Back to resultsSafety and Tolerability of Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome
Primary Purpose
Dravet Syndrome
Status
Terminated
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Clobazam
Sponsored by
About this trial
This is an interventional treatment trial for Dravet Syndrome
Eligibility Criteria
The inclusion and exclusion criteria for the patients who participated in lead-in Study 14362A will be transferred from the 14362A study and for the patients who did not participate in lead-in Study 14362A the inclusion/exclusion is separately listed below.
Inclusion Criteria:
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)
- 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.
Sites / Locations
- US010
- US012
- US001
- US003
- US005
- US0011
- US006
- US004
- MX003
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Clobazam
Arm Description
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
Outcomes
Primary Outcome Measures
Number of Participants With Adverse Events as a Measure of Safety and Tolerability
Number of Participants With Adverse Events of Special Interest as a Measure of Safety and Tolerability Based on Dose
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
Change in Behavioural, Neurocognitive Measures Using Vineland Adaptive Behaviour Scale (VABS)
Secondary Outcome Measures
Change in Mean Weekly Number of Tonic-clonic and Clonic Seizures
Number of Initial Treatment Responders Who Returned to Their Baseline Tonic-clonic and Clonic Seizure Rate During the Study (an Assessment of Tachyphylaxis)
Percentage of Initial Treatment Responders Who Returned to Their Baseline Tonic-clonic and Clonic Seizure Rate During the Study (an Assessment of Tachyphylaxis)
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02187809
Brief Title
Safety and Tolerability of Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome
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
Study Type
Interventional
2. Study Status
Record Verification Date
February 2017
Overall Recruitment Status
Terminated
Why Stopped
The study was terminated due to recruitment challenges
Study Start Date
March 2015 (undefined)
Primary Completion Date
October 2015 (Actual)
Study Completion Date
October 2015 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
H. Lundbeck A/S
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Dravet Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
3 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Clobazam
Arm Type
Experimental
Arm Description
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
Intervention Type
Drug
Intervention Name(s)
Clobazam
Other Intervention Name(s)
Onfi®
Primary Outcome Measure Information:
Title
Number of Participants With Adverse Events as a Measure of Safety and Tolerability
Time Frame
Up to Day 390
Title
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
Title
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
Title
Change in Behavioural, Neurocognitive Measures Using Vineland Adaptive Behaviour Scale (VABS)
Time Frame
Baseline and from Day 0 to Day 360
Secondary Outcome Measure Information:
Title
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
Title
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
Title
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
The inclusion and exclusion criteria for the patients who participated in lead-in Study 14362A will be transferred from the 14362A study and for the patients who did not participate in lead-in Study 14362A the inclusion/exclusion is separately listed below.
Inclusion Criteria:
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)
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Email contact via H. Lundbeck A/S
Organizational Affiliation
LundbeckClinicalTrials@lundbeck.com
Official's Role
Study Director
Facility Information:
Facility Name
US010
City
Los Angeles
State/Province
California
Country
United States
Facility Name
US012
City
Orange
State/Province
California
Country
United States
Facility Name
US001
City
Orlando
State/Province
Florida
Country
United States
Facility Name
US003
City
Rochester
State/Province
Minnesota
Country
United States
Facility Name
US005
City
Kansas City
State/Province
Missouri
Country
United States
Facility Name
US0011
City
Dallas
State/Province
Texas
Country
United States
Facility Name
US006
City
Dallas
State/Province
Texas
Country
United States
Facility Name
US004
City
Seattle
State/Province
Washington
Country
United States
Facility Name
MX003
City
Guadalajara
Country
Mexico
12. IPD Sharing Statement
Learn more about this trial
Safety and Tolerability of Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome
We'll reach out to this number within 24 hrs