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Efficacy and Safety of GWP42003-P Oral Solution in Children With Epilepsy With Myoclonic-atonic Seizures | Clinical Trials | Clareo Health

TERMINATEDPHASE3

Efficacy and Safety of GWP42003-P Oral Solution in Children With Epilepsy With Myoclonic-atonic Seizures

A Randomized, Double-blind, Placebo-controlled, Multisite, Phase 3 Study to Investigate the Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P) in Children and Adolescents With Epilepsy With Myoclonic-Atonic Seizures

NCT05288283•Jazz Pharmaceuticals

View on ClinicalTrials.gov

Summary

The primary aim of Part A of the study to assess the efficacy and tolerability of GWP42003-P compared to placebo as an adjunctive treatment for children with Epilepsy with myoclonic-atonic seizures (EMAS) -associated seizures. Part B of this study will be conducted to evaluate the long-term safety and tolerability of GWP42003-P in participants with EMAS.

Detailed Description

The duration of study participation in Part A will be approximately 26 weeks, which includes a 1- to 3-week Screening Period, 4-week Baseline Observation Period, 14-week Dose Optimization Treatment Period, 10-day Taper Period, and a Safety Follow-up Period (4 weeks after end of taper visit). Participants will be randomized centrally in a 1:1 ratio to receive either GWP42003-P or matching placebo. Randomization will be stratified by clobazam use (on/off) and age of seizure onset (3 years of age and younger or older than 3 years of age). Upon completion of the double-blind phase (Part A), participants will have an option to continue in a 54-week open-label extension (Part B).

Eligibility

Age

1 - 18 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Participant has a current diagnosis of epilepsy with myoclonic-atonic seizures (EMAS), also known as Doose syndrome, myoclonic-astatic epilepsy, or myoclonic-atonic epilepsy, consistent with the International League Against Epilepsy (ILAE) guidelines. Presence of myoclonic-atonic seizures is mandatory to support a diagnosis of EMAS as determined by medical history and independent approval by The Epilepsy Study Consortium (TESC).
•Participant's initial seizure onset occurred from ≥ 6 months to \< 6 years of age, with normal or mildly impaired/delayed neurodevelopment reported prior to onset of seizures. During the first year of seizure onset, the majority of seizures experienced by the participant were myoclonic-atonic seizures or generalized tonic-clonic seizures as determined by medical history.
•Participant is currently treated with one or more antiepileptic drug (AED) on a stable regimen (≥ 28 days prior to starting the baseline period \[Part A Visit 2\]) or on a stable ketogenic diet (≥ 3 months prior to starting the baseline period \[Part A Visit 2\]) and no changes to treatment are planned for the duration of the study.
•Participant is refractory to anticonvulsant medication and failed at least 1 AED (e.g., valproic acid, clobazam, clonazepam, and levetiracetam) at therapeutic doses.
•Participant is able to provide a historical electroencephalogram (EEG) report, which was performed within 12 months of Screening (Part A Visit 1), or is willing to complete an EEG at Screening (Part A Visit 1), that confirms a 3 to 6 Hertz (Hz) generalized spike-and-slow-wave or polyspike-and-slow-wave pattern.
•Contraceptive use by male and female participants should be consistent with Clinical Trial Facilitation Group (CTFG) guidelines and any applicable local regulations regarding the methods of contraception for those participating in clinical studies.
•1. Fertile male participants with partners of childbearing potential (CBP) must be willing to use a male barrier method of contraception in addition to a second method of acceptable contraception used by their female of CBP partners, from the time of Screening (Part A Visit 1) until 3 months after the follow-up visit.
•2. Female participants of CBP will not be pregnant or lactating and have a confirmed negative highly sensitive serum pregnancy test at Screening (Part A Visit 1).
•3. Female participants must also have a confirmed negative urine pregnancy test prior to receiving their first dose of blinded investigational medicinal product (IMP) at Part A Visit 3.
•4. Female participants who are continuing to Part B must have a confirmed negative urine pregnancy test prior to receiving their first dose of open-label GWP42003-P at Part B Visit 1.
+7 more criteria

Exclusion Criteria

•Has a history of psychogenic non-epileptic seizures that confounds the assessment of the primary efficacy measure
•Has clinically significant unstable medical condition(s), other than EMAS
•Has a clinically significant illness in the 28 days prior to Screening (Visit 1) or randomization (Part A Visit 3), other than epilepsy, which in the opinion of the investigator could affect seizure frequency
•Has presence of focal seizures or persistent focal epileptiform discharges on EEG
•Has a history of infantile spasms
•Has moderate to severe neurocognitive and/or developmental delay prior to seizure onset
•Has a progressive neurological condition
•Has known or suspected hypersensitivity to cannabinoids or any of the excipients of GWP42003-P such as sesame oil
•Is unwilling or unable to remain stable on concurrent AEDs throughout the study.
•Has, in the opinion of the investigator, clinically significant abnormalities in the electrocardiogram (ECG) measured at Screening (Part A Visit 1), or any concurrent cardiovascular conditions, which will interfere with the ability to read their ECGs
+33 more criteria

Locations (14)

Children's Hospital of Alabama

Birmingham, Alabama, United States

University of California Davis Health

Sacramento, California, United States

Healthcare of Atlanta

Atlanta, Georgia, United States

Ann & Robert H. Lurie Children's Hospital

Chicago, Illinois, United States

Mayo Clinic

Rochester, Minnesota, United States

Wake Forest Baptist Health Sciences, Department of Neurology

Winston-Salem, North Carolina, United States

Cincinnati Children's Hospital Medical Center - TS Clinic

Cincinnati, Ohio, United States

Medical University of South Carolina

Charleston, South Carolina, United States

Dell's Children's Hospital

Austin, Texas, United States

Seattle Children's Hospital

Seattle, Washington, United States

Key Dates

Start Date

October 31, 2022

Primary Completion Date

September 28, 2023

Completion Date

September 28, 2023

Last Updated

February 3, 2025

Enrollment

ACTUAL participants

Conditions

Seizures Associated With EMAS

Interventions

GWP42003-P

DRUG

Placebo

DRUG