Clinical trial NCT03936777: A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome
Official title: An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy for Seizures in Patients With Rare Seizure Disorders Such as Epileptic Encephalopathies Including Dravet Syndrome and Lennox-Gastaut Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Dravet Syndrome
Lennox Gastaut Syndrome
Epileptic Encephalopathy
Lead sponsor: UCB Pharma
Sponsor class: INDUSTRY
Collaborators: name: Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
First posted: 2019-05-03
Enrollment: 412
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Single Group Assisgnment
Primary purpose: TREATMENT

Brief summary: This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome, Lennox-Gastaut syndrome or epileptic encephalopathy

Detailed description: This is an international, multicenter, open-label, long-term safety study of ZX008 in patients with epileptic encephalopathy, including Dravet syndrome or Lennox-Gastaut syndrome. Subjects eligible for participation are those with Dravet syndrome who are currently enrolled in Study ZX008-1503, or those with Lennox-Gastaut syndrome who have successfully completed Study ZX008-1601-Part 2, and are candidates for continued treatment with ZX008 for an extended period of time, or those with Dravet syndrome, Lennox-Gastaut syndrome, or another epileptic encephalopathy who have completed participation in another Zogenix-sponsored study and have been invited to participate in this study.

Eligibility criteria: Inclusion Criteria:

* Male or nonpregnant, nonlactating female
* Satisfactory completion of a core study
* Has a rare seizure disorder, such as epileptic encephalopathy and has successfully completed another Zogenix-sponsored clinical trials with ZX008
* Subject's caregiver is willing and able to be compliant with study procedures, visit schedule and study drug accountability

Exclusion Criteria:

* Current cardiac valvulopathy or pulmonary hypertension that is clinically significant
* Moderate or severe hepatic impairment
* Receiving monoamine oxidase inhibitors, serotonin agonists, serotonin antagonists, and serotonin reuptake inhibitors within 14 days of receiving ZX008

Primary outcomes:
measure: Change in Adverse Events and Serious Adverse Events leading to study withdrawal in subjects receiving open label ZX008
timeFrame: Up to 36 months open-label
measure: Changes in laboratory test results
timeFrame: Up to 36 months open-label
measure: Changes in heart rate
timeFrame: Up to 36 months open-label
measure: Changes in respiratory rate
timeFrame: Up to 36 months open-label
measure: Changes in blood pressure
timeFrame: Up to 36 months open-label
measure: Change in body weight
timeFrame: Up to 36 months open-label
measure: Changes in heart rhythm
timeFrame: Up to 36 months open-label
measure: Changes in heart valve function
timeFrame: Up to 36 months open-label
Secondary outcomes:
measure: Change in convulsive seizure response
timeFrame: Up to 36 months open-label
measure: Change in Cognitive CGI by Parent/Caregiver
timeFrame: Up to 36 months open-label
measure: Change in Behavioral CGI by Parent/Caregiver
timeFrame: Up to 36 months open-label
measure: Change in Motor CGI by Parent/Caregiver
timeFrame: Up to 36 months open-label
measure: Change in Cognitive CGI by Investigator
timeFrame: Up to 36 months open-label
measure: Change in Behavioral CGI by Investigator
timeFrame: Up to 36 months open-label
measure: Change in Motor CGI by Investigator
timeFrame: Up to 36 months open-label
measure: Change in Clinical Global Impression by Parent/Caregiver
timeFrame: Up to 36 months open-label
measure: Change in Clinical Global Impression by Investigator
timeFrame: Up to 36 months open-label
Other outcomes:
Locations:
city: Tucson
country: United States
facility: Ep0215 107
state: Arizona
city: Los Angeles
country: United States
facility: Ep0215 144
state: California
city: San Diego
country: United States
facility: Ep0215 108
state: California
city: San Francisco
country: United States
facility: Ep0215 101
state: California
city: Aurora
country: United States
facility: Ep0215 103
state: Colorado
city: Gulf Breeze
country: United States
facility: Ep0215 115
state: Florida
city: Miami
country: United States
facility: Ep0215 104
state: Florida
city: Orlando
country: United States
facility: Ep0215 141
state: Florida
city: Winter Park
country: United States
facility: Ep0215 121
state: Florida
city: Atlanta
country: United States
facility: Ep0215 117
state: Georgia
city: Chicago
country: United States
facility: Ep0215 110
state: Illinois
city: Bethesda
country: United States
facility: Ep0215 140
state: Maryland
city: Boston
country: United States
facility: Ep0215 112
state: Massachusetts
city: Rochester
country: United States
facility: Ep0215 109
state: Minnesota
city: Roseville
country: United States
facility: Ep0215 132
state: Minnesota
city: Hackensack
country: United States
facility: Ep0215 105
state: New Jersey
city: Livingston
country: United States
facility: Ep0215 118
state: New Jersey
city: Hawthorne
country: United States
facility: Ep0215 150
state: New York
city: New York
country: United States
facility: Ep0215 142
state: New York
city: Cleveland
country: United States
facility: Ep0215 131
state: Ohio
city: Memphis
country: United States
facility: Ep0215 124
state: Tennessee
city: Dallas
country: United States
facility: Ep0215 146
state: Texas
city: Fort Worth
country: United States
facility: Ep0215 126
state: Texas
city: Salt Lake City
country: United States
facility: Ep0215 106
state: Utah
city: Seattle
country: United States
facility: Ep0215 119
state: Washington
city: Tacoma
country: United States
facility: Ep0215 125
state: Washington
city: Heidelberg
country: Australia
facility: Ep0215 301
city: South Brisbane
country: Australia
facility: Ep0215 302
city: Westmead
country: Australia
facility: Ep0215 303
city: Bruxelles
country: Belgium
facility: Ep0215 803
city: Edegem
country: Belgium
facility: Ep0215 801
city: Jette
country: Belgium
facility: Ep0215 802
city: Montreal
country: Canada
facility: Ep0215 202
city: Toronto
country: Canada
facility: Ep0215 204
city: Vancouver
country: Canada
facility: Ep0215 201
city: Dianalund
country: Denmark
facility: Ep0215 701
city: Bordeaux Cedex
country: France
facility: Ep0215 1004
city: Lille
country: France
facility: Ep0215 1005
city: Marseille Cedex 05
country: France
facility: Ep0215 1007
city: Paris
country: France
facility: Ep0215 1001
city: Paris
country: France
facility: Ep0215 1002
city: Salouel
country: France
facility: Ep0215 1008
city: Bielefeld
country: Germany
facility: Ep0215 902
city: Freiburg Im Breisgau
country: Germany
facility: Ep0215 906
city: Jena
country: Germany
facility: Ep0215 905
city: Kiel
country: Germany
facility: Ep0215 908
city: Radeberg
country: Germany
facility: Ep0215 903
city: Vogtareuth
country: Germany
facility: Ep0215 901
city: Firenze
country: Italy
facility: Ep0215 1201
city: Genova
country: Italy
facility: Ep0215 1204
city: Mantova
country: Italy
facility: Ep0215 1205
city: Milano
country: Italy
facility: Ep0215 1207
city: Roma
country: Italy
facility: Ep0215 1206
city: Roma
country: Italy
facility: Ep0215 1208
city: Verona
country: Italy
facility: Ep0215 1202
city: Guadalajara
country: Mexico
facility: Ep0215 1604
city: Heeze
country: Netherlands
facility: Ep0215 1402
city: Zwolle
country: Netherlands
facility: Ep0215 1401
city: Bydgoszcz
country: Poland
facility: Ep0215 1702
city: Krakow
country: Poland
facility: Ep0215 1701
city: Barcelona
country: Spain
facility: Ep0215 1105
city: Barcelona
country: Spain
facility: Ep0215 1107
city: Esplugues de Llobregat
country: Spain
facility: Ep0215 1103
city: Madrid
country: Spain
facility: Ep0215 1101
city: Pamplona
country: Spain
facility: Ep0215 1102
city: Göteborg
country: Sweden
facility: Ep0215 502
city: Birmingham
country: United Kingdom
facility: Ep0215 605
city: Glasgow
country: United Kingdom
facility: Ep0215 601
city: Liverpool
country: United Kingdom
facility: Ep0215 603
city: London
country: United Kingdom
facility: Ep0215 602
city: London
country: United Kingdom
facility: Ep0215 606
Study officials:
affiliation: 001 844 599 2273
name: UCB Cares
role: STUDY_DIRECTOR
References: