A Study of Lorcaserin as Adjunctive Treatment in Participants With Dravet Syndrome

NCT04572243 | Terminated Phase 3 Results FDA Drug

• 1 other identifier: E2023-A001-304
• Study Type: interventional
• Target: 22
• Locations: 2 countries
• Sites: 30

Brief Summary

The primary purpose of the study is to demonstrate that lorcaserin has superior efficacy compared to placebo on percent change in frequency of convulsive seizures per 28 days in participants with Dravet syndrome.

Conditions

Epilepsies, Myoclonic

Keywords

Dravet Syndrome; Lorcaserin; E2023; Epilepsy; Seizures

Outcome Measures

Primary Outcomes (1)

• Core Study: Percent Change From Baseline in Convulsive Seizure Frequency Per 28 Days During the Treatment Period

  Seizure frequency for convulsive seizures was based on number of seizures per 28 days, calculated during the baseline period (Week -4 to Week 0) and 14-week treatment period, as the number of seizures during each respective period divided by the number of non-missing days during each respective period, multiplied by 28. Percent change from baseline was calculated as: (\[post-baseline value minus the baseline value\] / baseline value) \*100.

  Baseline up to Week 14

Secondary Outcomes (4)

• Core Study: Percentage of Participants With 50% or Greater Response for Convulsive Seizures in the Treatment Period Compared to Baseline

  Baseline up to Week 14

• Core Study: Percentage of Participants Who Were Free From Convulsive Seizures in the Treatment Period

  Baseline up to Week 14

• Core Study: Plasma Concentrations of Lorcaserin

  Weeks 1, 2, 6, 15: Pre-dose and 1 to 2 hours post-dose; Weeks 4,10: Pre-dose, 1 to 2 hours and 3 to 6 hours post-dose

• Core Study: Number of Participants With Treatment-emergent Adverse Events (TEAEs)

  From first dose of study drug up to end of 4 weeks of follow up after last dose of study drug (up to Week 18)

Study Arms (2)

Lorcaserin (Core Study and Open-label Extension Phase)

EXPERIMENTAL

Participants will be randomized to receive lorcaserin administered as an oral suspension, twice daily for 14 weeks during the core treatment period. Dose will be based on body weight as follows: target dose for participants weighing 10 to less than (\<) 20, 20 to \<40, and greater than or equal to (\>=) 40 kilogram (kg) will be 5, 10, and 20 milligram per day (mg/day) respectively. Based on clinical response and tolerability and within 2 weeks of treatment, dose can be increased up to 10, 20 mg/day for participants weighing 10 to \<20, 20 to \<40 kg respectively. Participants completing the core treatment period will enter a 12-week extension phase and will receive lorcaserin.

Drug: Lorcaserin

Placebo (Core Study) + Lorcaserin (Open-label Extension Phase)

PLACEBO COMPARATOR

Participants will be randomized to receive lorcaserin matching placebo administered as an oral suspension, twice daily for 14 weeks during the core treatment period. Dose will be based on body weight as follows: target dose for participants weighing 10 to \<20, 20 to \<40, and \>=40 kg will be 5, 10, and 20 mg/day respectively. Based on clinical response and tolerability and within 2 weeks of treatment, dose can be increased up to 10, 20 mg/day for participants weighing 10 to \<20, 20 to \<40 kg respectively. Participants completing the core treatment period will enter a 12-week extension phase and will receive lorcaserin.

Drug: Placebo; Drug: Lorcaserin

Interventions

Placebo DRUG

Placebo matching to lorcaserin oral tablet, administered as oral suspension.

Placebo (Core Study) + Lorcaserin (Open-label Extension Phase)

Lorcaserin DRUG

Lorcaserin oral tablet, administered as oral suspension.

Also known as: E2023

Lorcaserin (Core Study and Open-label Extension Phase); Placebo (Core Study) + Lorcaserin (Open-label Extension Phase)

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants must meet all of the following criteria to be included in this study:
• Male or female, age 2 years and older at the time of informed consent
• Diagnosis of epilepsy with Dravet syndrome
• Has at least 4 convulsive seizures during the 4 weeks of baseline
• Current treatment with antiepileptic drugs must be stable for at least 4 weeks before screening, and be expected to remain stable throughout the study

You may not qualify if:

• Participants who meet any of the following criteria will be excluded from this study:
• Use of lorcaserin within 4 weeks before screening, or any history of it being discontinued due to lack of efficacy or adverse reactions
• Use of fenfluramine within 2 months before screening, any history of lack of fenfluramine efficacy, or any history of valvulopathy at baseline with history of fenfluramine use
• Recent or concomitant use of serotonergic medications or monoamine oxidase inhibitors
• Presence of progressive central nervous system disease other than Dravet syndrome

Sponsors & Collaborators

• Eisai Inc.: lead

Study Sites (30)

Children's of Alabama / University of Alabama at Birmingham

Birmingham, Alabama, 35226, United States

Location

University of California Los Angeles (UCLA)

Los Angeles, California, 90095, United States

Location

UCSD Rady's Children's Hosptial

San Diego, California, 92123, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Northwest Florida Clinical Research Group

Gulf Breeze, Florida, 32561, United States

Location

Joe DiMaggio Children's Hospital

Hollywood, Florida, 33021, United States

Location

Miami Children's Hospital - Nicklaus Children's Hospital

Miami, Florida, 33155, United States

Location

Pediatric Neurology, P.A.

Winter Park, Florida, 32789, United States

Location

Rare Disease Research Center Pediatrics, LLC

Atlanta, Georgia, 30318, United States

Location

Mid-Atlantic Epilepsy and Sleep Center - Bethesda

Bethesda, Maryland, 20817, United States

Location

Spectrum Health/ Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States

Location

University of Missouri, Department of Child Health, Division of Neurology

Columbia, Missouri, 65201, United States

Location

Institute of Neurology and Neurosurgery at Saint Barnabas

Livingston, New Jersey, 07039, United States

Location

Northwell Health - Neuroscience Institute at Great Neck

New Hyde Park, New York, 10075, United States

Location

NYU Langone Comprehensive Epilepsy Center

New York, New York, 10016, United States

Location

New York Medical College

New York, New York, 10019-1147, United States

Location

NorthWell Health - Lennox Hill Hospital

New York, New York, 11021, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

University of North Carolina

Chapel Hill, North Carolina, 27599-7025, United States

Location

Duke University Hospital Center

Durham, North Carolina, 27710, United States

Location

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States

Location

The University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

MultiCare Institute for Research & Innovation

Tacoma, Washington, 98405, United States

Location

Alberta Children's Hospital

Calgary, Alberta, AB T3B 6A8, Canada

Location

Stollery Children's Hospital

Edmonton, Alberta, T6G 1C9, Canada

Location

BC Children's Hospital

Vancouver, British Columbia, V6H 3N1, Canada

Location

Children's Hospital - VH, London Health Sciences Centre

London, Ontario, N6A 4G5, Canada

Location

University of Toronto Division of Hematology Oncology/The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

MeSH Terms

Conditions

Epilepsies, Myoclonic; Epilepsy; Seizures

Interventions

lorcaserin

Condition Hierarchy (Ancestors)

Epilepsy, Generalized; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epileptic Syndromes; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Limitations and Caveats

This study was terminated due to sponsor decision, and not due to safety concerns.

Results Point of Contact

• Title: Eisai Medical Information
• Organization: Eisai Inc.

esi_medinfo@eisai.com

+1-888-274-2378

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: September 30, 2020
• First Posted: October 1, 2020
• Study Start: September 23, 2020
• Primary Completion: August 15, 2024
• Study Completion: August 15, 2024
• Last Updated: October 8, 2025
• Results First Posted: October 8, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will share

Locations

CA (5); US (25)