Fenfluramine for the Treatment of Different Types of Developmental and Epileptic Encephalopathies: a Pilot Trial Exploring Epileptic and Non-epileptic Outcomes
Summary
This study is a pilot non-controlled clinical trial with adjunctive fenfluramine for the treatment of five different types of developmental and epileptic encephalopathies (DEEs) focused on epileptic and "non-epileptic outcomes": SYNGAP1 and STXBP1 encephalopathies, inv-dup(15) encephalopathy, multifocal or bilateral malformations of cortical development, and continuous spikes and waves during sleep. The main goal is to assess changes in seizure frequency comparing before and after treatment with fenfluramine in five specific types of developmental and epileptic encephalopathies (DEEs). Secondary objectives of this study are the analysis of changes in seizure intensity and duration, and "non-epileptic outcomes" such as variations in cognitive activity, level of alertness, impulsivity/self-control, gait stability and other alterations that might be detected during the interview and physical examination.
Timeline
- Start
- 2022-10-20
- Primary completion
- 2025-02-20
- Completion
- 2025-06-20
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Fenfluramine | Small molecule | 0.2 mg/kg | — |
| Subject | Fenfluramine | Small molecule | 0.4 mg/kg | — |
| Subject | Fenfluramine | Small molecule | 0.5 mg/kg | — |
| Subject | Fenfluramine | Small molecule | 0.6 mg/kg | — |
| Subject | Fenfluramine | Small molecule | 0.7 mg/kg | — |