Cannabidiol Oral Solution as an Adjunctive Treatment for Treatment-resistant Seizure Disorder
- Registration Number
- NCT02318602
- Lead Sponsor
- INSYS Therapeutics Inc
- Brief 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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 52
- 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
-
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:
- the safety or well-being of the participant or study staff
- the safety or well-being of the participant's offspring (such as through pregnancy or breast-feeding)
- the analysis of results
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Infants Cannabidiol Oral Solution Participants 1 to\<2 years of age. Participants who completed INS011-14-029 initiated this study on the dose with which they were being treated previously, and dose modifications were made at the Investigator's discretion if tolerability or efficacy issues were observed for a particular participant. The maximum daily dose was 40 mg/kg/day. Participants who enrolled from INS011-15-054 continued treatment with the dose at which they were being treated previously, and dose modifications were made at the Investigator's discretion if tolerability or efficacy issues were observed for a particular participant. The total daily dose, milligrams per kilograms per day (mg/kg/day), will be evenly split between morning and evening doses (12 hours apart). If tolerability issues arise, the participant's dose may be changed at the investigator's discretion. Children Cannabidiol Oral Solution Participants 2 to \<12 years of age. Participants who completed INS011-14-029 initiated this study on the dose with which they were being treated previously, and dose modifications were made at the Investigator's discretion if tolerability or efficacy issues were observed for a particular participant.The maximum daily dose was 40 mg/kg/day. Participants who enrolled from INS011-15-054 continued treatment with the dose at which they were being treated previously, and dose modifications were made at the Investigator's discretion if tolerability or efficacy issues were observed for a particular participant. The total daily dose (mg/kg/day) will be evenly split between morning and evening doses (12 hours apart). If tolerability issues arise, the participant's dose may be changed at the investigator's discretion. Adolescents Cannabidiol Oral Solution Participants 12 to \<17 years of age. Participants who completed INS011-14-029 initiated this study on the dose with which they were being treated previously, and dose modifications were made at the Investigator's discretion if tolerability or efficacy issues were observed for a particular participant. The maximum daily dose was 40 mg/kg/day. Participants who enrolled from INS011-15-054 continued treatment with the dose at which they were being treated previously, and dose modifications were made at the Investigator's discretion if tolerability or efficacy issues were observed for a particular participant. The total daily dose (mg/kg/day) will be evenly split between morning and evening doses (12 hours apart). If tolerability issues arise, the participant's dose may be changed at the investigator's discretion.
- Primary Outcome Measures
Name Time Method Percentage of Participants With Clinically Significant Change From Baseline in Laboratory Values Up to Week 50 Laboratory values include chemistry and hematology, and urinary analysis.
Percentage of Participants With Adverse Events Up to Week 50 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.
Percentage of Participants With Serious Adverse Events Up to Week 50 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.
Percentage of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG) Findings Up to Week 48 Percentage of Participants With Clinically Significant Change From Baseline in Vital Signs Up to Week 50 Change From Baseline in Trough Plasma Levels of Cannabidiol and Its 7-OH Metabolite Up to Week 50
- Secondary Outcome Measures
Name Time Method Vineland Adaptive Behavior Scales (VABS) Up to Week 48 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.Number of Participants With a Positive Response on the Columbia-Suicide Severity Rating Scale (C-SSRS) Up to Week 50 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.
Trial Locations
- Locations (11)
Granger Medical Clinic
🇺🇸Riverton, Utah, United States
Le Bonheur Children's Hospital
🇺🇸Memphis, Tennessee, United States
Child Neurology Center - NW F
🇺🇸Pensacola, Florida, United States
University of Chicago Medical Center
🇺🇸Chicago, Illinois, United States
Mary Bridge Children's Hospital
🇺🇸Tacoma, Washington, United States
Texas Scottish Rite Hospital for Children
🇺🇸Dallas, Texas, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
University of California San Francisco Medical Center
🇺🇸San Francisco, California, United States
Miami Children's Hospital
🇺🇸Miami, Florida, United States
Oregon Health Services University
🇺🇸Portland, Oregon, United States
Clinical Research Center of Nevada LLC
🇺🇸Las Vegas, Nevada, United States