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非在研适应症- |
最高研发阶段批准上市 |
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首次获批日期2020-06-25 |
Open-Label, Single-Arm, Phase 3 Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome
The primary purpose of this study is evaluate the safety and tolerability of fenfluramine hydrochloride (HCl) 0.2 to 0.8 mg/kg/day in infants 1 year to less than 2 years of age with Dravet syndrome.
A Phase II Study of Fenfluramine for Treatment of Refractory Infantile Spasms
This is a phase II clinical trial in which children with refractory infantile spasms (also called epileptic spasms or West syndrome) will be treated with fenfluramine, to evaluate efficacy, safety, and tolerability. Patients with infantile spasms that have not responded to treatment with vigabatrin and ACTH we will be invited to participate. Study participants will undergo baseline video-EEG, receive treatment with fenfluramine for 21 days, and then undergo repeat video-EEG to determine effectiveness. Patients with favorable response will have the opportunity to continue treatment for up to 6 months.
Fenfluramine for the Treatment of Refractory Epilepsy in Adult Dravet Patients
Full Title: Fenfluramine for the treatment of refractory Epilepsy in Adult Dravet patients
Short Title: Fenfluramine for Adult Dravet patients
Clinical Phase: Phase III
Sample Size: A total of 15 participants will be included in the study.
Study Population: Adult patients (18 years and older) with drug-resistant epilepsy (maintained on their existing medications, with exception of cannabidiol) and genetically confirmed Dravet syndrome will be recruited to participate in the study.
Accrual Period: 12 months Study Design: Open label, non-randomized and uncontrolled add-on trial in adults (18 years of age and older) residing in Ontario, with refractory motor seizures and maintained on their existing antiepileptic medications, with exception of cannabidiol.
Study Duration:
• Treatment period: 12 months Study duration: 28 months
Study Agent/ Intervention/ Procedure:
Name of study drug: fenfluramine (FINTEPLA)
Dose and frequency: starting at 0.1 mg/kg twice daily, maximum 26 mg/day, in patients not taking concomitant stiripentol; starting at 0.1 mg/kg twice daily, maximum of 17 mg/day in patients taking concomitant stiripentol. All doses are divided to twice a day.
Duration:
Baseline phase: 4 weeks (no study drug) Titration phase: 2 weeks (if not taking stiripentol) to 3 weeks (if the patient is taking stiripentol) Treatment phase: 12 weeks Extension phase: up to 38 weeks, for patients who had at least a 50% decrease in seizure frequency Post-trial washout phase: 2 weeks (if not taking stiripentol) to 3 weeks (if the patient is taking stiripentol)
Route of administration:
Oral
Efficacy and safety points of interest
Monthly convulsive seizure frequency (MCSF) reduction ≥ 50%
Improvement in motor function
Improvement in Cognition and Behavior
Improvement in Quality of Sleep
Improvement in Quality of life
Determination of Cardiovascular safety in adults
Responder analysis (≥25%, ≥75%, or 100% reduction in mean MCSF)
Longest period of seizure freedom
Number of Emergency room visits
Use of rescue medication (number of days in 28 day-periods)
Duration of post-ictal stage
Frequency of other seizure types
Body weight changes
Patient's global functioning prior to and after study (Clinical Global Impressions Scale)
Trial registration:
www.clinicaltrials.gov
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