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Anti-epileptic

Fenfluramine for Dravet Syndrome

Phase 4

Recruiting

Led By Kelly Knupp, MD

Research Sponsored by University of Colorado, Denver

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients must be between 12 and 23 months old to be eligible

Failure of at least one anti-seizure medication that is not a sodium channel blocker (lamotrigine, oxcarbazepine, carbamazepine, eslicarbazepine)

Must not have

Patients with mild or greater mitral valve regurgitation and/or trace or greater aortic valve regurgitation will not be eligible for participation

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months

Awards & highlights

Drug Has Already Been Approved

Pivotal Trial

No Placebo-Only Group

Summary

"This trial focuses on Dravet syndrome, a genetic epilepsy that causes prolonged seizures in children usually within the first year of life. Many anti-seizure medications worsen seizures in these patients because they

Who is the study for?

This trial is for infants and toddlers under 24 months old with Dravet Syndrome, a severe form of epilepsy. To join, they must have the genetic markers associated with this condition and cannot be effectively treated with common anti-seizure medications that block sodium channels.

What is being tested?

The trial tests fenfluramine's safety in young children with Dravet Syndrome. Fenfluramine has shown promise in older children by significantly reducing seizures. This study will explore if similar benefits occur in those under two years old.

What are the potential side effects?

While not specified here, fenfluramine may cause side effects such as heart-related issues, decreased appetite, drowsiness, fatigue, diarrhea, and potential interactions with other seizure medications.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My child is between 12 and 23 months old.

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I have tried an anti-seizure medication other than lamotrigine, oxcarbazepine, carbamazepine, or eslicarbazepine without success.

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I have a SCN1A mutation linked to long seizures or Dravet syndrome.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have significant heart valve issues.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Percentage of participants that have at least one adverse event

Awards & Highlights

Drug Has Already Been Approved

The FDA has already approved this drug, and is just seeking more data.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: FenfluramineExperimental Treatment1 Intervention

The dose of FENFLURAMINE for the duration of the Treatment Period will range from 0.2 mg/kg/day to a maximum of 0.7 mg/kg/day, not to exceed a total daily dose of 26 mg/day; for patients concurrently being prescribed stiripentol, the maximum will be 0.4 mg/kg/day, not to exceed a total daily dose of 17 mg/day. Under this treatment plan, FENFLURAMINE will be administered twice daily in equally divided doses, with at least 8 hours and no more than 12 hours between doses in a single day.

0 / 4Eligibility criteria met