Study About the Safety of Soticlestat and if it can Help Children and Adults With Dravet Syndrome or Lennox-Gastaut Syndrome who Have Already Taken Fenfluramine

2023-504104-29-00 Protocol TAK-935-3004 Therapeutic confirmatory (Phase III) Ended

Start 15 Apr 2024 · End 15 Aug 2024 · Status Ended · 1 EU/EEA countries · 1 sites · Protocol TAK-935-3004

Overview

Sponsor-declared trial summary

Phase Therapeutic confirmatory (Phase III)
Status Ended
Participants planned 15
Countries 1
Sites 1

Dravet Syndrome/Lennox-Gastaut Syndrome

To assess the efficacy of soticlestat in participants with DS or LGS who have been exposed to fenfluramine.

Key facts

Sponsor
Takeda Development Center Americas Inc.
Participant type
Pediatric, Patients
Age range
0-17 years, 18-64 years, 65+ years
Gender
Male and Female
Therapeutic area
Diseases [C] - Nervous System Diseases [C10]
Trial duration
15 Apr 2024 → 15 Aug 2024
Decision date (initial)
2023-11-14
Transition trial
No
Low-intervention
No
Rare-disease indication
Yes
Vulnerable population
Yes
Funding sources
Takeda Development Center Americas, Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Others

To assess the efficacy of soticlestat in participants with DS or LGS who have been exposed to fenfluramine.

Conditions and MedDRA coding

Dravet Syndrome/Lennox-Gastaut Syndrome

VersionLevelCodeTermSystem organ class
20.1 PT 10048816 Lennox-Gastaut syndrome 100000004852
20.0 LLT 10073682 Dravet syndrome 10010331

Regulatory references

EMA paediatric investigation plan (PIP)
EMEA-002572-PIP02-19
EU CT numberTitleSponsor
2021-002480-22 A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy, Safety, and Tolerability of Soticlestat as Adjunctive Therapy in Pediatric and Young Adult Subjects With Dravet Syndrome (DS), Studio multicentrico, randomizzato, in doppio cieco, controllato con placebo, a gruppi paralleli per valutare l’efficacia, la sicurezza e la tollerabilità di soticlestat come terapia aggiuntiva in soggetti pediatrici e giovani adulti affetti da sindrome di Dravet (SD), Estudio multicéntrico, aleatorizado, doble ciego, controlado con placebo y de grupos paralelos para evaluar la eficacia, la seguridad y la tolerabilidad de soticlestat como tratamiento adyuvante en sujetos pediátricos y en adultos jóvenes con síndrome de Dravet (SD), Multicentrikus, randomizált, kettős vak, placebo-kontrollos, párhuzamos csoportos vizsgálat a kiegészítő terápiaként alkalmazott szotiklesztát hatásosságának, biztonságosságának és tolerálhatóságának értékelésére Dravet-szindrómában (DS) szenvedő gyermek és fiatal felnőtt betegeknél
2021-002481-40 A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy, Safety, and Tolerability of Soticlestat as Adjunctive Therapy in Pediatric and Adult Subjects With Lennox-Gastaut Syndrome (LGS), Studio multicentrico, randomizzato, in doppio cieco, controllato con placebo, a gruppi paralleli per valutare l’efficacia, la sicurezza e la tollerabilità di soticlestat come terapia aggiuntiva in soggetti pediatrici e adulti affetti da sindrome di Lennox-Gastaut (LGS), Estudio multicéntrico, aleatorizado, doble ciego, controlado con placebo y de grupos paralelos para evaluar la eficacia, la seguridad y la tolerabilidad de soticlestat como tratamiento adyuvante en sujetos pediátricos y en adultos con síndrome de Lennox-Gastaut (SLG), Multicentrikus, randomizált, kettős vak, placebo-kontrollos, párhuzamos csoportos vizsgálat a kiegészítő terápiaként alkalmazott szotiklesztát hatásosságának, biztonságosságának és tolerálhatóságának értékelésére Lennox-Gastaut-szindrómában (LGS) szenvedő gyermek- és felnőtt betegeknél
2022-502801-13-00 A Phase 2, Prospective, Interventional, Open-Label, Multisite, Extension Study to Assess the Long-Term Safety and Tolerability of Soticlestat (TAK-935) as Adjunctive Therapy in Subjects with Developmental Epileptic Encephalopathies Including Dravet Syndrome, Lennox Gastaut Syndrome, CDKL5 Deficiency Disorder, and Chromosome 15 Duplication Syndrome (ENDYMION 1) Takeda Development Center Americas Inc.
2021-002482-17 A Phase 3, Prospective, Open-Label, Multisite, Extension of Phase 3 Studies To Assess the Long-Term Safety and Tolerability of Soticlestat as Adjunctive Therapy in Subjects With Dravet Syndrome or Lennox-Gastaut Syndrome (ENDYMION 2)., Estudio fase 3 prospectivo, abierto, multicéntrico y de extensión de los estudios Fase 3, para evaluar la seguridad y tolerabilidad a largo plazo de Soticlestat como tratamiento adyuvante en pacientes con Síndrome Dravet o Síndrome de Lennox-Gastaut (ENDYMION 2), Estensione di fase 3, prospettica, in aperto, multicentrica di studi di fase 3 per valutare la sicurezza e la tollerabilità a lungo termine di soticlestat come terapia aggiuntiva in soggetti con sindrome di Dravet o sindrome di Lennox-Gastaut (ENDYMION 2)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

  1. The participant, of any sex, and aged ≥2 years, at the time of informed consent.
  2. The participant has a documented clinical diagnosis of DS/LGS supported by variable combinations of typical clinical features and as determined by the investigator.
  3. The participant has been exposed to fenfluramine (currently on or used previously).

Exclusion criteria 2

  1. The participant has an unstable, clinically significant disease or other abnormality that may impact the ability to participate in the study or that may potentially confound the study results.
  2. The participant is considered by the investigator to be at imminent risk of suicide or injury to self, others, or property, or the participant has attempted suicide within 12 months before the screening visit (Visit 1).

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

  1. Percent change from baseline in convulsive (DS) and MMD (LGS) seizure frequency per 28 days during the initial 12 weeks of the maintenance period.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Soticlestat

PRD10263438 · Product

Active substance
Soticlestat
Pharmaceutical form
TABLET
Route of administration
ORAL
Max daily dose
600 mg milligram(s)
Max total dose
214.2 g gram(s)
Max treatment duration
52 Week(s)
Authorisation status
Not Authorised
MA holder
TAKEDA DEVELOPMENT CENTER AMERICAS, INC.,
Paediatric formulation
Yes
Orphan designation
Yes
Orphan designation number
EU/3/21/2529

Soticlestat

PRD10264465 · Product

Active substance
Soticlestat
Pharmaceutical form
TABLET
Route of administration
ORAL
Max daily dose
600 mg milligram(s)
Max total dose
214.2 g gram(s)
Max treatment duration
52 Week(s)
Authorisation status
Not Authorised
MA holder
TAKEDA DEVELOPMENT CENTER AMERICAS, INC.,
Paediatric formulation
No
Orphan designation
Yes
Orphan designation number
EU/3/21/2529

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Takeda Development Center Americas Inc.

65 Total trials 27 Recruiting 34 Ended
Commercial
Sponsor organisation
Takeda Development Center Americas Inc.
Address
95 Hayden Avenue
City
Lexington
Postcode
02421-7942
Country
United States

Scientific contact point

Organisation
Takeda Development Center Americas Inc.
Contact name
Medical Director

Public contact point

Organisation
Takeda Development Center Americas Inc.
Contact name
Takeda

Third parties 7

OrganisationCity, countryDuties
Endpoint Clinical Inc.
ORG-100040567
 Wakefield, United States Interactive response technologies (IRT)
Fisher Clinical Services GmbH
ORG-100017323
 Weil Am Rhein, Germany Code 14
Medidata Solutions Inc.
ORG-100016256
 New York, United States E-data capture
Greenphire LLC
ORG-100041621
 King Of Prussia, United States Other
PPD Development LP
ORG-100011560
 Wilmington, United States Code 10, Code 11, Code 12
MARKEN Germany GmbH
ORG-100017196
 Hamburg, Germany Code 14
Cogstate Inc.
ORG-100045256
 New Haven, United States Other

Locations

1 EU/EEA country · 1 investigational sites

By country

CountryMS statusPlanned subjectsSites
Denmark Ended 5 1
Rest of world
United Kingdom
 — 10 —

Investigational sites

Denmark

1 site · Ended
Filadelfia
Department of Epilepsy and personalized medicine, Kolonivej 1, 4293, Dianalund

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Country Trial startTrial end Recruitment startRecruitment end Early termination
Denmark 2024-04-15 2024-04-25

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

TitleSubmission dateStatusType
TAK-935-3004 Summary of Results
SUM-70873
 2025-02-14T12:44:02 Submitted Summary of Results

Layperson summary Annex V

TitleSubmission dateStatusType
TAK-935-3004 Plain Language Summary 2025-02-14T12:45:09 Submitted Laypersons Summary of Results

Documents 9 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

TypeTitleVersion
Laypersons summary of results (for publication) TAK-935-3004 Plain Language Summary 1
Recruitment arrangements (for publication) K1_TAK_935_3004_Justification-for-Incapactited-Adults_DNK_Public N/A
Recruitment arrangements (for publication) K1_TAK_935_3004_Justification-for-Pediatric-Population_DNK_Public N/A
Recruitment arrangements (for publication) K1_TAK-935-3004_Recruitment-Arrangement_DNK_Public n/a
Subject information and informed consent form (for publication) L1_TAK-935-3004_Assent-Age-15-17yr_ICF_DNK_Danish_Public 1.0
Subject information and informed consent form (for publication) L1_TAK-935-3004_Main-Adults-Parent-Legal-Guardian_ICF_DNK_Danish_Public 1.0
Subject information and informed consent form (for publication) L1_TAK-935-3004_Main-Minors-Parental-Legal-Guardian_ICF_DNK_Danish_Public 1.0
Subject information and informed consent form (for publication) L1_TAK-935-3004_Pregnant-Partner-Authorization_ICF_DNK_Danish_Public 1.0
Summary of results (for publication) TAK-935-3004 Summary of Results 1

Application history

3 submissions — initial application plus substantial / non-substantial modifications

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2023-08-17 Denmark Acceptable
2023-10-27
 2023-11-14
2 SUBSTANTIAL MODIFICATION SM-1 2023-11-29 Denmark Acceptable
2023-12-19
 2023-12-19
3 NON SUBSTANTIAL MODIFICATION NSM-1 2024-06-27 Denmark Acceptable
2023-12-19
 2024-06-27