GWPCARE2 A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet Syndrome

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Brief Title

GWPCARE2 A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet Syndrome

Official Title

A Randomized, Double-blind, Placebo-controlled Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet Syndrome.

Brief Summary

      To investigate the potential antiepileptic effects of cannabidiol (GWP42003-P) in children
      and young adults with Dravet syndrome.
    

Detailed Description

      This study was a 2:2:1:1 randomized, double-blind, 14-week comparison of two dose levels of
      GWP42003-P (10 milligram/kilogram [mg/kg]/day and 20 mg/kg/day) versus placebo (10 mg/kg/day
      dose-volume equivalent and 20 mg/kg/day dose-volume equivalent; presented as pooled placebo
      in the study). A 28-day screening period prior to randomization (to establish baseline
      parameters) preceded the treatment period, which consisted of a 2-week titration period
      followed by a 12-week maintenance period. The study aimed to determine the efficacy, safety,
      and tolerability of GWP42003-P compared with placebo. 20 mg/kg/day was recommended by the
      Data Safety Monitoring Committee (DSMC) after assessment of safety and pharmacokinetic data
      from Part A of study GWEP1332 (NCT02091206). The first participant did not enroll into this
      study until the DSMC reviewed the safety data from Part A of study GWEP1332 (NCT02091206).

      Following study completion, all participants were invited to continue to receive GWP42003-P
      in a separate open-label extension study (GWEP1415; NCT02224573).
    

Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Change In Convulsive Seizures During The Treatment Period Compared To Baseline

Secondary Outcome

 Change In Total Seizures During The Treatment Period Compared To Baseline

Condition

Epilepsy

Intervention

GWP42003-P

Study Arms / Comparison Groups

 10 mg/kg/day GWP42003-P
Description:  GWP42003-P oral solution (100 mg/milliliter [mL] cannabidiol in sesame oil with anhydrous ethanol with added sweetener [sucralose] and strawberry flavoring). The 10 mg/kg/day dose was defined as 50% of the 20 mg/kg/day dose.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

199

Start Date

April 13, 2015

Completion Date

April 9, 2018

Primary Completion Date

April 9, 2018

Eligibility Criteria

        Key Inclusion Criteria:

          -  Participant must have been male or female, aged between 2 and 18 years (inclusive).

          -  Participant must have had a documented history of Dravet syndrome that was not
             completely controlled by current antiepileptic drugs.

          -  Participant must have been taking 1 or more antiepileptic drugs at a dose that had
             been stable for at least 4 weeks.

          -  All medications or interventions for epilepsy (including ketogenic diet and vagus
             nerve stimulation) must have been stable for 4 weeks prior to screening and
             participant was willing to maintain a stable regimen throughout the study.

        Key Exclusion Criteria:

          -  Participant had clinically significant unstable medical conditions other than
             epilepsy.

          -  Participant had clinically relevant symptoms or a clinically significant illness in
             the 4 weeks prior to screening or randomization, other than epilepsy.

          -  Participant was currently using or had in the past used recreational cannabis,
             medicinal cannabis, or synthetic cannabinoid-based medications (including Sativex®)
             within the 3 months prior to study entry and was unwilling to abstain for the duration
             for the study.

          -  Participant had any known or suspected hypersensitivity to cannabinoids or any of the
             excipients of the investigational medicinal products.

          -  There were plans for the participant to travel outside their country of residence
             during the study.

          -  Any history of suicidal behavior or any suicidal ideation of type four or five on the
             Columbia-Suicide Severity Rating Scale (Children's) at screening.
      

Gender

All

Ages

2 Years - 18 Years

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

Australia

Location Countries

Australia

Administrative Informations


NCT ID

NCT02224703

Organization ID

GWEP1424

Secondary IDs

2014-002939-34

Responsible Party

Sponsor

Study Sponsor

Jazz Pharmaceuticals


Study Sponsor

, , 


Verification Date

July 2019