Safety and Tolerability of Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome

Phase 3

Terminated

• Conditions: Dravet Syndrome
• Interventions: Drug: Clobazam

• Registration Number: NCT02187809
• Lead Sponsor: H. Lundbeck A/S

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2014-07-09
• Study First Submitted QC: 2014-07-09
• Study First Posted: 2014-07-11
• Study Start: 2015-03
• Primary Completion: 2015-10
• Study Completion: 2015-10
• Results First Submitted: 2016-10-28
• Results First Submitted QC: 2016-12-22
• Status Verified: 2017-02
• Results First Posted: 2017-02-15
• Last Update Submitted: 2017-02-22
• Last Update Posted: 2017-03-27

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 3

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.

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Clobazam	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

Primary Outcome Measures

Name	Time	Method
Number of Participants With Adverse Events as a Measure of Safety and Tolerability	Up to Day 390
Change in Behavioural, Neurocognitive Measures Using Vineland Adaptive Behaviour Scale (VABS)	Baseline and from Day 0 to Day 360
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

Secondary Outcome Measures

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

Trial Locations

Locations (9)

US010; 🇺🇸 Los Angeles, California, United States

US003; 🇺🇸 Rochester, Minnesota, United States

US0011; 🇺🇸 Dallas, Texas, United States

US006; 🇺🇸 Dallas, Texas, United States

US004; 🇺🇸 Seattle, Washington, United States

US012; 🇺🇸 Orange, California, United States

US001; 🇺🇸 Orlando, Florida, United States

US005; 🇺🇸 Kansas City, Missouri, United States

MX003; 🇲🇽 Guadalajara, Mexico