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

Phase 3

Completed

• Conditions: seizures associated with Dravet syndrome and Lennox-Gastaut syndrome.; Dravet syndrome and Lennox-Gastaut syndrome; seizures

• Registration Number: NL-OMON54815
• Lead Sponsor: Zogenix International Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 16

Inclusion Criteria

1. Subject is currently enrolled in core Study ZX008-1503
ORSubject has successfully completed core Study ZX008-1601-Part 2
OR
Subject with a rare seizure disorder, such as epileptic encephalopathy, that
has successfully completed another Zogenix-sponsored clinical trial with ZX008,
and has been invited to participate in this study by the Sponsor.
2. Subjects must, in the medical opinion of the Investigator, be candidates for
continued treatment for an extended period of time with ZX008 (ie, subject has
demonstrated a clinically meaningful benefit with ZX008 in the prior trial, and
benefits of continued treatment outweigh potential risks).
3. Subject is male or a nonpregnant, nonlactating female. Female subjects of
childbearing potential must not be pregnant or breast-feeding. Female subjects
of childbearing potential must have a negative pregnancy test prior to study
entry. Subjects of childbearing or child-fathering potential must be willing to
use medically acceptable forms of birth control, which includes abstinence,
while being treated on this study and for 90 days after the last dose of study
drug.
4. Subject has been informed of the nature of the study and informed consent
has been obtained from the legally responsible parent/guardian.
5. Subject has provided assent in accordance with Institutional Review Board
(IRB)/Independent Ethics Committee (IEC) requirements, if capable.
6. Subject*s caregiver is willing and able to be compliant with study
procedures, visit schedule and study drug accountability.

Exclusion Criteria

1. Subject has a known hypersensitivity to fenfluramine or any of the
excipients in the study medication. 2. Subject has current cardiac valvulopathy
or pulmonary hypertension that the investigator, ICAB, IDSMC, or Sponsor deems
reason for exclusion. 3. Subject is at imminent risk of self-harm or harm to
others, in the investigator*s opinion, based on clinical interview and
responses provided on the Columbia-Suicide Severity Rating Scale (C-SSRS).
Subjects must be excluded if they report suicidal behavior as measured by the C
SSRS Since Last Visit, which includes suicidal ideation with intent and plan
(Item #5). If a subject reports suicidal ideation on Item 4 without specific
plan, and the investigator feels that the subject is appropriate for the study
considering the potential risks, the investigator must document appropriateness
for inclusion, and discuss with the parent/caregiver to be alert to mood or
behavioral changes, especially around times of dose adjustment. 4. Subject has
moderate or severe hepatic impairment. Asymptomatic subjects with mild hepatic
impairment (elevated liver enzymes < 3x upper limit of normal [ULN] and/or
elevated bilirubin < 2x ULN) may be entered into the study after review and
approval by the Medical Monitor in conjunction with the Sponsor, in
consideration of comorbidities and concomitant medications. 5. Administration
of monoamine oxidase inhibitors, serotonin agonists, serotonin antagonists, and
serotonin reuptake inhibitors within 14 days of receiving ZX008. 6. Subject is
unwilling or unable to comply with scheduled visits, drug administration plan,
laboratory tests, other study procedures, and study restrictions. 7. Subject
has a clinically significant condition, or has had clinically relevant symptoms
or a clinically significant illness at Visit 1, other than epilepsy, that would
negatively impact study participation, collection of study data, or pose a risk
to the subject, including chronic obstructive pulmonary disease, interstitial
lung disease, or portal hypertension. 8. Subject has participated in another
clinical treatment trial within the past 30 days (ie, the last visit of the
previous study was in the past 30 days), with the exception of a ZX008 clinical
study.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The primary objective of the study is:<br /><br>• To assess the long-term safety and tolerability of ZX008</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>The secondary objectives of the study are: • To assess the effect of ZX008 on<br /><br>the following effectiveness measures: - Investigator assessment of convulsive<br /><br>seizure response (<25%, >=25%, >=50%, >=75%, or 100% [ie, seizure-free]<br /><br>improvement) - Clinical Global Impression - Improvement (CGI-I) rating, as<br /><br>assessed by the investigator - CGI-I rating, as assessed by the<br /><br>parent/caregiver - Symptomatic CGI-I for cognition, behavior, motor abilities,<br /><br>as assessed by the investigator - Symptomatic CGI-I for cognition, behavior,<br /><br>motor abilities, as assessed by the parent/caregiver</p><br>