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

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

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2020-002238-34-DE

Enrollment

Registered

2020-12-04

Start date

2021-03-22

Completion date

Unknown

Last updated

2024-10-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Childhood epilepsy: Myoclonic Astatic Epilepsy (Doose-Syndrome) MedDRA version: 21.1 Level: LLT Classification code 10081183 Term: Myoclonic-astatic epilepsy System Organ Class: 100000004850

Interventions

Product Name: Fenfluramine hydrochloride Pharmaceutical Form: Oral solution INN or Proposed INN: FENFLURAMINE HYDROCHLORIDE CAS Number: 404-82-0 Current Sponsor code: FFA-MAE extension study Concentra

Eligibility

Sex/Gender

All

Inclusion criteria

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

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.

Design outcomes

Primary

Measure	Time frame
Main Objective: The aim of this study is to monitor the individual long-term safety and efficacy of low dose Fenfluramine (0.4 or 0.8 mg/kg/day, max 30.0 mg/day) as add-on therapy.;Secondary Objective: Not applicable;Timepoint(s) of evaluation of this end point: During the course of the trial and at the end of the trial;Primary end point(s): 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 Endpoints: - (Serious) Adverse Events - Laboratory measurements - Vital signs - Physical examination - 12-lead electrocardiogram (ECG) - Doppler echocardiogram (ECHO) - Body weight	—

Measure

Time frame

Main Objective: The aim of this study is to monitor the individual long-term safety and efficacy of low dose Fenfluramine (0.4 or 0.8 mg/kg/day, max 30.0 mg/day) as add-on therapy.;Secondary Objective: Not applicable;Timepoint(s) of evaluation of this end point: During the course of the trial and at the end of the trial;Primary end point(s): 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 Endpoints: - (Serious) Adverse Events - Laboratory measurements - Vital signs - Physical examination - 12-lead electrocardiogram (ECG) - Doppler echocardiogram (ECHO) - Body weight

—

Secondary

Measure	Time frame
Secondary end point(s): Not applicable;Timepoint(s) of evaluation of this end point: Not applicable	—

Countries

Germany

Contacts

Public ContactDepartment of Children and adolesce

UKSH

irene.lehmann@uksh.de+4943150024140

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026