Clinical Trials Register

Summary
EudraCT Number:	2020-003140-83
Sponsor's Protocol Code Number:	NBI-921352-DEE2012
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2022-01-26
Trial 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

2020-003140-83

A.3

Full title of the trial

A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of NBI-921352 as
Adjunctive Therapy in Subjects with SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)

Studio di fase 2 randomizzato, in doppio cieco, controllato con placebo, volto a valutare l’efficacia, la sicurezza, la tollerabilità e la farmacocinetica di NBI-921352 come terapia aggiuntiva in soggetti con sindrome dell’encefalopatia epilettica e dello sviluppo correlata a SCN8A (SCN8A-DEE)

A.3.1

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

Study to Evaluate NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8ADEE)

Studio volto a valutare NBI-921352 come terapia aggiuntiva in soggetti con sindrome dell’encefalopatia epilettica e dello sviluppo correlata a SCN8A (SCN8A-DEE)

A.3.2

Name or abbreviated title of the trial where available

NBI-921352-DEE2012

A.4.1

Sponsor's protocol code number

NBI-921352-DEE2012

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Neurocrine Biosciences, Inc.
B.1.3.4	Country	United States
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	Neurocrine Biosciences, Inc.
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Neurocrine Biosciences, Inc.
B.5.2	Functional name of contact point	Medical Information Call Center
B.5.3	Address:
B.5.3.1	Street Address	12780 El Camino Real
B.5.3.2	Town/ city	San Diego
B.5.3.3	Post code	CA 92130
B.5.3.4	Country	United States
B.5.4	Telephone number	0018776413461
B.5.5	Fax number	0000000
B.5.6	E-mail	medinfo@neurocrine.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	No
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	NBI-921352
D.3.2	Product code	[NBI-921352]
D.3.4	Pharmaceutical form	Pillules
D.3.4.1	Specific paediatric formulation	No
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	NBI-921352
D.3.9.2	Current sponsor code	NBI-921352
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	20
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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
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.IMP: 2
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	No
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	NBI-921352
D.3.2	Product code	[NBI-921352]
D.3.4	Pharmaceutical form	Pillules
D.3.4.1	Specific paediatric formulation	No
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	NBI-921352
D.3.9.2	Current sponsor code	NBI-921352
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	2
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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
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.IMP: 3
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	No
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	NBI-921352
D.3.2	Product code	[NBI-921352]
D.3.4	Pharmaceutical form	Pillules
D.3.4.1	Specific paediatric formulation	No
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	NBI-921352
D.3.9.2	Current sponsor code	NBI-921352
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	5
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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
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.IMP: 4
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	No
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	NBI-921352
D.3.2	Product code	[NBI-921352]
D.3.4	Pharmaceutical form	Pillules
D.3.4.1	Specific paediatric formulation	No
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	NBI-921352
D.3.9.2	Current sponsor code	NBI-921352
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	10
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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
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.IMP: 5
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	No
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	NBI-921352
D.3.2	Product code	[NBI-921352]
D.3.4	Pharmaceutical form	Pillules
D.3.4.1	Specific paediatric formulation	No
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	NBI-921352
D.3.9.2	Current sponsor code	NBI-921352
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	2
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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
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
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Pillules
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8ADEE)

Sindrome dell’encefalopatia epilettica e dello sviluppo correlata a SCN8A (SCN8A-DEE)

E.1.1.1

Medical condition in easily understood language

Epilepsy

Epilessia

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	PT
E.1.2	Classification code	10077380
E.1.2	Term	Epileptic encephalopathy
E.1.2	System Organ Class	10029205 - Nervous system disorders

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

To assess the efficacy of NBI-921352 as adjunctive therapy on the frequency of countable motor seizures

Valutare l’efficacia di NBI-921352 come terapia aggiuntiva sulla frequenza delle crisi convulsive motorie

E.2.2

Secondary objectives of the trial

• To evaluate the efficacy of NBI-921352 using the Clinical and Parent/Caregiver Global Impression of Change scales and the Clinical and Parent/Caregiver Global Impression of Severity scales.
• To characterize the pharmacokinetics of NBI-921352 and determine the effect of NBI-921352 on plasma levels of concomitant ASMs and evaluated metabolites.
• To evaluate the safety and tolerability of NBI-921352.

Valutare l’efficacia di NBI-921352 utilizzando la scala di impressione globale del cambiamento clinica e quella per genitori/caregiver e le scale di impressione globale di gravità da parte del genitore/caregiver e clinica.
Caratterizzare la farmacocinetica (PK) di NBI-921352 e determinare l’effetto di NBI-921352 sui livelli plasmatici di farmaci antiepilettici (ASM) concomitanti e dei metaboliti valutati.
Valutare la sicurezza e la tollerabilità di NBI-921352.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Be a male or female 12 to 21 years of age, inclusive.
• Have a diagnosis of SCN8A-DEE supported by both clinical and genetic findings
• Have on average at least 1 countable motor seizure per week and not
be seizure-free for more than 20 consecutive days.
• Being treated with at least 1 other ASM, but no more than 4 ASMs.
• Have failed to achieve seizure freedom with at least 2 ASMs.
• Must be using a nocturnal alerting system or practice consistent with standards of care at the time of screening and continue to use this for the duration of the study.
• Must have an adequate rescue medication regimen per the investigator's judgment in place at the time of screening and for the duration of the study.
• Have a body weight of at least 10 kg
• The subject's parent/caregiver is able to accurately identify seizure types, especially countable motor seizures and is able to complet

•Essere soggetti ambosessi di età tra 12 e 21 anni, compresi.
•Avere una diagnosi di SCN8A-DEE supportata sia da risultati clinici che genetici
•Il soggetto manifesta in media almeno 1 crisi convulsiva motoria numerabile ogni settimana e non presenta crisi convulsive per più di 20 giorni consecutivi.
•Essere trattati con almeno 1 altro ASM, ma non più di 4 ASM.
•Non sono riusciti a debellare le crisi convulsive nonostante il trattamento con almeno 2 ASM.
•Devono utilizzare un sistema di allerta notturna o un processo in linea con gli standard di cura al momento dello screening e continuare a usare il sistema o il processo per tutta la durata dello studio.
•Devono avere un adeguato regime farmacologico di soccorso in base al giudizio dello sperimentatore in vigore al momento dello screening e per la durata dello studio.
•Devono presentare un peso corporeo di almeno 10 kg
•Il genitore/la persona che assiste il soggetto deve essere in grado di identificare accuratamente i tipi di crisi convulsive, in particolare le crisi convulsive motorie numerabili, ed è in grado di compilare

E.4

Principal exclusion criteria

Have previously been enrolled in this study and received blinded treatment.
• Have participated in an interventional clinical trial <30 days prior to screening.
• Have symptoms that would be more consistent with another epilepsy disorder such as Dravet syndrome (eg, fever-induced episodes of status epilepticus, frequent myoclonic seizures, worsening on sodium channel blockers, absence seizures with generalized spike-and-wave EEG as the sole seizure type).
• Are currently receiving cannabinoids or medical marijuana except Epidiolex/Epidyolex, unless approved by the Sponsor.
• Are currently taking systemic steroids (excluding inhaled medication for asthma treatments). If subject has received these medications in the past, must be off these medications for at least 3 months prior to the screening visit and these drugs may not be initiated during the duration of the study. Intermittent steroids to treat nonepilepsy related diseases (such as allergies or dermatological conditions) are not exclusionary.
• Have a history of moderate or severe head trauma or other neurological disorders or systemic medical diseases that are, in the investigator's opinion, likely to affect nervous system functioning.
• Have a clinically significant medical condition or chronic disease that in the opinion of the investigator would preclude the subject from participating in and completing the study or that could confound interpretation of study outcome.
• Have clinically significant abnormal vital signs at the screening visit as determined by the investigator.
• Have one or more clinical laboratory test values outside the reference range, based on blood samples taken at the screening visit, that are of potential risk to the subject's safety
• Have, at the screening visit, an ECG finding of a corrected QT interval using Fridericia's formula (QTcF) >450 msec or presence of any significant cardiac abnormality

Essere stati precedentemente arruolati in questo studio e aver ricevuto un trattamento in cieco.
•Aver partecipato a una sperimentazione clinica interventistica <30 giorni prima dello screening.
•Presentare sintomi che sarebbero più coerenti con un altro disturbo dell’epilessia come la sindrome di Dravet (ad es. episodi indotti da febbre di stato epilettico, frequenti crisi convulsive miocloniche, peggioramento dei bloccanti dei canali del sodio,crisi convulsive di assenza con EEG punta-onda generalizzato come crisi convulsiva unica).
•Ricevere attualmente cannabinoidi o marijuana per uso medico, ad eccezione di Epidiolex/Epidyolex, a meno che ciò non sia approvato dallo sponsor.
•Assumere attualmente steroidi sistemici (esclusi farmaci per via inalatoria per i trattamenti dell’asma). Se il soggetto ha già ricevuto questi farmaci in passato, non deve averli assunti per almeno 3 mesi prima della visita di screening, e la somministrazione di questi farmaci non può essere iniziata nel corso dello studio. La terapia intermittente a base di steroidi per il trattamento di malattie non correlate ad epilessia (quali allergie o condizioni dermatologiche) non sono causa di esclusione.
•Presentare un’anamnesi di trauma cranico moderato o grave o altri disturbi neurologici o patologie mediche sistemiche che, a giudizio dello sperimentatore, potrebbero influire sul funzionamento del sistema nervoso.
•Presentare una condizione medica clinicamente significativa o una patologia cronica che, a giudizio dello sperimentatore, potrebbe impedire al soggetto di partecipare e completare lo studio o che potrebbe confondere l’interpretazione dell’esito dello studio.
•Presentare segni vitali anomali clinicamente significativi alla visita di screening, secondo la valutazione dello sperimentatore.
•Presentare uno o più valori dei test clinici di laboratorio al di fuori dell’intervallo di riferimento, sulla base dei campioni di sangue prelevati alla visita di screening, che costituiscono un potenziale rischio per la sicurezza del soggetto
•Presentare, alla visita di screening, un risultato ECG di un intervallo QT corretto usando la formula di Fridericia (QTcF) >450 msec o una qualsiasi anomalia cardiaca significativa.

E.5 End points

E.5.1

Primary end point(s)

Percentage change from baseline in 28-day seizure frequency for countable motor seizures during the treatment period of the study.

Variazione percentuale dal basale nella frequenza delle crisi convulsive ogni 28 giorni per le crisi convulsive computabili durante il periodo di trattamento dello studio.

E.5.1.1

Timepoint(s) of evaluation of this end point

Time Frame: Baseline, Treatment Period: Day 1 to Week 16

Finestra temporale: Basale, periodo di trattamento: dal giorno 1 alla settimana 16

E.5.2

Secondary end point(s)

• Treatment response of = 50% decrease for countable motor seizures
• Treatment response of = 25%, = 75%, or 100% decrease in countable motor seizures
• Clinical Global Impression of Change (CGIC) at each study visit
• Parent/Caregiver Global Impression of Change (GIC) at each study visit
• Change from Baseline in Clinical Global Impression of Severity (CGIS)
• Change from Baseline in Parent/Caregiver Global Impression of Severity (GIS)
Other secondary endpoints (maintenance period):
-Percentage Change from Baseline in 28-day Seizure Frequency for countable motor seizures
-Treatment response of = 25%, = 50%, = 75%, or 100% decrease for countable motor seizures

• Risposta al trattamento con una riduzione =50% per le crisi convulsive motorie computabili
• Risposta al trattamento di =25%, =75% o riduzione del 100% delle crisi convulsive motorie computabili
• Impressione clinica globale del cambiamento (CGIC) ad ogni visita dello studio
• Impressione globale del cambiamento (GIC) del genitore/della persona che assiste il soggetto a ogni visita dello studio
• Variazione rispetto al basale dell’impressione clinica globale della severità del genitore/tutore
Altri endpoint secondari (periodo di mantenimento):
- Variazione percentuale dal basale della frequenza delle crisi convulsive a 28 giorni delle le crisi convulsive motorie computabili
- Risposta al trattamento di =25%, =50%, =75%, o riduzione del 100% per le crisi convulsive motorie computabili

E.5.2.1

Timepoint(s) of evaluation of this end point

Time Frame: Baseline, Treatment Period: Day 1 to Week 16.
Other secondary endpoints (maintenance period):
Timeframe: Maintenance Period: Week 7 to Week 16

Finestra temporale: Basale, periodo di trattamento: Dal Giorno 1 alla Settimana 16.
Altri endpoint secondari (periodo di mantenimento): Finestra temporale: Periodo di mantenimento: Dalla Settimana 7 alla Settimana 16

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

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

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

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

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)

E.8.2.2

Placebo

Yes

E.8.2.3

Other

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

Information not present in EudraCT

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

Australia

Belgium

Brazil

Canada

Czechia

Denmark

France

Germany

Italy

Netherlands

New Zealand

Poland

Portugal

Spain

United Kingdom

United States

E.8.7

Trial has a data monitoring committee

Yes

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.1.1.1

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)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

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

Yes

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

The study population may include adult subjects who are not capable of providing consent due to developmental delay and cognitive impairment common in patients with SCN8A-DEE.

La popolazione in studio può includere soggetti adulti che non sono in grado di fornire il consenso a causa del ritardo dello sviluppo e del deterioramento cognitivo comuni nei pazienti con SCN8A-DEE.

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)

Normal treatment as discussed with their doctor or eligible subjects will have the option to enrol into an extension study to receive NBI-921352.

Il trattamento normale come discusso con il proprio medico oppure i soggetti idonei, avranno la possibilità di iscriversi a uno studio di estensione per ricevere NBI-921352.

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

2021-10-27

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-02-08

P. End of Trial
P.	End of Trial Status	Ongoing

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