A Phase II Study of Fenfluramine for Treatment of Refractory Infantile Spasms
Trial Snapshot
- Phase
- Phase 2
- Status
- Recruiting
- Enrollment
- 10
- Locations
- 3
- Primary Endpoint
- Electroclinical response (Efficacy)
Study Overview
Brief Summary
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 12 Months to 36 Months (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Children ages 12 to 36 months, inclusive
- •Clinical diagnosis of infantile spasms
- •Continued epileptic spasms despite adequate treatment with ACTH and vigabatrin.
Exclusion Criteria
- •Significant preexisting cardiovascular disease
- •Exposure to any cannabinoid product within 14 days of screening
- •Initiation or dose-titration of any second-line treatment for infantile spasms in the 14 days prior to screening.
- •Implantation of a vagal nerve simulator within 14 days of screening
- •Initiation and maintenance of the ketogenic diet within 3 months of screening
Arms & Interventions
Fenfluramine treatment
Open label treatment with fenfluramine. Dosage will be titrated to 0.8 mg/kg/day, for an initial duration of 21 days. Patients with favorable response will have an option to continue treatment for up to 6 months.
Intervention: Fenfluramine (Drug)
Outcomes
Primary Outcomes
Electroclinical response (Efficacy)
Time Frame: 12 months
Number of participants with resolution of epileptic spasms and hypsarrhythmia (if present at baseline) after 21 days of treatment, as determined by overnight video-electroencephalography (EEG) evaluation and caregiver seizure diary.
Secondary Outcomes
- Incidence of treatment emergent adverse events (Safety and tolerability)(12 months)
- Computational electroencephalography response (Efficacy)(12 months)
Investigators
Daniel Shrey
Associate Professor
Children's Hospital of Orange County