Dravet SyndromePhase 3NCT02174094
Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome
A Phase 3 study testing Clobazam in people with Dravet Syndrome, run by H. Lundbeck A/S at 9 sites in 2 countries.
Trial summary
The purpose of this study is to investigate the effect on the frequency of tonic-clonic and clonic seizures of clobazam as adjunctive therapy compared to placebo after 16 weeks of treatment in paediatric patients aged ≥1 to ≤16 years with Dravet Syndrome. Read more about this study
- Who is it for?
- Ages 1 year to 16 years
- Conditions the study is for
- Dravet Syndrome
- What is being tested?
- Clobazam
- 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
- 9 sitesin 2 countries
- Study dates
- Mar 2015to Aug 2015
- 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 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Onset of seizures in the first year of life
- History of fever-induced prolonged seizures as determined by the Investigator
- These may include prolonged (approximately 15 minutes or longer) hemi-clonic seizures
- Multiple seizure types which may include:
- generalised tonic-clonic (required for inclusion)
- clonic (required for inclusion)
Show all 9Show fewer
- myoclonic jerks/seizures
- history of normal development prior to seizure onset followed by development delay or regression after seizure onset
- abnormal EEG consistent with Dravet Syndrome 2. The patient has a history of approximately 2 tonic-clonic or clonic seizures in 2 weeks 3. The patient is treated with at least 1 but no more than 3 antiepileptic drugs (AEDs) [Vagal Nerve Stimulator (VNS) and ketogenic diet will not be considered an AED] 4. Patient has at least 2 seizures during the Baseline Period of either 2 or 4 weeks
Who cannot join
- The patient is taking stiripentol, verapamil, or felbatol. If patients have taken these drugs in the past, they need to have been off drug for 5 half-lives
- The patient is taking a sodium channel blocker including, but not limited to, phenytoin, fosphenytoin, carbamazepine, oxcarbamazepine, lamotrigine, lacosamide, and rufinamide. If patients have taken these drugs in the past, they need to have been off drug for 5 half-lives
- The patient is on cannabidiol, medical marijuana, or any drug that contains cannabinoids
- The patient has received chronic treatment (≥2 weeks for any indication) with a benzodiazepine within at least 5 half-lives prior to screening. Rescue therapy for prolonged seizures is allowed
- The patient has received clobazam within 3 months prior to the Screening Visit. If the patient has received clobazam in the past, discontinuation must not have been for adverse events or lack of efficacy
- Other protocol-defined inclusion and exclusion criteria may apply.
Trial locations and contacts
9 sites in 2 countries.
9 sites in 2 countries
United States8 sites
US0011
Dallas, Texas
US006
Dallas, Texas
US002
Houston, Texas
US005
Kansas City, Missouri
US010
Los Angeles, California
US001
Orlando, Florida
US003
Rochester, Minnesota
US004
Seattle, Washington
About this study
Official title: Multi-site, Prospective, Randomised, Double-blind, Placebo-controlled, Parallel-group, Interventional Study to Evaluate the Efficacy, Safety, and Tolerability of Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome
The purpose of this study is to investigate the effect on the frequency of tonic-clonic and clonic seizures of clobazam as adjunctive therapy compared to placebo after 16 weeks of treatment in paediatric patients aged ≥1 to ≤16 years with Dravet Syndrome.
- Conditions
- Dravet Syndrome
- Being tested
- Clobazam, Placebo
- Medical subject headings
- Epilepsies, Myoclonic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ClobazamDrug
Also called Onfi®
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| Clobazam | Experimental | Clobazam - 1.0, 1.5 or 2.0 mg/kg/day (maximum 60 or 80 mg/day) twice daily (BID); Clobazam oral suspension 2.5 mg/mL, clobazam scored tablets 10 mg, orally Receives: Drug: Clobazam |
| Placebo | Placebo comparator | Placebo to clobazam oral suspension 2.5 mg/mL and placebo to clobazam scored tablets 10 mg, orally Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent change from baseline to study completion/withdrawal in seizure rate for combined tonic-clonic and clonic seizure rates, based upon a calculation of seizure frequency determined from daily seizure diary counts
Measured: Baseline and from week 0 to week 16
Secondary outcomes (14)
Percent change from baseline to study completion/withdrawal in seizure rate for combined tonic-clonic and clonic seizure rates, based upon a calculation of seizure frequency determined from daily seizure diary counts during 4 weeks of maintenance
Measured: Baseline and from week 4 to week 16
Percent change in seizure rate for myoclonic seizures determined from daily seizure diary counts
Measured: Baseline and from week 0 to week 16
Percent change in seizure rate for atypical absence seizures determined from daily seizure diary counts
Measured: Baseline and from week 0 to week 16
Percent change in seizure rate for complex partial seizures determined from daily seizure diary counts
Measured: Baseline and from week 0 to week 16
Percent change in seizure rate for all seizure types determined from daily seizure diary counts
Measured: Baseline and from week 0 to week 16
Number of initial treatment responders who returned to their baseline tonic-clonic and clonic seizure rate during the study (an assessment of tachyphylaxis)
Measured: Baseline and from week 0 to week 16
Percentage of initial treatment responders who returned to their baseline tonic-clonic and clonic seizure rate during the study (an assessment of tachyphylaxis)
Measured: Baseline and from week 0 to week 16
Percent change in seizure rate for myoclonic seizures determined from video EEG
Measured: Baseline and from week 0 to week 16
Percent change in seizure rate for atypical absence seizures determined from video EEG
Measured: Baseline and from week 0 to week 16
Change in Symptom and Seizure Activity Scale (Investigator and Parent/caregiver versions)
Measured: Baseline and from week 0 to week 16
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Measured: Up to Week 32
Columbia Suicide Severity Rating Scale (C-SSRS), categorisation based on Columbia Classification Algorithm of Suicide Assessment (C-CASA) categories (1, 2, 3, 4 and 7) for patients aged ≥ 6 years
Measured: Baseline and from week 0 to week 16
Number of Participants with Adverse Events of special interest as a Measure of Safety and Tolerability based on dose
Measured: Baseline and Week 32
Change in Vineland Adaptive Behaviour Scale (VABS) - all adaptive behavior sub-domains and maladaptive behaviors
Measured: Baseline and from week 0 to week 16
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jun 2014 (estimated)
- Study startMar 2015
- Primary completionAug 2015
- Study completionAug 2015
Why it stopped: The study was terminated due to recruitment challenges
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.
- More trials run by H. Lundbeck A/SThe sponsor's other registered studies.
- 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
- H. Lundbeck A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Email contact via H. Lundbeck A/S, Study director, LundbeckClinicalTrials@lundbeck.com
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02174094
- Sponsor's protocol ID
- 14362A
- Status last verified
- Sep 2015
- First submitted
- 2 Jun 2014
- Last updated
- 24 Sep 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multi-site, Prospective, Randomised, Double-blind, Placebo-controlled, Parallel-group, Interventional Study to Evaluate the Efficacy, Safety, and Tolerability of Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome
- ClinicalTrials.gov ID
- NCT02174094
- Acronym / sponsor ID
- 14362A
- Conditions
- Dravet Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: The study was terminated due to recruitment challenges
- Enrolment
- 0 participants (actual)
- Lead sponsor
- H. Lundbeck A/SIndustry (pharma, biotech, devices)
- Study start
- Mar 2015
- Primary completion
- Aug 2015 (actual)
- Study completion
- Aug 2015 (actual)
- First posted
- 25 Jun 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.
The purpose of this study is to investigate the effect on the frequency of tonic-clonic and clonic seizures of clobazam as adjunctive therapy compared to placebo after 16 weeks of treatment in paediatric patients aged ≥1 to ≤16 years with Dravet Syndrome.
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 |
|---|---|---|
| Clobazam | Experimental | Drug: ClobazamClobazam - 1.0, 1.5 or 2.0 mg/kg/day (maximum 60 or 80 mg/day) twice daily (BID); Clobazam oral suspension 2.5 mg/mL, clobazam scored tablets 10 mg, orally |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo to clobazam oral suspension 2.5 mg/mL and placebo to clobazam scored tablets 10 mg, orally |
Who may qualify
- Ages
- 1 year to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- Onset of seizures in the first year of life
- History of fever-induced prolonged seizures as determined by the Investigator
- These may include prolonged (approximately 15 minutes or longer) hemi-clonic seizures
- Multiple seizure types which may include:
Key exclusion criteria (6 listed)
- The patient is taking stiripentol, verapamil, or felbatol. If patients have taken these drugs in the past, they need to have been off drug for 5 half-lives
- The patient is taking a sodium channel blocker including, but not limited to, phenytoin, fosphenytoin, carbamazepine, oxcarbamazepine, lamotrigine, lacosamide, and rufinamide. If patients have taken these drugs in the past, they need to have been off drug for 5 half-lives
- The patient is on cannabidiol, medical marijuana, or any drug that contains cannabinoids
- The patient has received chronic treatment (≥2 weeks for any indication) with a benzodiazepine within at least 5 half-lives prior to screening. Rescue therapy for prolonged seizures is allowed
- The patient has received clobazam within 3 months prior to the Screening Visit. If the patient has received clobazam in the past, discontinuation must not have been for adverse events or lack of efficacy
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 |
|---|---|---|
| ClobazamAlso: Onfi® | Drug | Clobazam |
| Placebo | Drug | Placebo |
Primary outcome
Percent change from baseline to study completion/withdrawal in seizure rate for combined tonic-clonic and clonic seizure rates, based upon a calculation of seizure frequency determined from daily seizure diary counts
Time frame: Baseline and from week 0 to week 16
14 secondary outcomes
Percent change from baseline to study completion/withdrawal in seizure rate for combined tonic-clonic and clonic seizure rates, based upon a calculation of seizure frequency determined from daily seizure diary counts during 4 weeks of maintenance
Time frame: Baseline and from week 4 to week 16
Percent change in seizure rate for myoclonic seizures determined from daily seizure diary counts
Time frame: Baseline and from week 0 to week 16
Percent change in seizure rate for atypical absence seizures determined from daily seizure diary counts
Time frame: Baseline and from week 0 to week 16
Percent change in seizure rate for complex partial seizures determined from daily seizure diary counts
Time frame: Baseline and from week 0 to week 16
Percent change in seizure rate for all seizure types determined from daily seizure diary counts
Time frame: Baseline and from week 0 to week 16
Number of initial treatment responders who returned to their baseline tonic-clonic and clonic seizure rate during the study (an assessment of tachyphylaxis)
Time frame: Baseline and from week 0 to week 16
Percentage of initial treatment responders who returned to their baseline tonic-clonic and clonic seizure rate during the study (an assessment of tachyphylaxis)
Time frame: Baseline and from week 0 to week 16
Percent change in seizure rate for myoclonic seizures determined from video EEG
Time frame: Baseline and from week 0 to week 16
Percent change in seizure rate for atypical absence seizures determined from video EEG
Time frame: Baseline and from week 0 to week 16
Change in Symptom and Seizure Activity Scale (Investigator and Parent/caregiver versions)
Time frame: Baseline and from week 0 to week 16
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Time frame: Up to Week 32
Columbia Suicide Severity Rating Scale (C-SSRS), categorisation based on Columbia Classification Algorithm of Suicide Assessment (C-CASA) categories (1, 2, 3, 4 and 7) for patients aged ≥ 6 years
Time frame: Baseline and from week 0 to week 16
Number of Participants with Adverse Events of special interest as a Measure of Safety and Tolerability based on dose
Time frame: Baseline and Week 32
Change in Vineland Adaptive Behaviour Scale (VABS) - all adaptive behavior sub-domains and maladaptive behaviors
Time frame: Baseline and from week 0 to week 16
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 24 Sep 2015.
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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.
Email contact via H. Lundbeck A/S study director
LundbeckClinicalTrials@lundbeck.com
9 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Clobazam in people with Dravet Syndrome, run by H. Lundbeck A/S at 9 sites in 2 countries.
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 9 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: withdrawn, as of the sponsor's last update on 24 Sep 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, Mexico. 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. 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 from baseline to study completion/withdrawal in seizure rate for combined tonic-clonic and clonic seizure rates, based upon a calculation of seizure frequency determined from daily seizure diary counts (time frame: Baseline and from week 0 to week 16). 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 24 Sep 2015.