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Phase 2 N=2 Treatment

Verapamil as Therapy for Children and Young Adults With Dravet Syndrome

Dravet Syndrome

Enrolled (actual)
2
Serious AEs
50.0%
Results posted
Mar 2021
Primary outcome: Primary: Change in Number of General Tonic-clonic Seizures From Week 8 (Baseline) Visit to Week 12 Visit — 39; 14 General tonic-clonic seizures

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Verapamil (Drug)
Age
Pediatric, Adult · 2+ yrs
Sex
All
Sponsor
Gillette Children's Specialty Healthcare
Primary completion
Jan 2015

Outcome Measures

OutcomeResultp-value
PRIMARY
Change in Number of General Tonic-clonic Seizures From Week 8 (Baseline) Visit to Week 12 Visit
39; 14
SECONDARY
Change in Number of Myoclonic Seizures From Week 8 (Baseline) to Week 12
116; 175
SECONDARY
Change in Number of Absence Seizures From Week 8 (Baseline) to Week 12
165; 101

Summary

This study will assess how well the drug verapamil can improve control of seizures and dysautonomia symptoms in children and young adults diagnosed with Dravet syndrome. The safety of verapamil when given with all concomitant medications will also be assessed.

Eligibility Criteria

Inclusion Criteria

  • 2 to 25 years old
  • Onset of seizures in first year of life
  • seizure type usually generalized tonic-clonic, clonic, or hemiclonic, often prolonged (>10 minutes)
  • myoclonic jerks/myoclonic seizures
  • history of normal development at seizure onset with subsequent developmental delay or regression which occurs after seizure onset
  • presence of documented abnormality on the SCN1A gene
  • medically intractable epilepsy: must have been on at least 2 prior antiepileptic medications without adequate control of epilepsy
  • subject is capable of giving informed consent (or assent if possible) or has an acceptable surrogate capable of giving informed consent on the subject's behalf

Exclusion Criteria

  • use of clonidine, propranolol, carbamazepine, oxcarbazine, stiripentol, lamotrigine, or cyclosporine
  • Abnormalities of cardiac conduction or rhythm (excluding sinus arrhythmia) on screening EKG
  • significant use of grapefruit juice
  • ketogenic diet
  • pregnancy
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01607073). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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