Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms (GWPCARE7)
- Registration Number
- NCT02953548
- Lead Sponsor
- Jazz Pharmaceuticals
- 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 available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 9
- 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
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description GWP42003-P 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.
- Primary Outcome Measures
Name Time Method Number of Participants With Severe Treatment-emergent Adverse Events (TEAEs) 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 Day 4 and Day 15 Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value Day 4 and Day 15 Number of Participant With Any Clinically Relevant Urinalysis Parameter Value Day 4 and Day 15 Clinical relevance was determined by the investigator.
Number of Participants With Clinically Significant Electrocardiogram Findings 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 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 From signing of informed consent up to Day 15 Clinical significance was determined by the investigator.
- Secondary Outcome Measures
Name Time Method Percentage of Participants Free of Clinical Spasms 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 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 Day 15 Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.
Number of Responders 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 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.
Number of Participants Free of Clinical Spasms Day 15 Clinical spasms were determined by video-electroencephalography (VEEG) for at least 8 hours and up to 24 hours.
Number of Participants Experiencing Spasms and Seizures by Subtype 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.
Caregiver Clinical Global Impression of Change (CGIC) 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) 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).
Average Time to Cessation of Spasms 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.
Trial Locations
- Locations (8)
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
The Childrens Hospital of San Antonio
🇺🇸San Antonio, Texas, United States
Wake Forest Baptist Medical Center
🇺🇸Winston-Salem, North Carolina, United States
Le Bonheur Children's Hospital
🇺🇸Memphis, Tennessee, United States
Uniwersyteckie Centrum Kliniczne
🇵🇱Gdańsk, Poland
Valley Health Clinical Research
🇺🇸Winchester, Virginia, United States
Centrum Medyczne POMOC
🇵🇱Łódź, Poland