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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Fenfluramine treatment | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Fenfluramine | Drug | Open-label |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Electroclinical response (Efficacy) | 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. | 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Computational electroencephalography response (Efficacy) | Median and range of response quantified using the probability-weighted response index (PWRI), a novel computational electroencephalography measure of hypsarrhythmia burden. | 12 months |
| Incidence of treatment emergent adverse events (Safety and tolerability) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Kellie Bacon, MPH | Contact | 714-509-8972 | kellie.bacon@choc.org | |
| Virginia Allhusen, PhD | Contact | vallhusen@choc.org |
| Name | Affiliation | Role |
|---|---|---|
| Daniel Shrey, MD | Children's Hospital of Orange County | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCLA Health | Not yet recruiting | Los Angeles | California | 90095 | United States |
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| ID | Term |
|---|---|
| D013036 | Spasms, Infantile |
| ID | Term |
|---|---|
| D004829 | Epilepsy, Generalized |
| D004827 | Epilepsy |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| ID | Term |
|---|---|
| D005277 | Fenfluramine |
| ID | Term |
|---|---|
| D010627 | Phenethylamines |
| D005021 | Ethylamines |
| D000588 | Amines |
| D009930 | Organic Chemicals |
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Detailed accounting of all treatment-emergent adverse events, including number of participants with clinically-significant valvulopathy and/or pulmonary hypertension, as determined by echocardiography. |
| 12 months |
| Children's Hospital of Orange County | Recruiting | Orange | California | 92868 | United States |
|
| D009422 |
| Nervous System Diseases |
| D000073376 | Epileptic Syndromes |