Purpose

To evaluate the efficacy of cannabidiol oral solution (GWP42003-P, CBD-OS) in reducing symptom severity when compared with placebo, in patients with Rett syndrome.

Conditions

Eligibility

Eligible Ages
Between 2 Years and 18 Years
Eligible Genders
Female
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Patient (if possessing adequate understanding, in the investigator's opinion) and/or her parent(s)/legal representative is willing and able to give informed consent/assent for participation in the trial.
  • Patient and her 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 weigh at least 9 kg.
  • Clinical diagnosis of Rett syndrome (typical or atypical), defined according to RettSearch Consortium criteria.
  • Confirmed genetic mutation of the MECP2 gene.
  • Patient must be post-regression (≥ 6 months since last loss of hand use or verbal language or gross motor regression).
  • All medications or interventions (including antiepileptic drugs [AEDs] and non-pharmacological interventions - dietary supplements, probiotics, physical therapy, speech therapy, etc.) for Rett syndrome-related symptoms must have been stable for 4 weeks prior to screening and the patient/caregiver must be willing to maintain a stable regimen throughout the trial.
  • Ability to swallow the investigational medicinal product (IMP) provided as a liquid solution, or the ability for 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 she has one) and consultant (if she has one) to be notified of participation in the trial, if the primary care practitioner/consultant is different to the investigator.

Exclusion Criteria

  • Patient meets exclusion criteria for Rett syndrome diagnosis (traumatic brain injury, neurometabolic disease, or severe infection that causes neurological problems; grossly abnormal psychomotor development in the first 6 months of life).
  • Patient has clinically significant abnormal laboratory values, in the investigator's opinion.
  • Patient is taking more than 2 concurrent AEDs.
  • Any history of suicidal behavior or any suicidal ideation in the last month or at screening.
  • Clinically relevant abnormalities in the ECG measured at screening or randomization.
  • Concurrent cardiovascular conditions which will, in the investigator's opinion, interfere with the ability to assess her ECGs or put the patient at risk because of participation in the trial.
  • First or second degree relative with a history of significant ECG abnormalities, in the opinion of the investigator (e.g. cardiac arrest, sudden death).
  • Any known or suspected hypersensitivity to cannabinoids or any of the excipients of the IMP (active or placebo), such as sesame oil.
  • Patient has significantly impaired hepatic function at screening, defined as alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3 × upper limit of normal (ULN) or total bilirubin > 2 × ULN.
  • Pregnant (positive pregnancy test) or lactating.
  • Received an IMP within the 3 months prior to screening.
  • Patient has been taking felbamate for less than 1 year prior to screening.
  • Currently using or has used recreational or medicinal cannabis, cannabinoid-based medications (including Sativex®), or cannabidiol oral solutions (including CBD-OS [GWP42003-P]) within the 3 months prior to screening and is unwilling to abstain for the duration of the trial.
  • Patient has a positive delta-9-tetrahydrocannabinol (THC) test at screening.
  • Any other systemic dysfunction (e.g., gastrointestinal, renal, respiratory) or significant disease or disorder which, in the opinion of the investigator, may either put the patient at risk because of participation in the trial, may influence the result of the trial, or the patient's ability to participate in the trial.
  • Any abnormalities identified following a physical examination of the patient that, in the opinion of the investigator, would jeopardize the safety of the patient if she took part in the trial.
  • Patient has been previously randomized into this trial.
  • Patient has travel outside the country of residence planned during the trial.

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
5 mg/kg/day GWP42003-P
100 mg/mL GWP42003-P oral solution. Taken twice daily (morning and evening).
  • Drug: GWP42003-P
    GWP42003-P presented as an oral solution containing cannabidiol in the excipients sesame oil and anhydrous ethanol with added sweetener (sucralose) and strawberry flavoring.
    Other names:
    • Cannabidiol
    • CBD
    • Epidiolex
    • CBD-OS
Experimental
15 mg/kg/day GWP42003-P
100 mg/mL GWP42003-P oral solution. Taken twice daily (morning and evening).
  • Drug: GWP42003-P
    GWP42003-P presented as an oral solution containing cannabidiol in the excipients sesame oil and anhydrous ethanol with added sweetener (sucralose) and strawberry flavoring.
    Other names:
    • Cannabidiol
    • CBD
    • Epidiolex
    • CBD-OS
Placebo Comparator
Placebo
Placebo oral solution (0 mg/mL GWP42003-P) volume matched to 5 mg/kg/day or 15 mg/kg/day GWP42003-P. Taken twice daily (morning and evening).
  • Drug: Placebo
    Placebo oral solution containing the excipients sesame oil and anhydrous ethanol with added beta-carotene, sweetener (sucralose) and strawberry flavoring.

Recruiting Locations

Montefiore Medical Center/Albert Einstein College of Medicine
Bronx, New York 10467

More Details

Status
Recruiting
Sponsor
GW Research Ltd

Study Contact

Medical Information
+44 (0) 1223 266 800
medinfo@gwpharm.com, medinfo@greenwichbiosciences.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.