Phase 3 Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms: Open-label Extension Phase (GWPCARE7)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 September 2022
• Main ID: NCT02954887
• Date of registration: 02/11/2016
• Prospective Registration: Yes
• Primary sponsor: Jazz Pharmaceuticals
• Public title: Phase 3 Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms: Open-label Extension Phase (GWPCARE7)
• Scientific 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
• Date of first enrolment: May 12, 2017
• Target sample size: 9
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02954887
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Poland, United States

Key inclusion & exclusion 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.



Age minimum: 1 Month
Age maximum: 24 Months
Gender: All

Health Condition(s) or Problem(s) studied

Infantile Spasms

Intervention(s)

Drug: GWP42003-P

Primary Outcome(s)

Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value [Time Frame: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389]

Number of Participants With Any Clinically Relevant Urinalysis Parameter Value [Time Frame: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389]

Number of Participants With Clinically Significant Vital Sign Findings [Time Frame: From signing of informed consent up to Day 389]

Number of Participants With Severe Treatment-emergent Adverse Events (TEAEs) [Time Frame: From signing of informed consent up to Day 417]

Number of Participants With Any Low or High Hematology Laboratory Parameter Value [Time Frame: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389]

Number of Participants With Clinically Significant Electrocardiogram Findings [Time Frame: From signing of informed consent up to Day 389]

Number of Participants With Clinically Significant Physical Examination Findings [Time Frame: From signing of informed consent up to Day 389]

Secondary Outcome(s)

Average Time to Cessation of Spasms [Time Frame: Day 1 to Day 379]

Change From Baseline in Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) Score [Time Frame: Baseline (Day 1 of Pilot Study); Day 211, Day 379]

Number of Participants Experiencing Spasms and Seizures by Subtype [Time Frame: Days 19, 29, 127, 211, 295, and 379]

Percentage of Participants With Relapse of Spasms [Time Frame: Day 16 to Day 379]

Percentage of Participants Free of Clinical Spasms [Time Frame: Days 29, 43, 127, 211, 295, and 379]

Physician Global Impression of Change (PGIC) [Time Frame: Baseline; Days 29, 43, 71, 127, 211, 295, and 379]

Percentage of Participants With a Resolution of Hypsarrhythmia [Time Frame: Days 29, 43, 127, 211, 295, and 379]

Change From Baseline in Head Circumference [Time Frame: Baseline (Day 1 of PIlot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389]

Change From Baseline in Body Weight. [Time Frame: Baseline (Day 1 of Pilot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389]

Average Time to Relapse [Time Frame: Day 16 to Day 379]

Number of Participants With a Resolution of Hypsarrhythmia [Time Frame: Days 29, 43, 127, 211, 295, and 379]

Number of Participants Free of Clinical Spasms [Time Frame: Days 29, 43, 127, 211, 295, and 379]

Percentage of Responders [Time Frame: Days 29, 43, 127, 211, 295, and 379]

Change From Baseline in Height [Time Frame: Baseline (Day 1 of Pilot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389]

Number of Participants With Relapse of Spasms [Time Frame: Day 16 to Day 379]

Caregiver Global Impression of Change (CGIC) [Time Frame: Baseline; Days 29, 43, 71, 127, 211, 295, and 379]

Number of Responders [Time Frame: Days 29, 43, 127, 211, 295, and 379]

Secondary ID(s)

GWEP15100 Open-label Extension

2015-004904-50

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 23/07/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02954887