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GWP42003-P for Epilepsy

Ann & Robert H. Lurie Children's Hospital, Chicago, IL
EpilepsyGWP42003-P - Drug
Eligibility
1 - 18
All Sexes
What conditions do you have?
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Study Summary

This trialwill assess a new epilepsy drug's effectiveness and safety in children with myoclonic-atonic seizures. Part A will assess efficacy & tolerability, Part B will assess long-term safety.

Eligible Conditions
  • Epilepsy-Associated Seizures (EMAS)

Treatment Effectiveness

Phase-Based Effectiveness

2 of 3
Phase 3
This is further along than 85% of similar trials

Similar Trials

1
LP352
1
Part B: Open-Label Extension Treatment 0.5mg/kg/day PRAX-562
1
BMS-986165

Study Objectives

9 Primary · 24 Secondary · Reporting Duration: Weeks 14, 24, and 48

Week 14
Part A: Change from Baseline in the number of EMAS-associated seizure-free days over the 14-week treatment period
Part A: Number of participants who achieve ≥ 25%, ≥ 50%, ≥ 75%, and 100% reduction from Baseline in total seizures over the 14-week treatment period
Part A: Number of participants who achieve ≥ 50% reduction from Baseline in EMAS-associated seizures over the 14-week treatment period
Part A: Number of participants with at least 25% and 50% reduction from Baseline in the number of days per week with myoclonic seizures during the 14-week treatment period
Part A: Percent change from Baseline in epilepsy with myoclonic-atonic seizures (EMAS) associated seizure frequency (myoclonic-atonic, atonic, tonic, clonic, or tonic-clonic) over the 14-week treatment period
Seizures
Week 48
Part B: Change from Baseline in the number of EMAS-associated seizure-free days over the 48-week open-label treatment period
Part B: Number of participants achieving ≥50% reduction from Baseline in EMAS-associated seizures over the 48-week open-label treatment period
Seizures
Part B: Number of participants with at least 25% and 50% reduction from Baseline in the number of days per week with myoclonic seizures during the 48-week open-label treatment period
Part B: Percent change from Baseline in EMAS-associated seizure frequency (myoclonic-atonic, atonic, tonic, clonic, or tonic-clonic) over the 48-week open-label treatment period
Week 14
Part A: Caregiver Global Impression of Change (CGIC) score at Week 14
Part A: Physician Global Impression of Change (PGIC) score at Week 14
Week 14
Part B: CGIC score at Weeks 14, 24, and 48
Part B: PGIC score at Weeks 14, 24, and 48
up to 14 weeks
Part A: Time to Baseline seizure frequency
up to Day 110
Part A: Number of participants with clinically significant vital sign values
up to Day 138
Cocaine
Part A: Number of participants with a change in the number of suicide attempts per C-SSRS scores
Part A: Number of participants with treatment-emergent adverse events
up to Day 99
Part A: Number of participants with changes in Tanner Staging
Part A: Number of participants with clinically significant 12-lead ECG values
Part A: Number of participants with clinically significant laboratory test values
Part A: Number of participants with clinically significant physical examination values
up to Week 48
Part B: Number of participants with changes in Tanner Staging
Electrocardiogram
Part B: Number of participants with clinically significant laboratory test values
Part B: Number of participants with clinically significant physical examination values
Part B: Total seizure frequency over the 48-week open-label treatment period
up to Week 50
Part B: Number of participants with clinically significant vital sign values
up to Week 54
Part B: Number of participants with a change in Columbia-Suicide Severity Rating Scale (C-SSRS) ideation scores
Part B: Number of participants with a change in the number of suicide attempts per C-SSRS scores
Part B: Number of participants with treatment-emergent adverse events

Trial Safety

Phase-Based Safety

3 of 3
This is further along than 85% of similar trials

Similar Trials

2
LP352
2
Part B: Open-Label Extension Treatment 0.5mg/kg/day PRAX-562
2
BMS-986165

Side Effects for

GWP42003-P 10 mg/kg/Day Dose-Safety Analysis Set
21%Somnolence
16%Upper respiratory tract infection
16%Decreased appetite
10%Status epilepticus
10%Diarrhoea
9%Pyrexia
9%Irritability
7%Fatigue
6%Vomiting
6%Insomnia
4%Nasopharyngitis
4%Lethargy
4%Pneumonia
3%Convulsion
3%Headache
1%Sleep apnoea syndrome
1%Pancreatitis
1%Cholecystitis chronic
1%Pneumonia bacterial
1%Pneumonia respiratory syncytial viral
1%Transaminases increased
1%Aspartate aminotransferase increased
1%Sedation
1%Dehydration
1%Hypoventilation
1%Hypoxia
This histogram enumerates side effects from a completed 2016 Phase 3 trial (NCT02224560) in the GWP42003-P 10 mg/kg/Day Dose-Safety Analysis Set ARM group. Side effects include: Somnolence with 21%, Upper respiratory tract infection with 16%, Decreased appetite with 16%, Status epilepticus with 10%, Diarrhoea with 10%.

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2 Treatment Groups

GWP42003-P
1 of 2
Placebo
1 of 2

Experimental Treatment

Non-Treatment Group

240 Total Participants · 2 Treatment Groups

Primary Treatment: GWP42003-P · Has Placebo Group · Phase 3

GWP42003-P
Drug
Experimental Group · 1 Intervention: GWP42003-P · Intervention Types: Drug
Placebo
Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
GWP42003-P
2016
Completed Phase 3
~1630

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: weeks 14, 24, and 48

Who is running the clinical trial?

Jazz PharmaceuticalsLead Sponsor
229 Previous Clinical Trials
32,173 Total Patients Enrolled
16 Trials studying Epilepsy
1,845 Patients Enrolled for Epilepsy

Eligibility Criteria

Age 1 - 18 · All Participants · 0 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
References

Frequently Asked Questions

Is there an age limit for participants in this particular research project?

"In order to take part in this research, potential participants must be aged 1-18. Out of the total 316 clinical trials, 126 are for people under 18 years old and 190 are for those over 65." - Anonymous Online Contributor

Unverified Answer

Are there any safety concerns associated with taking GWP42003-P?

"There is existing data that suggests GWP42003-P's efficacy and safety, so it was given a score of 3." - Anonymous Online Contributor

Unverified Answer

Are there any open slots for new volunteers in this clinical trial?

"Indeed, the most recent information available on clinicaltrials.gov points to this trial recruiting patients as we speak. This particular study was first advertised on October 31st 2020 and updated November 14th 2020. There is a need for 240 individuals at 1 site location." - Anonymous Online Contributor

Unverified Answer

How many individuals can join this clinical trial?

"The information available on clinicaltrials.gov verifies that this study is currently recruiting patients. The trial was posted on October 31st, 2020 and updated two weeks ago on November 14th. They are looking for a total of 240 subjects at a single location." - Anonymous Online Contributor

Unverified Answer

For whom is this trial designed?

"This trial is looking for 240 participants that suffer from seizures and are between 1 year old to 18 years old. Eligibility requirements include the following: contraceptive use by both male and female participants must follow Clinical Trial Facilitation Group (CTFG) guidelines, females cannot be pregnant or lactating, have a negative serum pregnancy test at Screening , confirmed negative urine pregnancy test before receiving IMP at Part A Visit 3, continuing to Part B with a negative urine pregnancy test result before receiving GWP42003-P, diagnosis of epilepsy with myoclonic-atonic seizures consistent with ILAE guidelines, initial seizure onset occurred 6 months to" - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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