Open-label Follow-up Study of Soticlestat in Adults and Children with Developmental Epileptic Encephalopathies

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Takeda Development Center Americas Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

26 Jul 2019 → 30 Jul 2025

Decision date (initial)

2023-09-27

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Takeda Development Center Americas, Inc.

External identifiers

EU CT number

2022-502801-13-00

EudraCT number

2018-002485-39

WHO UTN

U1111-1218-5515

ClinicalTrials.gov

NCT03635073

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Therapy, Others, Safety, Efficacy, Pharmacokinetic

Assess the long-term safety and tolerability of soticlestat when administered as adjunctive therapy to at least one anti-seizure therapy (ie, antiepileptic drugs [AEDs], vagal nerve stimulator, ketogenic diet or modified Atkins diet) in subjects with rare epilepsies.

Secondary objectives 1

1. The secondary objectives, in subjects receiving soticlestat as adjunctive therapy to at least one anti-seizure therapy, are the following: 1) To assess the effect of soticlestat on seizure frequency. 2) To assess the effect of soticlestat on the Clinical Global Impression of Severity (CGI-S) provided by the investigator.

Conditions and MedDRA coding

Epileptic Encephalopathies: Dravet Syndrome, Lennox Gastaut Syndrome, CDKL5 Deficiency Disorder, and Chromosome 15 Duplication Syndrome, other adult DEEs.

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-002572-PIP02-19

Plan to share IPD

Yes

IPD plan description

Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. 1. Subjects must have participated in a previous soticlestat study and meet one of the following conditions: -Successfully completed a Soticlestat clinical study. -Received at least 10 weeks of treatment (combined Dose Optimization and Maintenance Period) with the study drug in an antecedent placebo controlled blinded soticlestat clinical study and the subject did not have a serious or severe AE that, in the investigator's or sponsor's opinion, was related to the study drug and would make it unsafe for the subject to continue receiving the study drug. - In the opinion of the investigator, the subject has the potential to benefit from the administration of soticlestat (not applicable for Spain).

Exclusion criteria 1

1. 1. Clinically significant disease, that, in the investigator's opinion, precludes study participation. 2. Suicide attempt within the last year, at significant risk of suicide (either in the opinion of the investigator or defined as 'yes' to suicidal ideation question 4 or 5 on the C-SSRS at Screening) or appearing suicidal per investigator judgment.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 6

1. Incidence of AEs.
2. Change from Baseline in behavioral and adaptive functional measures using the Vineland Adaptive Behavior Scale (VABS).
3. Change from Baseline in behavior measures using total scores and subscale scores of the Aberrant Behavior Checklist-Community Edition (ABC-C) for subjects ≥6 years of age.
4. Change from Baseline in the Columbia-Suicide Severity Rating Scale (CSSRS) categorization based on Columbia Classification Algorithm of Suicide Assessment categories 1, 2, 3, 4, and 5 for subjects ≥6 years of age.
5. Absolute values and change from Baseline in clinical laboratory assessments, vital sign measurements, body weight, and electrocardiogram (ECG) parameters.
6. Incidence of potentially clinically significant clinical safety laboratory test values, vital signs, weight, height, and ECG evaluations.

Secondary endpoints 5

1. Percent change from Baseline in all seizure 28-day frequency.
2. Percent change from Baseline in drop seizure 28-day frequency (LGS subjects).
3. Percent change from Baseline in convulsive seizure 28-day frequency (DS subjects).
4. Percent change from Baseline in motor seizure 28-day frequency
5. Change from Baseline in CGI-S.

Investigational products

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

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Takeda Development Center Americas Inc.

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

Locations

3 EU/EEA countries · 8 investigational sites

By country

Investigational sites

Country notifications

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

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

Layperson summary Annex V

Documents 59 files

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

Application history

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