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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 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline up to week 16

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

Upper respiratory tract infection

Nasopharyngitis

Pyrexia

Decreased appetite

Fatigue

COVID-19

Constipation

Fall

Diarrhoea

Cataract

Status epilepticus

Urinary tract infection

Influenza

Oxygen saturation decreased

Pneumonia

Vomiting

Pyelonephritis

COVID-19 pneumonia

Acute respiratory failure

Rhinovirus infection

100%

80%

60%

40%

20%

Study treatment Arm

Placebo

Soticlestat

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

0 / 3Eligibility criteria met