SeizuresPhase 3NCT02318602

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

A Phase 3 study testing Cannabidiol Oral Solution in people with Seizures, run by INSYS Therapeutics Inc at 11 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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). Read more about this study

Who is it for?: Ages 1 year to 18 years
Conditions the study is for: Seizures
What is being tested?: Cannabidiol Oral Solution
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 52 participants
Where: 11 sitesUnited States
Study dates: 8 Jan 2016to 22 Jun 2017
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 1 year to 18 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

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

Who cannot join

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

Trial locations and contacts

11 sites in 1 country.

City, postcode or hospital

11 sites in 1 country

United States11 sites

University of Chicago Medical Center
Chicago, Illinois, 60637
Texas Scottish Rite Hospital for Children
Dallas, Texas, 79219
Clinical Research Center of Nevada LLC
Las Vegas, Nevada, 89104
Le Bonheur Children's Hospital
Memphis, Tennessee, 38103
Miami Children's Hospital
Miami, Florida, 33155
Child Neurology Center - NW F
Pensacola, Florida, 32504
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Oregon Health Services University
Portland, Oregon, 97239
Granger Medical Clinic
Riverton, Utah, 84096
University of California San Francisco Medical Center
San Francisco, California, 94143
Mary Bridge Children's Hospital
Tacoma, Washington, 98403

Other trials in United States

About this study

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

Conditions: Seizures
Being tested: Cannabidiol Oral Solution
Keywords: Lennox-Gastaut syndrome, Dravet syndrome, Treatment-resistant seizures
Medical subject headings: Seizures, Lennox Gastaut Syndrome, Epilepsies, Myoclonic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

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

Study groups in NCT02318602
Study group	Role	What they receive
Infants	Experimental	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. Receives: Drug: Cannabidiol Oral Solution
Children	Experimental	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. Receives: Drug: Cannabidiol Oral Solution
Adolescents	Experimental	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. Receives: Drug: Cannabidiol Oral Solution

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: Up to Week 50
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.
Measured: Up to Week 50
Percentage of Participants With Clinically Significant Change From Baseline in Laboratory Values
Laboratory values include chemistry and hematology, and urinary analysis.
Measured: Up to Week 50
Percentage of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG) Findings
Measured: Up to Week 48
Percentage of Participants With Clinically Significant Change From Baseline in Vital Signs
Measured: Up to Week 50
Change From Baseline in Trough Plasma Levels of Cannabidiol and Its 7-OH Metabolite
Measured: Up to Week 50

Secondary outcomes (2)

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.
Measured: Up to Week 48
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.
Measured: Up to Week 50

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

8 Jan 201622 Jun 2017

First posted17 Dec 2014 (estimated)
Study start8 Jan 2016
Primary completion22 Jun 2017
Study completion22 Jun 2017

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for SeizuresStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 3
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Enrolment: 52 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02318602
Sponsor's protocol ID: INS011-14-030
Status last verified: Jun 2018
First submitted: 12 Dec 2014
Last updated: 26 Jul 2018
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 3
Investigate Efficacy and Safety of Carisbamate as Adjunctive Treatment for Seizures Associated With LGS in Children and Adults
Seizures, Lennox Gastaut Syndrome
RecruitingPhase 3
Study Evaluating the Efficacy and Safety of RAP-219 in Adult Participants With Focal Seizures
Focal Seizure, Epilepsy
RecruitingPhase 3
A Study to Test the Efficacy and Safety of Staccato Alprazolam in Study Participants 12 Years of Age and Older With Stereotypical Prolonged Seizures
Stereotypical Prolonged Seizures
RecruitingPhase 3
Study Evaluating the Efficacy and Safety of RAP-219 in Adult Participants With Focal Seizures FOCUS-1
Focal Seizure, Epilepsy
Not yet recruitingPhase 4
Study of Electrocardiogram (ECG) Safety of Sezaby in Neonatal Seizures
Neonatal Seizure

All Seizures trials

Study at a glance

From the registry record, last updated by the sponsor on 26 Jul 2018. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT02318602
Acronym / sponsor ID: INS011-14-030
Conditions: Seizures
Keywords: Lennox-Gastaut syndrome, Dravet syndrome, Treatment-resistant seizures
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 52 participants (actual)
Lead sponsor: INSYS Therapeutics IncIndustry (pharma, biotech, devices)
Study start: 8 Jan 2016 (actual)
Primary completion: 22 Jun 2017 (actual)
Study completion: 22 Jun 2017 (actual)
First posted: 17 Dec 2014 (estimated)
Results: Posted 26 Jul 2018

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Number of arms: 3

Arm	Type	Receives
Infants	Experimental	Drug: Cannabidiol Oral SolutionParticipants 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.
Children	Experimental	Drug: Cannabidiol Oral SolutionParticipants 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.
Adolescents	Experimental	Drug: Cannabidiol Oral SolutionParticipants 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.

Who may qualify

Ages: 1 year to 18 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (5 listed)

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

Key exclusion criteria (6 listed)

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:

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Cannabidiol Oral SolutionAn oral solution containing pharmaceutical grade cannabidiol (nonplant-based).	Drug	Adolescents, Children, Infants

Primary outcome

Percentage of Participants With Adverse Events
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: Up to Week 48
Percentage of Participants With Clinically Significant Change From Baseline in Vital Signs
Time frame: Up to Week 50
Change From Baseline in Trough Plasma Levels of Cannabidiol and Its 7-OH Metabolite
Time frame: Up to Week 50

2 secondary outcomes

Vineland Adaptive Behavior Scales (VABS)
Time frame: Up to Week 48
Number of Participants With a Positive Response on the Columbia-Suicide Severity Rating Scale (C-SSRS)
Time frame: Up to Week 50

Results and safety

Results posted to the registry on 26 Jul 2018, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Infants	9	8
Children	26	22
Adolescents	17	15

Primary outcome: Percentage of Participants With Adverse Events

Time frame: Up to Week 50. Number. Unit: Percentage of participants

Measure	Infants n = 9	Children n = 26	Adolescents n = 17	All Participants n = 52
Value	88.9	92.3	88.2	90.4

Primary outcome: Percentage of Participants With Serious Adverse Events

Time frame: Up to Week 50. Number. Unit: Percentage of participants

Measure	Infants n = 9	Children n = 26	Adolescents n = 17	All Participants n = 52
Value	77.8	38.5	0	32.7

Primary outcome: Percentage of Participants With Clinically Significant Change From Baseline in Laboratory Values

Time frame: Up to Week 50. Number. Unit: Percentage of participants

Measure	Infants n = 9	Children n = 26	Adolescents n = 17
Hematology	0	0	0
Blood Chemistry	0	0	0
Urinalysis	0	0	0

Primary outcome: Percentage of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG) Findings

Time frame: Up to Week 48. Number. Unit: Percentage of participants

Measure	Infants n = 9	Children n = 26	Adolescents n = 17
Value	0	0	0

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: Up to Week 50. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Infants	7 of 9 (77.8%)	8 of 9 (88.9%)	1 of 9
Children	10 of 26 (38.5%)	24 of 26 (92.3%)	0 of 26
Adolescents	0 of 17 (0%)	15 of 17 (88.2%)	0 of 17

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 26 Jul 2018.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Seizures

RecruitingPhase 3
Investigate Efficacy and Safety of Carisbamate as Adjunctive Treatment for Seizures Associated With LGS in Children and Adults
Seizures, Lennox Gastaut Syndrome
RecruitingPhase 3
Study Evaluating the Efficacy and Safety of RAP-219 in Adult Participants With Focal Seizures
Focal Seizure, Epilepsy
RecruitingPhase 3
A Study to Test the Efficacy and Safety of Staccato Alprazolam in Study Participants 12 Years of Age and Older With Stereotypical Prolonged Seizures
Stereotypical Prolonged Seizures
RecruitingPhase 3
Study Evaluating the Efficacy and Safety of RAP-219 in Adult Participants With Focal Seizures FOCUS-1
Focal Seizure, Epilepsy
Not yet recruitingPhase 4
Study of Electrocardiogram (ECG) Safety of Sezaby in Neonatal Seizures
Neonatal Seizure

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Neha Parikh study director

INSYS Therapeutics Inc

11 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Cannabidiol Oral Solution in people with Seizures, run by INSYS Therapeutics Inc at 11 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 26 Jul 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 11 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 26 Jul 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of Participants With Adverse Events (time frame: Up to Week 50). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02318602.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jul 2018.