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

Primary Purpose

Infantile Spasms

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
GWP42003-P
Sponsored by
Jazz Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Infantile Spasms focused on measuring GWP42003-P, Cannabidiol

Eligibility Criteria

1 Month - 24 Months (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Arkansas Children's Hospital
  • Wake Forest Baptist Medical Center
  • Nationwide Children's Hospital
  • Le Bonheur Children's Hospital
  • The Childrens Hospital of San Antonio
  • Valley Health Clinical Research
  • Uniwersyteckie Centrum Kliniczne
  • Centrum Medyczne POMOC

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

GWP42003-P

Arm Description

Administered orally, titrating to a target dose of 40 mg/kg/day. Participants continue at the target dose, or the highest tolerated dose up to the target dose, for the remainder of the 2-week treatment period.

Outcomes

Primary Outcome Measures

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.
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Number of Participant With Any Clinically Relevant Urinalysis Parameter Value
Clinical relevance was determined by the investigator.
Number of Participants With Clinically Significant Electrocardiogram Findings
Clinical significance was determined by the investigator.
Number of Participants With Clinically Significant Physical Examination Findings
Clinical significance was determined by the investigator.
Number of Participants With Clinically Significant Vital Sign Findings
Clinical significance was determined by the investigator.

Secondary Outcome Measures

Number of Participants Free of Clinical Spasms
Clinical spasms were determined by video-electroencephalography (VEEG) for at least 8 hours and up to 24 hours.
Percentage of Participants Free of Clinical Spasms
Clinical spasms were determined by VEEG for at least 8 hours and up to 24 hours.
Number of Participants With Resolution of Hypsarrhythmia
Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.
Percentage of Participants With Resolution of Hypsarrhythmia
Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.
Number of Participants Experiencing Spasms and Seizures by Subtype
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.
Average Time to Cessation of Spasms
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.
Caregiver Clinical Global Impression of Change (CGIC)
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).
Physician Global Impression of Change (PGIC)
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).
Number of Responders
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.
Percentage of Responders
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.

Full Information

First Posted
November 1, 2016
Last Updated
August 31, 2022
Sponsor
Jazz Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT02953548
Brief Title
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

2. Study Status

Record Verification Date
August 2022
Overall Recruitment Status
Completed
Study Start Date
April 24, 2017 (Actual)
Primary Completion Date
May 7, 2018 (Actual)
Study Completion Date
May 7, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Jazz Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Infantile Spasms
Keywords
GWP42003-P, Cannabidiol

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Masking Description
Open-label
Allocation
N/A
Enrollment
9 (Actual)

8. Arms, Groups, and Interventions

Arm Title
GWP42003-P
Arm Type
Experimental
Arm Description
Administered orally, titrating to a target dose of 40 mg/kg/day. Participants continue at the target dose, or the highest tolerated dose up to the target dose, for the remainder of the 2-week treatment period.
Intervention Type
Drug
Intervention Name(s)
GWP42003-P
Other Intervention Name(s)
CBD, Cannabidiol
Intervention Description
Clear, colorless to yellow solution containing cannabidiol dissolved in the excipients sesame oil and anhydrous ethanol with added sweetener (sucralose) and strawberry flavoring.
Primary Outcome Measure Information:
Title
Number of Participants With Severe Treatment-emergent Adverse Events (TEAEs)
Description
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
Title
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Time Frame
Day 4 and Day 15
Title
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Time Frame
Day 4 and Day 15
Title
Number of Participant With Any Clinically Relevant Urinalysis Parameter Value
Description
Clinical relevance was determined by the investigator.
Time Frame
Day 4 and Day 15
Title
Number of Participants With Clinically Significant Electrocardiogram Findings
Description
Clinical significance was determined by the investigator.
Time Frame
From signing of informed consent up to Day 15
Title
Number of Participants With Clinically Significant Physical Examination Findings
Description
Clinical significance was determined by the investigator.
Time Frame
From signing of informed consent up to Day 15
Title
Number of Participants With Clinically Significant Vital Sign Findings
Description
Clinical significance was determined by the investigator.
Time Frame
From signing of informed consent up to Day 15
Secondary Outcome Measure Information:
Title
Number of Participants Free of Clinical Spasms
Description
Clinical spasms were determined by video-electroencephalography (VEEG) for at least 8 hours and up to 24 hours.
Time Frame
Day 15
Title
Percentage of Participants Free of Clinical Spasms
Description
Clinical spasms were determined by VEEG for at least 8 hours and up to 24 hours.
Time Frame
Day 15
Title
Number of Participants With Resolution of Hypsarrhythmia
Description
Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.
Time Frame
Day 15
Title
Percentage of Participants With Resolution of Hypsarrhythmia
Description
Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.
Time Frame
Day 15
Title
Number of Participants Experiencing Spasms and Seizures by Subtype
Description
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.
Time Frame
Day 4 and Day 15
Title
Average Time to Cessation of Spasms
Description
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.
Time Frame
Day 1 to start of Open-label Extension (OLE) Phase
Title
Caregiver Clinical Global Impression of Change (CGIC)
Description
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).
Time Frame
Day 15
Title
Physician Global Impression of Change (PGIC)
Description
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).
Time Frame
Day 15
Title
Number of Responders
Description
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.
Time Frame
Baseline to Day 15
Title
Percentage of Responders
Description
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.
Time Frame
Baseline to Day 15

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Month
Maximum Age & Unit of Time
24 Months
Accepts Healthy Volunteers
No
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.
Facility Information:
Facility Name
Arkansas Children's Hospital
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
Wake Forest Baptist Medical Center
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Facility Name
Le Bonheur Children's Hospital
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38103
Country
United States
Facility Name
The Childrens Hospital of San Antonio
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78207
Country
United States
Facility Name
Valley Health Clinical Research
City
Winchester
State/Province
Virginia
ZIP/Postal Code
22601
Country
United States
Facility Name
Uniwersyteckie Centrum Kliniczne
City
Gdańsk
Country
Poland
Facility Name
Centrum Medyczne POMOC
City
Łódź
Country
Poland

12. IPD Sharing Statement

Learn more about this trial

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

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