Phase 3
N=225
Efficacy and Safety of GWP42003-P for Seizures Associated With Lennox-Gastaut Syndrome in Children and Adults
Epilepsy · Lennox Gastaut Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT02224560 ↗Enrolled (actual)
225
Serious AEs
15.1%
Results posted
Jul 2018
Primary outcome: Primary: Percentage Change From Baseline In Drop Seizure Frequency During The Treatment Period — -41.86; -37.16; -17.17 percentage change — p=0.0047
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- GWP42003-P (Drug); Placebo control (Drug)
- Age
- Pediatric, Adult · 2+ yrs
- Sex
- All
- Sponsor
- Jazz Pharmaceuticals
- Primary completion
- May 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage Change From Baseline In Drop Seizure Frequency During The Treatment Period |
-41.86; -37.16; -17.17 | 0.0047 sig |
| SECONDARY Number Of Participants With A ≥50% Reduction From Baseline In Drop Seizure Frequency During The Treatment Period |
30; 26; 11 | 0.0006 sig |
| SECONDARY Percentage Change From Baseline In Total Seizure Frequency During The Treatment Period |
-38.40; -36.44; -18.47 | 0.0091 sig |
| SECONDARY Subject/Caregiver Global Impression Of Change (S/CGIC) Assessment |
6; 9; 1; 15; 14; 8 | 0.0439 sig |
Summary
The primary objective of this study was to evaluate the efficacy of GWP42003-P as adjunctive treatment in reducing the number of drop seizures when compared with placebo in participants with Lennox-Gastaut syndrome (LGS).
Eligibility Criteria
Key Inclusion Criteria
- Participant and/or parent(s)/legal representatives were willing and able to give informed assent/consent for participation in the study.
- Participant and his or her caregivers were willing and able (in the investigator's opinion) to comply with all study requirements.
- Participant was male or female aged between 2 and 55 years (inclusive).
- Participant had a documented history of LGS. This included written documentation of having met electroencephalogram (EEG) diagnostic criteria during the participant's history and evidence of at least 1 type of generalized seizure, including drop seizures (atonic, tonic, tonic-clonic or myoclonic) for at least 6 months.
- Participant had a history of slow ( 5 × upper limit of normal (ULN); ALT or AST > 3 × ULN and total bilirubin > 2 × ULN or international normalized ratio (INR) > 1.5; ALT or AST > 3 × ULN with the presence of fatigue, nausea, vomiting, right upper quadrant pain or tenderness, fever, rash, and/or eosinophilia (>5%). This criterion could only be confirmed once the laboratory results were available; participants randomized into the study who were later found not to meet this criterion were withdrawn from the study.
- Any history of suicidal behavior or any suicidal ideation of type 4 or 5 on the Columbia Suicide Severity Rating Scale in the last month or at screening.
- Participant was unwilling to abstain from donation of blood during the study.
- Participant planned to travel outside his or her country of residence during the study.
- Participant had previously randomized into the study.
- Participant was taking more than 4 concurrent AEDs.
- Participant had taken corticotropins in the 6 months prior to screening.
- Participant was currently taking long-term systemic steroids (excluding inhaled medication for asthma treatment) or any other daily medication known to exacerbate epilepsy. An exception was made of prophylactic medication, for example, idiopathic nephrotic syndrome or asthma.
- Participant was taking felbamate, and he or she had been taking it for less than 1 year prior to screening.
Data sourced from ClinicalTrials.gov (NCT02224560). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.