Dravet Syndrome
Conditions
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.
Interventions
DRUGClobazam
Sponsors
H. Lundbeck A/S
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to 16 Years
Healthy volunteers
No
Inclusion 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: 1. The patient has a diagnosis of Dravet Syndrome supported by: 1. onset of seizures in the first year of life 2. history of fever-induced prolonged seizures as determined by the Investigator * these may include prolonged (approximately 15 minutes or longer) hemi-clonic seizures 3. multiple seizure types which may include: * generalised tonic-clonic (required for inclusion) * clonic (required for inclusion) * myoclonic jerks/seizures 4. history of normal development prior to seizure onset followed by development delay or regression after seizure onset 5. abnormal EEG consistent with Dravet Syndrome 2. 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants With Adverse Events as a Measure of Safety and Tolerability | Up to Day 390 |
| Number of Participants With Adverse Events of Special Interest as a Measure of Safety and Tolerability Based on Dose | 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 | Baseline and from Day 0 to Day 360 |
| Change in Behavioural, Neurocognitive Measures Using Vineland Adaptive Behaviour Scale (VABS) | Baseline and from Day 0 to Day 360 |
Secondary
| Measure | Time frame |
|---|---|
| Percentage of Initial Treatment Responders Who Returned to Their Baseline Tonic-clonic and Clonic Seizure Rate During the Study (an Assessment of Tachyphylaxis) | Baseline and from Day 0 to Day 360 |
| Number of Initial Treatment Responders Who Returned to Their Baseline Tonic-clonic and Clonic Seizure Rate During the Study (an Assessment of Tachyphylaxis) | Baseline and from Day 0 to Day 360 |
| Change in Mean Weekly Number of Tonic-clonic and Clonic Seizures | Baseline and from Day 0 to Day 360 and upon Study Completion/Withdrawal |
Countries
Mexico, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Clobazam 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
Clobazam | 1 |
| Total | 1 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | The study was terminated | 3 |
Baseline characteristics
| Characteristic | Clobazam |
|---|---|
| Age, Continuous | 13 years |
| Sex: Female, Male Female | 1 Participants |
| Sex: Female, Male Male | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 0 / 1 |
| serious Total, serious adverse events | 0 / 1 |
Outcome results
Primary
Change in Behavioural, Neurocognitive Measures Using Vineland Adaptive Behaviour Scale (VABS)
Time frame: Baseline and from Day 0 to Day 360
Population: At the time of study termination, only one patient had received IMP. No VABS data were recorded for that single patient.
Primary
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: At the time of study termination, one patient had received IMP. No C-SSRS data were collected from that single patient.
Primary
Number of Participants With Adverse Events as a Measure of Safety and Tolerability
Time frame: Up to Day 390
Population: At the time of study termination, only one patient had received IMP. No adverse events were observed in the study.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Clobazam | Number of Participants With Adverse Events as a Measure of Safety and Tolerability | 0 participants |
Primary
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
Population: At the time of study termination, only one patient had received IMP. No adverse events were observed in the study
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Clobazam | Number of Participants With Adverse Events of Special Interest as a Measure of Safety and Tolerability Based on Dose | 0 participants |
Secondary
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
Population: At the time of study termination, only one patient had received IMP. No seizure data were summarised for that single patient.
Secondary
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
Population: At the time of study termination, only one patient had received IMP. No seizure data were summarised for that single patient.
Secondary
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
Population: At the time of study termination, only one patient had received IMP. No seizure data were summarised for that single patient.