Clinical trial NCT02318602: Cannabidiol Oral Solution as an Adjunctive Treatment for Treatment-resistant Seizure Disorder
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Seizures
Keywords: Lennox-Gastaut syndrome
Dravet syndrome
Treatment-resistant seizures
Lead sponsor: INSYS Therapeutics Inc
Sponsor class: INDUSTRY
First posted: 2014-12-17
Enrollment: 52
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).


Eligibility criteria: 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

Primary outcomes:
measure: Percentage of Participants With Adverse Events
timeFrame: Up to Week 50
measure: Percentage of Participants With Serious Adverse Events
timeFrame: Up to Week 50
measure: Percentage of Participants With Clinically Significant Change From Baseline in Laboratory Values
timeFrame: Up to Week 50
measure: Percentage of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG) Findings
timeFrame: Up to Week 48
measure: Percentage of Participants With Clinically Significant Change From Baseline in Vital Signs
timeFrame: Up to Week 50
measure: Change From Baseline in Trough Plasma Levels of Cannabidiol and Its 7-OH Metabolite
timeFrame: Up to Week 50
Secondary outcomes:
measure: Vineland Adaptive Behavior Scales (VABS)
timeFrame: Up to Week 48
measure: Number of Participants With a Positive Response on the Columbia-Suicide Severity Rating Scale (C-SSRS)
timeFrame: Up to Week 50
Other outcomes:
measure: Clinical Global Impression of Severity (CGI-S)
timeFrame: through study completion, up to 48 weeks or marketing approval, whichever is earlier
measure: Impact of Pediatric Epilepsy Scale (IPES)
timeFrame: through study completion, up to 48 weeks or marketing approval, whichever is earlier
measure: Clinical Global Impression of Improvement (CGI-I)
timeFrame: through study completion, up to 48 weeks or marketing approval, whichever is earlier
measure: Change From Baseline in Frequency of Seizures as a Measure of Seizure Control
timeFrame: through study completion, up to 48 weeks or marketing approval, whichever is earlier
Locations:
city: San Francisco
country: United States
facility: University of California San Francisco Medical Center
state: California
city: Miami
country: United States
facility: Miami Children's Hospital
state: Florida
city: Pensacola
country: United States
facility: Child Neurology Center - NW F
state: Florida
city: Chicago
country: United States
facility: University of Chicago Medical Center
state: Illinois
city: Las Vegas
country: United States
facility: Clinical Research Center of Nevada LLC
state: Nevada
city: Portland
country: United States
facility: Oregon Health Services University
state: Oregon
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Memphis
country: United States
facility: Le Bonheur Children's Hospital
state: Tennessee
city: Dallas
country: United States
facility: Texas Scottish Rite Hospital for Children
state: Texas
city: Riverton
country: United States
facility: Granger Medical Clinic
state: Utah
city: Tacoma
country: United States
facility: Mary Bridge Children's Hospital
state: Washington
Study officials:
affiliation: INSYS Therapeutics Inc
name: Neha Parikh
role: STUDY_DIRECTOR
References: