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Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms (GWPCARE7)

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ClinicalTrials.gov Identifier: NCT02953548
Recruitment Status : Completed
First Posted : November 2, 2016
Results First Posted : July 23, 2020
Last Update Posted : July 23, 2020
Sponsor:
Information provided by (Responsible Party):
GW Research Ltd

Tracking Information
First Submitted Date  ICMJE November 1, 2016
First Posted Date  ICMJE November 2, 2016
Results First Submitted Date  ICMJE June 10, 2020
Results First Posted Date  ICMJE July 23, 2020
Last Update Posted Date July 23, 2020
Actual Study Start Date  ICMJE April 24, 2017
Actual Primary Completion Date May 7, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 10, 2020)
  • Number of Participants With Severe Treatment-emergent Adverse Events (TEAEs) [ Time Frame: From signing of informed consent up to Day 15 ]
    TEAEs are defined as all adverse events not present prior to the first investigational medicinal product (IMP) or placebo administration or any event already present that worsened in severity or frequency following IMP.
  • Number of Participants With Any Low or High Hematology Laboratory Parameter Value [ Time Frame: Day 4 and Day 15 ]
  • Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value [ Time Frame: Day 4 and Day 15 ]
  • Number of Participant With Any Clinically Relevant Urinalysis Parameter Value [ Time Frame: Day 4 and Day 15 ]
    Clinical relevance was determined by the investigator.
  • Number of Participants With Clinically Significant Electrocardiogram Findings [ Time Frame: From signing of informed consent up to Day 15 ]
    Clinical significance was determined by the investigator.
  • Number of Participants With Clinically Significant Physical Examination Findings [ Time Frame: From signing of informed consent up to Day 15 ]
    Clinical significance was determined by the investigator.
  • Number of Participants With Clinically Significant Vital Sign Findings [ Time Frame: From signing of informed consent up to Day 15 ]
    Clinical significance was determined by the investigator.
Original Primary Outcome Measures  ICMJE
 (submitted: November 1, 2016)
  • Number of participants who experienced a treatment-emergent adverse event. [ Time Frame: 2 weeks ]
    The number of participants who experienced an adverse event that started, or worsened in severity or seriousness, following the first dose of study drug is presented.
  • Number of participants with a potentially clinically significant change in clinical laboratory tests. [ Time Frame: 2 weeks ]
    The number of participants with a potentially clinically significant change in clinical laboratory tests is presented.
  • Number of participants with a clinically significant change in 12-lead electrocardiogram (ECG). [ Time Frame: 2 weeks ]
    The number of participants with a clinically significant change in ECG is presented.
  • Number of participants with a clinically significant change in vital signs. [ Time Frame: 2 weeks ]
    The number of participants with a clinically significant change in vital signs (systolic and diastolic blood pressure, pulse rate, body temperature and respiratory rate) is presented.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 10, 2020)
  • Number of Participants Free of Clinical Spasms [ Time Frame: Day 15 ]
    Clinical spasms were determined by video-electroencephalography (VEEG) for at least 8 hours and up to 24 hours.
  • Percentage of Participants Free of Clinical Spasms [ Time Frame: Day 15 ]
    Clinical spasms were determined by VEEG for at least 8 hours and up to 24 hours.
  • Number of Participants With Resolution of Hypsarrhythmia [ Time Frame: Day 15 ]
    Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.
  • Percentage of Participants With Resolution of Hypsarrhythmia [ Time Frame: Day 15 ]
    Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.
  • Number of Participants Experiencing Spasms and Seizures by Subtype [ Time Frame: Day 4 and Day 15 ]
    Caregivers recorded the participant's spasms and seizures by category in a daily diary. Subtypes of spasms and seizures included: clonic, tonic-clonic, myoclonic, focal, and absence.
  • Average Time to Cessation of Spasms [ Time Frame: Day 1 to start of Open-label Extension (OLE) Phase ]
    Analysis could not be conducted for this outcome measure because the study met No Go Criteria. The Pilot Phase concluded after 9 participants completed treatment and demonstrated continued hypsarrhythmia and spasms on follow-up VEEG. The Pivotal Phase was not initiated; however, participants completing the Pilot Phase could roll into the Open Label Extension Phase (NCT02954887) for up to 1 year.
  • Caregiver Clinical Global Impression of Change (CGIC) [ Time Frame: Day 15 ]
    The CGIC is a single-question assessment completed by the caregiver. The question assessed the status of the participant's condition since treatment start. The caregiver provided a rating on a 7-point scale from 1 (very much improved) to 7 (very much worse).
  • Physician Global Impression of Change (PGIC) [ Time Frame: Day 15 ]
    The PGIC is a single-question assessment completed by the investigator. The question assesses the status of the participant's condition since treatment start. The investigator provided a rating on a 7-point scale from 1 (very much improved) to 7 (very much worse).
  • Number of Responders [ Time Frame: Baseline to Day 15 ]
    A responder is defined as a participant experiencing a resolution of hypsarrhythmia and free of spasms. Testing for responders was conducted by VEEG for at least 8 hours and up to 24 hours.
  • Percentage of Responders [ Time Frame: Baseline to Day 15 ]
    A responder is defined as a participant experiencing a resolution of hypsarrhythmia and free of spasms. Testing for responders was conducted by VEEG for at least 8 hours and up to 24 hours.
Original Secondary Outcome Measures  ICMJE
 (submitted: November 1, 2016)
  • Number of treatment responders. [ Time Frame: 2 weeks ]
    The number of participants who are free of spasms and have resolution of hypsarrhythmia is presented.
  • Number of participants who are spasm-free. [ Time Frame: 2 weeks ]
    The number of participants who are free of clinical spasms is presented.
  • Number of participants without hypsarrhythmia. [ Time Frame: 2 weeks ]
    The number of participants with resolution of hypsarrhythmia is presented.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms (GWPCARE7)
Official Title  ICMJE A Randomized, Double-blind, Placebo-controlled Trial to Investigate the Efficacy and Safety of Cannabidiol (CBD; GWP42003-P) in Infants With Infantile Spasms Following an Initial Open-label Pilot Study
Brief Summary This trial consists of 3 parts: a pilot safety phase, a pivotal randomized controlled phase, and an open-label extension phase. The pilot phase only will be described in this record. 2 cohorts of 5 participants will be enrolled sequentially. All participants will receive GWP42003-P.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Masking Description:
Open-label
Primary Purpose: Treatment
Condition  ICMJE Infantile Spasms
Intervention  ICMJE Drug: GWP42003-P
Clear, colorless to yellow solution containing cannabidiol dissolved in the excipients sesame oil and anhydrous ethanol with added sweetener (sucralose) and strawberry flavoring.
Other Names:
  • CBD
  • Cannabidiol
Study Arms  ICMJE Experimental: GWP42003-P
Administered orally, titrating to a target dose of 40 mg/kg/day. Participants continue at the target dose, or the highest tolerated dose up to the target dose, for the remainder of the 2-week treatment period.
Intervention: Drug: GWP42003-P
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 Completed
Actual Enrollment  ICMJE
 (submitted: April 23, 2020)
9
Original Estimated Enrollment  ICMJE
 (submitted: November 1, 2016)
10
Actual Study Completion Date  ICMJE May 7, 2018
Actual Primary Completion Date May 7, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • Participant is aged 6- 24 months (inclusive) in the first cohort or aged 1-24 months (inclusive) in the second cohort, at the time of consent.
  • Participant is diagnosed with IS and has failed to respond adequately following treatment with 1 or more approved IS therapies.
  • To be considered hypsarrhythmia, as defined for use in the study, the electroencephalography (EEG) background must be slowed and have multifocal spikes. In addition, it must be either high voltage (above 300 µV) or have electrodecrement/discontinuity.

Key Exclusion Criteria:

  • Participant is currently taking or has taken clobazam or any mammalian target of rapamycin (mTOR) inhibitor within the 2 weeks prior to the screening visit.
  • Participant has a QT interval, corrected for heart rate with Bazett's formula (QTcB), of 460 msec or greater on ECG.
  • Participant's caregiver is currently giving or has given recreational or medicinal cannabis, or synthetic cannabinoid-based medications, within the 1 month prior to the screening visit.
  • Participant's caregiver is unwilling to abstain from giving the participant (including the participant's mother abstaining themselves, if breastfeeding)recreational or medicinal cannabis, or synthetic cannabinoid-based medications (other than the study drug) during the trial.
  • Participant has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the study drug, such as sesame oil.
  • Participant has significantly impaired hepatic function at the screening visit.
  • Participant has received an investigational medicinal product as part of a clinical trial within a minimum of 5 half-lives prior to the screening visit.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Month to 24 Months   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Poland,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02953548
Other Study ID Numbers  ICMJE GWEP15100 Pilot Phase
2015-004904-50 ( 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 July 2020

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