Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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

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.

Condition or disease Intervention/treatment Phase
Infantile Spasms Drug: GWP42003-P Phase 3

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 9 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Masking Description: Open-label
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : April 24, 2017
Actual Primary Completion Date : May 7, 2018
Actual Study Completion Date : May 7, 2018


Arm Intervention/treatment
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.
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




Primary Outcome Measures :
  1. 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.

  2. Number of Participants With Any Low or High Hematology Laboratory Parameter Value [ Time Frame: Day 4 and Day 15 ]
  3. Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value [ Time Frame: Day 4 and Day 15 ]
  4. Number of Participant With Any Clinically Relevant Urinalysis Parameter Value [ Time Frame: Day 4 and Day 15 ]
    Clinical relevance was determined by the investigator.

  5. 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.

  6. 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.

  7. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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).

  8. 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).

  9. 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.

  10. 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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   1 Month to 24 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

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): NCT02953548


Locations
Layout table for location information
United States, Arkansas
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
United States, North Carolina
Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, United States, 27157
United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
United States, Tennessee
Le Bonheur Children's Hospital
Memphis, Tennessee, United States, 38103
United States, Texas
The Childrens Hospital of San Antonio
San Antonio, Texas, United States, 78207
United States, Virginia
Valley Health Clinical Research
Winchester, Virginia, United States, 22601
Poland
Uniwersyteckie Centrum Kliniczne
Gdańsk, Poland
Centrum Medyczne POMOC
Łódź, Poland
Sponsors and Collaborators
GW Research Ltd
  Study Documents (Full-Text)

Documents provided by GW Research Ltd:
Study Protocol  [PDF] September 20, 2016
Statistical Analysis Plan  [PDF] September 25, 2019

Layout table for additonal information
Responsible Party: GW Research Ltd
ClinicalTrials.gov Identifier: NCT02953548    
Other Study ID Numbers: GWEP15100 Pilot Phase
2015-004904-50 ( EudraCT Number )
First Posted: November 2, 2016    Key Record Dates
Results First Posted: July 23, 2020
Last Update Posted: July 23, 2020
Last Verified: July 2020

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by GW Research Ltd:
GWP42003-P
Cannabidiol
Additional relevant MeSH terms:
Layout table for MeSH terms
Epidiolex
Spasm
Spasms, Infantile
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Epilepsy, Generalized
Epilepsy
Brain Diseases
Central Nervous System Diseases
Epileptic Syndromes
Anticonvulsants