Assessment of Safety of the Use of Fenfluramine in Children With Dravet Syndrome Under 24 Months of Age

NCT06598449 | Recruiting Phase 4 FDA Drug

• 1 other identifier: 21-3972
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 3

Brief Summary

Dravet syndrome is a genetic epilepsy associated with pathogenic variants in SCN1A that codes for Nav1.1, a protein necessary for sodium channels. Children with Dravet syndrome classically present in the first year of life with prolonged seizures, often hemiclonic and in the setting of fever or temperature changes such as getting in or out of bath water. Many anti-seizure medications are sodium channel blockers and exacerbate seizures in this patient population. This creates some limitations in medication choices for this patient population. Recently fenfluramine was approved for use in Dravet syndrome for people 2 years and older. Randomized studies demonstrated a 74.9% reduction of convulsive motor seizures compared to 19.2% in the placebo group. Additionally, 16% of children treated with fenfluramine were seizure free. Fenfluramine is likely to be as effective in children under the age of 2 years. The current study has proposed a treatment protocol to allow access to fenfluramine for children under 24 months of age.

Conditions

Dravet Syndrome (DS); Children Under 2 Years

Keywords

Fenfluramine; Dravet Syndrome; Children under 2 years

Outcome Measures

Primary Outcomes (1)

• Percentage of participants that have at least one adverse event

  A description of the percentage of participants that have at least one adverse event.

  12 months

Study Arms (1)

Fenfluramine

EXPERIMENTAL

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.

Drug: fenfluramine

Interventions

fenfluramine DRUG

Clear, cherry flavored oral solution

Fenfluramine

Eligibility Criteria

• Age: 12 Months - 24 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• SCN1A with a known or presumed pathogenic variant or VUS with a history of prolonged seizure or a clinical diagnosis of Dravet syndrome.
• Failure of at least one anti-seizure medication that is not a sodium channel blocker (lamotrigine, oxcarbazepine, carbamazepine, eslicarbazepine)

You may not qualify if:

• Patients with mild or greater mitral valve regurgitation and/or trace or greater aortic valve regurgitation will not be eligible for participation. The clinical statement can be submitted first for initial, conditional approval and then ECHO results can be submitted at a later date for final approval.
• Patients with failure to thrive will not be eligible for participation as fenfluramine can suppress appetite and has a risk for weight loss. Failure to thrive will be evaluated on the following criteria:
• Weight less than the 2nd percentile.
• Lack of weight gain that crosses two or more of the major percentile lines and is not congruent with length.

Sponsors & Collaborators

• University of Colorado, Denver: lead
• UCB Pharma: collaborator

Study Sites (3)

University of California San Francisco

San Francisco, California, 94143, United States

NOT YET RECRUITING

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

RECRUITING

Mayo Clinic

Rochester, Minnesota, 55905, United States

NOT YET RECRUITING

MeSH Terms

Conditions

Epilepsies, Myoclonic

Interventions

Fenfluramine

Condition Hierarchy (Ancestors)

Epilepsy, Generalized; Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epileptic Syndromes

Intervention Hierarchy (Ancestors)

Phenethylamines; Ethylamines; Amines; Organic Chemicals

Study Officials

• Kelly Knupp, MD

  University of Colorado, Denver

  PRINCIPAL INVESTIGATOR

• Joseph Sullivan, MD

  University of California, San Francisco

  PRINCIPAL INVESTIGATOR

• Elaine Wirrel, MD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Rebecca Rochowiak

CONTACT

720-777-0717; rebecca.rochowiak@childrenscolorado.org

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 13, 2024
• First Posted: September 19, 2024
• Study Start: October 22, 2024
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): July 1, 2027
• Last Updated: December 10, 2024

Record last verified: 2024-12

Locations

US (3)