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Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Participants who have uncontrolled seizures, and who are currently receiving 1 or more antiseizure medication (ASMs)
Participants with TSC must have a diagnosis per the 2012 International Tuberous Sclerosis Complex Consensus Conference
Timeline
Screening 3 weeks
Treatment Varies
Follow Up predose, 3 hours and 6 hours post dose on days 1, 15, 29, 57, and end of treatment (week 52)
Awards & highlights
Study Summary
This trial will study a new seizure medication to see if it is safe and effective.
Who is the study for?
This trial is for children under 2 years old with TSC, LGS, or DS who have seizures not well-controlled by current medications. They must be diagnosed according to specific guidelines and their caregivers should consent and comply with study requirements. Children with certain health issues or hypersensitivity to cannabinoids are excluded.Check my eligibility
What is being tested?
The trial tests GWP42003-P, a cannabidiol oral solution as an additional treatment for young children with TSC, LGS, or DS experiencing poorly controlled seizures. It aims to assess the safety, how the body processes the drug (pharmacokinetics), and its effectiveness.See study design
What are the potential side effects?
Potential side effects of GWP42003-P may include sleepiness, decreased appetite, diarrhea, fever, vomiting and fatigue. Since it contains cannabidiol derived from cannabis plants there might also be risks related to mood changes or allergic reactions.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have seizures that aren't controlled even though I'm on medication.
Select...
I have been diagnosed with Tuberous Sclerosis Complex according to the 2012 guidelines.
Select...
My seizures are not fully controlled by my current medication.
Select...
My child is between 1 month and 2 years old and has TSC, DS, or LGS.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ predose, 3 hours and 6 hours post dose on days 1, 15, 29, 57, and end of treatment (week 52)
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~predose, 3 hours and 6 hours post dose on days 1, 15, 29, 57, and end of treatment (week 52)
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Clinician Global Impression of Change/Severity (CGIC/S) Score
Infant and Toddler Quality of Life Questionnaire Short Form 47 (ITQOL-47) Score
Mean Change From Baseline in Blood Pressure
+16 moreSecondary outcome measures
Number of Participants Who Achieved Seizure-Free Status
Number of Treatment Responders
Percentage Change from Baseline in Total Countable Seizures
+1 moreSide effects data
From 2016 Phase 3 trial • 225 Patients • NCT0222456021%
Somnolence
16%
Decreased appetite
16%
Upper respiratory tract infection
10%
Status epilepticus
10%
Diarrhoea
9%
Pyrexia
9%
Irritability
7%
Fatigue
6%
Insomnia
6%
Vomiting
4%
Nasopharyngitis
4%
Pneumonia
4%
Lethargy
3%
Convulsion
3%
Headache
1%
Sedation
1%
Hypoventilation
1%
Cholecystitis chronic
1%
Sleep apnoea syndrome
1%
Dehydration
1%
Aspartate aminotransferase increased
1%
Transaminases increased
1%
Pancreatitis
1%
Pneumonia bacterial
1%
Pneumonia respiratory syncytial viral
1%
Hypoxia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo-Safety Analysis Set
GWP42003-P 10 mg/kg/Day Dose-Safety Analysis Set
GWP42003-P 20 mg/kg/Day Dose-Safety Analysis Set
Trial Design
1Treatment groups
Experimental Treatment
Group I: GWP42003-PExperimental Treatment1 Intervention
The 52-week treatment period includes a fixed 2-week titration schedule followed by flexible dose optimization.
Day 1: 5 mg/kg/day (2.5 mg/kg twice daily (b.i.d.))
Day 8: 10 mg/kg/day (5 mg/kg b.i.d.)
Day 15 to Week 52: Flexible dosing based on the participant's observed efficacy, safety, and tolerability per the investigator's clinical judgement. Up to a maximum of 20 mg/kg/day (10 mg/kg b.i.d.) for LGS and DS or 25 mg/kg/day (12.5 mg/kg b.i.d.) for TSC, in maximum weekly increments of 5 mg/kg/day (≤ 2.5 mg/kg b.i.d.).
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
GWP42003-P
2016
Completed Phase 3
~1740
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Tuberous Sclerosis Complex (TSC) often involve modulation of the endocannabinoid system, anti-inflammatory properties, and potential neuroprotective effects, as seen with Cannabidiol (CBD). CBD modulates the endocannabinoid system, which helps regulate pain, mood, and memory, and has anti-inflammatory properties that reduce neuroinflammation.
Additionally, its neuroprotective effects may protect against neuronal damage and improve brain function. These mechanisms are crucial for TSC patients who experience seizures and other neurological symptoms, offering a comprehensive approach to symptom management.
Impact of Endocannabinoid System Manipulation on Neurodevelopmental Processes Relevant to Schizophrenia.Chronic cannabis promotes pro-hallucinogenic signaling of 5-HT2A receptors through Akt/mTOR pathway.Hippocampal mammalian target of rapamycin is implicated in stress-coping behavior induced by cannabidiol in the forced swim test.
Impact of Endocannabinoid System Manipulation on Neurodevelopmental Processes Relevant to Schizophrenia.Chronic cannabis promotes pro-hallucinogenic signaling of 5-HT2A receptors through Akt/mTOR pathway.Hippocampal mammalian target of rapamycin is implicated in stress-coping behavior induced by cannabidiol in the forced swim test.
Find a Location
Who is running the clinical trial?
GW Research LtdLead Sponsor
34 Previous Clinical Trials
3,181 Total Patients Enrolled
Jazz PharmaceuticalsLead Sponsor
249 Previous Clinical Trials
34,872 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have seizures that aren't controlled even though I'm on medication.You have important irregularities in your heart's electrical activity as shown on an ECG test.You had an appropriate VEEG test within the past year.I have been diagnosed with Tuberous Sclerosis Complex according to the 2012 guidelines.My seizures are not fully controlled by my current medication.My epilepsy diagnosis aligns with international guidelines and has been confirmed by a specialist group.My child is between 1 month and 2 years old and has TSC, DS, or LGS.You have important abnormal test results, according to the doctor.My liver is not working well.I have heart conditions that may affect ECG test results.
Research Study Groups:
This trial has the following groups:- Group 1: GWP42003-P
Awards:
This trial has 2 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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