Open label extension study for monitoring long-term safety in patients with Myoclonic Astatic Epilepsy (Doose-Syndrome) receiving Fenfluramine as add-on therapy
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 10
- 试验地点
- 3
- 主要终点
- 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)
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
结局指标
主要结局
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
次要结局
未报告次要终点
研究者
Department of Children's and Adolescence Medicine II
Scientific
Universitaetsklinikum Schleswig-Holstein AöR