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

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ClinicalTrials.gov Identifier: NCT02318602
Recruitment Status : Active, not recruiting
First Posted : December 17, 2014
Last Update Posted : December 5, 2017
Sponsor:
Information provided by (Responsible Party):

Study Description
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 and adult 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), INS011-14-024 (NCT02318537), or INS011- 14-025 (NCT02318563).

Condition or disease Intervention/treatment Phase
Seizures Drug: Cannabidiol Oral Solution Phase 3

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 54 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 and Adult Subjects With a Treatment-resistant Seizure Disorder Who Complete INS011-14-029 or Part A of INS011-15-054
Actual Study Start Date : January 2016
Estimated Primary Completion Date : December 2017
Estimated Study Completion Date : December 2017


Arms and Interventions

Arm Intervention/treatment
Experimental: Cannabidiol Oral Solution

Participants who completed INS011-14-029 will begin this study on the dose with which they were being treated previously, unless they experienced tolerability issues. When the dose is selected for INS011-14-024 and INS011-14-025, participants will be transitioned to that dose (a maximum of 40 mg/kg/day).

Participants who completed INS011-14-024 and INS011-14-025 will continue treatment with the dose at which they were being treated previously according to those protocols, unless they experienced tolerability issues. This dose will be based on the results of INS011-014-029 and established prior to recruitment of the first participant in these studies.

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.

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


Outcome Measures

Primary Outcome Measures :
  1. Percentage of participants with adverse events [ Time Frame: through study completion, up to 48 weeks or marketing approval, whichever is earlier ]
    Categories: adverse events (AEs), treatment-emergent AEs (TEAEs), and serious adverse events (SAEs)

  2. Percentage of participants with severe adverse events [ Time Frame: through study completion, up to 48 weeks or marketing approval, whichever is earlier ]
    Categories: adverse events (AEs), treatment-emergent AEs (TEAEs), and serious adverse events (SAEs)

  3. Percentage of participants with clinically significant change from baseline in safety parameters [ Time Frame: through study completion, up to 48 weeks or marketing approval, whichever is earlier ]
    Safety parameters will include 12-lead electrocardiogram (ECG), chemistry and hematology, and urinary analysis.

  4. Change from baseline in trough plasma levels of cannabidiol and its 7-OH metabolite [ Time Frame: through study completion, up to 48 weeks or marketing approval, whichever is earlier ]

Secondary Outcome Measures :
  1. 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.


  2. 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.


  3. 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.


  4. Vineland Adaptive Behavior Scales (VABS) [ Time Frame: through study completion, up to 48 weeks or marketing approval, whichever is earlier ]

    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. 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, regardless of chronological and developmental age.


  5. Columbia-Suicide Severity Rating Scale (C-SSRS) [ Time Frame: through study completion, up to 48 weeks or marketing approval, whichever is earlier ]
    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.

  6. 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 ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 30 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Completed all scheduled visits in the treatment phase of their core study
  • 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
  • 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
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02318602


Locations
United States, California
University of California San Francisco Medical Center
San Francisco, California, United States, 94143
United States, Florida
Miami Children's Hospital
Miami, Florida, United States, 33155
Child Neurology Center - NW F
Pensacola, Florida, United States, 32504
United States, Illinois
University of Chicago Medical Center
Chicago, Illinois, United States, 60637
United States, Nevada
Clinical Research Center of Nevada LLC
Las Vegas, Nevada, United States, 89104
United States, Oregon
Oregon Health Services University
Portland, Oregon, United States, 97239
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, Tennessee
Le Bonheur Children's Hospital
Memphis, Tennessee, United States, 38103
United States, Texas
Texas Scottish Rite Hospital for Children
Dallas, Texas, United States, 79219
United States, Utah
Granger Medical Clinic
Riverton, Utah, United States, 84096
United States, Washington
Mary Bridge Children's Hospital
Tacoma, Washington, United States, 98403
Sponsors and Collaborators
INSYS Therapeutics Inc
Investigators
Study Director: Neha Parikh INSYS Therapeutics Inc
More Information

Responsible Party: INSYS Therapeutics Inc
ClinicalTrials.gov Identifier: NCT02318602     History of Changes
Other Study ID Numbers: INS011-14-030
First Posted: December 17, 2014    Key Record Dates
Last Update Posted: December 5, 2017
Last Verified: December 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by INSYS Therapeutics Inc:
Lennox-Gastaut syndrome
Dravet syndrome
Treatment-resistant seizures

Additional relevant MeSH terms:
Seizures
Epilepsy
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurologic Manifestations
Signs and Symptoms
Pharmaceutical Solutions