Cannabidiol Oral Solution as an Adjunctive Treatment for Treatment-resistant Seizure Disorder
Sponsor
INSYS Therapeutics Inc (Industry)
Overall Status
Completed
CT.gov ID
NCT02318602
Collaborator
(none)
52
11
3
17.4
4.7
0.3
Study Details
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 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).
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
Study Design
Study Type:
Interventional
Actual Enrollment
:
52 participants
Allocation:
Non-Randomized
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 Subjects With a Treatment-resistant Seizure Disorder Who Complete INS011-14-029 or Part A of INS011-15-054
Actual Study Start Date
:
Jan 8, 2016
Actual Primary Completion Date
:
Jun 22, 2017
Actual Study Completion Date
:
Jun 22, 2017
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| 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
An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).
|
| 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
An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).
|
| 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
An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).
|
Outcome Measures
Primary Outcome Measures
- 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 Laboratory Values [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 [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
- 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.
Other Outcome Measures
- Clinical Global Impression of Severity (CGI-S) [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) [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) [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 [through study completion, up to 48 weeks or marketing approval, whichever is earlier]
Eligibility Criteria
Criteria
Ages Eligible for Study:
1 Year
to 18 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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:
-
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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | University of California San Francisco Medical Center | San Francisco | California | United States | 94143 |
| 2 | Miami Children's Hospital | Miami | Florida | United States | 33155 |
| 3 | Child Neurology Center - NW F | Pensacola | Florida | United States | 32504 |
| 4 | University of Chicago Medical Center | Chicago | Illinois | United States | 60637 |
| 5 | Clinical Research Center of Nevada LLC | Las Vegas | Nevada | United States | 89104 |
| 6 | Oregon Health Services University | Portland | Oregon | United States | 97239 |
| 7 | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | United States | 19104 |
| 8 | Le Bonheur Children's Hospital | Memphis | Tennessee | United States | 38103 |
| 9 | Texas Scottish Rite Hospital for Children | Dallas | Texas | United States | 79219 |
| 10 | Granger Medical Clinic | Riverton | Utah | United States | 84096 |
| 11 | Mary Bridge Children's Hospital | Tacoma | Washington | United States | 98403 |
Sponsors and Collaborators
- INSYS Therapeutics Inc
Investigators
- Study Director: Neha Parikh, INSYS Therapeutics Inc
Study Documents (Full-Text)
More Information
Publications
None provided.Responsible Party:
INSYS Therapeutics Inc
ClinicalTrials.gov Identifier:
NCT02318602
Other Study ID Numbers:
- INS011-14-030
First Posted:
Dec 17, 2014
Last Update Posted:
Jul 26, 2018
Last Verified:
Jun 1, 2018
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by INSYS Therapeutics Inc
Additional relevant MeSH terms:
Study Results
Participant Flow
| Recruitment Details | |
|---|---|
| Pre-assignment Detail |
| Arm/Group Title | Infants | Children | Adolescents |
|---|---|---|---|
| Arm/Group Description | 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. 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. | 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. 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. | 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. 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. |
| Period Title: Overall Study | |||
| STARTED | 9 | 26 | 17 |
| COMPLETED | 8 | 22 | 15 |
| NOT COMPLETED | 1 | 4 | 2 |
Baseline Characteristics
| Arm/Group Title | Infants | Children | Adolescents | Total |
|---|---|---|---|---|
| Arm/Group Description | 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. 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. | 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. 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. | 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. 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. | Total of all reporting groups |
| Overall Participants | 9 | 26 | 17 | 52 |
| Age (Years) [Mean (Standard Deviation) ] | ||||
| Mean (Standard Deviation) [Years] |
1.00
(0.00)
|
6.7
(2.84)
|
13.9
(1.58)
|
8.1
(5.07)
|
| Sex: Female, Male (Count of Participants) | ||||
| Female |
4
44.4%
|
11
42.3%
|
9
52.9%
|
24
46.2%
|
| Male |
5
55.6%
|
15
57.7%
|
8
47.1%
|
28
53.8%
|
| Ethnicity (NIH/OMB) (Count of Participants) | ||||
| Hispanic or Latino |
2
22.2%
|
2
7.7%
|
1
5.9%
|
5
9.6%
|
| Not Hispanic or Latino |
7
77.8%
|
24
92.3%
|
16
94.1%
|
47
90.4%
|
| Unknown or Not Reported |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Race (NIH/OMB) (Count of Participants) | ||||
| American Indian or Alaska Native |
0
0%
|
1
3.8%
|
1
5.9%
|
2
3.8%
|
| Asian |
0
0%
|
1
3.8%
|
0
0%
|
1
1.9%
|
| Native Hawaiian or Other Pacific Islander |
0
0%
|
1
3.8%
|
0
0%
|
1
1.9%
|
| Black or African American |
0
0%
|
2
7.7%
|
0
0%
|
2
3.8%
|
| White |
8
88.9%
|
19
73.1%
|
16
94.1%
|
43
82.7%
|
| More than one race |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Unknown or Not Reported |
1
11.1%
|
2
7.7%
|
0
0%
|
3
5.8%
|
| Region of Enrollment (participants) [Number] | ||||
| United States |
9
100%
|
26
100%
|
17
100%
|
52
100%
|
Outcome Measures
| Title | Percentage of Participants With Adverse Events |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Infants | Children | Adolescents | All Participants |
|---|---|---|---|---|
| Arm/Group Description | 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. 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. | 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. 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. | 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. 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. | All participants who participated in the study. |
| Measure Participants | 9 | 26 | 17 | 52 |
| Number [Percentage of participants] |
88.9
987.8%
|
92.3
355%
|
88.2
518.8%
|
90.4
173.8%
|
| Title | Percentage of Participants With Serious Adverse Events |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Infants | Children | Adolescents | All Participants |
|---|---|---|---|---|
| Arm/Group Description | 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. 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. | 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. 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. | 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. 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. | All participants who participated in the study. |
| Measure Participants | 9 | 26 | 17 | 52 |
| Number [Percentage of participants] |
77.8
864.4%
|
38.5
148.1%
|
0
0%
|
32.7
62.9%
|
| Title | Percentage of Participants With Clinically Significant Change From Baseline in Laboratory Values |
|---|---|
| Description | Laboratory values include chemistry and hematology, and urinary analysis. |
| Time Frame | Up to Week 50 |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Infants | Children | Adolescents |
|---|---|---|---|
| Arm/Group Description | 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. 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. | 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. 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. | 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. 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. |
| Measure Participants | 9 | 26 | 17 |
| Hematology |
0
0%
|
0
0%
|
0
0%
|
| Blood Chemistry |
0
0%
|
0
0%
|
0
0%
|
| Urinalysis |
0
0%
|
0
0%
|
0
0%
|
| Title | Percentage of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG) Findings |
|---|---|
| Description | |
| Time Frame | Up to Week 48 |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Infants | Children | Adolescents |
|---|---|---|---|
| Arm/Group Description | 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. 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. | 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. 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. | 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. 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. |
| Measure Participants | 9 | 26 | 17 |
| Number [Percentage of participants] |
0
0%
|
0
0%
|
0
0%
|
| Title | Percentage of Participants With Clinically Significant Change From Baseline in Vital Signs |
|---|---|
| Description | |
| Time Frame | Up to Week 50 |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Infants | Children | Adolescents |
|---|---|---|---|
| Arm/Group Description | 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. 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. | 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. 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. | 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. 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. |
| Measure Participants | 9 | 26 | 17 |
| Number [Percentage of participants] |
0
0%
|
0
0%
|
0
0%
|
| Title | Change From Baseline in Trough Plasma Levels of Cannabidiol and Its 7-OH Metabolite |
|---|---|
| Description | |
| Time Frame | Up to Week 50 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Insufficient data was collected to perform study analysis. |
| Arm/Group Title | Infants | Children | Adolescents |
|---|---|---|---|
| Arm/Group Description | 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. 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. | 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. 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. | 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. 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. |
| Measure Participants | 0 | 0 | 0 |
| Title | Vineland Adaptive Behavior Scales (VABS) |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Safety Analysis Population (SAF) consisted of all participants in the enrolled population group (ENR) who received at least one dose of the investigational product. |
| Arm/Group Title | Infants | Children | Adolescents |
|---|---|---|---|
| Arm/Group Description | 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. 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. | 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. 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. | 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. 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. |
| Measure Participants | 9 | 26 | 17 |
| Communication: Expressive (Day 1) |
6.8
(7.34)
|
40.4
(37.53)
|
40.3
(42.76)
|
| Communication: Expressive (Week 36) |
8.3
(9.03)
|
51.7
(41.79)
|
36.0
(41.63)
|
| Communication: Expressive (Week 48) |
9.9
(9.30)
|
48.6
(38.91)
|
34.3
(40.62)
|
| Communication: Receptive (Day 1) |
6.0
(7.35)
|
17.4
(13.10)
|
18.8
(14.88)
|
| Communication: Receptive (Week 36) |
8.6
(7.23)
|
21.1
(13.15)
|
15.7
(15.95)
|
| Communication: Receptive (Week 48) |
11.5
(8.73)
|
20.6
(13.15)
|
16.8
(14.62)
|
| Communication: Written (Day 1) |
0.0
(0.00)
|
9.2
(13.47)
|
12.9
(17.09)
|
| Communication: Written (Week 36) |
1.3
(2.31)
|
11.8
(15.98)
|
12.8
(17.33)
|
| Communication: Written (Week 48) |
0.0
(0.00)
|
10.8
(13.88)
|
10.5
(17.15)
|
| Daily Living Skills: Community (Day 1) |
0.4
(1.33)
|
13.8
(18.62)
|
19.1
(26.03)
|
| Daily Living Skills: Community (Week 36) |
0.6
(1.51)
|
17.8
(19.99)
|
18.8
(26.58)
|
| Daily Living Skills: Community (Week 48) |
1.0
(2.14)
|
16.6
(21.29)
|
18.8
(29.07)
|
| Daily Living Skills: Domestic (Day 1) |
0.4
(1.33)
|
9.1
(11.85)
|
9.7
(15.70)
|
| Daily Living Skills: Domestic (Week 36) |
0.9
(2.27)
|
9.6
(12.45)
|
10.7
(16.42)
|
| Daily Living Skills: Domestic (Week 48) |
0.8
(2.12)
|
9.9
(13.43)
|
9.2
(15.71)
|
| Daily Living Skills: Personal (Day 1) |
3.6
(6.65)
|
31.7
(29.37)
|
28.6
(27.98)
|
| Daily Living Skills: Personal (Week 36) |
5.0
(8.93)
|
39.2
(29.64)
|
26.2
(29.19)
|
| Daily Living Skills: Personal (Week 48) |
5.6
(8.09)
|
36.2
(30.66)
|
25.4
(29.78)
|
| Socialization: Coping Skills (Day 1) |
2.1
(2.67)
|
14.6
(18.34)
|
16.9
(20.33)
|
| Socialization: Coping Skills (Week 36) |
2.6
(3.99)
|
16.2
(16.37)
|
16.7
(20.81)
|
| Socialization: Coping Skills (Week 48) |
3.1
(4.32)
|
15.0
(17.08)
|
15.9
(20.63)
|
| Socialization: Interpersonal Relationships (Day 1) |
9.4
(10.14)
|
28.9
(22.93)
|
31.1
(25.41)
|
| Socialization: Interpersonal Relationships (Wk 36) |
11.9
(10.67)
|
35.7
(21.85)
|
25.8
(22.75)
|
| Socialization: Interpersonal Relationships (Wk 48) |
14.0
(12.62)
|
33.3
(22.67)
|
26.7
(24.35)
|
| Socialization: Play and Leisure Time (Day 1) |
5.3
(9.53)
|
20.5
(20.11)
|
19.7
(20.62)
|
| Socialization: Play and Leisure Time (Week 36) |
6.6
(10.55)
|
24.4
(19.77)
|
19.6
(22.71)
|
| Socialization: Play and Leisure Time (Week 48) |
6.4
(10.16)
|
21.8
(18.96)
|
17.9
(22.66)
|
| Motor Skills: Fine (Day 1) |
4.2
(9.72)
|
26.7
(27.29)
|
23.1
(26.20)
|
| Motor Skills: Fine (Week 36) |
5.1
(12.29)
|
33.6
(27.38)
|
20.9
(24.32)
|
| Motor Skills: Fine (Week 48) |
6.0
(8.67)
|
32.2
(27.56)
|
24.0
(25.21)
|
| Motor Skills: Gross (Day 1) |
8.0
(18.45)
|
42.5
(31.90)
|
31.0
(29.57)
|
| Motor Skills: Gross (Week 36) |
11.0
(22.96)
|
46.5
(32.43)
|
31.9
(26.74)
|
| Motor Skills: Gross (Week 48) |
12.8
(22.42)
|
46.6
(32.44)
|
33.3
(29.03)
|
| Maladaptive Behavior Index: Externalizing (Day 1) |
3.5
(3.51)
|
3.5
(3.10)
|
|
| Maladaptive Behavior Index: Externalizing (Wk 36) |
0.0
(0.00)
|
4.5
(4.81)
|
3.1
(3.42)
|
| Maladaptive Behavior Index: Externalizing (Wk 48) |
0.0
(0.00)
|
4.4
(4.10)
|
2.2
(2.95)
|
| Maladaptive Behavior Index: Internalizing (Day 1) |
5.2
(4.01)
|
3.6
(3.24)
|
|
| Maladaptive Behavior Index: Internalizing (Wk 36) |
0.0
(0.00)
|
5.4
(3.06)
|
3.7
(3.63)
|
| Maladaptive Behavior Index: Internalizing (Wk 48) |
1.0
(2.00)
|
5.3
(4.10)
|
3.6
(3.62)
|
| Maladaptive Behavior Index: MBI Score (Day 1) |
14.8
(7.53)
|
13.0
(8.75)
|
|
| Maladaptive Behavior Index: MBI Score (Week 36) |
0.0
(0.00)
|
16.6
(8.65)
|
10.8
(9.04)
|
| Maladaptive Behavior Index: MBI Score (Week 48) |
1.0
(2.00)
|
16.1
(9.17)
|
10.2
(8.47)
|
| Maladaptive Behavior Index: Other (Day 1) |
6.2
(4.17)
|
6.2
(4.76)
|
|
| Maladaptive Behavior Index: Other (Week 36) |
0.0
(0.00)
|
6.7
(4.01)
|
4.1
(3.60)
|
| Maladaptive Behavior Index: Other (Week 48) |
0.0
(0.00)
|
6.5
(3.30)
|
4.3
(3.88)
|
| Title | Number of Participants With a Positive Response on the Columbia-Suicide Severity Rating Scale (C-SSRS) |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Infants | Children | Adolescents |
|---|---|---|---|
| Arm/Group Description | 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. 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. | 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. 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. | 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. 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. |
| Measure Participants | 9 | 26 | 17 |
| Suicidal Ideation |
0
0%
|
0
0%
|
0
0%
|
| Suicidal Behavior |
0
0%
|
0
0%
|
0
0%
|
| Title | Clinical Global Impression of Severity (CGI-S) |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title |
|---|
| Arm/Group Description |
| Title | Impact of Pediatric Epilepsy Scale (IPES) |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title |
|---|
| Arm/Group Description |
| Title | Clinical Global Impression of Improvement (CGI-I) |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title |
|---|
| Arm/Group Description |
| Title | Change From Baseline in Frequency of Seizures as a Measure of Seizure Control |
|---|---|
| Description | |
| Time Frame | through study completion, up to 48 weeks or marketing approval, whichever is earlier |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title |
|---|
| Arm/Group Description |
Adverse Events
| Time Frame | Up to Week 50 | |||||
|---|---|---|---|---|---|---|
| Adverse Event Reporting Description | ||||||
| Arm/Group Title | Infants | Children | Adolescents | |||
| Arm/Group Description | 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. 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. | 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. 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. | 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. 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. | |||
All Cause Mortality |
||||||
| Infants | Children | Adolescents | ||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
Serious Adverse Events |
||||||
| Infants | Children | Adolescents | ||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 7/9 (77.8%) | 10/26 (38.5%) | 0/17 (0%) | |||
| Gastrointestinal disorders | ||||||
| Constipation | 0/9 (0%) | 1/26 (3.8%) | 0/17 (0%) | |||
| General disorders | ||||||
| Multi-organ failure | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Infections and infestations | ||||||
| Adenovirus infection | 0/9 (0%) | 1/26 (3.8%) | 0/17 (0%) | |||
| Pneumonia | 0/9 (0%) | 1/26 (3.8%) | 0/17 (0%) | |||
| Pneumonia viral | 0/9 (0%) | 1/26 (3.8%) | 0/17 (0%) | |||
| Pulmonary sepsis | 0/9 (0%) | 1/26 (3.8%) | 0/17 (0%) | |||
| Pyelonephritis | 0/9 (0%) | 1/26 (3.8%) | 0/17 (0%) | |||
| Respiratory syncytial virus infection | 0/9 (0%) | 1/26 (3.8%) | 0/17 (0%) | |||
| Respiratory tract infection viral | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Sepsis | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Tracheobronchitis | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Injury, poisoning and procedural complications | ||||||
| Accidental overdose | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Skull fracture | 0/9 (0%) | 1/26 (3.8%) | 0/17 (0%) | |||
| Nervous system disorders | ||||||
| Seizure | 5/9 (55.6%) | 5/26 (19.2%) | 0/17 (0%) | |||
| Status epilepticus | 2/9 (22.2%) | 0/26 (0%) | 0/17 (0%) | |||
| Ataxia | 0/9 (0%) | 1/26 (3.8%) | 0/17 (0%) | |||
| Change in seizure presentation | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Psychiatric disorders | ||||||
| Mental status changes | 1/9 (11.1%) | 1/26 (3.8%) | 0/17 (0%) | |||
| Aggression | 0/9 (0%) | 1/26 (3.8%) | 0/17 (0%) | |||
| Anxiety | 0/9 (0%) | 1/26 (3.8%) | 0/17 (0%) | |||
| Respiratory, thoracic and mediastinal disorders | ||||||
| Pneumonia aspiration | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Respiratory distress | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Respiratory failure | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Status asthmaticus | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Social circumstances | ||||||
| Physical assault | 0/9 (0%) | 1/26 (3.8%) | 0/17 (0%) | |||
Other (Not Including Serious) Adverse Events |
||||||
| Infants | Children | Adolescents | ||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 8/9 (88.9%) | 24/26 (92.3%) | 15/17 (88.2%) | |||
| Blood and lymphatic system disorders | ||||||
| Anaemia | 0/9 (0%) | 6/26 (23.1%) | 1/17 (5.9%) | |||
| Microcytic anaemia | 0/9 (0%) | 0/26 (0%) | 1/17 (5.9%) | |||
| Cardiac disorders | ||||||
| Bradycardia | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Pulmonary valve incompetence | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Tachycardia | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Tricuspid valve incompetence | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Congenital, familial and genetic disorders | ||||||
| Atrial septal defect | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Cryptorchism | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Gastrointestinal disorders | ||||||
| Diarrhoea | 0/9 (0%) | 5/26 (19.2%) | 2/17 (11.8%) | |||
| Constipation | 0/9 (0%) | 4/26 (15.4%) | 1/17 (5.9%) | |||
| Haematochezia | 1/9 (11.1%) | 1/26 (3.8%) | 0/17 (0%) | |||
| Vomiting | 0/9 (0%) | 2/26 (7.7%) | 0/17 (0%) | |||
| Inguinal hernia | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Haematemesis | 0/9 (0%) | 0/26 (0%) | 1/17 (5.9%) | |||
| General disorders | ||||||
| Pyrexia | 3/9 (33.3%) | 4/26 (15.4%) | 0/17 (0%) | |||
| Device expulsion | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Device failure | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Multi-organ failure | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Infections and infestations | ||||||
| Upper respiratory tract infection | 1/9 (11.1%) | 6/26 (23.1%) | 1/17 (5.9%) | |||
| Nasopharyngitis | 1/9 (11.1%) | 3/26 (11.5%) | 1/17 (5.9%) | |||
| Otitis media | 0/9 (0%) | 5/26 (19.2%) | 0/17 (0%) | |||
| Influenza | 1/9 (11.1%) | 3/26 (11.5%) | 0/17 (0%) | |||
| Urinary tract infection | 0/9 (0%) | 1/26 (3.8%) | 2/17 (11.8%) | |||
| Escherichia urinary tract infection | 0/9 (0%) | 2/26 (7.7%) | 0/17 (0%) | |||
| Pharyngitis streptococcal | 0/9 (0%) | 2/26 (7.7%) | 0/17 (0%) | |||
| Pneumonia | 0/9 (0%) | 1/26 (3.8%) | 1/17 (5.9%) | |||
| Conjunctivitis | 0/9 (0%) | 0/26 (0%) | 1/17 (5.9%) | |||
| Ear infection | 0/9 (0%) | 0/26 (0%) | 1/17 (5.9%) | |||
| Folliculitis | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Mycoplasma infection | 0/9 (0%) | 0/26 (0%) | 1/17 (5.9%) | |||
| Otitis media acute | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Pharyngitis | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Respiratory tract infection | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Respiratory tract infection viral | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Sepsis | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Tracheobronchitis | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Vestibulitis | 0/9 (0%) | 0/26 (0%) | 1/17 (5.9%) | |||
| Viral upper respiratory tract infection | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Injury, poisoning and procedural complications | ||||||
| Skin abrasion | 0/9 (0%) | 3/26 (11.5%) | 0/17 (0%) | |||
| Ligament sprain | 0/9 (0%) | 2/26 (7.7%) | 0/17 (0%) | |||
| Accidental overdose | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Ankle fracture | 0/9 (0%) | 0/26 (0%) | 1/17 (5.9%) | |||
| Investigations | ||||||
| Weight increased | 0/9 (0%) | 4/26 (15.4%) | 0/17 (0%) | |||
| Human rhinovirus test positive | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Weight decreased | 0/9 (0%) | 0/26 (0%) | 1/17 (5.9%) | |||
| Metabolism and nutrition disorders | ||||||
| Decreased appetite | 0/9 (0%) | 3/26 (11.5%) | 0/17 (0%) | |||
| Musculoskeletal and connective tissue disorders | ||||||
| Pain in extremity | 0/9 (0%) | 2/26 (7.7%) | 0/17 (0%) | |||
| Musculoskeletal stiffness | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Nervous system disorders | ||||||
| Seizure | 4/9 (44.4%) | 9/26 (34.6%) | 3/17 (17.6%) | |||
| Somnolence | 2/9 (22.2%) | 4/26 (15.4%) | 1/17 (5.9%) | |||
| Ataxia | 0/9 (0%) | 2/26 (7.7%) | 0/17 (0%) | |||
| Epilepsy | 1/9 (11.1%) | 1/26 (3.8%) | 0/17 (0%) | |||
| Status epilepticus | 2/9 (22.2%) | 0/26 (0%) | 0/17 (0%) | |||
| Change in seizure presentation | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Clumsiness | 0/9 (0%) | 0/26 (0%) | 1/17 (5.9%) | |||
| Dizziness | 0/9 (0%) | 0/26 (0%) | 1/17 (5.9%) | |||
| Fontanelle bulging | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Partial seizures | 0/9 (0%) | 0/26 (0%) | 1/17 (5.9%) | |||
| Postictal paralysis | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Psychiatric disorders | ||||||
| Aggression | 0/9 (0%) | 5/26 (19.2%) | 0/17 (0%) | |||
| Insomnia | 1/9 (11.1%) | 2/26 (7.7%) | 1/17 (5.9%) | |||
| Irritability | 1/9 (11.1%) | 0/26 (0%) | 1/17 (5.9%) | |||
| Mental status changes | 1/9 (11.1%) | 1/26 (3.8%) | 0/17 (0%) | |||
| Agitation | 0/9 (0%) | 0/26 (0%) | 1/17 (5.9%) | |||
| Flat affect | 0/9 (0%) | 0/26 (0%) | 1/17 (5.9%) | |||
| Head banging | 0/9 (0%) | 0/26 (0%) | 1/17 (5.9%) | |||
| Restlessness | 0/9 (0%) | 0/26 (0%) | 1/17 (5.9%) | |||
| Renal and urinary disorders | ||||||
| Nephrolithiasis | 0/9 (0%) | 0/26 (0%) | 1/17 (5.9%) | |||
| Respiratory, thoracic and mediastinal disorders | ||||||
| Cough | 0/9 (0%) | 3/26 (11.5%) | 1/17 (5.9%) | |||
| Asthma | 0/9 (0%) | 2/26 (7.7%) | 0/17 (0%) | |||
| Sleep apnea syndrome | 1/9 (11.1%) | 0/26 (0%) | 1/17 (5.9%) | |||
| Adenoidal hypertrophy | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Apnoea | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Nasal congestion | 0/9 (0%) | 0/26 (0%) | 1/17 (5.9%) | |||
| Hypoxia | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Pneumonia aspiration | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Respiratory distress | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Respiratory failure | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Respiratory tract congestion | 0/9 (0%) | 0/26 (0%) | 1/17 (5.9%) | |||
| Sinus congestion | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Status asthmaticus | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Upper respiratory tract inflammation | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Wheezing | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Skin and subcutaneous tissue disorders | ||||||
| Ecchymosis | 0/9 (0%) | 2/26 (7.7%) | 0/17 (0%) | |||
| Cold sweat | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Decubitus ulcer | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Dermatitis diaper | 1/9 (11.1%) | 0/26 (0%) | 0/17 (0%) | |||
| Rash | 0/9 (0%) | 0/26 (0%) | 1/17 (5.9%) | |||
Limitations/Caveats
[Not Specified]
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
| Name/Title | Director, Clinical Development |
|---|---|
| Organization | Insys Therapeutics, Inc. |
| Phone | 480-500-3105 |
| gdecastro@insysrx.com |
Responsible Party:
INSYS Therapeutics Inc
ClinicalTrials.gov Identifier:
NCT02318602
Other Study ID Numbers:
- INS011-14-030
First Posted:
Dec 17, 2014
Last Update Posted:
Jul 26, 2018
Last Verified:
Jun 1, 2018