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
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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
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Verification Date
July 2018