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

NCT02318563 | Withdrawn Phase 3 DMC FDA Drug

• 1 other identifier: INS011-14-025
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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).

Conditions

Dravet Syndrome

Keywords

Treatment-resistant seizures

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (7)

• 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

Study Arms (2)

Cannabidiol Oral Solution

EXPERIMENTAL

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

Placebo Solution

PLACEBO COMPARATOR

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

Drug: Placebo Solution

Interventions

Cannabidiol Oral Solution DRUG

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

Cannabidiol Oral Solution

Placebo Solution DRUG

A matching oral solution containing no cannabidiol

Placebo Solution

Eligibility Criteria

• Age: 1 Year - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• INSYS Therapeutics Inc: lead

MeSH Terms

Conditions

Epilepsies, Myoclonic

Interventions

Cannabidiol

Condition Hierarchy (Ancestors)

Epilepsy, Generalized; Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epileptic Syndromes

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals

Study Officials

• Neha Parikh

  INSYS Therapeutics Inc

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 12, 2014
• First Posted: December 17, 2014
• Study Start: December 30, 2017
• Primary Completion: June 17, 2018
• Study Completion: June 17, 2018
• Last Updated: January 4, 2018

Record last verified: 2018-01