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

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ClinicalTrials.gov Identifier: NCT02174094
Recruitment Status : Withdrawn (The study was terminated due to recruitment challenges)
First Posted : June 25, 2014
Last Update Posted : September 24, 2015
Sponsor:
Information provided by (Responsible Party):
H. Lundbeck A/S

Brief Summary:
The purpose of this study is to investigate the effect on the frequency of tonic-clonic and clonic seizures of clobazam as adjunctive therapy compared to placebo after 16 weeks of treatment in paediatric patients aged ≥1 to ≤16 years with Dravet Syndrome.

Condition or disease Intervention/treatment Phase
Dravet Syndrome Drug: Clobazam Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: Multi-site, Prospective, Randomised, Double-blind, Placebo-controlled, Parallel-group, Interventional Study to Evaluate the Efficacy, Safety, and Tolerability of Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome
Study Start Date : March 2015
Actual Primary Completion Date : August 2015
Actual Study Completion Date : August 2015


Arm Intervention/treatment
Experimental: Clobazam
Clobazam - 1.0, 1.5 or 2.0 mg/kg/day (maximum 60 or 80 mg/day) twice daily (BID); Clobazam oral suspension 2.5 mg/mL, clobazam scored tablets 10 mg, orally
Drug: Clobazam
Other Name: Onfi®

Placebo Comparator: Placebo
Placebo to clobazam oral suspension 2.5 mg/mL and placebo to clobazam scored tablets 10 mg, orally
Drug: Placebo



Primary Outcome Measures :
  1. Percent change from baseline to study completion/withdrawal in seizure rate for combined tonic-clonic and clonic seizure rates, based upon a calculation of seizure frequency determined from daily seizure diary counts [ Time Frame: Baseline and from week 0 to week 16 ]

Secondary Outcome Measures :
  1. Percent change from baseline to study completion/withdrawal in seizure rate for combined tonic-clonic and clonic seizure rates, based upon a calculation of seizure frequency determined from daily seizure diary counts during 4 weeks of maintenance [ Time Frame: Baseline and from week 4 to week 16 ]
  2. Percent change in seizure rate for myoclonic seizures determined from daily seizure diary counts [ Time Frame: Baseline and from week 0 to week 16 ]
  3. Percent change in seizure rate for atypical absence seizures determined from daily seizure diary counts [ Time Frame: Baseline and from week 0 to week 16 ]
  4. Percent change in seizure rate for complex partial seizures determined from daily seizure diary counts [ Time Frame: Baseline and from week 0 to week 16 ]
  5. Percent change in seizure rate for all seizure types determined from daily seizure diary counts [ Time Frame: Baseline and from week 0 to week 16 ]
  6. 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 week 0 to week 16 ]
  7. 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 week 0 to week 16 ]
  8. Percent change in seizure rate for myoclonic seizures determined from video EEG [ Time Frame: Baseline and from week 0 to week 16 ]
  9. Percent change in seizure rate for atypical absence seizures determined from video EEG [ Time Frame: Baseline and from week 0 to week 16 ]
  10. Change in Symptom and Seizure Activity Scale (Investigator and Parent/caregiver versions) [ Time Frame: Baseline and from week 0 to week 16 ]
  11. Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: Up to Week 32 ]
  12. 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 week 0 to week 16 ]
  13. Number of Participants with Adverse Events of special interest as a Measure of Safety and Tolerability based on dose [ Time Frame: Baseline and Week 32 ]
  14. Change in Vineland Adaptive Behaviour Scale (VABS) - all adaptive behavior sub-domains and maladaptive behaviors [ Time Frame: Baseline and from week 0 to week 16 ]


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Ages Eligible for Study:   1 Year to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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 2. The patient has a history of approximately 2 tonic-clonic or clonic seizures in 2 weeks 3. The patient is treated with at least 1 but no more than 3 antiepileptic drugs (AEDs) [Vagal Nerve Stimulator (VNS) and ketogenic diet will not be considered an AED] 4. Patient has at least 2 seizures during the Baseline Period of either 2 or 4 weeks

Exclusion Criteria:

  1. The patient is taking stiripentol, verapamil, or felbatol. If patients have taken these drugs in the past, they need to have been off drug for 5 half-lives
  2. The patient is taking a sodium channel blocker including, but not limited to, phenytoin, fosphenytoin, carbamazepine, oxcarbamazepine, lamotrigine, lacosamide, and rufinamide. If patients have taken these drugs in the past, they need to have been off drug for 5 half-lives
  3. The patient is on cannabidiol, medical marijuana, or any drug that contains cannabinoids
  4. The patient has received chronic treatment (≥2 weeks for any indication) with a benzodiazepine within at least 5 half-lives prior to screening. Rescue therapy for prolonged seizures is allowed
  5. The patient has received clobazam within 3 months prior to the Screening Visit. If the patient has received clobazam in the past, discontinuation must not have been for adverse events or lack of efficacy

Other protocol-defined inclusion and exclusion criteria may apply.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02174094


Locations
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United States, California
US010
Los Angeles, California, United States
United States, Florida
US001
Orlando, Florida, United States
United States, Minnesota
US003
Rochester, Minnesota, United States
United States, Missouri
US005
Kansas City, Missouri, United States
United States, Texas
US006
Dallas,, Texas, United States
US0011
Dallas, Texas, United States
US002
Houston, Texas, United States
United States, Washington
US004
Seattle, Washington, United States
Mexico
MX003
Guadalajara, Mexico
Sponsors and Collaborators
H. Lundbeck A/S
Investigators
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Study Director: Email contact via H. Lundbeck A/S LundbeckClinicalTrials@lundbeck.com

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Responsible Party: H. Lundbeck A/S
ClinicalTrials.gov Identifier: NCT02174094     History of Changes
Other Study ID Numbers: 14362A
First Posted: June 25, 2014    Key Record Dates
Last Update Posted: September 24, 2015
Last Verified: September 2015

Additional relevant MeSH terms:
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Syndrome
Epilepsies, Myoclonic
Spasms, Infantile
Rett Syndrome
Disease
Pathologic Processes
Epilepsy, Generalized
Epilepsy
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Epileptic Syndromes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Clobazam
Anticonvulsants
Anti-Anxiety Agents
Tranquilizing Agents
Central Nervous System Depressants
Physiological Effects of Drugs
Psychotropic Drugs
GABA-A Receptor Agonists
GABA Agonists
GABA Agents
Neurotransmitter Agents