Dravet SyndromePhase 3NCT02187809

Safety and Tolerability of 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.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Trial summary

To investigate the long-term safety and tolerability of clobazam when administered for 1 year as adjunctive therapy 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?: No, everyone receives an active treatment
How many people: 3 participants
Where: 9 sitesin 2 countries
Study dates: Mar 2015to Oct 2015
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 16 years
All sexes
No healthy volunteers
Child

Who may be able to join

The patient has a diagnosis of Dravet Syndrome supported by:
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)

Show all 12Show fewer

clonic (required for inclusion)
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
The patient is currently receiving a stable dose of clobazam of at least 0.5 mg/kg/day (maximum 20 mg/day) for at least 3 months
Other protocol-defined inclusion and exclusion criteria may apply.

Who cannot join

None listed.

Trial locations and contacts

9 sites in 2 countries.

Country

City, postcode or hospital

9 sites in 2 countries

United States8 sites

US0011
Dallas, Texas
US006
Dallas, Texas
US005
Kansas City, Missouri
US010
Los Angeles, California
US012
Orange, California
US001
Orlando, Florida
US003
Rochester, Minnesota
US004
Seattle, Washington

Other trials in United States

Mexico1 site

MX003
Guadalajara

Other trials in Mexico

About this study

Official title: Multi-site, Prospective, Open-label, Long-term, Flexible Dose, Interventional Study to Evaluate the Safety and Tolerability of Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome

To investigate the long-term safety and tolerability of clobazam when administered for 1 year as adjunctive therapy in paediatric patients aged ≥1 to ≤16 years with Dravet Syndrome.

Conditions: Dravet Syndrome
Being tested: Clobazam
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®

Study groups in NCT02187809
Study group	Role	What they receive
Clobazam	Experimental	A maximum of 2.0 mg/kg/day (maximum 80 mg/day) twice daily (BID); clobazam oral suspension (2.5 mg/mL) or clobazam scored tablets (10 mg), orally Receives: Drug: Clobazam

What is being measured

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

Number of Participants With Adverse Events as a Measure of Safety and Tolerability
Measured: Up to Day 390
Number of Participants With Adverse Events of Special Interest as a Measure of Safety and Tolerability Based on Dose
Measured: Up to Day 390
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 Day 0 to Day 360
Change in Behavioural, Neurocognitive Measures Using Vineland Adaptive Behaviour Scale (VABS)
Measured: Baseline and from Day 0 to Day 360

Secondary outcomes (3)

Change in Mean Weekly Number of Tonic-clonic and Clonic Seizures
Measured: Baseline and from Day 0 to Day 360 and upon Study Completion/Withdrawal
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 Day 0 to Day 360
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 Day 0 to Day 360

Timeline

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

Mar 2015Oct 2015

First posted11 Jul 2014 (estimated)
Study startMar 2015
Primary completionOct 2015
Study completionOct 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.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
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: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 3 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02187809
Sponsor's protocol ID: 14362B
Status last verified: Feb 2017
First submitted: 9 Jul 2014
Last updated: 27 Mar 2017
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

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

Not yet recruitingPhase 3
Open-label, Single-arm Trial of Ganaxolone in CDD Patients 6 Months to Less Than 2 Years Old
CDKL5 Deficiency Disorder
RecruitingPhase 1/2
ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
Dravet Syndrome
RecruitingPhase 3
A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome
Dravet Syndrome
Active, not recruitingPhase 3
A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)
Dravet Syndrome
RecruitingPhase 1/2
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
Dravet Syndrome

All Dravet Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 27 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Multi-site, Prospective, Open-label, Long-term, Flexible Dose, Interventional Study to Evaluate the Safety and Tolerability of Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome
ClinicalTrials.gov ID: NCT02187809
Acronym / sponsor ID: 14362B
Conditions: Dravet Syndrome
Phase: Phase 3
Study type: Interventional
Recruitment status: TerminatedReason stopped: The study was terminated due to recruitment challenges
Enrolment: 3 participants (actual)
Lead sponsor: H. Lundbeck A/SIndustry (pharma, biotech, devices)
Study start: Mar 2015
Primary completion: Oct 2015 (actual)
Study completion: Oct 2015 (actual)
First posted: 11 Jul 2014 (estimated)
Results: Posted 15 Feb 2017

Clinical summary

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

To investigate the long-term safety and tolerability of clobazam when administered for 1 year as adjunctive therapy in paediatric patients aged ≥1 to ≤16 years with Dravet Syndrome.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Clobazam	Experimental	Drug: ClobazamA maximum of 2.0 mg/kg/day (maximum 80 mg/day) twice daily (BID); clobazam oral suspension (2.5 mg/mL) or 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 (12 listed)

The patient has a diagnosis of Dravet Syndrome supported by:
The patient is currently receiving a stable dose of clobazam of at least 0.5 mg/kg/day (maximum 20 mg/day) for at least 3 months
Other protocol-defined inclusion and exclusion criteria may apply.

Key exclusion criteria (0 listed)

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

Primary outcome

Number of Participants With Adverse Events as a Measure of Safety and Tolerability
Time frame: Up to Day 390
Number of Participants With Adverse Events of Special Interest as a Measure of Safety and Tolerability Based on Dose
Time frame: Up to Day 390
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 Day 0 to Day 360
Change in Behavioural, Neurocognitive Measures Using Vineland Adaptive Behaviour Scale (VABS)
Time frame: Baseline and from Day 0 to Day 360

3 secondary outcomes

Change in Mean Weekly Number of Tonic-clonic and Clonic Seizures
Time frame: Baseline and from Day 0 to Day 360 and upon Study Completion/Withdrawal
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 Day 0 to Day 360
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 Day 0 to Day 360

Results and safety

Results posted to the registry on 15 Feb 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Clobazam	3	0

Primary outcome: Number of Participants With Adverse Events as a Measure of Safety and Tolerability

Time frame: Up to Day 390. Number. Unit: participants

Measure	Clobazam n = 1
Value	0

Population analysed: At the time of study termination, only one patient had received IMP. No adverse events were observed in the study.

Primary outcome: Number of Participants With Adverse Events of Special Interest as a Measure of Safety and Tolerability Based on Dose

Time frame: Up to Day 390. Number. Unit: participants

Measure	Clobazam n = 1
Value	0

Population analysed: At the time of study termination, only one patient had received IMP. No adverse events were observed in the study

Primary outcome: 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 Day 0 to Day 360

Population analysed: At the time of study termination, one patient had received IMP. No C-SSRS data were collected from that single patient.

Primary outcome: Change in Behavioural, Neurocognitive Measures Using Vineland Adaptive Behaviour Scale (VABS)

Time frame: Baseline and from Day 0 to Day 360

Population analysed: At the time of study termination, only one patient had received IMP. No VABS data were recorded for that single patient.

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: One patient was treated for 33 days before study termination. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Clobazam	0 of 1 (0%)	0 of 1 (0%)	Not reported

Limitations and caveats (from the sponsor): Early termination leading to small numbers of subjects treated and no analysis. One patient was treated for 33 days.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 27 Mar 2017.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Dravet Syndrome

Not yet recruitingPhase 3
Open-label, Single-arm Trial of Ganaxolone in CDD Patients 6 Months to Less Than 2 Years Old
CDKL5 Deficiency Disorder
RecruitingPhase 1/2
ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
Dravet Syndrome
RecruitingPhase 3
A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome
Dravet Syndrome
Active, not recruitingPhase 3
A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)
Dravet Syndrome
RecruitingPhase 1/2
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
Dravet Syndrome

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 12 inclusion and 0 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: stopped early, as of the sponsor's last update on 27 Mar 2017. 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. 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 15 Feb 2017). 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?

Number of Participants With Adverse Events as a Measure of Safety and Tolerability (time frame: Up to Day 390). The study lists 4 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 NCT02187809.

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 27 Mar 2017.