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Trial to Investigate the Safety and Efficacy of Cannabidiol Oral Solution (GWP42003-P; CBD-OS) in Children and Adolescents With Autism Spectrum Disorder

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ClinicalTrials.gov Identifier: NCT04745026
Recruitment Status : Recruiting
First Posted : February 9, 2021
Last Update Posted : March 10, 2023
Sponsor:
Information provided by (Responsible Party):
Jazz Pharmaceuticals

Study Description
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Brief Summary:
This study will be conducted to evaluate the efficacy of GWP42003-P, compared with placebo, in reducing symptom severity in children with Autism Spectrum Disorder (ASD).

Condition or disease Intervention/treatment Phase
Autism Spectrum Disorder Drug: GWP42003-P Drug: Placebo Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 160 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: An Exploratory, Phase 2, Randomized, Double-blind, Placebo-controlled Trial to Investigate the Safety and Efficacy of Cannabidiol Oral Solution (GWP42003-P; CBD-OS) in Children and Adolescents With Autism Spectrum Disorder
Actual Study Start Date : May 31, 2021
Estimated Primary Completion Date : February 28, 2023
Estimated Study Completion Date : March 30, 2023

Resource links provided by the National Library of Medicine

Drug Information available for: Cannabidiol

Arms and Interventions
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Arm Intervention/treatment
Experimental: GWP42003-P 10 mg/kg/day
Participants will be stratified based on their age (6 to 11 years old, 12 to 17 years old), use of antipsychotics (on versus off), and region (North America versus Rest of the World) and will be randomized to receive 5 milligrams per kilogram per day (mg/kg/day) GWP42003-P for 1 week and then 10 mg/kg/day GWP42003-P for 11 weeks.
 Drug: GWP42003-P
GWP42003-P oral solution (100 milligrams per milliliter [mg/mL] cannabidiol [CBD] in sesame oil with anhydrous ethanol, ethanol [10% v/v] sweetener [sucralose], and strawberry flavoring), administered twice a day (morning and evening)
Other Names:
  • Epidiolex®
  • cannabidiol
  • CBD-OS
Placebo Comparator: Placebo
Participants will be stratified based on their age (6 to 11 years old, 12 to 17 years old), use of antipsychotics (on versus off), and region (North America versus Rest of the World) and will be randomized to receive matching placebo for 12 weeks.
 Drug: Placebo
Oral placebo to match GWP42003-P oral solution containing sesame oil with anhydrous ethanol, sweetener (sucralose), strawberry flavoring, and beta carotene, administered twice a day (morning and evening)


Outcome Measures
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Primary Outcome Measures :
  1. Change from Screening in Aberrant Behavior Checklist (ABC) Subscale Scores [ Time Frame: Screening; Day 85 ]
  2. Change from Day 1 in Vineland Adaptive Behavior Scales-3 (VABS-3) Scores [ Time Frame: Day 1; Day 85 ]
  3. Change from Day 29 in Clinical Global Impression Improvement (CGI-I) Scores [ Time Frame: Day 29; Day 85 ]
  4. Change from Screening in Clinical Global Impression Severity (CGI-S) Scores [ Time Frame: Screening; Day 85 ]

Secondary Outcome Measures :
  1. Number of Participants with Severe Treatment-emergent Adverse Events [ Time Frame: up to Day 106 ]
  2. Number of Participants with Clinically Significant Clinical Laboratory Parameter Values [ Time Frame: up to Day 92 ]
  3. Number of Participants with Clinically Significant Vital Sign Values [ Time Frame: up to Day 92 ]
  4. Number of Participants with Clinically Significant Physical Examination Procedure Findings [ Time Frame: up to Day 85 ]
  5. Number of Participants with Clinically Significant 12-lead Electrocardiogram Findings [ Time Frame: up to Day 85 ]
  6. Number of Participants with a Positive Response to Questions Regarding Suicidal Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS) [ Time Frame: up to Day 92 ]

Eligibility Criteria
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Ages Eligible for Study:   6 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Participant weight is at least 12 kilograms (kg).
  • Participants (if possessing adequate understanding, in the investigator's opinion) and their parent(s)/legal representative are willing and able to give informed assent and consent for participation in the trial.
  • Participant and their caregiver are willing and able (in the investigator's opinion) to comply with all trial requirements.
  • Participant has a diagnosis of Autism Spectrum Disorder (ASD) as per Diagnostic and Statistical Manual of Mental Disorders, 5th Edition (DSM-5) criteria for ASD, confirmed by Autism Diagnostic Observational Schedule (ADOS-2) criteria (conducted within 2 years at the trial site or at screening by a qualified assessor). Note: During special circumstances (e.g., COVID-19 pandemic) where the ADOS-2 cannot be performed due to site restrictions (e.g., mandatory use of face masks) and an ADOS- 2 conducted within 2 years at the trial site by a qualified assessor is not available, eligibility can be confirmed using: 1) an ADOS-2 performed within 2 years by a qualified assessor (external to the site); 2) if 1) is not available, eligibility may be confirmed using the Autism Diagnostic Interview, Revised (ADI-R) at screening.
  • Clinical Global Impressions - Improvement Scale (CGI-S) ≥ 4 (moderately ill) at screening and randomization.
  • Aberrant Behavior Checklist Irritability (ABC-I) subscale score ≥ 15 at screening.
  • Intelligence quotient (IQ) ≥ 70 at screening, or measured within 1 year of screening, using Wechsler Abbreviated Scale of Intelligence Scale Second Edition (WASI-II).
  • All medications or interventions (including psychosocial interventions, dietary supplements, probiotics, speech therapy, etc.) for ASD related symptoms must have been stable for 4 weeks prior to screening and randomization, and the patient/caregiver should be willing to maintain a stable regimen throughout the trial.
  • Participants must have the ability to swallow the investigational medicinal product (IMP), provided as a liquid solution.
  • 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/are willing to allow the participant's primary care practitioner (if they have one) and consultant (if they have one) to be notified of participation in the trial if the primary care practitioner/consultant is different from the investigator.

Exclusion Criteria:

  • Current diagnosis of bipolar disorder, psychosis, schizophrenia, schizoaffective disorder, or major depression (participants with depression in remission may be included)
  • Has a diagnosis other than ASD that dominates the clinical presentation (e.g., Attention Deficit Hyperactivity Disorder [ADHD])
  • Has a progressive neurological condition
  • Seizures in the past 24 weeks
  • Changes in anticonvulsive therapy within the last 12 weeks
  • Currently taking more than 2 anti-epileptic drugs (AEDs)
  • Taking sirolimus, everolimus, temsirolimus, or tacrolimus
  • Taking clobazam
  • Taking omeprazole, lansoprazole, tolbutamide, or warfarin
  • Taking repaglinide, pioglitazone, rosiglitazone, montelukast, bupropion, or efavirenz
  • Currently using or has used recreational or medicinal cannabis, cannabinoid-based medications (including Sativex®, or Epidiolex®) within the 12 weeks prior to screening and is unwilling to abstain for the duration of the trial
  • Participant has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the IMP, such as sesame oil.
  • Participant has moderately impaired hepatic function at screening, defined as serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2 × upper limit of normal (ULN) or total bilirubin (TBL) > 2 × ULN. This criterion can only be confirmed once the laboratory results are available; participants enrolled into the trial who are later found to meet this criterion must be screen-failed.
  • Participant is male and fertile (i.e., after puberty unless permanently sterile by bilateral orchidectomy) unless willing to ensure that they use male contraception (condom) or remain sexually abstinent during the trial and for 12 weeks thereafter.
  • Participant is female and of childbearing potential (i.e., following menarche and until becoming postmenopausal for ≥ 12 consecutive months unless permanently sterile by hysterectomy, bilateral salpingectomy, or bilateral oophorectomy) unless willing to ensure that they use a highly effective method of birth control (e.g., hormonal contraception, intrauterine device/hormone-releasing system, bilateral tubal occlusion, vasectomized partner, sexual abstinence) during the trial and for 12 weeks thereafter.
  • Female participant who is pregnant (positive pregnancy test), lactating or planning pregnancy during the course of the trial or within 12 weeks thereafter.
  • Participant has received an IMP within the 12 weeks prior to the screening visit.
  • Participant had brain surgery or traumatic brain injury within 1 year of screening.
  • Participant has any other significant disease or disorder which, in the opinion of the investigator, may either put the participant, other participants, or site staff at risk because of participation in the trial, may influence the result of the trial, or may affect the participant's ability to take part in the trial.
  • Any abnormalities identified following a physical examination of the participant that, in the opinion of the investigator, would jeopardize the safety of the participant if they took part in the trial
  • Any history of suicidal behavior (lifelong) or any suicidal ideation of type 4 or 5 on the Columbia-Suicide Severity Rating Scale (C-SSRS) in the last 4 weeks or at screening or randomization
  • Participant has donated blood during the past 12 weeks and is unwilling to abstain from donation of blood during the trial.
  • Participant has any known or suspected history of alcohol or substance abuse or positive drugs of abuse test at screening (not justified by a known concurrent medication).
  • Participant has previously been randomized into this trial.
  • Participant has plans to travel outside their country of residence during the trial, unless the participant has confirmation that the IMP is permitted in the destination country/state
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04745026


Contacts
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Contact: Clinical Trial Disclosure & Transparency 215-832-3750 ClinicalTrialDisclosure@JazzPharma.com

Locations
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Sponsors and Collaborators
Jazz Pharmaceuticals
More Information
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Responsible Party: Jazz Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04745026    
Other Study ID Numbers: GWND19189
2020-002819-21 ( EudraCT Number )
First Posted: February 9, 2021    Key Record Dates
Last Update Posted: March 10, 2023
Last Verified: March 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Jazz Pharmaceuticals:
Cannabidiol oral solution
CBD-OS
GWP42003-P
Children
Adolescents
Additional relevant MeSH terms:
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Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
 Mental Disorders
Cannabidiol
Anticonvulsants