Clinical Trials Register

Summary
EudraCT Number:	2016-001072-29
Sponsor's Protocol Code Number:	BIA-2093-211/EXT
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2018-02-13
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2016-001072-29

A.3

Full title of the trial

OPEN-LABEL, 2-DOSE LEVEL TRIAL TO EVALUATE PHARMACOKINETICS, SAFETY, AND TOLERABILITY OF ESLICARBAZEPINE ACETATE (ESL) AS ADJUNCTIVE THERAPY IN INFANTS WITH REFRACTORY EPILEPSY WITH PARTIAL-ONSET SEIZURES AGED FROM 1 MONTH TO <2 YEARS – 1-YEAR EXTENSION

Studio clinico in aperto con 2 livelli di dosaggio per valutare la farmacocinetica, la sicurezza e la tollerabilità di eslicarbazepina acetato (ESL) come terapia aggiuntiva nei neonati affetti da epilessia refrattaria con crisi parziali di età compresa tra 1 mese e <2 anni – estensione di 1 anno

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

Clinical Trial to investigate the safety and tolerability of
Eslicarbazepine Acetate as additive therapy in babies aged from 1 month to <2 years with refractory epilepsy with partial-onset seizures for one year

Studio clinico per valutare la sicurezza e la tollerabilità di eslicarbazepina acetato come terapia aggiuntiva nei neonati affetti da epilessia refrattaria con crisi parziali di età compresa tra 1 mese e <2 anni per un anno

A.4.1

Sponsor's protocol code number

BIA-2093-211/EXT

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/015/2015

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	BIAL - Portela & Ca, S.A.
B.1.3.4	Country	Portugal
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	BIAL - Portela & Ca, S.A.
B.4.2	Country	Portugal
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	BIAL - Portela & Ca, S.A.
B.5.2	Functional name of contact point	Sponsor
B.5.3	Address:
B.5.3.1	Street Address	À Av. Siderurgia Nacional
B.5.3.2	Town/ city	Coronado (S. Romão e S. Mamede)
B.5.3.3	Post code	4745-457
B.5.3.4	Country	Portugal
B.5.4	Telephone number	+351229866100
B.5.5	Fax number	+351229866192
B.5.6	E-mail	jose.rocha@bial.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.2	Country which granted the Marketing Authorisation	European Union
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Eslicarbazepine Acetate
D.3.2	Product code	BIA 2-093
D.3.4	Pharmaceutical form	Oral suspension
D.3.4.1	Specific paediatric formulation	Yes
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	ESLICARBAZEPINE ACETATE
D.3.9.1	CAS number	236395-14-5
D.3.9.2	Current sponsor code	BIA 2-093
D.3.9.3	Other descriptive name	ESLICARBAZEPINE ACETATE
D.3.9.4	EV Substance Code	SUB30424
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	50
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

refractory epilepsy with partial-onset seizures in children aged from 1
month to < 2 years

E.1.1.1

Medical condition in easily understood language

epilepsy

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10065336
E.1.2	Term	Partial epilepsy
E.1.2	System Organ Class	100000004852

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To assess the safety and tolerability of 1 year of treatment with ESL in the defined patient population and to perform exploratory analyses of efficacy.

E.2.2

Secondary objectives of the trial

Not applicable

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Have an informed consent signed by their parent(s) or guardian(s) before undergoing any study-related activities.
- Current treatment with 1 to 2 anti-epileptic drugs (except oxcarbazepine; vagus nerve stimulation if present and functioning will not be counted as an anti-epileptic drug).
- Have completed the Evaluation Period in Study 211.

E.4

Principal exclusion criteria

- Diagnosis of treatable seizure aetiology such as metabolic, toxic, and infectious disorders.
- Primarily generalised seizures.
- Known rapidly progressive neurological disorders (e.g. rapidly progressive brain disease, epilepsy secondary to rapidly progressive cerebral lesion).
- Seizures of non-epileptic origin.
- Diagnosis with Epileptiform Encephalopathies (Early Myoclonic Encephalopathy, Ohtahara syndrome, West syndrome [current], Dravet’s syndrome, or Lennox-Gastaut syndrome).
- Uncontrolled cardiac (including atrioventricular block and other clinically significant electrocardiographic abnormalities), renal, hepatic, endocrine, gastrointestinal, metabolic, haematological, or oncology disorders.
- Hypersensitivity to the active substance, to other carboxamide derivatives (e.g. carbamazepine, oxcarbazepine), or to any of the excipients.
- Relevant clinical laboratory abnormalities (e.g. sodium <130 mmol/L, alanine or aspartate transaminases >2 x the upper limit of normal, white blood cell count <3000 cells/mm3) (measured at Visit 1).
- Any other condition or circumstance that, in the opinion of the investigator, could compromise the subject’s ability to comply with the study protocol.

E.5 End points

E.5.1

Primary end point(s)

Efficacy:
• Seizures (date, time, type, and duration) recorded by the parent(s) or guardian(s) in study diaries.

Safety:
• Treatment-emergent adverse events (TEAEs) defined as all AEs with onset or worsening after first intake of study treatment until 4 weeks
after last intake of study treatment.
• Clinical laboratory safety tests:
- Biochemistry: sodium, potassium, calcium, creatinine, blood urea nitrogen, aspartate transaminase, alanine transaminase, gamma-glutamyl
transferase, albumin, total protein, total bilirubin, and glucose.
- Haematology: haemoglobin, haematocrit, erythrocyte count, leucocyte
count with differential, and platelet count.
• Urinalysis: local dipstick test of pH, specific gravity, protein, blood, glucose, ketones, bilirubin, and urobilinogen. If dipstick results indicate a significant abnormality, then microscopy and other appropriate tests (as needed) will be performed by the central laboratory.
• Physical examination, vital signs, neurological examination, and electrocardiogram.

E.5.1.1

Timepoint(s) of evaluation of this end point

Ath the end of the Trial.

E.5.2

Secondary end point(s)

Not applicable

E.5.2.1

Timepoint(s) of evaluation of this end point

Not applicable

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

Information not present in EudraCT

E.8.2.2

Placebo

Information not present in EudraCT

E.8.2.3

Other

Information not present in EudraCT

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Croatia

Czech Republic

Italy

Portugal

Romania

Russian Federation

Serbia

Ukraine

E.8.7

Trial has a data monitoring committee

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1

Number of subjects for this age range:

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

Yes

F.1.1.4.1

Number of subjects for this age range:

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

F.1.3

Elderly (>=65 years)

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

infants with refractory epilepsy with partial-onset seizures aged from 1
month to < 2 years

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

Patients will be treated according to local standard practice.

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2017-12-01

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2017-04-12

P. End of Trial
P.	End of Trial Status	Completed

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IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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