- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02953548
Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms (GWPCARE7)
August 31, 2022 updated by: Jazz Pharmaceuticals
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
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.
Study Overview
Study Type
Interventional
Enrollment (Actual)
9
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Gdańsk, Poland
- Uniwersyteckie Centrum Kliniczne
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Łódź, Poland
- Centrum Medyczne POMOC
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Arkansas
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Little Rock, Arkansas, United States, 72202
- Arkansas Children's Hospital
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North Carolina
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Winston-Salem, North Carolina, United States, 27157
- Wake Forest Baptist Medical Center
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Ohio
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Columbus, Ohio, United States, 43205
- Nationwide Children's Hospital
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Tennessee
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Memphis, Tennessee, United States, 38103
- Le Bonheur Children's Hospital
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Texas
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San Antonio, Texas, United States, 78207
- The Childrens Hospital of San Antonio
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Virginia
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Winchester, Virginia, United States, 22601
- Valley Health Clinical Research
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
1 month to 2 years (CHILD)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NA
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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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.
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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:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Participants With Severe Treatment-emergent Adverse Events (TEAEs)
Time Frame: From signing of informed consent up to Day 15
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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.
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From signing of informed consent up to Day 15
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Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Time Frame: Day 4 and Day 15
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Day 4 and Day 15
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Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Time Frame: Day 4 and Day 15
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Day 4 and Day 15
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Number of Participant With Any Clinically Relevant Urinalysis Parameter Value
Time Frame: Day 4 and Day 15
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Clinical relevance was determined by the investigator.
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Day 4 and Day 15
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Number of Participants With Clinically Significant Electrocardiogram Findings
Time Frame: From signing of informed consent up to Day 15
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Clinical significance was determined by the investigator.
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From signing of informed consent up to Day 15
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Number of Participants With Clinically Significant Physical Examination Findings
Time Frame: From signing of informed consent up to Day 15
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Clinical significance was determined by the investigator.
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From signing of informed consent up to Day 15
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Number of Participants With Clinically Significant Vital Sign Findings
Time Frame: From signing of informed consent up to Day 15
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Clinical significance was determined by the investigator.
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From signing of informed consent up to Day 15
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Participants Free of Clinical Spasms
Time Frame: Day 15
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Clinical spasms were determined by video-electroencephalography (VEEG) for at least 8 hours and up to 24 hours.
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Day 15
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Percentage of Participants Free of Clinical Spasms
Time Frame: Day 15
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Clinical spasms were determined by VEEG for at least 8 hours and up to 24 hours.
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Day 15
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Number of Participants With Resolution of Hypsarrhythmia
Time Frame: Day 15
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Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.
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Day 15
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Percentage of Participants With Resolution of Hypsarrhythmia
Time Frame: Day 15
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Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.
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Day 15
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Number of Participants Experiencing Spasms and Seizures by Subtype
Time Frame: Day 4 and Day 15
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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.
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Day 4 and Day 15
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Average Time to Cessation of Spasms
Time Frame: Day 1 to start of Open-label Extension (OLE) Phase
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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.
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Day 1 to start of Open-label Extension (OLE) Phase
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Caregiver Clinical Global Impression of Change (CGIC)
Time Frame: Day 15
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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).
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Day 15
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Physician Global Impression of Change (PGIC)
Time Frame: Day 15
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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).
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Day 15
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Number of Responders
Time Frame: Baseline to Day 15
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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.
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Baseline to Day 15
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Percentage of Responders
Time Frame: Baseline to Day 15
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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.
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Baseline to Day 15
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (ACTUAL)
April 24, 2017
Primary Completion (ACTUAL)
May 7, 2018
Study Completion (ACTUAL)
May 7, 2018
Study Registration Dates
First Submitted
November 1, 2016
First Submitted That Met QC Criteria
November 1, 2016
First Posted (ESTIMATE)
November 2, 2016
Study Record Updates
Last Update Posted (ACTUAL)
September 2, 2022
Last Update Submitted That Met QC Criteria
August 31, 2022
Last Verified
August 1, 2022
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- GWEP15100 Pilot Phase
- 2015-004904-50 (EUDRACT_NUMBER)
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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