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Summary

for people ages 2–18 (full criteria)
at San Francisco, California and other locations
study started
estimated completion:

Description

Summary

This is a multicenter, double-blind, parallel-group, placebo-controlled, study to assess the efficacy, safety, and PK of ZX008 when used as adjunctive therapy for uncontrolled seizures in pediatric and young adult subjects with Dravet syndrome. After an initial Screening and Baseline charting of seizure frequency, subjects who qualify for the study will be randomized (1:1:1) to receive either ZX008 (0.2 mg/kg/day, 0.8 mg/kg/day; maximum dose: 30 mg/day) or placebo. Randomization will be stratified by age group (< 6 years, ≥6 to 18 years). All subjects will be titrated to their randomized dose over a 14-day Titration Period. Following titration, subjects will continue treatment at their randomly assigned dose over a 12-week Maintenance Period. Subjects exiting the study will undergo a 2-week taper, unless they enroll in a follow-on study. Subjects will be followed for post-study safety monitoring. Parents/caregivers will use a diary daily to record the number/type of seizures, dosing, and use of rescue medication.

Official Title

A Multicenter, Randomized, Double-blind, Parallel Group, Placebo-controlled Trial of Two Fixed Doses of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy in Children and Young Adults With Dravet Syndrome

Keywords

Dravet Syndrome Seizure Disorder seizure tonic-atonic clonic epilepsy myoclonic encephalopathy Syndrome Seizures Epilepsies, Myoclonic Pharmaceutical Solutions Fenfluramine

Eligibility

For people ages 2–18

Key Inclusion Criteria:

  • Male or non-pregnant, non-lactating female, age 2 to 18 years, inclusive as of the day of the Screening Visit.
  • Clinical diagnosis of Dravet syndrome, where convulsive seizures are not completely controlled by current antiepileptic drugs.
  • Must have a minimum # of convulsive seizures per 4-week period for past 12 weeks prior to screening
  • All medications or interventions for epilepsy must be stable for at least 4 weeks prior to screening and expected to remain stable throughout the study.
  • No cardiovascular or cardiopulmonary abnormality based on ECHO, ECG or physical examination
  • Parent/caregiver is willing and able to be compliant with diary completion, visit schedule and study drug accountability.

Key Exclusion Criteria:

  • Pulmonary arterial hypertension.
  • Current or past history of cardiovascular or cerebrovascular disease, such as cardiac valvulopathy, myocardial infarction or stroke.
  • Current or past history of glaucoma.
  • Moderate or severe hepatic impairment.
  • Receiving concomitant therapy with: anorectic agents; monoamine-oxidase inhibitors;medications that act via serotonin including serotonin reuptake inhibitors;atomoxetine, or other centrally-acting noradrenergic agonist; or cyproheptadine.
  • Currently receiving or has received stiripentol in the past 21 days prior to Screening.
  • Currently taking carbamazepine, oxcarbamazepine, eslicarbazepine, phenobarbital, or phenytoin, or has taken any of these within the past 30 days.
  • Positive result on tetrahydrocannabinol (THC) or cannabidiol (CBD) test at the Screening Visit.
  • A clinically significant medical condition,that would interfere with study participation, collection of study data, or pose a risk to the subject.

Locations

  • University of California San Francisco
    San Francisco, California, 94143, United States
  • Rady Children's Hospital San Diego
    San Diego, California, 92123, United States
  • Phoenix Children's
    Phoenix, Arizona, 85016, United States
  • University of Utah
    Salt Lake City, Utah, 84113, United States
  • Center for Neurosciences - Tucson
    Tucson, Arizona, 85718, United States
  • MultiCare Institute for Research & Innovation
    Tacoma, Washington, 98405, United States
  • Seattle Children's Hospital
    Seattle, Washington, 98105, United States
  • British Columbia Children's Hospital BCCH
    Vancouver, British Columbia, V6H3V4, Canada
  • The Children's Hospital Colorado
    Aurora, Colorado, 80045, United States
  • Alberta Children's Hospital Childrens Comprehensive Epilepsy Centre
    Calgary, Alberta, T3B6A8, Canada
  • Cook Children's Medical Center
    Fort Worth, Texas, 76087, United States
  • Mayo Clinic
    Rochester, Minnesota, 55905, United States
  • Centre Hospitalier Universitaire Sainte-Justine
    Quebec, H3T 1C5, Canada
  • Children's Hospital of Chicago
    Chicago, Illinois, 60611, United States
  • NW FL Clinical Research Group, LLC
    Gulf Breeze, Florida, 32561, United States
  • Panda Neurology
    Atlanta, Georgia, 30328, United States
  • Neurology and Epilepsy Research Center
    Orlando, Florida, 32819, United States
  • Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania, 19104, United States
  • Miami Children's Hospital Brain Institute
    Miami, Florida, 33155, United States
  • Saint Barnabas Medical Center
    Livingston, New Jersey, 07039, United States
  • Boston Children's Hospital
    Boston, Massachusetts, 02467, United States
  • Massachusetts General Hospital
    Boston, Massachusetts, 02114, United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
ID
NCT02682927
Phase
Phase 3
Lead Scientist
Joseph Sullivan
Study Type
Interventional
Last Updated
March 8, 2018