Dravet SyndromePhase 3NCT02318563
Cannabidiol Oral Solution as an Adjunctive Therapy for Treatment of Participants With Inadequately Controlled Dravet Syndrome
A Phase 3 study testing Cannabidiol Oral Solution in people with Dravet Syndrome, run by INSYS Therapeutics Inc.
Trial summary
This Phase 3 study will enroll participants diagnosed with Dravet Syndrome (DS) who are still experiencing at least one tonic-clonic, clonic, and/or focal seizures with motor components (FSMC) per week, despite ongoing treatment with up to three antiepileptic drugs (AEDs), and meet the other inclusion/exclusion… Read more about this study
- Who is it for?
- Ages 1 year to 30 years
- Conditions the study is for
- Dravet Syndrome
- What is being tested?
- Cannabidiol Oral Solution
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 30 Dec 2017to 17 Jun 2018
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Meets protocol-specified criteria for qualification and contraception, including clinical diagnosis of refractory DS and onset of seizures according to protocol-specified criteria
- Is able to speak and understand the language in which the study is being conducted, is able to understand the procedures and study requirements and has voluntarily signed and dated an informed consent form approved by the Institutional Review Board before the conduct of any study procedure
- In the opinion of the Investigator, the subject and/or parent(s)/caregiver(s) are able to keep accurate seizure diaries and the participant is able to take study drug and comply with the protocol, including dosing, medications and diet
Who cannot join
- Medical history is outside protocol-specified parameters
- Clinically significant history of allergic reactions or significant sensitivities to cannabinoids or to any of the other ingredients in the study drug
- Inadequate supervision by parents or guardians
- 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: 1) the safety or well-being of the participant or study staff; 2) the safety or well-being of the participant's offspring (such as through pregnancy or breast-feeding); 3) the analysis of results
About this study
Official title: A Multicenter, Randomized, Double-blind, Placebo- Controlled, Interventional Study to Assess the Safety and Efficacy of Pharmaceutical Cannabidiol Oral Solution as an Adjunctive Therapy for Treatment of Subjects With Inadequately Controlled Dravet Syndrome
This Phase 3 study will enroll participants diagnosed with Dravet Syndrome (DS) who are still experiencing at least one tonic-clonic, clonic, and/or focal seizures with motor components (FSMC) per week, despite ongoing treatment with up to three antiepileptic drugs (AEDs), and meet the other inclusion/exclusion criteria.
Following a 28-day baseline period, participants will begin an 84-day treatment period. Participants will be assigned to receive twice-daily doses of placebo or cannabidiol oral solution at the highest dose determined to be safe in a previous trial.
Following study completion, all participants will be invited to receive Cannabidiol Oral Solution in an open label extension study (under a separate protocol).
- Conditions
- Dravet Syndrome
- Being tested
- Cannabidiol Oral Solution, Placebo Solution
- Keywords
- Treatment-resistant seizures
- Medical subject headings
- 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)
Placebo SolutionDrug
A matching oral solution containing no cannabidiol
| Study group | Role | What they receive |
|---|---|---|
| Cannabidiol Oral Solution | Experimental | Participants will receive cannabidiol oral solution at an appropriate dose (no higher than 40 mg/kg/day) determined by data from a previous trial. The total daily dose will be administered in twice daily doses, approximately 12 hours apart. Receives: Drug: Cannabidiol Oral Solution |
| Placebo Solution | Placebo comparator | Participants will receive matching placebo solution administered twice daily, approximately 12 hours apart. Receives: Drug: Placebo Solution |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent change in the frequency of tonic-clonic, clonic, and focal seizures with motor components
Measured: Data point for observation period to data point for treatment period Weeks 9 through 12
Secondary outcomes (7)
Percent change from baseline in the severity of tonic-clonic, clonic, and focal seizures with motor components
Measured: Data point for observation period to data point for treatment period Weeks 9 through 12
Percent change from baseline in the duration of tonic-clonic, clonic, and focal seizures with motor components
Measured: Data point for observation period to data point for treatment period Weeks 9 through 12
Percent change from baseline in the frequency of all seizure activity independent of seizure type
Measured: Data point for observation period to data point for treatment period Weeks 9 through 12
Change from baseline in parent(s)/caregiver(s) Clinical Global Impressions of Improvement (CGI-I)
Measured: Data point for observation period to data point for treatment period Weeks 9 through 12
Change from baseline in parent(s)/caregiver(s) Clinical Global Impressions of Severity (CGI-S)
Measured: Data point for observation period to data point for treatment period Weeks 9 through 12
Change from baseline in Investigator CGI-I
Measured: Data point for observation period to data point for treatment period Weeks 9 through 12
Change from baseline in Investigator CGI-S
Measured: Data point for observation period to data point for treatment period Weeks 9 through 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2014 (estimated)
- Study start30 Dec 2017 (estimated)
- Primary completion17 Jun 2018 (estimated)
- Study completion17 Jun 2018 (estimated)
Why it stopped: Sponsor elected not to continue with study.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dravet SyndromeStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- INSYS Therapeutics Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Neha Parikh, Study director, INSYS Therapeutics Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02318563
- Sponsor's protocol ID
- INS011-14-025
- Status last verified
- Jan 2018
- First submitted
- 12 Dec 2014
- Last updated
- 4 Jan 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Randomized, Double-blind, Placebo- Controlled, Interventional Study to Assess the Safety and Efficacy of Pharmaceutical Cannabidiol Oral Solution as an Adjunctive Therapy for Treatment of Subjects With Inadequately Controlled Dravet Syndrome
- ClinicalTrials.gov ID
- NCT02318563
- Acronym / sponsor ID
- INS011-14-025
- Conditions
- Dravet Syndrome
- Keywords
- Treatment-resistant seizures
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Sponsor elected not to continue with study.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- INSYS Therapeutics IncIndustry (pharma, biotech, devices)
- Study start
- 30 Dec 2017 (estimated)
- Primary completion
- 17 Jun 2018 (estimated)
- Study completion
- 17 Jun 2018 (estimated)
- First posted
- 17 Dec 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This Phase 3 study will enroll participants diagnosed with Dravet Syndrome (DS) who are still experiencing at least one tonic-clonic, clonic, and/or focal seizures with motor components (FSMC) per week, despite ongoing treatment with up to three antiepileptic drugs (AEDs), and meet the other inclusion/exclusion criteria.
Following a 28-day baseline period, participants will begin an 84-day treatment period. Participants will be assigned to receive twice-daily doses of placebo or cannabidiol oral solution at the highest dose determined to be safe in a previous trial.
Following study completion, all participants will be invited to receive Cannabidiol Oral Solution in an open label extension study (under a separate protocol).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cannabidiol Oral Solution | Experimental | Drug: Cannabidiol Oral SolutionParticipants will receive cannabidiol oral solution at an appropriate dose (no higher than 40 mg/kg/day) determined by data from a previous trial. The total daily dose will be administered in twice daily doses, approximately 12 hours apart. |
| Placebo Solution | Placebo comparator | Drug: Placebo SolutionParticipants will receive matching placebo solution administered twice daily, approximately 12 hours apart. |
Who may qualify
- Ages
- 1 year to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Meets protocol-specified criteria for qualification and contraception, including clinical diagnosis of refractory DS and onset of seizures according to protocol-specified criteria
- Is able to speak and understand the language in which the study is being conducted, is able to understand the procedures and study requirements and has voluntarily signed and dated an informed consent form approved by the Institutional Review Board before the conduct of any study procedure
- In the opinion of the Investigator, the subject and/or parent(s)/caregiver(s) are able to keep accurate seizure diaries and the participant is able to take study drug and comply with the protocol, including dosing, medications and diet
Key exclusion criteria (5 listed)
- Medical history is outside protocol-specified parameters
- Clinically significant history of allergic reactions or significant sensitivities to cannabinoids or to any of the other ingredients in the study drug
- Inadequate supervision by parents or guardians
- 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: 1) the safety or well-being of the participant or study staff; 2) the safety or well-being of the participant's offspring (such as through pregnancy or breast-feeding); 3) the analysis of results
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 | Cannabidiol Oral Solution |
| Placebo SolutionA matching oral solution containing no cannabidiol | Drug | Placebo Solution |
Primary outcome
Percent change in the frequency of tonic-clonic, clonic, and focal seizures with motor components
Time frame: Data point for observation period to data point for treatment period Weeks 9 through 12
7 secondary outcomes
Percent change from baseline in the severity of tonic-clonic, clonic, and focal seizures with motor components
Time frame: Data point for observation period to data point for treatment period Weeks 9 through 12
Percent change from baseline in the duration of tonic-clonic, clonic, and focal seizures with motor components
Time frame: Data point for observation period to data point for treatment period Weeks 9 through 12
Percent change from baseline in the frequency of all seizure activity independent of seizure type
Time frame: Data point for observation period to data point for treatment period Weeks 9 through 12
Change from baseline in parent(s)/caregiver(s) Clinical Global Impressions of Improvement (CGI-I)
Time frame: Data point for observation period to data point for treatment period Weeks 9 through 12
Change from baseline in parent(s)/caregiver(s) Clinical Global Impressions of Severity (CGI-S)
Time frame: Data point for observation period to data point for treatment period Weeks 9 through 12
Change from baseline in Investigator CGI-I
Time frame: Data point for observation period to data point for treatment period Weeks 9 through 12
Change from baseline in Investigator CGI-S
Time frame: Data point for observation period to data point for treatment period Weeks 9 through 12
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jan 2018.
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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
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Cannabidiol Oral Solution in people with Dravet Syndrome, run by INSYS Therapeutics Inc.
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 3 inclusion and 5 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: withdrawn, as of the sponsor's last update on 4 Jan 2018. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. 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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Percent change in the frequency of tonic-clonic, clonic, and focal seizures with motor components (time frame: Data point for observation period to data point for treatment period Weeks 9 through 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 4 Jan 2018.