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

Primary Purpose

Dravet Syndrome

Status

Withdrawn

Phase

Phase 3

Locations

Study Type

Interventional

Intervention

Cannabidiol Oral Solution

Placebo Solution

About this trial

This is an interventional treatment trial for Dravet Syndrome focused on measuring Treatment-resistant seizures

Eligibility Criteria

1 Year - 30 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Cannabidiol Oral Solution

Placebo Solution

Arm Description

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.

Participants will receive matching placebo solution administered twice daily, approximately 12 hours apart.

Outcomes

Primary Outcome Measures

Percent change in the frequency of tonic-clonic, clonic, and focal seizures with motor components

Secondary Outcome Measures

Percent change from baseline in the severity of tonic-clonic, clonic, and focal seizures with motor components

Percent change from baseline in the duration of tonic-clonic, clonic, and focal seizures with motor components

Percent change from baseline in the frequency of all seizure activity independent of seizure type

Change from baseline in parent(s)/caregiver(s) Clinical Global Impressions of Improvement (CGI-I)

Change from baseline in parent(s)/caregiver(s) Clinical Global Impressions of Severity (CGI-S)

Change from baseline in Investigator CGI-I

Change from baseline in Investigator CGI-S

Full Information

NCT ID

NCT02318563

First Posted

December 12, 2014

Last Updated

January 2, 2018

Sponsor

INSYS Therapeutics Inc

1. Study Identification

Unique Protocol Identification Number

NCT02318563

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

January 2018

Overall Recruitment Status

Withdrawn

Why Stopped

Sponsor elected not to continue with study.

Study Start Date

December 30, 2017 (Anticipated)

Primary Completion Date

June 17, 2018 (Anticipated)

Study Completion Date

June 17, 2018 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

INSYS Therapeutics Inc

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Dravet Syndrome

Keywords

Treatment-resistant seizures

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigator

Allocation

Randomized

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Cannabidiol Oral Solution

Arm Type

Experimental

Arm Description

Arm Title

Placebo Solution

Arm Type

Placebo Comparator

Arm Description

Participants will receive matching placebo solution administered twice daily, approximately 12 hours apart.

Intervention Type

Drug

Intervention Name(s)

Cannabidiol Oral Solution

Intervention Description

An oral solution containing pharmaceutical grade cannabidiol (nonplant-based)

Intervention Type

Drug

Intervention Name(s)

Placebo Solution

Intervention Description

A matching oral solution containing no cannabidiol

Primary Outcome Measure Information:

Title

Percent change in the frequency of tonic-clonic, clonic, and focal seizures with motor components

Time Frame