Long-Term Safety of Pharmaceutical Grade Synthetic Cannab... | NCT03355300

Status Module

Name	Class
Benuvia Therapeutics Inc.	INDUSTRY

Label	Type	Description	Intervention Names
Cannabidiol Oral Solution	Experimental	Cannabidiol Oral solution, dose as assigned in INS-17-103.	Drug: Cannabidiol Oral Solution

Name	Type	Description	Arm Group Labels	Other Names
Cannabidiol Oral Solution	Drug	An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).	Cannabidiol Oral Solution

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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.	Baseline (Visit 6 of INS011-17-103) and Visit 10 (up to approximately 54 weeks)

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Completed all activities through Visit 6 (End of Study) of INS011-17-103.
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.
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.
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.
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:

Participant or parent(s)/caregiver(s) have daily commitments during the study duration that would interfere with attending all study visits.
Experienced an anoxic episode related to study drug requiring resuscitation during their previous study.
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.
Evidence of other clinically significant disease such as unstable hepatic, hematological, renal, cardiovascular, gastrointestinal, immunological, or pulmonary diseases or ongoing malignancies.
Compromised respiratory function or severe respiratory insufficiency.
Clinically significant abnormal laboratory values within the past 14 days.
In the opinion of the investigator, the participant is unsuitable in any other way to participate in this study.

Name	Affiliation	Role
Ahmed Elkashef, MD	INSYS Therapeutics Inc	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Nicklaus Children's Hospital		Miami	Florida	33155	United States
Pediatric Epilepsy and Neurology Specialists

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	Canabidiol Oral Solution 20 to <30 mg/kg/Day	Participants who completed the INS011-17-103 further received Cannabidiol Oral Solution 20 milligrams per kilogram per day (mg/kg/day) divided twice daily (BID) for 4 weeks for a long-term safety study.
FG001	Canabidiol Oral Solution 30 to <40 mg/kg/Day

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot_SAP	Yes	Yes	No	Study Protocol and Statistical Analysis Plan	Aug 23, 2018

Arm/Group Information

ID	Title	Description
BG000	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.
BG001	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.
BG002	Total	Total of all reporting groups

Units	Counts
Participants	BG0005 BG0016 BG00211

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Continuous

Mean

Standard Deviation

years

Title

Denominators

Categories

Title	Measurements
	BG0008.4± 2.41 BG00110.7± 3.01 BG0029.6± 2.87

Sex: Female, Male

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Female	BG0004 BG0012 BG002

Ethnicity (NIH/OMB)

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Hispanic or Latino	BG0000 BG0010 BG002

Race (NIH/OMB)

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
American Indian or Alaska Native	BG0000 BG0010 BG002

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

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.

All participants who were enrolled and received at least 1 dose of study drug.

Posted

Number

participants

Baseline (Visit 6 of INS011-17-103) and Visit 10 (up to approximately 54 weeks)

ID	Title	Description
OG000	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.
OG001	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.

Units	Counts
Participants	OG0005 OG0016

Title

Denominators

Categories

Number of participants with AEs

Title	Measurements
	OG0005 OG0014

Number of participants with SAEs

Title	Measurements
	OG000

Arm/Groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	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.	0	5	0	5	4	5
EG001	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.	0	6	0	6	4	6

Other Adverse Events

Term	Organ System	Assessment Type	Statistical Information
Vomiting	Gastrointestinal disorders	Systematic Assessment	EG0000 affected5 at risk EG0011 affected6 at risk
Fatigue	General disorders	Systematic Assessment	EG0000 affected5 at risk EG0011 affected6 at risk
Pyrexia	General disorders	Systematic Assessment	EG0001 affected5 at risk EG0010 affected6 at risk
Seasonal allergy	Immune system disorders	Systematic Assessment	EG0001 affected5 at risk EG0010 affected6 at risk
Pharyngitis streptococcal	Infections and infestations	Systematic Assessment	EG0000 affected5 at risk EG0011 affected6 at risk
Upper respiratory tract infection	Infections and infestations	Systematic Assessment	EG0000 affected5 at risk EG0011 affected6 at risk
Coordination abnormal	Nervous system disorders	Systematic Assessment	EG0001 affected5 at risk EG0010 affected6 at risk
Headache	Nervous system disorders	Systematic Assessment	EG0001 affected5 at risk EG0010 affected6 at risk
Seizure	Nervous system disorders	Systematic Assessment	EG0001 affected5 at risk EG0010 affected6 at risk
Somnolence	Nervous system disorders	Systematic Assessment	EG0001 affected5 at risk EG0010 affected6 at risk
Hallucination	Psychiatric disorders	Systematic Assessment	EG0001 affected5 at risk EG0010 affected6 at risk

ID	Term
D004829	Epilepsy, Generalized
D004827	Epilepsy
D001927	Brain Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases
D000073376	Epileptic Syndromes

ID	Term
D002186	Cannabinoids
D013729	Terpenes
D006838	Hydrocarbons
D009930	Organic Chemicals