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: EUCTR
• Last refreshed on: 10 January 2022
• Main ID: EUCTR2019-001331-31-DE
• Date of registration: 13/11/2019
• Prospective Registration: Yes
• Primary sponsor: Zogenix International Limited
• 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: 07/04/2020
• Target sample size: 650
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2019-001331-31
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

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

Contacts

• Name: Kunal Kanani
• Affiliation: Zogenix, Inc.

• Name: Kunal Kanani
• Affiliation: Zogenix, Inc.

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
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


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Dravet syndrome or Lennox-Gastaut syndrome
MedDRA version: 20.1 Level: PT Classification code 10048816 Term: Lennox-Gastaut syndrome System Organ Class: 10029205 - Nervous system disorders
MedDRA version: 20.0 Level: LLT Classification code 10073682 Term: Dravet syndrome System Organ Class: 100000004850

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Intervention(s)

Product Name: Fenfluramine hydrochloride (colorless)
Product Code: ZX008
Pharmaceutical Form: Oral solution
INN or Proposed INN: FENFLURAMINE HYDROCHLORIDE
CAS Number: 404-82-0
Current Sponsor code: ZX008
Other descriptive name: FENFLURAMINE HYDROCHLORIDE
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 2.5-

Primary Outcome(s)

Main Objective: To assess the long-term safety and tolerability of ZX008

Primary end point(s): The safety endpoints of the study are:
• AEs
• Laboratory safety (hematology, chemistry)
• Vital signs (blood pressure, heart rate, temperature, and respiratory rate)
• Physical examination
• Neurological examination
• Electrocardiogram (ECGs)
• Doppler echocardiogram (ECHOs)
• Body weight/height
• Chest x-ray (subjects in France and Netherlands only)
• Electroencephalogram (EEG) (in Italy only)

Timepoint(s) of evaluation of this end point: Up to 36 months

Secondary Objective: To assess the effect of ZX008 on the following effectiveness measures:
- Investigator assessment of convulsive seizure response (<25%, =25%, =50%, =75%, or 100% [ie, seizure-free] improvement)
- Clinical Global Impression – Improvement (CGI-I) rating, global and symptomatic, as assessed by the investigator
- CGI-I rating, global and symptomatic, as assessed by the parent/caregiver

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Up to 36 months

Secondary end point(s): The effectiveness endpoints of the study are:
• CGI-I, global and symptomatic, as assessed by parent/caregiver
• CGI-I, global and symptomatic, as assessed by investigator (or designee)
• Percent improvement in seizure burden as assessed by the investigator (or designee)

Secondary ID(s)

NCT03936777

125797; 132604

2019-001331-31-GB

ZX008-1900

Source(s) of Monetary Support

Zogenix, Inc

Ethics review

Status: Approved
Approval date: 07/04/2020
Contact: