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

Phase 1

• Conditions: Dravet syndrome or Lennox-Gastaut syndrome; MedDRA version: 20.1Level: PTClassification code 10048816Term: Lennox-Gastaut syndromeSystem Organ Class: 10029205 - Nervous system disorders; MedDRA version: 20.0Level: LLTClassification code 10073682Term: Dravet syndromeSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2019-001331-31-SE
• Lead Sponsor: Zogenix International Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-01-08
• Last Update Posted: 18 March 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 650

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
Are the trial subjects under 18? yes
Number of subjects for this age range: 445
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 205
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

• Current cardiac valvulopathy or pulmonary hypertension that is clinically significant
• Moderate or severe hepatic impairment
• Receiving prohibited medication (please see protocol section 5.6.2), within 14 days of receiving ZX008

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the long-term safety and tolerability of ZX008;Secondary Objective: To assess the effect of ZX008 on the following effectiveness measures:<br>- Investigator assessment of convulsive seizure response (<25%, =25%, =50%, =75%, or 100% [ie, seizure-free] improvement)<br>- Clinical Global Impression – Improvement (CGI-I) rating, global and symptomatic, as assessed by the investigator<br>- CGI-I rating, global and symptomatic, as assessed by the parent/caregiver;Primary end point(s): The safety endpoints of the study are:<br>• AEs<br>• Laboratory safety (hematology, chemistry)<br>• Vital signs (blood pressure, heart rate, temperature, and respiratory rate)<br>• Physical examination<br>• Neurological examination<br>• Electrocardiogram (ECGs)<br>• Doppler echocardiogram (ECHOs)<br>• Body weight/height<br>• Chest x-ray (subjects in France and Netherlands only)<br>• Electroencephalogram (EEG) (in Italy only);Timepoint(s) of evaluation of this end point: Up to 36 months

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): The effectiveness endpoints of the study are:<br>- CGI-I, global and symptomatic, as assessed by parent/caregiver<br>- CGI-I, global and symptomatic, as assessed by investigator (or designee)<br>- Percent improvement in seizure burden as assessed by the investigator (or designee);Timepoint(s) of evaluation of this end point: Up to 36 months