Clinical trial NCT02187809: 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
Overall status: TERMINATED
Phase: PHASE3
Conditions: Dravet Syndrome
Lead sponsor: H. Lundbeck A/S
Sponsor class: INDUSTRY
First posted: 2014-07-11
Enrollment: 3
Sex: ALL
Minimum age (years): 1
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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:

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 exclusion criteria may apply.

Primary outcomes:
measure: Number of Participants With Adverse Events as a Measure of Safety and Tolerability
timeFrame: Up to Day 390
measure: Number of Participants With Adverse Events of Special Interest as a Measure of Safety and Tolerability Based on Dose
timeFrame: Up to Day 390
measure: 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
timeFrame: Baseline and from Day 0 to Day 360
measure: Change in Behavioural, Neurocognitive Measures Using Vineland Adaptive Behaviour Scale (VABS)
timeFrame: Baseline and from Day 0 to Day 360
Secondary outcomes:
measure: Change in Mean Weekly Number of Tonic-clonic and Clonic Seizures
timeFrame: Baseline and from Day 0 to Day 360 and upon Study Completion/Withdrawal
measure: Number of Initial Treatment Responders Who Returned to Their Baseline Tonic-clonic and Clonic Seizure Rate During the Study (an Assessment of Tachyphylaxis)
timeFrame: Baseline and from Day 0 to Day 360
measure: Percentage of Initial Treatment Responders Who Returned to Their Baseline Tonic-clonic and Clonic Seizure Rate During the Study (an Assessment of Tachyphylaxis)
timeFrame: Baseline and from Day 0 to Day 360
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: US010
state: California
city: Orange
country: United States
facility: US012
state: California
city: Orlando
country: United States
facility: US001
state: Florida
city: Rochester
country: United States
facility: US003
state: Minnesota
city: Kansas City
country: United States
facility: US005
state: Missouri
city: Dallas
country: United States
facility: US0011
state: Texas
city: Dallas
country: United States
facility: US006
state: Texas
city: Seattle
country: United States
facility: US004
state: Washington
city: Guadalajara
country: Mexico
facility: MX003
Study officials:
affiliation: LundbeckClinicalTrials@lundbeck.com
name: Email contact via H. Lundbeck A/S
role: STUDY_DIRECTOR
References: