A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
Condition: Rett Syndrome; RTT
MeSH terms: Rett Syndrome; Syndrome
Intervention: GWP42003-P
Intervention description: GWP42003-P presented as an oral solution containing cannabidiol in the excipients sesame oil with anhydrous ethanol, sweetener (sucralose), and strawberry flavoring
Intervention MeSH terms: Cannabidiol
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 21.0
Sex: All
Minimum age: 2 Years
Maximum age: 18 Years
Healthy volunteers: No
Keywords: Cannabidiol; CBD; Epidiolex; GWP42003-P

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.
    

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