Clinical Trials Register

Summary
EudraCT Number:	2019-002787-27
Sponsor's Protocol Code Number:	20190530
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:	2021-06-07
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

2019-002787-27

A.3

Full title of the trial

A Phase 3, Multicenter, Double-blind, Randomized, Placebo-controlled,Parallel Group Study, Followed by an Active Treatment Phase to Evaluate the Efficacy and Safety of Apremilast in Children From 2 to Less Than 18 Years of Age With Active Oral Ulcers Associated With Behçet's Disease (BEAN)

Studio di fase 3, multicentrico, in doppio cieco, randomizzato, controllato verso placebo, a gruppi paralleli, seguito da una fase di trattamento attivo volto a valutare l'efficacia e la sicurezza di apremilast in bambini di età compresa tra 2 e meno di 18 anni con ulcere orali in fase attiva associate alla malattia di Behçet (BEAN)

A.3.1

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

Apremilast study in children with active oral ulcers associated with Behçet's Disease

Studio con apremilast in bambini con ulcere orali in fase attiva associate alla malattia di Behçet (BEAN)

A.3.2

Name or abbreviated title of the trial where available

BEAN

A.4.1

Sponsor's protocol code number

20190530

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/389/2019

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	AMGEN 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	Amgen 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	Amgen S.r.l.
B.5.2	Functional name of contact point	Medical Information
B.5.3	Address:
B.5.3.1	Street Address	Via Tazzoli 6
B.5.3.2	Town/ city	Milano
B.5.3.3	Post code	20154
B.5.3.4	Country	Italy
B.5.4	Telephone number	0039026241121
B.5.5	Fax number	0039026241121
B.5.6	E-mail	medicalinformationitaly@amgen.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.2.1.1.1	Trade name	OTEZLA - 10 MG + 20 MG + 30 MG COMPRESSA RIVESTITA CON FILM USO ORALE - BLISTER (PVC/ALU) IN UN ASTUCCIO - 4 COMPRESSE DA 10 MG + 4 COMPRESSE DA 20 MG +19 COMPRESSE DA 30 MG
D.2.1.1.2	Name of the Marketing Authorisation holder	AMGEN EUROPE B.V
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	Apremilast
D.3.2	Product code	[AMG407]
D.3.4	Pharmaceutical form	Film-coated tablet
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	Apremilast
D.3.9.1	CAS number	608141-41-9
D.3.9.2	Current sponsor code	AMG407
D.3.9.4	EV Substance Code	SUB130837
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: 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	Yes
D.2.1.1.1	Trade name	OTEZLA - 10 MG + 20 MG + 30 MG COMPRESSA RIVESTITA CON FILM USO ORALE - BLISTER (PVC/ALU) IN UN ASTUCCIO - 4 COMPRESSE DA 10 MG + 4 COMPRESSE DA 20 MG +19 COMPRESSE DA 30 MG
D.2.1.1.2	Name of the Marketing Authorisation holder	AMGEN EUROPE B.V
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	Apremilast
D.3.2	Product code	[AMG407]
D.3.4	Pharmaceutical form	Film-coated tablet
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	Apremilast
D.3.9.1	CAS number	608141-41-9
D.3.9.2	Current sponsor code	AMG407
D.3.9.4	EV Substance Code	SUB130837
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: 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	Yes
D.2.1.1.1	Trade name	OTEZLA - 10 MG + 20 MG + 30 MG COMPRESSA RIVESTITA CON FILM USO ORALE - BLISTER (PVC/ALU) IN UN ASTUCCIO - 4 COMPRESSE DA 10 MG + 4 COMPRESSE DA 20 MG +19 COMPRESSE DA 30 MG
D.2.1.1.2	Name of the Marketing Authorisation holder	AMGEN EUROPE B.V
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	Apremilast
D.3.2	Product code	[AMG 407]
D.3.4	Pharmaceutical form	Film-coated tablet
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	Apremilast
D.3.9.1	CAS number	608141-41-9
D.3.9.2	Current sponsor code	AMG 407
D.3.9.4	EV Substance Code	SUB130837
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	30
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	Apremilast
D.3.2	Product code	[AMG407]
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	Apremilast
D.3.9.1	CAS number	608141-41-9
D.3.9.2	Current sponsor code	AMG 407
D.3.9.4	EV Substance Code	SUB130837
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	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	Yes
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	Film-coated tablet
D.8.4	Route of administration of the placebo	Oral use
D.8 Placebo: 2
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Film-coated tablet
D.8.4	Route of administration of the placebo	Oral use
D.8 Placebo: 3
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Film-coated tablet
D.8.4	Route of administration of the placebo	Oral use
D.8 Placebo: 4
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Oral suspension
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

Subjects with active Behçet's Disease

Soggetti con malattia di Behçet attiva

E.1.1.1

Medical condition in easily understood language

Behçet's Disease causes inflammation in blood vessels throughout the body and manifests mainly as mouth or genital ulcers with eye and skin lesions.

La malattia di Behçet causa infiammazione dei vasi sanguigni in tutto il corpo e si manifesta principalmente come ulcere orali o genitali con lesioni agli occhi e alla pelle

E.1.1.2

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	LLT
E.1.2	Classification code	10004212
E.1.2	Term	Behcet's disease
E.1.2	System Organ Class	100000004866

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 estimate the efficacy of apremilast in pediatric subjects from 2 to less then 18 years of age with oral ulcers associated with Behçet's Disease (BD)

Valutare l’efficacia di apremilast in soggetti pediatrici di età compresa tra 2 e < 18 anni con ulcere orali associate alla malattia di Behçet (BD)

E.2.2

Secondary objectives of the trial

- Estimate other measures of the efficacy of apremilast for oral ulcers in pediatric subjects (2 to less 18 years);
- Estimate efficacy of apremilast in the treatment of genital ulcers in pediatric subjects with oral ulcers;
- Estimate efficacy of apremilast for overall BD related disease activity in pediatric subjects with oral ulcers;
- Estimate effect of apremilast on BD manifestations (other than oral and genital ulcers) in pediatric subjects with oral ulcers;
- Estimate effect of apremilast on health-related quality of life (HRQoL) in pediatric subjects with oral ulcers;
- Estimate safety and tolerability of apremilast, in pediatric subjects with oral ulcers;
- Characterize the pharmacokinetics of apremilast in pediatric subjects with oral ulcers;

*Please refer to the protocol for the full list

- Valutare altre misure di efficacia di apremilast in soggetti pediatrici di età compresa tra 2 e < 18 anni con ulcere orali associate alla malattia di Behçet (BD);
- Valutare l'efficacia di apremilast nel trattamento delle ulcere genitali in soggetti pediatrici con ulcere orali;
- Valutare l'efficacia di apremilast per l'attività complessiva della malattia correlata alla malattia di Behçet in soggetti pediatrici con ulcere orali;
- Valutare l'effetto di apremilast sulle manifestazioni di BD (diverse dalle ulcere orali e genitali) in soggetti pediatrici con ulcere orali;
- Valutare l'effetto di apremilast sulla qualità di vita correlata alla salute (HRQoL) in soggetti pediatrici con ulcere orali;
- Valutare la sicurezza e la tollerabilità di apremilast, in soggetti pediatrici con ulcere orali;
- Caratterizzare la farmacocinetica di apremilast in soggetti pediatrici con ulcere orali;

*Fare riferimento al protocollo per la lista completa

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Male or Female subjects 2 to less than 18 years of age at randomization.
2. Diagnosed with BD meeting the ISGBD criteria at any time prior to the screening visit.
3. Oral ulcers that occurred more or equal to 3 times within the 12-month period prior to the screening visit.
4. Subject must have more or equal to 2 oral ulcers at both the screening visit and on day 1.
5. Subject has had prior treatment with >= 1 non-biologic BD therapy, such as, but not limited to, topical corticosteroids or systemic treatment.
6. Subject is a candidate for systemic therapy for the treatment of oral ulcers.

*Please refer to the protocol for the full list

1. Soggetti di sesso maschile o femminile di età compresa tra 2 e < 18 anni al momento della randomizzazione
2. Diagnosi di BD che soddisfa i criteri ISGBD in qualsiasi momento prima della visita di screening
3. Ulcere orali che si sono verificate 3 volte o più nei 12 mesi precedenti la visita di screening
4. Il soggetto deve avere 2 o più ulcere orali sia alla visita di screening che al giorno 1.
5. Il soggetto ha avuto un precedente trattamento con >= 1 terapia non biologica per BD, come, ma non limitato a, corticosteroidi topici o trattamento sistemico.
6. Il soggetto è un candidato per la terapia sistemica per il trattamento delle ulcere orali.

*Fare riferimento al protocollo per la lista completa

E.4

Principal exclusion criteria

1. Behçet's disease-related active major organ involvement – pulmonary (eg, pulmonary artery aneurysm), vascular (eg, thrombophlebitis), gastrointestinal (eg, ulcers along the gastrointestinal tract), and CNS (eg, meningoencephalitis) manifestations, and ocular lesions (eg, uveitis) requiring immunosuppressive therapy; however:
- Previous major organ involvement is allowed if it occurred >= 1 year prior to the screening visit and is not active at time of enrollment;
- Subjects with mild BD-related ocular lesions not requiring systemic immunosuppressive therapy are allowed;
- Subjects with BD-related arthritis and BD-skin manifestations are also allowed.
2. Subjects with BD-related arthritis and BD-skin manifestations are also allowed. Previous exposure to biologic therapies for the treatment of BD
oral ulcers, previous biologic exposure is allowed for other indications (including other manifestations of BD).
3. History or evidence of any other clinically significant disorder, condition or disease that could pose a risk to subject safety or interfere with the study evaluation, procedures or completion.
4. Female subject who is (or plans to become) pregnant or breastfeeding.
5. Female subject of childbearing potential unwilling to use 1 highly effective method of contraception.

*Please refer to the protocol for the full list

1. Coinvolgimento degli organi principali nella malattia di Behçet attiva - manifestazioni a livello polmonare (es, aneurisma delle arterie polmonari), vascolare (es, tromboflebite), gastrointestinale (es, ulcere del tratto gastrointestinale, e del SNC (es, meningoencefalopatia) e lesioni oculari (es, uveite) che richiedono comunque una terapia immunosoppressiva:
- Il precedente coinvolgimento di organi principali è consentito se si è verificato >= 1 anno prima della visita di screening e non è attivo al momento dell'arruolamento;
- Sono ammessi soggetti con lievi lesioni oculari correlate alla BD che non richiedono terapia immunosoppressiva sistemica;
- Sono ammessi anche soggetti con artrite correlata a BD e manifestazioni cutanee BD.
2. Sono ammessi anche soggetti con artrite e manifestazioni cutanee correlate a BD. La precedente esposizione a terapie biologiche per il trattamento di ulcere orali correlati a BD, è consentita una precedente esposizione biologica per altre indicazioni (comprese altre manifestazioni di BD).
3. Storia o evidenza di qualsiasi altro disturbo, condizione o malattia clinicamente significativa che potrebbe rappresentare un rischio per la sicurezza dei soggetti o interferire con la valutazione, le procedure o il completamento dello studio.
4. Soggetto di sesso femminile che è (o sta pianificando) una gravidanza o sta allattando.
5. Soggetto di sesso femminile in età fertile che non desidera utilizzare 1 metodo contraccettivo altamente efficace.

*Fare riferimento al protocollo per la lista completa

E.5 End points

E.5.1

Primary end point(s)

- Area under the curve (AUC) for the number of oral ulcers

- Area sotto la curva (AUC) per il numero di ulcere orali

E.5.1.1

Timepoint(s) of evaluation of this end point

from baseline (week 0) to week 12

dal basale alla settimana 12

E.5.2

Secondary end point(s)

- Number of oral ulcers (a);
- Change in the pain of oral ulcers as measured by a visual analog scale (VAS) (a);
- Complete response rate for oral ulcers (defined as the proportion of subjects who are oral ulcer free) (b);
- Proportion of subjects whose number of oral ulcers is reduced by more or equal to 50% (a);
- Complete response rate for genital ulcers (defined as the proportion of subjects who are genital ulcer free) (b);
- Change in disease activity as measured by Behçet's Disease Current Activity score (a);
- Proportion of subjects who have new-onset or recurrence of Behçet'srelated manifestations (other than oral and genital ulcers) (b);
- Change on the Short form Survey (SF-10) (a)

*Please refer to the protocol for the full list

- Numero di ulcere orali (a);
- Variazione del dolore delle ulcere orali misurata con una scala analogica visiva (VAS) (a);
- Percentuale di risposta completa per ulcere orali (definita come la proporzione di soggetti privi di ulcera orale) (b);
- Proporzione di soggetti il ¿¿cui numero di ulcere orali è ridotto di più o del 50% (a);
- Tasso di risposta completa per le ulcere genitali (definito come la proporzione di soggetti che sono liberi da ulcera genitale) (b);
- Variazione dell'attività della malattia misurata dal punteggio dell'attività corrente della malattia di Behçet (a);
- Proporzione di soggetti che hanno nuova insorgenza o recidiva delle manifestazioni correlate a Behçet (diverse dalle ulcere orali e genitali) (b);
- Modifica sul sondaggio in forma breve (SF-10) (a)

*Fare riferimento al protocollo per la lista completa

E.5.2.1

Timepoint(s) of evaluation of this end point

(a) from baseline (week 0) to week 12
(b) at week 12
(c) from baseline (week 0) to week 56
(d) from baseline (week 0) to week 52
(e) from week 2 and to week 52
(f) at baseline (week 0) and at week 2

(a) dal basale (settimana 0) alla settimana 12
(b) settimana 12
(c) dal basale (settimana 0) alla settimana 56
(d) dal basale (settimana 0) alla settimana 56
(e) dalla settimana 2 alla settimana 52
(f) al basale (settimana 0) e alla settimana 52

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

Yes

E.6.13.1

Other scope of the trial description

Taste and Acceptability

Sapore e accettabilità

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)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

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

Israel

Switzerland

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)

Yes

F.1.1.5.1

Number of subjects for this age range:

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)

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

Subjects under age incapable of giving consent personally.

Soggetti minorenni incapaci di dare il consenso personalmente.

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)

All subjects who complete the week 52 visit and all assessments at this visit will have the opportunity to enroll in a separate optional openlabel, long-term study. Subjects may continue in the optional openlabel, long-term study for 4 years or until apremilast is commercially available for BD (whichever comes earlier) or until the benefit/risk of apremilast is found not to be acceptable for BD, according to either the Sponsor or health authority.

Tutti i pz che completano la visita alla settimana 52 e tutte le valutazioni avranno la possibilità di essere arruolati in uno studio a lungo termine in aperto separato e opzionale. I soggetti possono continuare nello studio a lungo termine in aperto separato e opzionale per 4 anni o fino a quando apremilast sarà disponibile sul mercato per la BD o fino a quando il beneficio/rischio di apremilast non viene considerato accettabile per la BD, secondo sponsor e autorità competente.

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

2021-04-20

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-05-24

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

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