Cannabidiol Oral Solution as an Adjunctive Therapy for Treatment of Participants With Inadequately Controlled Dravet Syndrome

A Multicenter, Randomized, Double-blind, Placebo- Controlled, Interventional Study to Assess the Safety and Efficacy of Pharmaceutical Cannabidiol Oral Solution as an Adjunctive Therapy for Treatment of Subjects With Inadequately Controlled Dravet Syndrome

This Phase 3 study will enroll participants diagnosed with Dravet Syndrome (DS) who are still experiencing at least one tonic-clonic, clonic, and/or focal seizures with motor components (FSMC) per week, despite ongoing treatment with up to three antiepileptic drugs (AEDs), and meet the other inclusion/exclusion criteria.

Following a 28-day baseline period, participants will begin an 84-day treatment period. Participants will be assigned to receive twice-daily doses of placebo or cannabidiol oral solution at the highest dose determined to be safe in a previous trial.

Following study completion, all participants will be invited to receive Cannabidiol Oral Solution in an open label extension study (under a separate protocol).

Study Overview

• Status: Withdrawn
• Conditions: Dravet Syndrome
• Intervention / Treatment: Drug: Cannabidiol Oral Solution; Drug: Placebo Solution

• Study Type: Interventional
• Phase: Phase 3

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 30 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Meets protocol-specified criteria for qualification and contraception, including clinical diagnosis of refractory DS and onset of seizures according to protocol-specified criteria
• Is able to speak and understand the language in which the study is being conducted, is able to understand the procedures and study requirements and has voluntarily signed and dated an informed consent form approved by the Institutional Review Board before the conduct of any study procedure
• In the opinion of the Investigator, the subject and/or parent(s)/caregiver(s) are able to keep accurate seizure diaries and the participant is able to take study drug and comply with the protocol, including dosing, medications and diet

Exclusion Criteria:

• Medical history is outside protocol-specified parameters
• Clinically significant history of allergic reactions or significant sensitivities to cannabinoids or to any of the other ingredients in the study drug
• Inadequate supervision by parents or guardians
• History or current use of dietary supplements, drugs or over-the counter medications outside protocol-specified parameters
• Signs, symptoms or history of any condition that, per protocol or in the opinion of the investigator, might compromise: 1) the safety or well-being of the participant or study staff; 2) the safety or well-being of the participant's offspring (such as through pregnancy or breast-feeding); 3) the analysis of results

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cannabidiol Oral Solution; Participants will receive cannabidiol oral solution at an appropriate dose (no higher than 40 mg/kg/day) determined by data from a previous trial. The total daily dose will be administered in twice daily doses, approximately 12 hours apart.	Drug: Cannabidiol Oral Solution; An oral solution containing pharmaceutical grade cannabidiol (nonplant-based)
Placebo Comparator: Placebo Solution; Participants will receive matching placebo solution administered twice daily, approximately 12 hours apart.	Drug: Placebo Solution; A matching oral solution containing no cannabidiol

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Percent change in the frequency of tonic-clonic, clonic, and focal seizures with motor components	Data point for observation period to data point for treatment period Weeks 9 through 12

Secondary Outcome Measures

Outcome Measure	Time Frame
Percent change from baseline in the severity of tonic-clonic, clonic, and focal seizures with motor components	Data point for observation period to data point for treatment period Weeks 9 through 12
Percent change from baseline in the duration of tonic-clonic, clonic, and focal seizures with motor components	Data point for observation period to data point for treatment period Weeks 9 through 12
Percent change from baseline in the frequency of all seizure activity independent of seizure type	Data point for observation period to data point for treatment period Weeks 9 through 12
Change from baseline in parent(s)/caregiver(s) Clinical Global Impressions of Improvement (CGI-I)	Data point for observation period to data point for treatment period Weeks 9 through 12
Change from baseline in parent(s)/caregiver(s) Clinical Global Impressions of Severity (CGI-S)	Data point for observation period to data point for treatment period Weeks 9 through 12
Change from baseline in Investigator CGI-I	Data point for observation period to data point for treatment period Weeks 9 through 12
Change from baseline in Investigator CGI-S	Data point for observation period to data point for treatment period Weeks 9 through 12

Collaborators and Investigators

• Sponsor: INSYS Therapeutics Inc
• Investigators: Study Director: Neha Parikh, INSYS Therapeutics Inc

Study record dates

Study Major Dates

• Study Start (Anticipated): December 30, 2017
• Primary Completion (Anticipated): June 17, 2018
• Study Completion (Anticipated): June 17, 2018

Study Registration Dates

• First Submitted: December 12, 2014
• First Submitted That Met QC Criteria: December 12, 2014
• First Posted (Estimate): December 17, 2014

Study Record Updates

• Last Update Posted (Actual): January 4, 2018
• Last Update Submitted That Met QC Criteria: January 2, 2018
• Last Verified: January 1, 2018

More Information

Terms related to this study

• Keywords: Treatment-resistant seizures
• Additional Relevant MeSH Terms: Pathologic Processes; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epilepsy, Generalized; Epileptic Syndromes; Disease; Epilepsy; Epilepsies, Myoclonic; Syndrome; Anticonvulsants; Pharmaceutical Solutions; Cannabidiol
• Other Study ID Numbers: INS011-14-025

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No