Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2015-004904-50 | EudraCT Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| GWP42003-P | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | From signing of informed consent up to Day 15 |
| Number of Participants With Any Low or High Hematology Laboratory Parameter Value | Day 4 and Day 15 | |
| Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value | Day 4 and Day 15 | |
| Number of Participant With Any Clinically Relevant Urinalysis Parameter Value | Clinical relevance was determined by the investigator. | Day 4 and Day 15 |
| Number of Participants With Clinically Significant Electrocardiogram Findings | Clinical significance was determined by the investigator. | 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. | 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. | From signing of informed consent up to Day 15 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Day 15 |
| Percentage of Participants Free of Clinical Spasms |
Not provided
Key Inclusion Criteria:
Key Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | 72202 | United States | ||
| Wake Forest Baptist Medical Center |
Not provided
Participants were screened to assess their eligibility to enter the trial within 7 days prior to the first dose administration.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Cohort 1: GWP42003-P OS | Participants 6 months to 24 months of age received GWP42003-P oral solution (OS) for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 milligrams per kilograms per day (mg/kg/day) of GWP42003-P. |
| FG001 | Cohort 2: GWP42003-P OS | Participants 1 month to 24 months of age received GWP42003-P OS for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Safety Analysis Set
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | GWP42003-P OS | Participants received GWP42003-P oral solution (OS) for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Safety Analysis Set: all participants who received at least 1 dose of GWP42003-P | Posted | Count of Participants | Participants | From signing of informed consent up to Day 15 |
|
From signing of informed consent up to Day 15
Treatment-emergent adverse events (TEAEs) were collected in members of the Safety Population, comprised of all participants who received at least 1 dose of GWP42003-P. 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.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | GWP42003-P OS | Participants received GWP42003-P oral solution (OS) for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Status epilepticus | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Application site erosion | General disorders | MedDRA (19.0) | Systematic Assessment |
This study met No Go Criteria. The Pilot Phase concluded after 9 participants completed treatment, and the Pivotal Phase was not initiated. Participants completing the Pilot Phase could roll into the Open Label Extension Phase for up to 1 year.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Enquires | GW Research Ltd | +44 01223 238170; 1877886281 | medinfo@gwpharm.com, medinfo@greenwichbiosciences.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Sep 20, 2016 | Jun 10, 2020 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 25, 2019 | Jun 10, 2020 | SAP_001.pdf |
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D013036 | Spasms, Infantile |
| ID | Term |
|---|---|
| D004829 | Epilepsy, Generalized |
| D004827 | Epilepsy |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D002185 | Cannabidiol |
| ID | Term |
|---|---|
| D002186 | Cannabinoids |
| D013729 | Terpenes |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
Not provided
Not provided
Not provided
Not provided
Not provided
Open-label
Not provided
|
Clinical spasms were determined by VEEG for at least 8 hours and up to 24 hours.
| Day 15 |
| Number of Participants With Resolution of Hypsarrhythmia | Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours. | Day 15 |
| Percentage of Participants With Resolution of Hypsarrhythmia | Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours. | Day 15 |
| 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. | Day 4 and Day 15 |
| 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. | Day 1 to start of Open-label Extension (OLE) Phase |
| 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). | Day 15 |
| 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). | Day 15 |
| 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. | Baseline to Day 15 |
| 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. | Baseline to Day 15 |
| Winston-Salem |
| North Carolina |
| 27157 |
| United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Le Bonheur Children's Hospital | Memphis | Tennessee | 38103 | United States |
| The Childrens Hospital of San Antonio | San Antonio | Texas | 78207 | United States |
| Valley Health Clinical Research | Winchester | Virginia | 22601 | United States |
| Uniwersyteckie Centrum Kliniczne | Gdansk | Poland |
| Centrum Medyczne POMOC | Lodz | Poland |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Primary | Number of Participants With Any Low or High Hematology Laboratory Parameter Value | Safety Analysis Set. Only participants with evaluable data were analyzed. | Posted | Count of Participants | Participants | Day 4 and Day 15 |
|
|
|
| Primary | Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value | Safety Analysis Set. Only participants with evaluable data were analyzed. | Posted | Count of Participants | Participants | Day 4 and Day 15 |
|
|
|
| Primary | Number of Participant With Any Clinically Relevant Urinalysis Parameter Value | Clinical relevance was determined by the investigator. | Safety Analysis Set | Posted | Count of Participants | Participants | Day 4 and Day 15 |
|
|
|
| Primary | Number of Participants With Clinically Significant Electrocardiogram Findings | Clinical significance was determined by the investigator. | Safety Analysis Set | Posted | Count of Participants | Participants | From signing of informed consent up to Day 15 |
|
|
|
| Primary | Number of Participants With Clinically Significant Physical Examination Findings | Clinical significance was determined by the investigator. | Safety Analysis Set | Posted | Count of Participants | Participants | From signing of informed consent up to Day 15 |
|
|
|
| Primary | Number of Participants With Clinically Significant Vital Sign Findings | Clinical significance was determined by the investigator. | Safety Analysis Set | Posted | Count of Participants | Participants | From signing of informed consent up to Day 15 |
|
|
|
| Secondary | 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. | Safety Analysis Set | Posted | Count of Participants | Participants | Day 15 |
|
|
|
| Secondary | Percentage of Participants Free of Clinical Spasms | Clinical spasms were determined by VEEG for at least 8 hours and up to 24 hours. | Safety Analysis Set | Posted | Number | percentage of participants | Day 15 |
|
|
|
| Secondary | Number of Participants With Resolution of Hypsarrhythmia | Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours. | Safety Analysis Set | Posted | Count of Participants | Participants | Day 15 |
|
|
|
| Secondary | Percentage of Participants With Resolution of Hypsarrhythmia | Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours. | Safety Analysis Set | Posted | Number | percentage of participants | Day 15 |
|
|
|
| Secondary | 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. | Safety Analysis Set | Posted | Count of Participants | Participants | Day 4 and Day 15 |
|
|
|
| Secondary | 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. | Safety Analysis Set | Posted | Day 1 to start of Open-label Extension (OLE) Phase |
|
|
| Secondary | 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). | Safety Analysis Set | Posted | Number | participants | Day 15 |
|
|
|
| Secondary | 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). | Safety Analysis Set | Posted | Number | participants | Day 15 |
|
|
|
| Secondary | 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. | Safety Analysis Set | Posted | Number | responders | Baseline to Day 15 |
|
|
|
| Secondary | 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. | Safety Analysis Set | Posted | Number | responders | Baseline to Day 15 |
|
|
|
| 0 |
| 9 |
| 1 |
| 9 |
| 5 |
| 9 |
| Irritability | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Somnolence | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Haematuria | Renal and urinary disorders | MedDRA (19.0) | Systematic Assessment |
|
| Increased appetite | Metabolism and nutrition disorders | MedDRA (19.0) | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
|
Not provided
| D009422 |
| Nervous System Diseases |
| D000073376 | Epileptic Syndromes |
|
| Day 15, Low |
|
|
| Day 15, High |
|
|
|
| Day 15, Low |
|
|
| Day 15, High |
|
|
| Title | Measurements |
|---|---|
|
| Day 4, Myoclonic |
|
| Day 4, Focal |
|
| Day 4, Absence |
|
| Day 4, Not Done |
|
| Day 15, Clonic |
|
| Day 15, Tonic-Clonic |
|
| Day 15, Atonic |
|
| Day 15, Myoclonic |
|
| Day 15, Focal |
|
| Day 15, Absence |
|
| Day 15, Not Done |
|
| Title | Measurements |
|---|---|
|
| No Change |
|
| Slightly Worse |
|
| Much Worse |
|
| Very Much Worse |
|
| Not Done |
|
| Title | Measurements |
|---|---|
|
| No Change |
|
| Slightly Worse |
|
| Much Worse |
|
| Very Much Worse |
|
| Not Done |
|