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

• Register: ClinicalTrials.gov
• Last refreshed on: 24 February 2025
• Main ID: NCT03936777
• Date of registration: 12/04/2019
• Prospective Registration: Yes
• Primary sponsor: Zogenix, Inc.
• Public title: 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
• Scientific 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
• Date of first enrolment: April 22, 2019
• Target sample size: 412
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03936777
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

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

Contacts

• Name: UCB Cares
• Affiliation: 001 844 599 2273

Key inclusion & exclusion 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


Age minimum: 2 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Dravet Syndrome

Epileptic Encephalopathy

Lennox Gastaut Syndrome

Intervention(s)

Drug: ZX008 (Fenfluramine Hydrochloride)

Primary Outcome(s)

Change in Adverse Events and Serious Adverse Events leading to study withdrawal in subjects receiving open label ZX008 [Time Frame: Up to 36 months open-label]

Changes in heart rate [Time Frame: Up to 36 months open-label]

Change in body weight [Time Frame: Up to 36 months open-label]

Changes in heart valve function [Time Frame: Up to 36 months open-label]

Changes in blood pressure [Time Frame: Up to 36 months open-label]

Changes in heart rhythm [Time Frame: Up to 36 months open-label]

Changes in laboratory test results [Time Frame: Up to 36 months open-label]

Changes in respiratory rate [Time Frame: Up to 36 months open-label]

Secondary Outcome(s)

Change in Motor CGI by Parent/Caregiver [Time Frame: Up to 36 months open-label]

Change in Clinical Global Impression by Investigator [Time Frame: Up to 36 months open-label]

Change in Clinical Global Impression by Parent/Caregiver [Time Frame: Up to 36 months open-label]

Change in Cognitive CGI by Parent/Caregiver [Time Frame: Up to 36 months open-label]

Change in Behavioral CGI by Investigator [Time Frame: Up to 36 months open-label]

Change in Behavioral CGI by Parent/Caregiver [Time Frame: Up to 36 months open-label]

Change in Motor CGI by Investigator [Time Frame: Up to 36 months open-label]

Change in Cognitive CGI by Investigator [Time Frame: Up to 36 months open-label]

Change in convulsive seizure response [Time Frame: Up to 36 months open-label]

Secondary ID(s)

2024-515680-61

U1111-1309-4338

2019-001331-31

ZX008-1900

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.