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

NCT ID: NCT06598449

Last Updated: 2024-12-10

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE4
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-10-22
• Study Completion Date: 2027-07-31

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Fenfluramine

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.

Group Type: EXPERIMENTAL

fenfluramine

Intervention Type: DRUG

Clear, cherry flavored oral solution

Interventions

fenfluramine

Clear, cherry flavored oral solution

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. SCN1A with a known or presumed pathogenic variant or VUS with a history of prolonged seizure or a clinical diagnosis of Dravet syndrome.

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

Exclusion Criteria

1. 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.

2. 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:

1. Weight less than the 2nd percentile.

2. Lack of weight gain that crosses two or more of the major percentile lines and is not congruent with length.

Inclusion of patients will be at the sole discretion of the multi-PIs based on a majority vote.

• Minimum Eligible Age: 12 Months
• Maximum Eligible Age: 24 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

UCB Pharma

INDUSTRY

Sponsor Role: collaborator

University of Colorado, Denver

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Kelly Knupp, MD

Role: PRINCIPAL_INVESTIGATOR

University of Colorado, Denver

Joseph Sullivan, MD

Role: PRINCIPAL_INVESTIGATOR

University of California, San Francisco

Elaine Wirrel, MD

Role: PRINCIPAL_INVESTIGATOR

Mayo Clinic

Locations

University of California San Francisco

San Francisco, California, United States

Site Status: NOT_YET_RECRUITING

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status: RECRUITING

Mayo Clinic

Rochester, Minnesota, United States

Site Status: NOT_YET_RECRUITING

Countries

United States

Central Contacts

Rebecca Rochowiak

Role: CONTACT

Phone: 720-777-0717

Email: rebecca.rochowiak@childrenscolorado.org

Facility Contacts

Antoinette Swanson

Role: primary

Rebecca Rochowiak

Role: primary

Other Identifiers

21-3972

Identifier Type: -

Identifier Source: org_study_id