A long-term investigation to oversee safety of patients with Doose syndrome, receiving Fenfluramine as additional medication.

Phase 1

• Conditions: Childhood epilepsy: Myoclonic Astatic Epilepsy (Doose-Syndrome); MedDRA version: 21.1Level: LLTClassification code 10081183Term: Myoclonic-astatic epilepsySystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2020-002238-34-DE
• Lead Sponsor: niversity Hospital Schleswig-Holstein (UKSH)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-12-04
• Last Update Posted: 8 October 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

1. Subjects is currently enrolled in FFA-MAE-study.
2. Subject and parents/caregiver have been informed of the nature of the study and written
informed consent has been obtained from the patient and the legally responsible
parents/caregiver.
3. Subject is between 1 (12 months) and 17 years.
4. In the medical opinion of the Investigator, subject must be a candidate for continued treatment
for an extended period of time with Fenfluramine (i.e. subject has demonstrated a clinically meaningful benefit with Fenfluramine in the prior trial (FFA-MAE), and benefits of continued treatment outweigh potential risks).
5. Clinically meaningful benefit is defined as follows: at least 50% reduction of total number of seizures (sum of GTKA, TS, AS, AB, MS) compared to baseline in FFA-MAE-study.
6. Subjects receives >= 1 AED in addition to Fenfluramine.
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1) Subject has a known hypersensitivity to Fenfluramine hydrochloride or other components in
the study formulation
2) Weight loss of 10 percent or more compared to visit 2 (first intake of IMP) of the FFA-MAE
study.
3) Certain drugs, as listed in the prohibited food & medication section in the protocol.
4) Intake of any investigational medicinal product (IMP) other than Fenfluramine.

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Main Objective: The aim of this study is to monitor the individual long-term safety and efficacy of low dose<br>Fenfluramine (0.4 or 0.8 mg/kg/day, max 30.0 mg/day) as add-on therapy.;Secondary Objective: Not applicable;Primary end point(s): Efficacy Endpoint:<br>- Change of individual (per subject) number and frequency of countable seizures compared to the baseline visit of the previous FFA-MAE study (before onset of Fenfluramine treatment).<br><br>Safety Endpoints:<br>- (Serious) Adverse Events<br>- Laboratory measurements<br>- Vital signs<br>- Physical examination<br>- 12-lead electrocardiogram (ECG)<br>- Doppler echocardiogram (ECHO)<br>- Body weight;Timepoint(s) of evaluation of this end point: During the course of the trial and at the end of the trial

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Not applicable;Timepoint(s) of evaluation of this end point: Not applicable