Childhood Absence Epilepsy
Conditions
Brief summary
The primary purpose of this study is to assess the long-term safety and tolerability of Cannabidiol Oral Solution (CBD) in pediatric participants with treatment-resistant childhood absence seizures.
Interventions
An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).
Sponsors
Benuvia Therapeutics Inc.
Radius Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
1. Completed all activities through Visit 6 (End of Study) of INS011-17-103. 2. Participant and/or parent(s)/caregiver(s) fully comprehend the informed consent form (ICF) and assent form, understand all study procedures, and can communicate satisfactorily with the investigator and study coordinator, in accordance with applicable laws, regulations, and local requirements. 3. A female participant is eligible to participate in the study if she is premenarchal, or of childbearing potential with a negative urine pregnancy test at the Screening Visit. If sexually active, she must agree to either complete abstinence from intercourse or use acceptable methods of contraception throughout the study and for 4 weeks after completion of study participation or discontinuation from investigational product. 4. A sexually active male participant or partner of enrolled participant must be willing to use acceptable methods of contraception throughout the study and for 4 weeks after completion of study participation or discontinuation from investigational product. 5. In the opinion of the investigator, the parent(s)/caregiver(s) is (are) willing and able to comply with the study procedures and visit schedules, including venipuncture, and the visit schedules.
Exclusion criteria
1. Participant or parent(s)/caregiver(s) have daily commitments during the study duration that would interfere with attending all study visits. 2. Experienced an anoxic episode related to study drug requiring resuscitation during their previous study. 3. Developed an adverse event thought to be related to CBD in the previous study and for whom the Investigator determines that continuing treatment with CBD would not be in the best interest of the participant. 4. Evidence of other clinically significant disease such as unstable hepatic, hematological, renal, cardiovascular, gastrointestinal, immunological, or pulmonary diseases or ongoing malignancies. 5. Compromised respiratory function or severe respiratory insufficiency. 6. Clinically significant abnormal laboratory values within the past 14 days. 7. In the opinion of the investigator, the participant is unsuitable in any other way to participate in this study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Baseline (Visit 6 of INS011-17-103) and Visit 10 (up to approximately 54 weeks) | An AE is defined as any untoward medical occurrence in a participant administered a pharmaceutical product during the course of a clinical investigation. An AE can therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of an investigational product, whether or not thought to be related to the investigational product. An SAE is any untoward medical occurrence that results in death, is life-threatening, requires the participant be at a risk of death at the time of the event, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital abnormality/birth defect, or other serious event that requires medical or surgical intervention. A summary of SAEs and all other non-serious AEs, regardless of causality, is located in the Reported AEs module. |
Countries
United States
Participant flow
Pre-assignment details
There were 11 participants who completed the INS011-17-103 study and who further received cannabidiol oral solution for this long-term safety study (INS011-17-113). Of which, 5 participants received 20 to \<30 milligram/kilogram/day (mg/kg/day) and 6 participants received 30 to \<40 mg/kg/day.
Participants by arm
| Arm | Count |
|---|---|
| Canabidiol Oral Solution 20 to <30 mg/kg/Day Participants who completed the INS011-17-103 further received Cannabidiol Oral Solution 20 mg/kg/day divided BID for 4 weeks for a long-term safety study. | 5 |
| Canabidiol Oral Solution 30 to <40 mg/kg/Day Participants who completed the INS011-17-103 study further received Cannabidiol Oral Solution 20 mg/kg/day divided BID for 4 weeks for a long-term study. | 6 |
| Total | 11 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Lack of Efficacy | 2 | 1 |
| Overall Study | Other reason | 1 | 0 |
| Overall Study | Physician Decision | 0 | 1 |
Baseline characteristics
| Characteristic | Canabidiol Oral Solution 30 to <40 mg/kg/Day | Total | Canabidiol Oral Solution 20 to <30 mg/kg/Day |
|---|---|---|---|
| Age, Continuous | 10.7 years STANDARD_DEVIATION 3.01 | 9.6 years STANDARD_DEVIATION 2.87 | 8.4 years STANDARD_DEVIATION 2.41 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 6 Participants | 11 Participants | 5 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 6 Participants | 11 Participants | 5 Participants |
| Sex: Female, Male Female | 2 Participants | 6 Participants | 4 Participants |
| Sex: Female, Male Male | 4 Participants | 5 Participants | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 5 | 0 / 6 |
| other Total, other adverse events | 4 / 5 | 4 / 6 |
| serious Total, serious adverse events | 0 / 5 | 0 / 6 |
Outcome results
Primary
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE is defined as any untoward medical occurrence in a participant administered a pharmaceutical product during the course of a clinical investigation. An AE can therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of an investigational product, whether or not thought to be related to the investigational product. An SAE is any untoward medical occurrence that results in death, is life-threatening, requires the participant be at a risk of death at the time of the event, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital abnormality/birth defect, or other serious event that requires medical or surgical intervention. A summary of SAEs and all other non-serious AEs, regardless of causality, is located in the Reported AEs module.
Time frame: Baseline (Visit 6 of INS011-17-103) and Visit 10 (up to approximately 54 weeks)
Population: All participants who were enrolled and received at least 1 dose of study drug.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Canabidiol Oral Solution 20 to <30 mg/kg/Day | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Number of participants with AEs | 5 participants |
| Canabidiol Oral Solution 20 to <30 mg/kg/Day | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Number of participants with SAEs | 0 participants |
| Canabidiol Oral Solution 30 to <40 mg/kg/Day | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Number of participants with AEs | 4 participants |
| Canabidiol Oral Solution 30 to <40 mg/kg/Day | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Number of participants with SAEs | 0 participants |