Antiepileptic Efficacy Study of GWP42003-P in Children and Young Adults With Dravet Syndrome (GWPCARE1)

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Brief Title

Antiepileptic Efficacy Study of GWP42003-P in Children and Young Adults With Dravet Syndrome (GWPCARE1)

Official Title

A Double Blind, Placebo Controlled Two-part Study to Investigate the Dose-ranging Safety and Pharmacokinetics, Followed by 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

      GWEP1332 Part B recruited an entirely new group of participants than GWEP1332 Part A.
      Participants who failed the entry criteria for Part A were eligible to take part in Part B.

      Part B was a 1:1 randomized, double-blind, placebo-controlled, 14-week comparison of
      GWP42003-P versus placebo. The aim of Part B was to assess the antiepileptic efficacy of
      GWP42003-P as an adjunctive antiepileptic treatment compared with placebo, with respect to
      the percentage change from baseline during the treatment period of the study in convulsive
      seizure frequency in children and young adults.

      Following the establishment of initial eligibility and baseline measurements, participants
      entered Part B and began a 28-day baseline observation period.

      Eligible participants were then randomized to receive either GWP42003-P or placebo on a 1:1
      basis and titrated up to the target dose that was identified in Part A (up to 20 milligrams
      [mg] per kilogram [kg] per day), which was confirmed following completion of Part A by an
      independent Data Safety Monitoring Committee who reviewed unblinded safety and
      pharmacokinetic data from Part A.

      Participants received investigational medicinal product for 14 weeks, consisting of a
      titration period followed by a 12-week maintenance period.

      Efficacy and safety were monitored at various clinic visits and via telephone. After 14 weeks
      of treatment, all participants were offered the option of entering an open label extension
      (OLE) study. Entry was within seven days of the final treatment visit. Participants who did
      not immediately enter the OLE study commenced a down-titration taper period lasting up to 10
      days. The taper period was interrupted if the participant wished to enter the open label
      extension study within the seven-day timeframe.

      For participants who opted not to enter the OLE study, a follow-up telephone call was made 28
      days after the end of dosing and weekly safety telephone calls were made during the 28-day
      follow-up period.
    

Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Percentage Change From Baseline In Convulsive Seizure Frequency During The Treatment Period

Secondary Outcome

 Number Of Participants With A ≥50% Reduction From Baseline In Convulsive Seizure Frequency During The Treatment Period

Condition

Epilepsy

Intervention

GWP42003-P 20 mg/kg/day Dose

Study Arms / Comparison Groups

 GWP42003-P 20 mg/kg/day Dose
Description:  Participants received 20 mg/kg/day of GWP42003-P administered orally, half in the morning and half in the evening. Participants titrated GWP42003-P to 20 mg/kg/day over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day) period.

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

120

Start Date

March 30, 2015

Completion Date

November 26, 2015

Primary Completion Date

November 26, 2015

Eligibility Criteria

        Key Inclusion Criteria:

          -  Participants were male or female aged between 2 and 18 years (inclusive).

          -  Participants had a documented history of Dravet Syndrome that was not completely
             controlled by current antiepileptic drugs.

          -  Participants took one or more antiepileptic drugs at a dose that had been stable for
             at least four weeks.

          -  All medications or interventions for epilepsy (including ketogenic diet and vagus
             nerve stimulation) were stable for four weeks prior to screening and participants were
             willing to maintain a stable regimen throughout the study.

        Key Exclusion Criteria:

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

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

          -  Participants were currently using or had in the past used recreational or medicinal
             cannabis or synthetic cannabinoid based medications (including Sativex®) within the
             three 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 investigational medicinal products.

          -  Participants had been part of a previous clinical trial involving another
             investigational product in the previous six months.

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

          -  Participants previously randomized into this study. In particular, participants who
             participated in Part A of the study could not enter Part B.
      

Gender

All

Ages

2 Years - 18 Years

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

France

Location Countries

France

Administrative Informations


NCT ID

NCT02091375

Organization ID

GWEP1332 Part B

Secondary IDs

2014-002941-23

Responsible Party

Sponsor

Study Sponsor

Jazz Pharmaceuticals


Study Sponsor

, , 


Verification Date

July 2018