Cannabidiol Oral Solution as an Adjunctive Treatment for Treatment-resistant Seizure Disorder

INSYS Therapeutics Inc52 enrolled

Overview

This is a multicenter, open-label trial to assess the long-term safety and efficacy of Cannabidiol Oral Solution as adjunctive therapy for pediatric participants with treatment-resistant seizure disorders, including Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS). All participants have rolled over from previous trials: INS011-14-029 (NCT02324673) and INS011-15-054 (NCT02551731).

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Seizures

Interventions

Cannabidiol Oral SolutionDRUG

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

Eligibility

Sex: ALLMin age: 1 YearMax age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Completed activities through Day 11 in INS011-14-029 or Part A (Visit 6) INS011-15-054 * Informed consent/assent (as applicable) was voluntarily provided by the participant and/or parent(s)/caregiver(s) in accordance with applicable laws, regulations, and local requirements * Is medically stable with no anticipated changes in chronic medications in the opinion of the Investigator * Continues to meet protocol-specified criteria for qualification and contraception, including treatment-resistant seizure disorder * In the opinion of the Investigator, the subject and/or parent(s)/caregiver(s) are able to continue keeping accurate seizure diaries Exclusion Criteria: * Inadequate supervision by parent(s)/caregiver(s) * 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

Locations (11)

University of California San Francisco Medical Center

San Francisco, California, United States

Miami Children's Hospital

Miami, Florida, United States

Child Neurology Center - NW F

Pensacola, Florida, United States

Outcomes

Primary Outcomes

Percentage of Participants With Adverse Events

An Adverse Event (AE) is any untoward medical occurrence in a subject administered a pharmaceutical product. It does not necessarily have a causal relationship with this treatment.

Time frame: Up to Week 50

Percentage of Participants With Serious Adverse Events

A serious adverse event is any untoward medical occurrence ( whether considered to be related to investigational product or not) that at any dose results in death, is life threatening, requires inpatient hospitalization, results in disability/incapacity, is a congenital abnormality/ birth defect, or medically significant as determined by an investigator.

Time frame: Up to Week 50

Percentage of Participants With Clinically Significant Change From Baseline in Laboratory Values

Laboratory values include chemistry and hematology, and urinary analysis.

Time frame: Up to Week 50

Percentage of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG) Findings

Time frame: Up to Week 48

Percentage of Participants With Clinically Significant Change From Baseline in Vital Signs

Time frame: Up to Week 50

Change From Baseline in Trough Plasma Levels of Cannabidiol and Its 7-OH Metabolite

Time frame: Up to Week 50

Secondary Outcomes

Vineland Adaptive Behavior Scales (VABS)

The Vineland Adaptive Behavior Scales measures the personal and social skills of individuals from birth through adulthood. Because adaptive behavior refers to an individual's typical performance of the day-to-day activities required for personal and social sufficiency, these scales assess what a person actually does, rather than what he or she is able to do. The Vineland Adaptive Behavior Scales, Second Edition (Survey Interview Form) is a measure of adaptive behavior in children, adolescents and adults. It yields an overall standard score (Adaptive Behavior Composite, ABC) and age standard scores in four domains. The 4 domains are Communication, Daily Living Skills, Motor Skills, and Maladaptive Behaviour Index. ABC scores have a mean of 100 and a standard deviation of 15 (range = 20 to 160). Higher scores suggest a higher level of adaptive functioning. A rise in standard scores from Baseline indicates improvement. The VABS will be completed for all participants.

Data from ClinicalTrials.gov

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