Brief Title
An Open-label Extension Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
Official 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
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.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Number of participants with any adverse event
Secondary Outcome
Change from prerandomization Baseline of the Randomized Controlled Trial (RCT) to Week 105 in Rett Syndrome Behaviour Questionnaire (RSBQ) scores
Condition
Rett Syndrome
Intervention
GWP42003-P
Study Arms / Comparison Groups
GWP42003-P
Description: 100 milligrams per milliliter (mg/mL) GWP42003-P oral solution, taken twice daily (morning and evening).
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
21
Start Date
February 28, 2020
Completion Date
June 9, 2021
Primary Completion Date
June 9, 2021
Eligibility 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
Gender
All
Ages
2 Years - 18 Years
Accepts Healthy Volunteers
No
Contacts
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Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT04252586
Organization ID
GWND19002
Secondary IDs
2019-001605-24
Responsible Party
Sponsor
Study Sponsor
Jazz Pharmaceuticals
Study Sponsor
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Verification Date
August 2021