A Study to Assess the Long-Term Safety of Pharmaceutical... | NCT03458416

NCT03458416

Status Module

Sponsor/Collaborators Module

Name	Class
Benuvia Therapeutics Inc.	INDUSTRY

Oversight Module

Description Module

Conditions Module

Design Module

Interventional Study Design

Biospecimen

No data available

No data is available for this block.

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Cannabidiol Oral Solution: 20-40 mg/kg/day	Experimental	Participants will receive total daily doses between 20 milligrams per kilograms per day (mg/kg/day), 30 mg/kg/day, and 40 mg/kg/day. The two equivalent doses will be administered twice a day with a standard meal approximately every 12 hours.	Drug: Cannabidiol Oral Solution

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Cannabidiol Oral Solution	Drug	An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).	Cannabidiol Oral Solution: 20-40 mg/kg/day

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Incidence Of Adverse Events (AEs) And Serious Adverse Events (SAEs) Associated With Cannabidiol Oral Solution	AE is defined as any untoward medical occurrence in a participant administered with a pharmaceutical product which does not necessarily have a causal relationship with the treatment. An SAE is defined as any event that results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, or a congenital anomaly/birth defect. A summary of other non-serious AEs and all serious AEs regardless of causality, is located in the Reported AE section.	Up to Week 52

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Completed activities up to and including Visit 10 (Study Completion) of INS011-16- 085.
Participant and/or parent(s)/caregiver(s) fully comprehend the informed consent form 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.
If female, is either not of childbearing potential or practicing medically acceptable methods of birth control.
Psychotropic treatment will be permitted if the participant has been on a stable dose during the INS011-16-085 and does not anticipate a dose change during the course of the study.
Growth hormone treatment will be permitted if the participant has been on a stable dose during INS01-16-085.
Any other treatment including thyroid hormones should be stable prior to entering the INS011-17-115 study.
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 commitments during the study duration that would interfere with attending all study visits.
Experienced an anoxic episode related to study drug requiring resuscitation during the previous study.
Uncontrolled Type I and Type II Diabetes.
Developed an adverse event thought to be related to CBD in the previous study and the investigator determines that continuing treatment with CBD would not be in the best interest of the partient.
History of clinically significant gastrointestinal, renal, hepatic, neurologic, hematologic, endocrine, oncologic, pulmonary, immunologic, psychiatric, or cardiovascular disease, or other condition which would jeopardize safety or impact validity of results (per investigator).
Currently taking felbamate.
Compromised respiratory function or severe respiratory insufficiency.
Pregnant or lactating female.
In the opinion of the investigator, the participant is unsuitable in any other way to participate in this study.

Contacts/Locations Module

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

Facility	Status	City	State	ZIP	Country	Contacts
Institute for Research and Innovation \| MultiCare Health System		Tacoma	Washington	98405	United States

References Module

No data available

No data is available for this block.

IPD Sharing Statement Module

No data available

No data is available for this block.

Results Section

Participant Flow Module

ID	Title	Description
FG000	Cannabidiol Oral Solution	Participants received 20 milligrams (mg)/kilogram (kg)/day to 40 mg/kg/day of CBD given twice daily with standard meals.

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Annotation Section

No data available

No data is available for this block.

Document Section

Large Document Module

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Feb 27, 2018	Nov 23, 2022

Derived Section

Miscellaneous Info Module

Submission Tracking

Condition Browse Module

Intervention Browse Module

Masking Info

This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.

ID	Title	Description
BG000	Cannabidiol Oral Solution	Participants received 20 mg/kg/day to 40 mg/kg/day of CBD given twice daily with standard meals.

Units	Counts
Participants	BG0000

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Continuous

Sex: Female, Male

Title

Denominators

Categories

Title	Measurements
Hispanic or Latino
Not Hispanic or Latino
Unknown or Not Reported

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

Incidence Of Adverse Events (AEs) And Serious Adverse Events (SAEs) Associated With Cannabidiol Oral Solution

AE is defined as any untoward medical occurrence in a participant administered with a pharmaceutical product which does not necessarily have a causal relationship with the treatment. An SAE is defined as any event that results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, or a congenital anomaly/birth defect. A summary of other non-serious AEs and all serious AEs regardless of causality, is located in the Reported AE section.

Posted

Up to Week 52

ID	Title	Description
OG000	Cannabidiol Oral Solution	Participants received total daily doses between 20 mg/kg/day to 40 mg/kg/day given twice daily with standard meals.

Units	Counts
Participants	OG0000

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	Cannabidiol Oral Solution	Participants received total daily doses between 20 to 40 mg/kg/day given twice daily with standard meals.	0	0	0	0	0	0

Certain Agreements

Title	Organization	Phone	Extension	Email
Tarek El Akkad, Head of Clinical Development	Radius Pharmaceuticals, Inc.	6175514000		telakkad@radiuspharm.com

First MCP Posted Date

ID	Term
D011218	Prader-Willi Syndrome

Ancestor Terms

ID	Term
D008607	Intellectual Disability
D019954	Neurobehavioral Manifestations
D009461	Neurologic Manifestations
D009422	Nervous System Diseases
D000015	Abnormalities, Multiple
D000013	Congenital Abnormalities
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D025063	Chromosome Disorders
D030342	Genetic Diseases, Inborn
D000096803	Imprinting Disorders
D009765	Obesity
D050177	Overweight
D044343	Overnutrition
D009748	Nutrition Disorders
D009750	Nutritional and Metabolic Diseases

ID	Term
D002185	Cannabidiol

ID	Term
D002186	Cannabinoids
D013729	Terpenes
D006838	Hydrocarbons
D009930	Organic Chemicals