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

Phase 3

Terminated

• Conditions: Epilepsies, Myoclonic
• Interventions: Drug: Placebo; Drug: Lorcaserin

• Registration Number: NCT04572243
• Lead Sponsor: Eisai Inc.

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2020-09-23
• Study First Submitted: 2020-09-30
• Study First Submitted QC: 2020-09-30
• Study First Posted: 2020-10-01
• Primary Completion: 2024-08-15
• Study Completion: 2024-08-15
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-30
• Last Update Posted: 2025-01-31

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 28

Inclusion Criteria

Participants must meet all of the following criteria to be included in this study:

1. Male or female, age 2 years and older at the time of informed consent
2. Diagnosis of epilepsy with Dravet syndrome
3. Has at least 4 convulsive seizures during the 4 weeks of baseline
4. Current treatment with antiepileptic drugs must be stable for at least 4 weeks before screening, and be expected to remain stable throughout the study

Key

Exclusion Criteria

Participants who meet any of the following criteria will be excluded from this study:

1. Use of lorcaserin within 4 weeks before screening, or any history of it being discontinued due to lack of efficacy or adverse reactions
2. 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
3. Recent or concomitant use of serotonergic medications or monoamine oxidase inhibitors
4. Presence of progressive central nervous system disease other than Dravet syndrome

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo (Core Study) + Lorcaserin (Open-label Extension Phase)	Lorcaserin	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.
Placebo (Core Study) + Lorcaserin (Open-label Extension Phase)	Placebo	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.
Lorcaserin (Core Study and Open-label Extension Phase)	Lorcaserin	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.

Primary Outcome Measures

Name	Time	Method
Percent Change From Baseline in Convulsive Seizure Frequency Per 28 Days During the Core Treatment Period (14 Weeks)	Baseline to Week 14	Seizure frequency will be based on number of seizures per 28 days, calculated during the baseline period and 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.

Secondary Outcome Measures

Name	Time	Method
Maximum Lorcaserin Plasma Concentration at Steady-state (Cmax,ss) in the Core Treatment Period (14 Weeks)	Up to 14 Weeks
Percentage of 50% Responders for Convulsive Seizures in the Core Treatment Period (14 Weeks) Compared to Baseline	Baseline to Week 14	A 50 percent (%) responder is defined as a participant with at least 50% reduction in frequency of convulsive seizures per 28 days compared to baseline.
Percentage of Participants who are Free From Convulsive Seizures in the Core Treatment Period (14 Weeks)	Up to 14 Weeks
Area Under the Plasma Lorcaserin Concentration-time Curve at Steady-state (AUC,ss) in the Core Treatment Period (14 Weeks)	Up to 14 Weeks

Trial Locations

Locations (30)

Children's of Alabama / University of Alabama at Birmingham; 🇺🇸 Birmingham, Alabama, United States

University of California Los Angeles (UCLA); 🇺🇸 Los Angeles, California, United States

UCSD Rady's Children's Hosptial; 🇺🇸 San Diego, California, United States

Children's Hospital Colorado; 🇺🇸 Aurora, Colorado, United States

Northwest Florida Clinical Research Group; 🇺🇸 Gulf Breeze, Florida, United States

Joe DiMaggio Children's Hospital; 🇺🇸 Hollywood, Florida, United States

Miami Children's Hospital - Nicklaus Children's Hospital; 🇺🇸 Miami, Florida, United States

Pediatric Neurology, P.A.; 🇺🇸 Winter Park, Florida, United States

Rare Disease Research Center Pediatrics, LLC; 🇺🇸 Atlanta, Georgia, United States

Mid-Atlantic Epilepsy and Sleep Center - Bethesda; 🇺🇸 Bethesda, Maryland, United States

Spectrum Health/ Helen DeVos Children's Hospital; 🇺🇸 Grand Rapids, Michigan, United States

University of Missouri, Department of Child Health, Division of Neurology; 🇺🇸 Columbia, Missouri, United States

Institute of Neurology and Neurosurgery at Saint Barnabas; 🇺🇸 Livingston, New Jersey, United States

Northwell Health - Neuroscience Institute at Great Neck; 🇺🇸 New Hyde Park, New York, United States

NYU Langone Comprehensive Epilepsy Center; 🇺🇸 New York, New York, United States

New York Medical College; 🇺🇸 New York, New York, United States

NorthWell Health - Lennox Hill Hospital; 🇺🇸 New York, New York, United States

University of Rochester Medical Center; 🇺🇸 Rochester, New York, United States

University of North Carolina; 🇺🇸 Chapel Hill, North Carolina, United States

Duke University Hospital Center; 🇺🇸 Durham, North Carolina, United States

University Hospitals Cleveland Medical Center; 🇺🇸 Cleveland, Ohio, United States

Children's Hospital of Pittsburgh of UPMC; 🇺🇸 Pittsburgh, Pennsylvania, United States

The University of Texas Health Science Center at Houston; 🇺🇸 Houston, Texas, United States

Seattle Children's Hospital; 🇺🇸 Seattle, Washington, United States

MultiCare Institute for Research & Innovation; 🇺🇸 Tacoma, Washington, United States

Alberta Children's Hospital; 🇨🇦 Calgary, Alberta, Canada

Stollery Children's Hospital; 🇨🇦 Edmonton, Alberta, Canada

BC Children's Hospital; 🇨🇦 Vancouver, British Columbia, Canada

Children's Hospital - VH, London Health Sciences Centre; 🇨🇦 London, Ontario, Canada

University of Toronto Division of Hematology Oncology/The Hospital for Sick Children; 🇨🇦 Toronto, Ontario, Canada