Extension Study for Dravet and Lennox-Gastaut Syndrome

Principal Investigator: Francis  Filloux
Keywords: Cannabidioal , Dravet , GW Pharmaceuticals , Lennox-Gastaut , CBD Department: Pediatric Neurology
IRB Number: 00080756 Co Investigator:  
Specialty: Pediatric Neurology, Pediatric Neurology
Sub Specialties: Pediatric Epilepsy,
Recruitment Status: Recruiting

Contact Information

Fumiko Alger

Brief Summary

To evaluate the long term safety and tolerability of GWP42003-P, as adjunctive treatment, in children and adults with inadequately controlled DS or LGS.

All Patients:
To evaluate the effect of GWP42003-P, as adjunctive treatment, on:
• Quality of life.
• Adaptive behavior.
• Need for hospitalizations due to epilepsy.
• Usage of rescue medication.
• Maintenance of seizure frequency reduction and freedom from
seizures during the open label extension (OLE) study.
• Frequency of total and subtypes of seizures.
• Change in duration of subtypes of seizures.
• Number of episodes of status epilepticus.
• Cognitive function.
• Signals indicating drug abuse liability of GWP42003-P.
DS Patients Only:
To evaluate the effect of GWP42003-P, as adjunctive treatment, on:
• Total convulsive seizure frequency.
• Total non-convulsive seizure frequency.
• Number of patients convulsive seizure free.
• Responder rate (defined in terms of percentage reduction in total
convulsive seizure frequency).
LGS Patients Only:
To evaluate the effect of GWP42003-P, as adjunctive treatment, on:
• Drop seizure frequency.
• Non-drop seizure frequency.
• Number of patients drop seizure free.
• Responder rate (defined in terms of percentage reduction in drop
seizure frequency).

Inclusion Criteria

Inclusion Criteria

For inclusion in the study patients must fulfill ALL of the following criteria:

1. Patient has completed all scheduled visits in the treatment phase of their
Core Study.
2. Patient and/or parent(s)/legal representative must be willing and able to
give informed consent/assent for participation in the study (see
Section 15.2).
3. Patient and their caregiver must be willing and able (in the
investigator’s opinion) to comply with all study requirements.
4. Patient and/or parent(s)/legal representative is willing to allow his or her
primary care practitioner and consultant to be notified of participation in
the study.

Exclusion Criteria

The patient may not enter the study if ANY of the following apply:
1.     Patient is currently using or has in the past used recreational or medicinal cannabis, or 
synthetic cannabinoid based medications (including Sativex®) within the three months prior to study 
entry, not including IMP received during the Core study.
2.     Patient is unwilling to abstain from using recreational or medicinal cannabis, or 
synthetic cannabinoid based medications (including Sativex) during the study.
3. Patient has a history of symptoms (e.g., dizziness, light-headedness, blurred vision, 
palpitations, weakness, syncope) related to a drop in blood pressure due to postural changes.
4.     Any history of suicidal behavior or any suicidal ideation of type four o five on the 
C-SSRS at Visit 1.

5.     Patient has been part of a clinical trial involving an IMP during the inter-study period.
6.     Patient has previously been enrolled and dosed in this study.
7.     Female patient is of child bearing potential and must have a negative pregnancy test and 
be willing and able to use a reliable method of contraception throughout the trial and for three 
months after last dose. Male patient’s partner is of child bearing potential; unless willing to

ensure that they or their partner use a highly effective contraception. In the context of this 
trial, a highly effective method is defined as those which result in low failure rate (i.e. less 
than 1% per year) when used consistently and correctly such as: combined or progesterone only oral 
contraceptives, intrauterine device, intrauterine hormone-releasing system, bilateral tubal 
occlusion, vasectomized partner or sexual abstinence.
8.     Female patient is pregnant, lactating or planning pregnancy during the course of the 
study and for three months thereafter.
9.     Any other significant disease or disorder which, in the opinion of the investigator, may 
either put the patient at risk because of participation in the study, may influence the result of 
the study, or affect the patient’s ability to participate in the study.
10.   Following a physical examination the patient has any abnormalities that, in the opinion of 
the investigator, would prevent the patient from safe participation in the study.
11.   Patient is unwilling to abstain from donation of blood during the study.
12.   Patient has significantly impaired hepatic function at the ‘End of Treatment’ visit of their Core Study or at Visit 1 if re-assessed, defined as any of the following:

  1. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >5 × upper limit of normal (ULN).
  2. ALT or AST >3 × ULN and (total bilirubin [TBL] >2 × ULN or international normalized ratio [INR] >1.5).
  3. ALT or AST >3 × ULN with the presence of fatigue, nausea, vomiting, right upper quadrant pain or tenderness, fever, rash, and/or eosinophilia (>5%).

If the Core Study ‘End of Treatment’ / ‘End of Taper Period’ visit assessments or Visit 1 re-assessments (as applicable) raise any safety concerns, the Investigator should consider whether it will be appropriate for the patient to continue to participate in the OLE study, or if the patient should be withdrawn.