Infantile Spasm
Conditions
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.
Interventions
DRUGFenfluramine
Open-label
Sponsors
Children's Hospital of Orange County
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Months to 36 Months
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Electroclinical response (Efficacy) | 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
| Measure | Time frame | Description |
|---|---|---|
| Computational electroencephalography response (Efficacy) | 12 months | Median and range of response quantified using the probability-weighted response index (PWRI), a novel computational electroencephalography measure of hypsarrhythmia burden. |
| Incidence of treatment emergent adverse events (Safety and tolerability) | 12 months | Detailed accounting of all treatment-emergent adverse events, including number of participants with clinically-significant valvulopathy and/or pulmonary hypertension, as determined by echocardiography. |
Countries
United States
Contacts
CONTACTKellie Bacon, MPH
CONTACTVirginia Allhusen, PhD
PRINCIPAL_INVESTIGATORDaniel Shrey, MD
Children's Hospital of Orange County
Outcome results
None listed