An extension study to learn about the long-term safety of fenfluramine hydrochloride for seizures in children and adults with rare seizure disorders such as epileptic encephalopathies Including Dravet Syndrome and Lennox-Gastaut Syndrome

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Zogenix International Limited

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

7 Feb 2020 → 9 May 2025

Decision date (initial)

2024-11-05

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Zogenix International Limited, an indirectly wholly owned subsidiary of UCB Biosciences

External identifiers

EU CT number

2024-515680-61-00

EudraCT number

2019-001331-31

WHO UTN

U1111-1309-4338

ClinicalTrials.gov

NCT03936777

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To assess the long-term safety and tolerability of ZX008

Secondary objectives 1

1. 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

Conditions and MedDRA coding

Dravet syndrome or Lennox-Gastaut syndrome

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. • 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 1

1. • 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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 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)

Secondary endpoints 1

1. 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)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Zogenix International Limited

Sponsor organisation

Zogenix International Limited

Address

The Pearce Building, West Street West Street

City

Maidenhead

Postcode

SL6 1RL

Country

United Kingdom

Scientific contact point

Organisation

Zogenix International Limited

Contact name

UCB Cares

Public contact point

Organisation

Zogenix International Limited

Contact name

UCB Cares

Third parties 11

Sponsor responsibilities

Contact point sponsor

Zogenix International Limited

Locations

4 EU/EEA countries · 4 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 39 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications