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An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution in Children and Young Adults With Dravet Syndrome

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ClinicalTrials.gov Identifier: NCT02823145
Recruitment Status : Enrolling by invitation
First Posted : July 6, 2016
Last Update Posted : November 15, 2018
Sponsor:
Information provided by (Responsible Party):
Zogenix, Inc. ( Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. )

Brief Summary:
This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome.

Condition or disease Intervention/treatment Phase
Dravet Syndrome Drug: ZX008 (Fenfluramine Hydrochloride) Phase 3

Detailed Description:
This is an international, multicenter, open-label, long-term safety study of ZX008 in pediatric and young adult subjects with Dravet syndrome who participated in one of the core studies (ZX008-1501 and ZX008-1502) and are candidates for continuous treatment for an extended period of time. This trial will consist of a 36-month Open-Label Extension (OLE) Treatment Period and a 2-week Post-Dosing Period.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 340 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution as an Adjunctive Therapy in Children and Young Adults With Dravet Syndrome
Study Start Date : June 2016
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019


Arm Intervention/treatment
Experimental: ZX008
ZX008 is supplied as an oral solution in a concentration of 2.5 mg/mL. Subjects will be titrated to an effective dose beginning with 0.2 mg/kg/day (maximum: 30 mg/day).
Drug: ZX008 (Fenfluramine Hydrochloride)



Primary Outcome Measures :
  1. Long-term safety and tolerability as measured by treatment emergent adverse events, including clinical labs, vital signs, and examination findings. [ Time Frame: Pre-baseline Up to 156 weeks ]
    Sensitivity of the key efficacy analysis will be assessed for changes in dose or type of concomitant AED medications


Secondary Outcome Measures :
  1. Proportion of subjects who achieve reduction from baseline in convulsive seizure frequency [ Time Frame: Baseline up to 156 weeks ]
  2. The longest interval between convulsive seizures will be calculated for each subject over the entire open-label treatment period. [ Time Frame: Up to 156 weeks ]


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Ages Eligible for Study:   2 Years to 35 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Male or non-pregnant, non-lactating female, age 2 to 18 years, inclusive as of the day of the core study Screening Visit.
  • Satisfactory completion of the core study in the opinion of the investigator and the sponsor.
  • Subjects who are >18 to ≤35 years of age at the time of screening and did not participate in one of the core studies may be eligible for participation.
  • A documented medical history to support a clinical diagnosis of Dravet syndrome, where convulsive seizures are not completely controlled by current antiepileptic drugs.
  • Parent/caregiver is willing and able to be compliant with diary completion, visit schedule and study drug accountability.
  • Subject's parent/caregiver has been compliant with diary completion during the core study, in the opinion of the investigator (eg, at least 90% compliant).

Key Exclusion Criteria:

  • Current or past history of cardiovascular or cerebrovascular disease, myocardial infarction or stroke.
  • Current cardiac valvulopathy or pulmonary hypertension that is clinically significant and warrants discontinuation of study medication.
  • Current or past history of glaucoma.
  • Moderate or severe hepatic impairment.
  • Receiving concomitant therapy with: centrally-acting anorectic agents; monoamineoxidase inhibitors; any centrally-acting compound with clinically appreciable amount of serotonin agonist or antagonist properties, including serotonin reuptake inhibition; atomoxetine, or other centrally-acting noradrenergic agonist; cyproheptadine, and/or cytochrome P450 (CYP) 2D6/3A4/2B6 inhibitors/substrates.
  • Currently taking carbamazepine, oxcarbamazepine, eslicarbazepine, phenobarbital, or phenytoin, or has taken any of these within the past 30 days, as maintenance therapy.
  • A clinically significant condition, or has had clinically relevant symptoms or a clinically significant illness at Visit 1, other than epilepsy, that would negatively impact study participation, collection of study data, or pose a risk to the subject.

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): NCT02823145


  Show 49 Study Locations
Sponsors and Collaborators
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.

Responsible Party: Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
ClinicalTrials.gov Identifier: NCT02823145     History of Changes
Other Study ID Numbers: ZX008-1503
First Posted: July 6, 2016    Key Record Dates
Last Update Posted: November 15, 2018
Last Verified: November 2018

Keywords provided by Zogenix, Inc. ( Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. ):
seizure
tonic clonic
epilepsy
myoclonic
encephalopathy

Additional relevant MeSH terms:
Syndrome
Epilepsies, Myoclonic
Disease
Pathologic Processes
Epilepsy
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Fenfluramine
Serotonin Uptake Inhibitors
Neurotransmitter Uptake Inhibitors
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Serotonin Agents
Physiological Effects of Drugs