A Study Evaluating Soticlestat in Participants With Dravet Syndrome or Lennox-Gastaut Syndrome Who Have Been Exposed to Fenfluramine

NCT ID: NCT06422377

Last Updated: 2025-07-29

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 1 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-04-25
• Study Completion Date: 2024-08-14

Brief Summary

The purpose of this study is to check how soticlestat impacts symptoms of Dravet syndrome [DS] and Lennox-Gastaut syndrome [LGS] in participants who have been exposed to fenfluramine.

Detailed Description

The drug being tested in this study is called soticlestat. Soticlestat is being tested to treat people who have DS or LGS and have been exposed to fenfluramine. This study will assess the efficacy and safety of soticlestat in addition to standard care in the treatment of DS or LGS.

The study will enroll approximately 45 patients. This study comprises a screening period of up to 6 weeks, a 4-week titration period, a 48-week maintenance period, a taper period of up to 1 week and a follow-up safety visit. Participants will be enrolled to receive soticlestat along with the standard of care:

• Soticlestat 100-300 milligrams (mg)

Participants will receive oral administration of soticlestat Dose 1 (days 1 to 7), Dose 2 (days 8 to 14), and Dose 3 (Days 15 to 28) with a minimum dose of 100 mg to a maximum dose of 300 mg depending on participant's body weight in the titration period followed by maintenance period up to end of treatment (up to approximately 52 weeks). Percent change from baseline in convulsive in participants with DS and major motor drop (MMD) in participants with LGS seizure frequency per 28 days during the initial 12 weeks of the maintenance period will be assessed.

This multi-center trial will be conducted in the United Kingdom and Europe. The overall time to participate in this study is approximately 60 weeks. Participants will make multiple visits to the clinic and will be followed up for safety by visiting the clinic or by telephone approximately 2 weeks after the last dose of the study drug.

Conditions

Dravet Syndrome (DS); Lennox-Gastaut Syndrome (LGS)

Study Design

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

Study Groups

Soticlestat

Participant received soticlestat at a starting dose of 100 mg to 200 mg in the 4-week titration. As a part of maintenance (initially planned for 48 weeks per protocol), participant remained on the 200 mg BID dose for 9 days followed by a 1-week taper to receive soticlestat 100 mg BID.

Group Type: EXPERIMENTAL

Soticlestat

Intervention Type: DRUG

Soticlestat tablets or mini-tablets

Interventions

Soticlestat

Soticlestat tablets or mini-tablets

Intervention Type: DRUG

Other Intervention Names

TAK-935

Eligibility Criteria

Inclusion Criteria

1. The participant has been exposed to fenfluramine (currently on or used previously).

2. The participant has a clinical diagnosis of LGS and a history of, on average, ≥12 MMD seizures in the last 90 days immediately before screening based on historical information, and the participant has ≥4 MMD seizures during a minimum of 4 weeks of seizure data collection during the prospective baseline period.

3. The participant is currently taking 0 to 5 antiseizure treatments (eg. antiseizure medications [ASMs], vagus nerve stimulation [VNS], ketogenic diet) at stable doses.

Exclusion Criteria

1. The participant is currently enrolled in a clinical study involving an investigational product or treatment device (ie, not approved in that country, other than soticlestat), or concurrently enrolled in any other type of medical research judged not to be scientifically or medically compatible with this study. Note: Compatibility will be determined on the basis of consultation with the sponsor/designee.

2. The participant has a known hypersensitivity to any component of the soticlestat formulation.

3. Participants aged ≥6 years who have positive answers on item numbers 4 or 5 on the Columbia-Suicide Severity Rating Scale (C-SSRS) before dosing are excluded. This scale will only be administered to participants aged ≥6 years at the time of enrollment or participants who turn 6 after enrollment.

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Takeda

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Director

Role: STUDY_DIRECTOR

Takeda

Locations

Epilepsihospitalet Filadelfia

Dianalund, Region Sjælland, Denmark

Countries

Denmark

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

https://clinicaltrials.takeda.com/study-detail/289390a307ed459b??page=1&idFilter=TAK-935-3004

To obtain more information on the study, click this link.

Other Identifiers

2023-504104-29-00

Identifier Type: CTIS

Identifier Source: secondary_id

TAK-935-3004

Identifier Type: -

Identifier Source: org_study_id