Fenfluramine hydrochloride for Myoclonic-astatic epilepsy (Doose syndrome)

Inclusion criteria

• {"criterion_text":"- Subjects is currently enrolled in FFA-MAE-study"}
• {"criterion_text":"- 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"}
• {"criterion_text":"- Subject is between 1 (12 months) and 17 years"}
• {"criterion_text":"- 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)"}
• {"criterion_text":"- 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"}
• {"criterion_text":"- Subjects receives >= 1 AED in addition to Fenfluramine"}

Exclusion criteria

• {"criterion_text":"- Subject has a known hypersensitivity to Fenfluramine hydrochloride or other components in the study formulation"}
• {"criterion_text":"- Weight loss of 10 percent or more compared to visit 2 (first intake of IMP) of the FFA-MAE study"}
• {"criterion_text":"- Certain drugs, as listed in the prohibited food & medication section in the protocol"}
• {"criterion_text":"- Intake of any investigational medicinal product (IMP) other than Fenfluramine"}
• {"criterion_text":"- Any cardiovascular abnormality"}

Primary endpoints

• {"endpoint_text":"- 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)","definition_or_measurement_approach":"Change per subject in number and frequency of countable seizures compared to the baseline visit of the previous FFA-MAE study (before onset of Fenfluramine treatment); seizure counts and frequency measured relative to prior-study baseline."}
• {"endpoint_text":"- Safety: (Serious) Adverse Events; Laboratory measurements; Vital signs; Physical examination; 12-lead electrocardiogram (ECGs); Doppler echocardiogram (ECHOs); Body weight","definition_or_measurement_approach":"Monitoring and recording of (serious) adverse events, laboratory measurements, vital signs, physical examinations, 12-lead ECGs, Doppler echocardiograms (ECHOs), and body weight."}

Germany

Earliest CTIS Part Ii Submission Date

10-10-2024

Latest Decision Or Authorization Date

02-06-2025

Processing Time Days

235

Number Of Sites

3

Number Of Participants

10

Primary sponsor

Full Name

Universitaetsklinikum Schleswig-Holstein AöR

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Germany

Third parties

• {"country":"Belgium","full_name":"UCB Biopharma SRL, Belgium","duties_or_roles":"Source of monetary support","organisation_type":""}