Clinical trial NCT02953548: Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms (GWPCARE7)
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Infantile Spasms
Keywords: GWP42003-P
Cannabidiol
Lead sponsor: Jazz Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2016-11-02
Enrollment: 9
Sex: ALL
Minimum age (years): 1
Maximum age (years): 24
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Number of Participants With Severe Treatment-emergent Adverse Events (TEAEs)
timeFrame: From signing of informed consent up to Day 15
measure: Number of Participants With Any Low or High Hematology Laboratory Parameter Value
timeFrame: Day 4 and Day 15
measure: Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
timeFrame: Day 4 and Day 15
measure: Number of Participant With Any Clinically Relevant Urinalysis Parameter Value
timeFrame: Day 4 and Day 15
measure: Number of Participants With Clinically Significant Electrocardiogram Findings
timeFrame: From signing of informed consent up to Day 15
measure: Number of Participants With Clinically Significant Physical Examination Findings
timeFrame: From signing of informed consent up to Day 15
measure: Number of Participants With Clinically Significant Vital Sign Findings
timeFrame: From signing of informed consent up to Day 15
Secondary outcomes:
measure: Number of Participants Free of Clinical Spasms
timeFrame: Day 15
measure: Percentage of Participants Free of Clinical Spasms
timeFrame: Day 15
measure: Number of Participants With Resolution of Hypsarrhythmia
timeFrame: Day 15
measure: Percentage of Participants With Resolution of Hypsarrhythmia
timeFrame: Day 15
measure: Number of Participants Experiencing Spasms and Seizures by Subtype
timeFrame: Day 4 and Day 15
measure: Average Time to Cessation of Spasms
timeFrame: Day 1 to start of Open-label Extension (OLE) Phase
measure: Caregiver Clinical Global Impression of Change (CGIC)
timeFrame: Day 15
measure: Physician Global Impression of Change (PGIC)
timeFrame: Day 15
measure: Number of Responders
timeFrame: Baseline to Day 15
measure: Percentage of Responders
timeFrame: Baseline to Day 15
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Winston-Salem
country: United States
facility: Wake Forest Baptist Medical Center
state: North Carolina
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Memphis
country: United States
facility: Le Bonheur Children's Hospital
state: Tennessee
city: San Antonio
country: United States
facility: The Childrens Hospital of San Antonio
state: Texas
city: Winchester
country: United States
facility: Valley Health Clinical Research
state: Virginia
city: Gdańsk
country: Poland
facility: Uniwersyteckie Centrum Kliniczne
city: Łódź
country: Poland
facility: Centrum Medyczne POMOC
Study officials:
References: