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Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome (ARCH)

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ClinicalTrials.gov Identifier: NCT03848832
Recruitment Status : Recruiting
First Posted : February 21, 2019
Last Update Posted : August 20, 2019
Sponsor:
Information provided by (Responsible Party):
GW Research Ltd

Tracking Information
First Submitted Date  ICMJE February 19, 2019
First Posted Date  ICMJE February 21, 2019
Last Update Posted Date August 20, 2019
Actual Study Start Date  ICMJE July 29, 2019
Estimated Primary Completion Date August 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 19, 2019)
  • Rett Syndrome Behaviour Questionnaire (RSBQ) [ Time Frame: 24 weeks ]
    RSBQ is a caregiver-completed questionnaire that measures the frequency of current disease characteristics (45 items); each item rated on a 3-point scale (0-2). Higher scores represent greater severity.
  • Clinical Global Impressions - Improvement (CGI-I) [ Time Frame: 24 weeks ]
    CGI-I is a 7-point scale that requires the clinician to assess how much a patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention. (1 = very much improved, to 7 = very much worse.)
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03848832 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: February 19, 2019)
  • Rett Syndrome Behavior Questionnaire subscales [ Time Frame: 24 weeks ]
    The 45 items in the caregiver-completed Rett Syndrome Behavior Questionnaire encompass 8 subscales: 1) general mood, 2) breathing problems, 3) hand behaviors, 4) face movements, 5) body rocking/expressionless face, 6) night-time behaviors, 7) anxiety/fear, 8) walking/standing. Higher scores represent greater severity.
  • Clinician Global Impressions - Severity Scale (CGI-S) [ Time Frame: 24 weeks ]
    CGI-S is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment relative to the clinician's experience with patients who have the same diagnosis, rating from 1 = normal, not at all ill, to 7 = extremely ill.
  • 9-items Motor Behavioral Assessment (MBA-9) [ Time Frame: 24 weeks ]
    MBA-9 was derived from the full MBA scale (37 Rett syndrome symptoms) by selecting the items deemed amenable to change and which reflect areas of meaningful clinical change. The severity of current symptoms are rated by the investigator on a 5-point numerical scale, from 0 = normal or never, to 4 = very severe or constant. Higher total scores represent greater severity.
  • Children's Sleep Habits Questionnaire (CSHQ) [ Time Frame: 24 weeks ]
    CSHQ is a caregiver-completed sleep screening instrument designed for school-aged children, including 33 items within 8 subscales reflecting the following sleep domains:1) bedtime resistance, 2) sleep onset delay, 3) sleep duration, 4) sleep anxiety, 5) night wakings, 6) parasomnias, 7) sleep-disordered breathing, 8) daytime sleepiness. Each item is answered with 1 of 3 markers: "usually" for 5 or more times a week, "sometimes" for 2-4 times a week, and "rarely" for never or 1 time a week. Higher scores reflect more disturbed sleep behaviour.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
Official Title  ICMJE A Randomized, Double-blind, Placebo-controlled Trial to Investigate the Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
Brief Summary 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Rett Syndrome
  • RTT
Intervention  ICMJE
  • 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
  • Drug: Placebo
    Placebo oral solution containing the excipients sesame oil and anhydrous ethanol with added beta-carotene, sweetener (sucralose) and strawberry flavoring.
Study Arms  ICMJE
  • Experimental: 5 mg/kg/day GWP42003-P
    100 mg/mL GWP42003-P oral solution. Taken twice daily (morning and evening).
    Intervention: Drug: GWP42003-P
  • Experimental: 15 mg/kg/day GWP42003-P
    100 mg/mL GWP42003-P oral solution. Taken twice daily (morning and evening).
    Intervention: Drug: GWP42003-P
  • 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).
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: February 19, 2019)
252
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE September 2021
Estimated Primary Completion Date August 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key 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.

Key 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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Female
Ages  ICMJE 2 Years to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Medical Information +44 (0) 1223 266 800 medinfo@gwpharm.com, medinfo@greenwichbiosciences.com
Listed Location Countries  ICMJE United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03848832
Other Study ID Numbers  ICMJE GWND18064
2018-003370-27 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party GW Research Ltd
Study Sponsor  ICMJE GW Research Ltd
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account GW Research Ltd
Verification Date August 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP