Phase 2
N=34
A Dose-ranging Pharmacokinetics and Safety Study of GWP42003-P in Children With Dravet Syndrome (GWPCARE1)
Epilepsy · Dravet Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT02091206 ↗Enrolled (actual)
34
Serious AEs
14.7%
Results posted
Jul 2018
Primary outcome: Primary: Number Of Participants Who Experienced Severe Treatment-Emergent Adverse Events (TEAEs) — 2; 1; 0; 1 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- GWP42003-P 5 mg/kg/day Dose (Drug); Placebo control (Drug); GWP42003-P 10 mg/kg/day Dose (Drug); GWP42003-P 20 mg/kg/day Dose (Drug)
- Age
- Pediatric · 4+ yrs
- Sex
- All
- Sponsor
- Jazz Pharmaceuticals
- Primary completion
- Mar 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number Of Participants Who Experienced Severe Treatment-Emergent Adverse Events (TEAEs) |
2; 1; 0; 1 | — |
| SECONDARY Area Under The Concentration-Time Curve Calculated To The Last Observable Concentration At Time T (AUC0-t) For CBD And Its Metabolites At Days 1 And 22 |
70.61; 66.35; 73.69; 240.8; 721.8; 962.6 | — |
| SECONDARY Mean Percentage Change From Baseline To End Of Treatment In Plasma Clobazam (CLB) And N-Desmethylclobazam (N-CLB) Concentrations |
-1.2; 18.0; 29.6; 15.1; 258.7; 170.7 | — |
Summary
To evaluate the safety and pharmacokinetics (PK) of multiple doses of GWP42003-P compared with placebo in children with Dravet syndrome.
Eligibility Criteria
Key Inclusion Criteria
- Participants were male or female aged between 4 and 10 years (inclusive).
- Participants had a documented history of Dravet Syndrome that was not completely controlled by AEDs.
- Participants took one or more AEDs at a dose which had been stable for at least 4 weeks.
- Participants had experienced fewer than 4 convulsive seizures (tonic-clonic, tonic, clonic, atonic seizures) during the 28-day baseline period.
- All medications or interventions for epilepsy (including ketogenic diet and vagus nerve stimulation [VNS]) were stable for four weeks prior to screening and participants were willing to maintain a stable regimen throughout the study. The ketogenic diet and VNS treatments were not counted as an AED.
Key Exclusion Criteria
- Participants had clinically significant unstable medical conditions other than epilepsy.
- Participants had clinically relevant abnormalities in the 12-lead electrocardiogram measured at screening or randomization.
- Participants were currently using or had in the past used recreational or medicinal cannabis, or synthetic CBD based medications (including Sativex®) within the 3 months prior to study entry and were unwilling to abstain for the duration for the study.
- Participants had any known or suspected hypersensitivity to cannabinoids or any of the excipients of the IMP.
- Participants who had been part of a clinical trial involving another investigational product in the previous 6 months.
- There were plans for the participants to travel outside their country of residence during the study.
- Participants were previously randomized into this study. In particular, participants participating in Part A of the study cannot enter Part B.
Data sourced from ClinicalTrials.gov (NCT02091206). 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.