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A Study to Investigate the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) as an Adjunctive Therapy in Children and Adults With Lennox-Gastaut Syndrome

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ClinicalTrials.gov Identifier: NCT03355209
Recruitment Status : Recruiting
First Posted : November 28, 2017
Last Update Posted : July 16, 2018
Sponsor:
Information provided by (Responsible Party):
Zogenix, Inc. ( Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. )

Brief Summary:
This is a two-part, multicenter, double-blind, parallel-group, placebo controlled study to evaluate the effect of ZX008 when used as adjunctive therapy for the treatment of uncontrolled seizures in children and adults with Lennox-Gastaut syndrome (LGS).

Condition or disease Intervention/treatment Phase
Lennox Gastaut Syndrome Drug: ZX008 02 and 0.8 mg/kg/day Other: Placebo Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 225 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Part 1: Double-Blind ZX008 - 0.2 mg/kg/day or Placebo and Part 2: Open-Label
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Part 1: double-Blind Part 2: Open-Label
Primary Purpose: Treatment
Official Title: A Two-Part Study of ZX008 in Children and Adults With Lennox-Gastaut Syndrome (LGS); Part 1: A Multicenter, Randomized, Double-blind, Parallel Group, Placebo Controlled Trial of Two Fixed Doses of ZX008 (Fenfluramine Hydrochloride) Oral Solution as Adjunctive Therapy for Seizures in Children and Adults With LGS, Followed by Part 2: An Open-Label Extension Trial to Assess Long-Term Safety of ZX008 in Children and Adults With LGS
Actual Study Start Date : November 27, 2017
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Seizures

Arm Intervention/treatment
Experimental: ZX008 0.2 and 0.8 mg/kg/day
Part 1: ZX008 is supplied as an oral solution. Subjects will be randomized to receive 1 of 2 doses of ZX008 (0.2 mg/kg/day, 0.8 mg/kg/day or placebo.
Drug: ZX008 02 and 0.8 mg/kg/day
ZX008 drug product is an oral aqueous solution of fenfluramine hydrochloride. The product is sugar free and is intended to be compatible with KD.

Placebo Comparator: Matching Placebo
Part 1: Matching ZX008 placebo is supplied as an oral solution.
Other: Placebo
Placebo will be administered twice a day (BID) in equally divided doses.
Other Names:
  • Placebo Comparator
  • Matching Placebo

Placebo Comparator: Open-Label
Part 2: ZX008 is supplied as an oral solution. Study medication will be administered twice a day (BID) in equally divided doses.
Drug: ZX008 02 and 0.8 mg/kg/day
ZX008 drug product is an oral aqueous solution of fenfluramine hydrochloride. The product is sugar free and is intended to be compatible with KD.




Primary Outcome Measures :
  1. Part 1: Change from baseline in frequency of seizures that result in drops in subjects receiving ZX008 compared to placebo [ Time Frame: Up to 20 weeks maintenance and taper period (T+M) ]
    Parent/caregiver seizure diary record will be used to assess frequency, type and duration of seizure activity


Secondary Outcome Measures :
  1. Part 2: Adverse events and related safety parameters in subjects receiving ZX008 compared to placebo [ Time Frame: Up to 12 months open label ]
    Safety and tolerability evaluated by reported adverse events, laboratory parameters, physical and neurological examination, vital signs, electrocardiograms, echocardiograms, and body weight



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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 35 years, inclusive as of the day of the Screening Visit.
  • Clinical diagnosis of Lennox-Gastaut syndrome, where seizures that result in drops are not completely controlled by current antiepileptic treatments.
  • Onset of seizures at 11 years of age or younger.
  • Abnormal cognitive development.
  • Must be receiving at least 1 concomitant AED and up to 4 concomitant anti-epileptic treatments.

Key Exclusion Criteria:

  • Etiology of seizures is a degenerative neurological disease.
  • History of hemiclonic seizures in the first year of life.
  • Subject only has drop seizures in clusters, where individual seizures cannot be counted reliably.
  • Pulmonary arterial hypertension.
  • Current or past history of cardiovascular or cerebrovascular disease, such as cardiac valvulopathy, myocardial infarction or stroke.
  • 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.
  • Taking felbamate for less than 1 year prior to screening and/or does not have stable liver function and hematology laboratory tests, and/or the dose has not been stable for at least 60 days prior to the Screening Visit.
  • Currently receiving an investigational product.
  • Institutionalized in a general nursing home (ie, in a facility that does not specialize in epilepsy care).
  • A clinically significant condition, or has had clinically relevant symptoms or a clinically significant illness in the 4 weeks prior to the Screening Visit, 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): NCT03355209


Contacts
Contact: Betty J Quarles, B.S. 510-375-5357 Bquarles@zogenix.com
Contact: Peter St. Wecker, Ph.D. 510-388-9968 Pstwecker@zogenix.com

  Show 35 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: NCT03355209     History of Changes
Other Study ID Numbers: ZX008-1601
First Posted: November 28, 2017    Key Record Dates
Last Update Posted: July 16, 2018
Last Verified: July 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Zogenix, Inc. ( Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. ):
LGS

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