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

Dravet Syndrome Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03936777
• Other study ID #: ZX008-1900
• Secondary ID: 2019-001331-31
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: April 22, 2019
• Est. completion date: June 28, 2024

Study information

• Verified date: May 2024
• Source: UCB Pharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 412
• Est. completion date: June 28, 2024
• Est. primary completion date: June 28, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Brain Diseases
• Dravet Syndrome
• Epilepsies, Myoclonic
• Epilepsy
• Epileptic Encephalopathy
• Lennox Gastaut Syndrome
• Syndrome

Intervention

Drug:
• ZX008 (Fenfluramine Hydrochloride)
Fenfluramine hydrochloride provided in a concentration of 2.5 mg/mL.

Locations

Country	Name	City	State
Australia	Ep0215 301	Heidelberg
Australia	Ep0215 302	South Brisbane
Australia	Ep0215 303	Westmead
Belgium	Ep0215 803	Bruxelles
Belgium	Ep0215 801	Edegem
Belgium	Ep0215 802	Jette
Canada	Ep0215 202	Montreal
Canada	Ep0215 204	Toronto
Canada	Ep0215 201	Vancouver
Denmark	Ep0215 701	Dianalund
France	Ep0215 1004	Bordeaux Cedex
France	Ep0215 1005	Lille
France	Ep0215 1007	Marseille Cedex 05
France	Ep0215 1001	Paris
France	Ep0215 1002	Paris
France	Ep0215 1008	Salouel
Germany	Ep0215 902	Bielefeld
Germany	Ep0215 906	Freiburg Im Breisgau
Germany	Ep0215 905	Jena
Germany	Ep0215 908	Kiel
Germany	Ep0215 903	Radeberg
Germany	Ep0215 901	Vogtareuth
Italy	Ep0215 1201	Firenze
Italy	Ep0215 1204	Genova
Italy	Ep0215 1205	Mantova
Italy	Ep0215 1207	Milano
Italy	Ep0215 1206	Roma
Italy	Ep0215 1208	Roma
Italy	Ep0215 1202	Verona
Mexico	Ep0215 1604	Guadalajara
Netherlands	Ep0215 1402	Heeze
Netherlands	Ep0215 1401	Zwolle
Poland	Ep0215 1702	Bydgoszcz
Poland	Ep0215 1701	Krakow
Spain	Ep0215 1105	Barcelona
Spain	Ep0215 1107	Barcelona
Spain	Ep0215 1103	Esplugues de Llobregat
Spain	Ep0215 1101	Madrid
Spain	Ep0215 1102	Pamplona
Sweden	Ep0215 502	Göteborg
United Kingdom	Ep0215 605	Birmingham
United Kingdom	Ep0215 601	Glasgow
United Kingdom	Ep0215 603	Liverpool
United Kingdom	Ep0215 602	London
United Kingdom	Ep0215 606	London
United States	Ep0215 117	Atlanta	Georgia
United States	Ep0215 103	Aurora	Colorado
United States	Ep0215 140	Bethesda	Maryland
United States	Ep0215 112	Boston	Massachusetts
United States	Ep0215 110	Chicago	Illinois
United States	Ep0215 131	Cleveland	Ohio
United States	Ep0215 146	Dallas	Texas
United States	Ep0215 126	Fort Worth	Texas
United States	Ep0215 115	Gulf Breeze	Florida
United States	Ep0215 105	Hackensack	New Jersey
United States	Ep0215 150	Hawthorne	New York
United States	Ep0215 118	Livingston	New Jersey
United States	Ep0215 144	Los Angeles	California
United States	Ep0215 124	Memphis	Tennessee
United States	Ep0215 104	Miami	Florida
United States	Ep0215 142	New York	New York
United States	Ep0215 141	Orlando	Florida
United States	Ep0215 109	Rochester	Minnesota
United States	Ep0215 132	Roseville	Minnesota
United States	Ep0215 106	Salt Lake City	Utah
United States	Ep0215 108	San Diego	California
United States	Ep0215 101	San Francisco	California
United States	Ep0215 119	Seattle	Washington
United States	Ep0215 125	Tacoma	Washington
United States	Ep0215 107	Tucson	Arizona
United States	Ep0215 121	Winter Park	Florida

Sponsors (2)

Lead Sponsor	Collaborator
Zogenix, Inc.	Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.

Countries where clinical trial is conducted

United States,  Australia,  Belgium,  Canada,  Denmark,  France,  Germany,  Italy,  Mexico,  Netherlands,  Poland,  Spain,  Sweden,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Adverse Events and Serious Adverse Events leading to study withdrawal in subjects receiving open label ZX008	Evaluate the long term safety and tolerability of oral dose administration of ZX008	Up to 36 months open-label
Primary	Changes in laboratory test results	The analysis will include abnormal hematology and chemistry laboratory results outside of reference ranges	Up to 36 months open-label
Primary	Changes in heart rate	The analysis will include change in heart rate using standard measure	Up to 36 months open-label
Primary	Changes in respiratory rate	The analysis will include change in resting respiratory rate using standard measure	Up to 36 months open-label
Primary	Changes in blood pressure	The analysis will include change in resting blood pressure using standard measure	Up to 36 months open-label
Primary	Change in body weight	The analysis will include change in body weight and height by report of BMI in kg/m^2	Up to 36 months open-label
Primary	Changes in heart rhythm	The analysis will include changes in heart beat as measured with 12-lead electrocardiogram	Up to 36 months open-label
Primary	Changes in heart valve function	The analysis will include change in heart valves as measured with standard echocardiogram	Up to 36 months open-label
Secondary	Change in convulsive seizure response	The analysis will include percent improvement per investigator rating	Up to 36 months open-label
Secondary	Change in Cognitive CGI by Parent/Caregiver	The rating by parent/caregiver of the patient's change in cognitive symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".	Up to 36 months open-label
Secondary	Change in Behavioral CGI by Parent/Caregiver	The rating by parent/caregiver of the patient's change in behavioral symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".	Up to 36 months open-label
Secondary	Change in Motor CGI by Parent/Caregiver	The rating by parent/caregiver of the patient's change in motor symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".	Up to 36 months open-label
Secondary	Change in Cognitive CGI by Investigator	The rating by investigator of the patient's change in cognitive symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".	Up to 36 months open-label
Secondary	Change in Behavioral CGI by Investigator	The rating by investigator of the patient's change in behavioral symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".	Up to 36 months open-label
Secondary	Change in Motor CGI by Investigator	The rating by investigator of the patient's change in motor symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".	Up to 36 months open-label
Secondary	Change in Clinical Global Impression by Parent/Caregiver	The rating by parent/caregiver of the patient's overall change in symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse"	Up to 36 months open-label
Secondary	Change in Clinical Global Impression by Investigator	The rating by investigator of the patient's overall change in symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse"	Up to 36 months open-label