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Soticlestat for Dravet Syndrome

Phase 3
Waitlist Available
Research Sponsored by Takeda
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Has documented clinical diagnosis of DS
Weighs ≥10 kg at the screening visit
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline up to week 16
Awards & highlights
Pivotal Trial

Summary

This trial is testing soticlestat, a new medication, to see if it can help reduce seizures in children and young adults with Dravet Syndrome. The medication works by affecting brain chemicals involved in causing seizures. Participants will continue their usual treatments and add either soticlestat or another substance for a few months.

Who is the study for?
This trial is for children and young adults with Dravet Syndrome who weigh at least 10 kg. They should have had a minimum of 12 convulsive seizures in the past 12 weeks, be on stable doses of up to four antiseizure medications, and not have any other unstable health conditions that could interfere with the study.
What is being tested?
The trial tests if Soticlestat can reduce seizure frequency when added to standard therapy compared to a placebo. Participants will take either Soticlestat or placebo alongside their regular antiseizure drugs for 16 weeks, with an option to join an extension study afterwards.
What are the potential side effects?
While specific side effects are not listed here, participants may experience unexpected reactions due to adding Soticlestat or placebo to their current treatment regimen. These could range from mild discomforts like headaches or nausea to more serious issues depending on individual responses.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with Down syndrome.
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I weigh at least 10 kg.
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My seizures are not controlled despite trying at least one antiseizure medication.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline up to week 16
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to week 16 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Convulsive Seizures
Percent Change From Baseline in Convulsive Seizure Frequency per 28 days During the Maintenance Period (EMA Region Specific)
Secondary study objectives
CGI-I Nonseizure Symptoms Score
CGI-I Seizure Intensity and Duration Score
Caregiver Global Impression of Improvement (Care GI-I) Score
+9 more

Side effects data

From 2024 Phase 3 trial • 270 Patients • NCT04938427
14%
Somnolence
13%
Change in seizure presentation
8%
Upper respiratory tract infection
7%
Nasopharyngitis
7%
Pyrexia
6%
Decreased appetite
6%
Fatigue
6%
COVID-19
5%
Constipation
5%
Fall
1%
Vomiting
1%
Influenza
1%
Pneumonia
1%
Urinary tract infection
1%
Status epilepticus
1%
Pyelonephritis
1%
COVID-19 pneumonia
1%
Cataract
1%
Oxygen saturation decreased
1%
Acute respiratory failure
1%
Diarrhoea
1%
Rhinovirus infection
100%
80%
60%
40%
20%
0%
Study treatment Arm
Soticlestat
Placebo

Awards & Highlights

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

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: SoticlestatExperimental Treatment1 Intervention
Participants weighing \<45kg: Soticlestat, mini-tablets, at the dose of 40mg to 200mg, orally or via gastrostomy tube (G-tube) or low-profile gastric tube (MIC-KEY button) or jejunostomy tube (J-tube), twice daily (BID) based on the body weight up to 4 weeks in Titration Period. Participants will continue to receive dose that they are on at the end of Titration Period, for 12 weeks in Maintenance Period. The total duration of the treatment will be up to 16 weeks (Treatment Period). The dose will be tapered down if participants decide to discontinue the treatment. Participants weighing ≥45kg: Soticlestat mini-tablets or tablets with a starting dose of 100mg BID followed by 200 mg BID and, then 300mg BID, up to 4 weeks in Titration Period. Participants will continue to receive 300mg BID for 12 weeks in the Maintenance Period. The total duration of the treatment will be up to 16 weeks (Treatment Period). The dose will be tapered down if participants decide to discontinue the treatment.
Group II: PlaceboPlacebo Group1 Intervention
Soticlestat placebo-matching mini-tablets or tablets, orally or via G-tube or MIC-KEY button or J-tube, BID, up to 4 weeks in the Titration Period. Participants will continue to receive the soticlestat placebo-matching mini-tablets or tablets for 12 weeks in the Maintenance Period. The total duration of the treatment will be up to 16 weeks. Soticlestat matching tapering will be done to maintain the blind if participants decide to discontinue the treatment.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Soticlestat
2022
Completed Phase 3
~640

Find a Location

Who is running the clinical trial?

TakedaLead Sponsor
1,233 Previous Clinical Trials
4,148,082 Total Patients Enrolled
Study DirectorStudy DirectorTakeda
1,267 Previous Clinical Trials
503,941 Total Patients Enrolled

Media Library

Soticlestat Clinical Trial Eligibility Overview. Trial Name: NCT04940624 — Phase 3
Dravet Syndrome Research Study Groups: Soticlestat, Placebo
Dravet Syndrome Clinical Trial 2023: Soticlestat Highlights & Side Effects. Trial Name: NCT04940624 — Phase 3
Soticlestat 2023 Treatment Timeline for Medical Study. Trial Name: NCT04940624 — Phase 3
~37 spots leftby Oct 2025