Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms (GWPCARE7)

Jazz Pharmaceuticals9 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Infantile Spasms

Interventions

GWP42003-PDRUG

Clear, colorless to yellow solution containing cannabidiol dissolved in the excipients sesame oil and anhydrous ethanol with added sweetener (sucralose) and strawberry flavoring.

Eligibility

Sex: ALLMin age: 1 MonthMax age: 24 Months

Medical Language ↔ Plain English

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.

Locations (8)

Arkansas Children's Hospital

Little Rock, Arkansas, United States

Wake Forest Baptist Medical Center

Winston-Salem, North Carolina, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

Le Bonheur Children's Hospital

Memphis, Tennessee, United States

Outcomes

Primary Outcomes

Number of Participants With Severe Treatment-emergent Adverse Events (TEAEs)

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.

Time frame: From signing of informed consent up to Day 15

Number of Participants With Any Low or High Hematology Laboratory Parameter Value

Time frame: Day 4 and Day 15

Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value

Time frame: Day 4 and Day 15

Number of Participant With Any Clinically Relevant Urinalysis Parameter Value

Clinical relevance was determined by the investigator.

Time frame: Day 4 and Day 15

Number of Participants With Clinically Significant Electrocardiogram Findings

Clinical significance was determined by the investigator.

Time frame: From signing of informed consent up to Day 15

Number of Participants With Clinically Significant Physical Examination Findings

Clinical significance was determined by the investigator.

Time frame: From signing of informed consent up to Day 15

Number of Participants With Clinically Significant Vital Sign Findings

Clinical significance was determined by the investigator.

Time frame: From signing of informed consent up to Day 15

Secondary Outcomes

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.