Open label extension study for monitoring long-term safety in patients with Myoclonic Astatic Epilepsy (Doose-Syndrome) receiving Fenfluramine as add-on therapy

Phase 2

Recruiting

• Conditions: Myclonic-astatic epilepsy

• Registration Number: 2024-518520-77-00
• Lead Sponsor: Universitaetsklinikum Schleswig-Holstein AöR, Universitaetsklinikum Schleswig-Holstein AöR

Brief Summary

The aim of this study is to monitor the individual long-term safety and efficacy of low dose Fenfluramine (minimum 0.2 mg/kg/day BID up to a maximum of 0.8 mg/kg/day BID, max 30.0 mg/day) as add-on therapy. At the discretion of the investigator, an individual, if necessary repeated, gradual adaptation of the dose to the respective situation of the seizures is possible in this extension study.

Detailed Description

Not available

Study Timeline

• Study First Posted: 2024-10-24
• Last Update Posted: 2025-06-02

Recruitment & Eligibility

• Status: Authorised, recruiting
• Sex: Not specified
• Target Recruitment: 10

Inclusion Criteria

Subjects is currently enrolled in FFA-MAE-study

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

Subject is between 1 (12 months) and 17 years

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)

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

Subjects receives >= 1 AED in addition to Fenfluramine

Exclusion Criteria

Subject has a known hypersensitivity to Fenfluramine hydrochloride or other components in the study formulation

Weight loss of 10 percent or more compared to visit 2 (first intake of IMP) of the FFA-MAE study

Certain drugs, as listed in the prohibited food & medication section in the protocol

Intake of any investigational medicinal product (IMP) other than Fenfluramine

Any cardiovascular abnormality

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Efficacy Endpoint. 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)		Efficacy Endpoint. 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)
Safety: (Serious) Adverse Events; Laboratory measurements; Vital signs; Physical examination; 12-lead electrocardiogram (ECGs); Doppler echocardiogram (ECHOs); Body weight		Safety: (Serious) Adverse Events; Laboratory measurements; Vital signs; Physical examination; 12-lead electrocardiogram (ECGs); Doppler echocardiogram (ECHOs); Body weight

Trial Locations

Locations (3)

Schoen Klinik Vogtareuth SE & Co. KG; 🇩🇪 Vogtareuth, Germany

Krankenhaus Mara gGmbH; 🇩🇪 Bielefeld, Germany

Universitaetsklinikum Schleswig-Holstein AöR; 🇩🇪 Kiel, Germany

Schoen Klinik Vogtareuth SE & Co. KG

🇩🇪Vogtareuth, Germany

Milka Pringsheim

Site contact

00498038904064

mpringsheim@schoen-kliniken.de