Health TrialsbyHealthPanel

Treatment of Refractory Infantile Spasms With Fenfluramine

ConditionInfantile Spasm
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age12-36
SponsorChildren's Hospital of Orange County

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Children ages 12 to 36 months, inclusive

Clinical diagnosis of infantile spasms

Continued epileptic spasms despite adequate treatment with ACTH and vigabatrin.

Disqualifiers

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

Trial design

Design model

Single group

Treatments tested in this trial

  • Fenfluramine

    Drug

    Open-label

Treatment groups

10 Participants
are divided into 1 treatment group
Group A: Fenfluramine treatmentExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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.

Time frame
12 months

Secondary outcomes

1

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.

Time frame
12 months
2

Incidence of treatment emergent adverse events (Safety and tolerability)

Detailed accounting of all treatment-emergent adverse events, including number of participants with clinically-significant valvulopathy and/or pulmonary hypertension, as determined by echocardiography.

Time frame
12 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Children's Hospital of Orange County

Lead sponsor

Source ClinicalTrials.gov
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