ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02318602
Recruitment Status : Completed
First Posted : December 17, 2014
Results First Posted : July 25, 2018
Last Update Posted : July 25, 2018
Sponsor:
Information provided by (Responsible Party):
INSYS Therapeutics Inc

December 12, 2014
December 17, 2014
June 26, 2018
July 25, 2018
July 25, 2018
January 8, 2016
June 22, 2017   (Final data collection date for primary outcome measure)
  • Percentage of Participants With Adverse Events [ Time Frame: 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 [ Time Frame: 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 Laboratory Values [ Time Frame: Up to Week 50 ]
    Laboratory values include chemistry and hematology, and urinary analysis.
  • 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 ]
Number of participants experiencing adverse events [ Time Frame: through study completion, up to 48 weeks ]
Incidence and severity of adverse events (AEs), treatment-emergent AEs (TEAEs), and serious adverse events (SAEs)
Complete list of historical versions of study NCT02318602 on ClinicalTrials.gov Archive Site
  • Vineland Adaptive Behavior Scales (VABS) [ Time Frame: 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) [ Time Frame: 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.
  • Clinical Global Impression of Improvement (CGI-I) [ Time Frame: through study completion, up to 48 weeks ]
  • Clinical Global Impression of Severity (CGI-S) [ Time Frame: through study completion, up to 48 weeks ]
  • Child Behavior Checklist (CBCL) Total Problems Scale [ Time Frame: through study completion, up to 48 weeks ]
  • Impact of Pediatric Epilepsy Scale (IPES) [ Time Frame: through study completion, up to 48 weeks ]
  • Vineland Adaptive Behavior Scales (VABS) [ Time Frame: through study completion, up to 48 weeks ]
  • Vineland Maladaptive Behavior Scales (VMBS) [ Time Frame: through study completion, up to 48 weeks ]
  • Columbia-Suicide Severity Rating Scale (C-SSRS) [ Time Frame: through study completion, up to 48 weeks ]
  • Change from baseline in frequency of seizures as a measure of seizure control [ Time Frame: through study completion, up to 48 weeks ]
  • Clinical Global Impression of Severity (CGI-S) [ Time Frame: through study completion, up to 48 weeks or marketing approval, whichever is earlier ]

    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.

  • Impact of Pediatric Epilepsy Scale (IPES) [ Time Frame: through study completion, up to 48 weeks or marketing approval, whichever is earlier ]

    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.

  • Clinical Global Impression of Improvement (CGI-I) [ Time Frame: through study completion, up to 48 weeks or marketing approval, whichever is earlier ]

    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.

  • Change From Baseline in Frequency of Seizures as a Measure of Seizure Control [ Time Frame: through study completion, up to 48 weeks or marketing approval, whichever is earlier ]
Not Provided
 
Cannabidiol Oral Solution as an Adjunctive Treatment for Treatment-resistant Seizure Disorder
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
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).
Not Provided
Interventional
Phase 3
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Seizures
Drug: Cannabidiol Oral Solution
An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).
  • Experimental: Infants

    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.

    Intervention: Drug: Cannabidiol Oral Solution
  • Experimental: Children

    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.

    Intervention: Drug: Cannabidiol Oral Solution
  • Experimental: Adolescents

    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.

    Intervention: Drug: Cannabidiol Oral Solution
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
52
196
June 22, 2017
June 22, 2017   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
1 Year to 18 Years   (Child, Adult)
No
Contact information is only displayed when the study is recruiting subjects
United States
 
 
NCT02318602
INS011-14-030
Yes
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Not Provided
INSYS Therapeutics Inc
INSYS Therapeutics Inc
Not Provided
Study Director: Neha Parikh INSYS Therapeutics Inc
INSYS Therapeutics Inc
June 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP