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

Lundbeck

Status and phase: Withdrawn; Phase 3

Conditions: Dravet Syndrome

Treatments: Drug: Clobazam; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02174094

14362A

Details and patient eligibility

About

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.

Sex: All

Ages: 1 to 16 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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0 participants in 2 patient groups, including a placebo group

Clobazam

Experimental group

Description:

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

Treatment:

Drug: Clobazam

Placebo

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo