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

INSYS Therapeutics

Status and phase: Withdrawn; Phase 3

Conditions: Dravet Syndrome

Treatments: Drug: Placebo Solution; Drug: Cannabidiol Oral Solution

Study type: Interventional

Funder types: Industry

Identifiers

NCT02318563

INS011-14-025

Details and patient eligibility

About

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).

Sex: All

Ages: 1 to 30 years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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0 participants in 2 patient groups, including a placebo group

Cannabidiol Oral Solution

Experimental group

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.

Treatment:

Drug: Cannabidiol Oral Solution

Placebo Solution

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo Solution