Change from prerandomization Baseline of the RCT to Week 105 in Clinician Global Impressions - Severity Scale (CGI-S) scores
Timepoint [3] 0 0
from Baseline up to Week 105
Secondary outcome [4] 0 0
Change from prerandomization Baseline of the RCT to Week 105 in 9-item Motor Behavioral Assessment (MBA-9) scores
Timepoint [4] 0 0
from Baseline up to Week 105
Secondary outcome [5] 0 0
Change from prerandomization Baseline of the RCT to Week 105 in Children's Sleep Habits Questionnaire (CSHQ) scores
Timepoint [5] 0 0
from Baseline up to Week 105

Eligibility
Key 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.
Minimum age
2 Years
Maximum age
18 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Clinical Trial Site - Perth
Recruitment hospital [2] 0 0
Clinical Trial Site - South Brisbane
Recruitment postcode(s) [1] 0 0
- Perth
Recruitment postcode(s) [2] 0 0
- South Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
Missouri
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
Pennsylvania
Country [12] 0 0
United States of America
State/province [12] 0 0
South Carolina
Country [13] 0 0
United States of America
State/province [13] 0 0
Tennessee
Country [14] 0 0
United States of America
State/province [14] 0 0
Texas
Country [15] 0 0
Canada
State/province [15] 0 0
Toronto
Country [16] 0 0
Canada
State/province [16] 0 0
Vancouver
Country [17] 0 0
Italy
State/province [17] 0 0
Genova
Country [18] 0 0
Italy
State/province [18] 0 0
Messina
Country [19] 0 0
Italy
State/province [19] 0 0
Milano
Country [20] 0 0
Italy
State/province [20] 0 0
Rome
Country [21] 0 0
Italy
State/province [21] 0 0
Siena
Country [22] 0 0
Spain
State/province [22] 0 0
Barcelona
Country [23] 0 0
Spain
State/province [23] 0 0
Madrid
Country [24] 0 0
Spain
State/province [24] 0 0
Valencia
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Edinburgh
Country [26] 0 0
United Kingdom
State/province [26] 0 0
Liverpool
Country [27] 0 0
United Kingdom
State/province [27] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
GW Research Ltd
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This study will be conducted to evaluate the long-term safety of cannabidiol oral solution
(GWP42003-P, CBD-OS) in participants with Rett syndrome.
Trial website
https://clinicaltrials.gov/show/NCT04252586
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT04252586