Clinical trial NCT02954887: Phase 3 Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms: Open-label Extension Phase (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: 2016-11-04
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 open-label extension phase only will be described in this record. All participants will receive GWP42003-P.


Eligibility 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.

Primary outcomes:
measure: Number of Participants With Severe Treatment-emergent Adverse Events (TEAEs)
timeFrame: From signing of informed consent up to Day 417
measure: Number of Participants With Any Low or High Hematology Laboratory Parameter Value
timeFrame: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389
measure: Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
timeFrame: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389
measure: Number of Participants With Any Clinically Relevant Urinalysis Parameter Value
timeFrame: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389
measure: Number of Participants With Clinically Significant Electrocardiogram Findings
timeFrame: From signing of informed consent up to Day 389
measure: Number of Participants With Clinically Significant Vital Sign Findings
timeFrame: From signing of informed consent up to Day 389
measure: Number of Participants With Clinically Significant Physical Examination Findings
timeFrame: From signing of informed consent up to Day 389
Secondary outcomes:
measure: Number of Participants Free of Clinical Spasms
timeFrame: Days 29, 43, 127, 211, 295, and 379
measure: Percentage of Participants Free of Clinical Spasms
timeFrame: Days 29, 43, 127, 211, 295, and 379
measure: Number of Participants With a Resolution of Hypsarrhythmia
timeFrame: Days 29, 43, 127, 211, 295, and 379
measure: Percentage of Participants With a Resolution of Hypsarrhythmia
timeFrame: Days 29, 43, 127, 211, 295, and 379
measure: Number of Participants Experiencing Spasms and Seizures by Subtype
timeFrame: Days 19, 29, 127, 211, 295, and 379
measure: Caregiver Global Impression of Change (CGIC)
timeFrame: Baseline; Days 29, 43, 71, 127, 211, 295, and 379
measure: Physician Global Impression of Change (PGIC)
timeFrame: Baseline; Days 29, 43, 71, 127, 211, 295, and 379
measure: Number of Responders
timeFrame: Days 29, 43, 127, 211, 295, and 379
measure: Percentage of Responders
timeFrame: Days 29, 43, 127, 211, 295, and 379
measure: Change From Baseline in Height
timeFrame: Baseline (Day 1 of Pilot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389
measure: Change From Baseline in Body Weight.
timeFrame: Baseline (Day 1 of Pilot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389
measure: Change From Baseline in Head Circumference
timeFrame: Baseline (Day 1 of PIlot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389
measure: Change From Baseline in Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) Score
timeFrame: Baseline (Day 1 of Pilot Study); Day 211, Day 379
measure: Number of Participants With Relapse of Spasms
timeFrame: Day 16 to Day 379
measure: Percentage of Participants With Relapse of Spasms
timeFrame: Day 16 to Day 379
measure: Average Time to Cessation of Spasms
timeFrame: Day 1 to Day 379
measure: Average Time to Relapse
timeFrame: Day 16 to Day 379
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: 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: