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Completed

NCT Number: NCT02953548

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

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.

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Key information

Conditions

Infantile Spasms Brain Diseases Central Nervous System Diseases Epilepsy Epilepsy, Generalized Epileptic Syndromes Nervous System Diseases Spasms, Infantile

Age range

1 month–24 month

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Uniwersyteckie Centrum Kliniczne, Gdansk, Poland

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

GWP42003-P

Drug

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

Other names: CBD, Cannabidiol

Primary outcomes

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

    Time frame: 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.

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

    Time frame: Day 4 and Day 15

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

    Time frame: Day 4 and Day 15

  4. Number of Participant With Any Clinically Relevant Urinalysis Parameter Value

    Time frame: Day 4 and Day 15

    Clinical relevance was determined by the investigator.

  5. Number of Participants With Clinically Significant Electrocardiogram Findings

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

    Clinical significance was determined by the investigator.

  6. Number of Participants With Clinically Significant Physical Examination Findings

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

    Clinical significance was determined by the investigator.

  7. Number of Participants With Clinically Significant Vital Sign Findings

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

    Clinical significance was determined by the investigator.

Secondary outcomes

  1. Number of Participants Free of Clinical Spasms

    Time frame: Day 15

    Clinical spasms were determined by video-electroencephalography (VEEG) for at least 8 hours and up to 24 hours.

  2. Percentage of Participants Free of Clinical Spasms

    Time frame: Day 15

    Clinical spasms were determined by VEEG for at least 8 hours and up to 24 hours.

  3. Number of Participants With Resolution of Hypsarrhythmia

    Time frame: Day 15

    Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.

  4. Percentage of Participants With Resolution of Hypsarrhythmia

    Time frame: Day 15

    Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.

  5. Number of Participants Experiencing Spasms and Seizures by Subtype

    Time frame: 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.

  6. Average Time to Cessation of Spasms

    Time frame: 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.

  7. Caregiver Clinical Global Impression of Change (CGIC)

    Time frame: 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).

  8. Physician Global Impression of Change (PGIC)

    Time frame: 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).

  9. Number of Responders

    Time frame: 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.

  10. Percentage of Responders

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

Jazz Pharmaceuticals

Industry

Registry information

Official study 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

Important dates

Study start
2017
Primary completion
2018
Study completion
2018
First posted
Nov 2, 2016
Registry last updated
Sep 2, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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