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

Browse trials for

• Seizures 766
• Lennox Gastaut Syndrome 68
• Epilepsies, Myoclonic 92

Keywords

• Lennox-Gastaut syndrome
• Dravet syndrome
• Treatment-resistant seizures

Seizures

766 studies on the registry are indexed under Seizures; 136 are open to participants now.

This study's enrollment of 52 is below the median of 64 across 519 interventional studies indexed under Seizures.

Lead sponsor

INSYS Therapeutics Inc is the lead sponsor of 45 studies on the registry; none are open to participants now.

Of its 5 completed or terminated interventional studies of FDA-regulated products, 3 (60%) have results posted.

Study arms

• 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.

  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.

  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.

  Drug: Cannabidiol Oral Solution

Interventions

• DrugCannabidiol Oral Solution

  An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).

Primary outcomes

1. 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.

   Time frame: Up to Week 50

2. 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.

   Time frame: Up to Week 50

3. Percentage of Participants With Clinically Significant Change From Baseline in Laboratory Values

   Laboratory values include chemistry and hematology, and urinary analysis.

   Time frame: Up to Week 50

4. Percentage of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG) Findings

   Time frame: Up to Week 48

5. Percentage of Participants With Clinically Significant Change From Baseline in Vital Signs

   Time frame: Up to Week 50

6. Change From Baseline in Trough Plasma Levels of Cannabidiol and Its 7-OH Metabolite

   Time frame: Up to Week 50

Secondary outcomes

1. 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.

   Time frame: Up to Week 48

2. 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.

   Time frame: Up to Week 50

Other outcomes

1. 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.

   Time frame: through study completion, up to 48 weeks or marketing approval, whichever is earlier

2. 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.

   Time frame: through study completion, up to 48 weeks or marketing approval, whichever is earlier

3. 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.

   Time frame: through study completion, up to 48 weeks or marketing approval, whichever is earlier

4. 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

Baseline characteristics

Study documents

• Study protocol ↗ · Mar 6, 2017
• Statistical analysis plan ↗ · Dec 4, 2017

PDFs hosted by the registry, ClinicalTrials.gov.