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Trial record 78 of 249 for:    epidiolex

Phase 3 Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms: Open-label Extension Phase (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: NCT02954887
Recruitment Status : Completed
First Posted : November 4, 2016
Results First Posted : June 29, 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 open-label extension phase only will be described in this record. All participants will receive GWP42003-P.

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

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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 : May 12, 2017
Actual Primary Completion Date : June 13, 2019
Actual Study Completion Date : June 13, 2019


Arm Intervention/treatment
Experimental: GWP42003-P

Administered orally, up to the target dose recommended by the data safety monitoring committee.

Participants continue at the target dose, or the highest tolerated dose up to the target dose, for a total of 12 months' treatment.

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 417 ]
    TEAEs were collected in members of the Safety Population, comprised of all participants who received at least 1 dose of GWP42003-P. 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: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389 ]
  3. Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value [ Time Frame: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389 ]
  4. Number of Participants With Any Clinically Relevant Urinalysis Parameter Value [ Time Frame: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389 ]
    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 389 ]
    Clinical significance was determined by the investigator.

  6. Number of Participants With Clinically Significant Vital Sign Findings [ Time Frame: From signing of informed consent up to Day 389 ]
    Clinical significance was determined by the investigator.

  7. Number of Participants With Clinically Significant Physical Examination Findings [ Time Frame: From signing of informed consent up to Day 389 ]
    Clinical significance was determined by the investigator.


Secondary Outcome Measures :
  1. Number of Participants Free of Clinical Spasms [ Time Frame: Days 29, 43, 127, 211, 295, and 379 ]
    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: Days 29, 43, 127, 211, 295, and 379 ]
    Clinical spasms were determined by VEEG for at least 8 hours and up to 24 hours.

  3. Number of Participants With a Resolution of Hypsarrhythmia [ Time Frame: Days 29, 43, 127, 211, 295, and 379 ]
    Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.

  4. Percentage of Participants With a Resolution of Hypsarrhythmia [ Time Frame: Days 29, 43, 127, 211, 295, and 379 ]
    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: Days 19, 29, 127, 211, 295, and 379 ]
    Caregivers recorded the participant's spasms and seizures by category in a daily diary. Subtypes of spasms and seizure included, clonic, tonic-clonic, myoclonic, focal, and absence.

  6. Caregiver Global Impression of Change (CGIC) [ Time Frame: Baseline; Days 29, 43, 71, 127, 211, 295, and 379 ]
    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: 1, very much improved; 2, much Improved; 3, slightly improved; 4, no change; 5, slightly worse; 6, much worse; 7, very much worse.

  7. Physician Global Impression of Change (PGIC) [ Time Frame: Baseline; Days 29, 43, 71, 127, 211, 295, and 379 ]
    The PGIC is a single-question assessment completed by the investigator. The question assessed the status of the participant's condition since treatment start. The investigator provided a rating on a 7-point scale: 1, very much improved; 2, much Improved; 3, slightly improved; 4, no change; 5, slightly worse; 6, much worse; 7, very much worse.

  8. Number of Responders [ Time Frame: Days 29, 43, 127, 211, 295, and 379 ]
    A responder is defined as a participant experiencing a resolution of hypsarrhythmia and free of spasms. Test for responders was conducted by VEEG for at least 8 hours and up to 24 hours.

  9. Percentage of Responders [ Time Frame: Days 29, 43, 127, 211, 295, and 379 ]
    A responder is defined as a participant experiencing a resolution of hypsarrhythmia and free of spasms. Test for responders was conducted by VEEG for at least 8 hours and up to 24 hours.

  10. Change From Baseline in Height [ Time Frame: Baseline (Day 1 of Pilot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389 ]
    A positive change indicates an increase in the average participant's height. A negative change indicates a decrease in the average participant's height. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.

  11. Change From Baseline in Body Weight. [ Time Frame: Baseline (Day 1 of Pilot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389 ]
    A positive change indicates an increase in the average participant's weight. A negative change indicates a decrease in the average participant's weight. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.

  12. Change From Baseline in Head Circumference [ Time Frame: Baseline (Day 1 of PIlot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389 ]
    A positive change indicates an increase in the average participant's head circumference. A negative change indicates a decrease in the average participant's head circumference. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.

  13. Change From Baseline in Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) Score [ Time Frame: Baseline (Day 1 of Pilot Study); Day 211, Day 379 ]
    The Vineland-II scores were assessed by the participant's caregiver. Caregivers were asked to score questions in the following categories: the participant's communication, daily living, physical activity, problem behaviors, and social skills and relationships. Scoring was slightly different for each section, but generally ranged from "usually" (2) to "never" (0). The total score is calculated as the sum of standard scores from the domains and converted into the adaptive behavior composite score (ranging from 20 to 160). Higher scores represent greater levels of functioning, and lower scores represent lower levels of functioning. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.

  14. Number of Participants With Relapse of Spasms [ Time Frame: Day 16 to Day 379 ]
    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 for up to 1 year.

  15. Percentage of Participants With Relapse of Spasms [ Time Frame: Day 16 to Day 379 ]
    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 for up to 1 year.

  16. Average Time to Cessation of Spasms [ Time Frame: Day 1 to Day 379 ]
    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 for up to 1 year.

  17. Average Time to Relapse [ Time Frame: Day 16 to Day 379 ]
    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 for up to 1 year.



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.


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

Only participants who completed the pilot or pivotal phases of the trial may proceed to take part in this open-label extension phase of the trial.

Key eligibility criteria for the blinded phase were as follows:

Key Inclusion Criteria:

  • Participant is diagnosed with IS and has failed to respond adequately following treatment with 1 or more approved IS therapies.

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): NCT02954887


Locations
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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, 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

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Responsible Party: GW Research Ltd
ClinicalTrials.gov Identifier: NCT02954887    
Other Study ID Numbers: GWEP15100 Open-label Extension
2015-004904-50 ( EudraCT Number )
First Posted: November 4, 2016    Key Record Dates
Results First Posted: June 29, 2020
Last Update Posted: July 23, 2020
Last Verified: July 2020

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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 GW Research Ltd:
GWP42003-P
Cannabidiol
Additional relevant MeSH terms:
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Epidiolex
Spasm
Spasms, Infantile
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Epilepsy, Generalized
Epilepsy
Brain Diseases
Central Nervous System Diseases
Epileptic Syndromes
Anticonvulsants