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

Seizures Clinical Trial

Official title:

A Multicenter, Open-label, Flexible Dose Study to Assess the Long-term Safety of Pharmaceutical Cannabidiol Oral Solution as an Adjunctive Treatment for Pediatric Subjects With a Treatment-resistant Seizure Disorder Who Complete INS011-14-029 or Part A of INS011-15-054

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02318602
• Other study ID #: INS011-14-030
• Status: Completed
• Phase: Phase 3
• Start date: January 8, 2016
• Est. completion date: June 22, 2017

Study information

• Verified date: June 2018
• Source: INSYS Therapeutics Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 52
• Est. completion date: June 22, 2017
• Est. primary completion date: June 22, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Epilepsy
• Seizures

Intervention

Drug:
• Cannabidiol Oral Solution
An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).

Locations

Country	Name	City	State
United States	University of Chicago Medical Center	Chicago	Illinois
United States	Texas Scottish Rite Hospital for Children	Dallas	Texas
United States	Clinical Research Center of Nevada LLC	Las Vegas	Nevada
United States	Le Bonheur Children's Hospital	Memphis	Tennessee
United States	Miami Children's Hospital	Miami	Florida
United States	Child Neurology Center - NW F	Pensacola	Florida
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Oregon Health Services University	Portland	Oregon
United States	Granger Medical Clinic	Riverton	Utah
United States	University of California San Francisco Medical Center	San Francisco	California
United States	Mary Bridge Children's Hospital	Tacoma	Washington

Sponsors (1)

Lead Sponsor	Collaborator
INSYS Therapeutics Inc

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Clinical Global Impression of Severity (CGI-S)	The severity of the participant's illness is rated on a seven-point scale, where 1=normal, not at all ill, and 7=among the most extremely ill patients. This rating is based upon observed and reported symptoms, behavior, and function in the past seven days. The score reflects the average severity level across the seven days.; The CGI-S will be completed for all participants, regardless of chronological and developmental age.	through study completion, up to 48 weeks or marketing approval, whichever is earlier
Other	Impact of Pediatric Epilepsy Scale (IPES)	The IPES assesses the impact on academic achievement, participation in activities, health, relationships with family and with peers and siblings, social activities, self-esteem, and the caregiver's hopes for their child's future. It takes about 3 minutes for the parent to complete. Each of the 11 items is given a severity score of 0 (not at all) to 3 (a lot). The higher the score, the higher is the impact of epilepsy on that item. The highest total score possible is 33 (range 0-33).; The Impact of Pediatric Epilepsy Scale (IPES) is validated for subjects who are 2 to 16 years of age. Due to developmental delay characteristic of the study population, subjects through 18 years of chronological age will complete the IPES. Subjects over 18 years of chronological age will not complete the IPES.	through study completion, up to 48 weeks or marketing approval, whichever is earlier
Other	Clinical Global Impression of Improvement (CGI-I)	The participant's overall clinical condition is compared to the one week period just before the start of medication (the baseline visit). The participant's condition is compared to the patient's condition at admission to the project [prior to starting treatment] on a 7-point scale, where 1=very much improved since the initiation of treatment; and 7=very much worse since the initiation of treatment.; The CGI-I will be completed for all participants, regardless of chronological and developmental age.	through study completion, up to 48 weeks or marketing approval, whichever is earlier
Other	Change From Baseline in Frequency of Seizures as a Measure of Seizure Control		through study completion, up to 48 weeks or marketing approval, whichever is earlier
Primary	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.	Up to Week 50
Primary	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.	Up to Week 50
Primary	Percentage of Participants With Clinically Significant Change From Baseline in Laboratory Values	Laboratory values include chemistry and hematology, and urinary analysis.	Up to Week 50
Primary	Percentage of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG) Findings		Up to Week 48
Primary	Percentage of Participants With Clinically Significant Change From Baseline in Vital Signs		Up to Week 50
Primary	Change From Baseline in Trough Plasma Levels of Cannabidiol and Its 7-OH Metabolite		Up to Week 50
Secondary	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.	Up to Week 48
Secondary	Number of Participants With a Positive Response on the Columbia-Suicide Severity Rating Scale (C-SSRS)	For subjects 7 years of developmental age or older, the Columbia-Suicide Severity Rating Scale (C-SSRS) will be administered to access suicidality. The appropriate adult version will be used in subjects 12 years of (developmental) age and older. The investigator will determine the participant's developmental age.	Up to Week 50