Clinical Trials Register

Summary
EudraCT Number:	2016-004130-24
Sponsor's Protocol Code Number:	M15-572
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:	2021-06-15
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

2016-004130-24

A.3

Full title of the trial

A Phase 3, Randomized, Double-Blind, Study Comparing Upadacitinib (ABT-494) to Placebo and to Adalimumab in Subjects with Active Psoriatic Arthritis Who Have a History of Inadequate Response to at Least One Non-Biologic Disease Modifying Anti-Rheumatic Drug (DMARD) ¿ SELECT ¿ PsA 1

Sperimentazione di Fase 3, Randomizzata ed in Doppio Cieco per Confrontare Upadacitinib (ABT-494) rispetto a Placebo e rispetto ad Adalimumab in Soggetti Affetti da Artrite Psoriasica in Fase Attiva con una Storia di Risposta Inadeguata ad almeno un Farmaco Antireumatico Modificante la Malattia (DMARD) Non Biologico ¿ SELECT ¿ PsA 1.

A.3.1

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

A Study Comparing ABT-494 to Placebo and to Adalimumab in Subjects with Psoriatic Arthritis Who Have an Inadequate Response to at Least One Non-Biologic Disease Modifying Anti-Rheumatic Drug

Sperimentazione per Confrontare ABT-494 rispetto a Placebo e rispetto ad Adalimumab in Soggetti Affetti da Artrite Psoriasica con una Storia di Risposta Inadeguata ad almeno un Farmaco Antireumatico Modificante la Malattia Non Biologico.

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

M15-572

A.5.4

Other Identifiers

Name:

Number:

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	ABBVIE DEUTSCHLAND GMBH & CO. KG
B.1.3.4	Country	Germany
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	AbbVie 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	AbbVie Ltd
B.5.2	Functional name of contact point	EU Clinical Trials Helpdesk
B.5.3	Address:
B.5.3.1	Street Address	AbbVie House, Vanwall Business Park, Vanwall Road
B.5.3.2	Town/ city	Maidenhead, Berkshire
B.5.3.3	Post code	SL6 4UB
B.5.3.4	Country	United Kingdom
B.5.4	Telephone number	441628561090
B.5.5	Fax number	441628461153
B.5.6	E-mail	eu-clinical-trials@abbvie.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	ABT-494
D.3.2	Product code	[ABT-494]
D.3.4	Pharmaceutical form	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	Upadacitinib
D.3.9.1	CAS number	1310726-60-3
D.3.9.2	Current sponsor code	ABT-494
D.3.9.4	EV Substance Code	SUB125895
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	Yes
D.3.11.13.1	Other medicinal product type	Janus Kinase (Jak) 1 inhibitor
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	ABT-494
D.3.2	Product code	ABT-494
D.3.4	Pharmaceutical form	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	Upadacitinib
D.3.9.1	CAS number	1310726-60-3
D.3.9.2	Current sponsor code	ABT-494
D.3.9.4	EV Substance Code	SUB125895
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	15
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	Yes
D.3.11.13.1	Other medicinal product type	Janus Kinase (Jak) 1 inhibitor
D.IMP: 3
D.1.2 and D.1.3	IMP Role	Comparator
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	Humira 40mg solution for injection in pre-filled syringe
D.2.1.1.2	Name of the Marketing Authorisation holder	AbbVie Ltd.
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	na
D.3.2	Product code	[na]
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	ADALIMUMAB
D.3.9.1	CAS number	331731-18-1
D.3.9.2	Current sponsor code	NA
D.3.9.4	EV Substance Code	SUB20016
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	40
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	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)	Yes
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	Yes
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	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	Solution for infusion
D.8.4	Route of administration of the placebo	Subcutaneous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Moderately to Severely Active Psoriatic Arthritis

Artrite Psoriasica in Fase Attiva e di Grado da Moderato a Severo

E.1.1.1

Medical condition in easily understood language

Psoriatic Arthritis

Artrite psoriasica

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	21.0
E.1.2	Level	LLT
E.1.2	Classification code	10037160
E.1.2	Term	Psoriatic arthritis
E.1.2	System Organ Class	100000004859

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

Primary Objective
1. To compare the efficacy of ABT-494 15 mg QD and 30 mg QD versus placebo and versus adalimumab (ADA) for the treatment of signs and
symptoms of PsA in subjects with moderately to severely active PsA who have an inadequate response or intolerance to 1 or more non-biologic
DMARD (DMARD-IR).
Secondary Objective
2. To compare the efficacy of ABT-494 15 mg QD and 30 mg QD versus placebo for the prevention of structural progression in subjects with moderately to severely active PsA who have an inadequate response or intolerance to 1 or more non-biologic DMARD (DMARD-IR).
3. To compare the safety and tolerability of ABT-494 15 mg QD and 30 mg QD versus placebo and versus adalimumab in subjects with moderately to severely active PsA who have an inadequate response or intolerance to 1 or more non-biologic DMARD (DMARD-IR).

Obiettivo Principale
1. Confrontare l¿efficacia di ABT-494 15 mg QD e 30 mg QD rispetto a placebo e rispetto ad adalimumab (ADA) per il trattamento dei segni e sintomi di PsA in soggetti affetti da PsA in fase attiva e di grado da moderato a grave e con risposta inadeguata o intolleranza a uno o pi¿ DMARD (DMARD-IR) non biologici.
Obiettivo Secondario
2. Confrontare l¿efficacia di ABT-494 15 mg QD e 30 mg QD rispetto a placebo per la prevenzione della progressione strutturale in soggetti con PsA in fase attiva e di grado da moderato a grave, che hanno avuto una risposta inadeguata o intolleranza a uno o pi¿ DMARD (DMARD-IR) non biologici.
3. Confrontare la sicurezza e la tollerabilit¿ di ABT-494 15 mg QD e 30 mg QD rispetto a placebo e rispetto ad adalimumab in soggetti con PsA in fase attiva di grado da moderato a grave che hanno avuto una risposta inadeguata o intolleranza a uno o pi¿ DMARD non biologici (DMARD-IR).

E.2.2

Secondary objectives of the trial

To evaluate the long-term safety, tolerability and efficacy of ABT-494 15 mg QD and 30 mg QD in subjects with PsA who have completed Period 1.

Valutare nel lungo termine la sicurezza, la tollerabilit¿ e l¿efficacia di ABT-494 15 mg QD e 30 mg QD in soggetti con PsA che abbiano completato il Periodo 1.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Male or female, at least 18 years of age
2. Diagnosed with psoriatic arthritis with symptom onset at least 6 months prior to the Screening Visit and fulfillment of the Classification Criteria for PsA (CASPAR)
3. Subject has active disease at Baseline defined as = 3 tender joints (based on 68 joint counts) and = 3 swollen joints (based on 66 joint counts) at Screening and Baseline Visits
4. Diagnosis of active plaque psoriasis or documented history of plaque psoriasis
5. Subject is on current treatment with = 2 nonbiologic DMARDs
6. Subject has had an inadequate response to previous or current treatment with at least 1 non-biologic DMARD (MTX, SSZ, LEF,cyclosporine, apremilast, bucillamine, or iguratimod), or subject has an intolerance to or contraindication for DMARDs
7. Presence of either at Screening:
• = 1 erosion on x-ray
• hs-CRP > laboratory-defined upper limit of normal (ULN)

1. Soggetti adulti di ambo i sessi, di età = 18 anni allo Screening
2. Diagnosi clinica di PsA, con esordio dei sintomi almeno 6 mesi prima della Visita di Screening e conformità con i criteri CASPAR (Classification Criteria for PsA).
3. Soggetto con malattia in fase attiva al Baseline definita in base alla presenza di = 3 articolazioni dolenti alla palpazione (su conta totale di 68 articolazioni) e = 3 articolazioni tumefatte (su conta totale di 66 articolazioni) alle Visite di Screening e Baseline
4. Diagnosi di psoriasi a placche in fase attiva oppure storia documentata di psoriasi a placche.
5. il soggetto deve essere in trattamento con = 2 DMARD non biologici
6. Soggetto che ha avuto una risposta inadeguata al trattamento pregresso o in atto con almeno un DMARD non biologico (MTX,SSZ, LEF, ciclosporine, apremilast, bucillamina o iguratimod), oppure soggetto che presenta intolleranza o controindicazione ai DMARD.
7. Presenza allo Screening di uno dei seguenti criteri:
- = 1 erosione radiografica;
- valore di hs-CRP > al limite superiore della norma (ULN) stabilito dal laboratorio.

E.4

Principal exclusion criteria

1. Prior exposure to any Janus Kinase (JAK) inhibitor
2. Current treatment with > 2 non-biologic DMARDs; or use of DMARDs other than MTX, SSZ, LEF, apremilast, HCQ, bucillamine, or iguratimod; or use of MTX in combination with LEF.

1. Pregressa esposizione a qualsiasi inibitore delle proteine Janus chinasi (JAK)
2. Trattamento in atto con > 2 DMARD non biologici; oppure utilizzo di DMARD diversi da MTX, SSZ, LEF, apremilast, HCQ, bucillamina o iguratimod; oppure utilizzo di MTX in combinazione con LEF.

E.5 End points

E.5.1

Primary end point(s)

The proportion of subjects achieving ACR20 response

Percentuale di soggetti che ottengono la risposta ACR20

E.5.1.1

Timepoint(s) of evaluation of this end point

12 weeks

alla settimana 12.

E.5.2

Secondary end point(s)

1. Change from baseline in HAQ-DI
2. Proportion of subjects achieving a static Investigator Global Assessment (sIGA) of Psoriasis of 0 or 1 and at least a 2-point
improvement from baseline
3. Psoriasis Area Severity Index (PASI) 75 response (for subjects with = 3% BSA psoriasis at baseline)
4. Change from baseline in modified PsA Sharp/van der Heijde Score (SHS)
5. Proportion of subjects achieving Minimal Disease Activity (MDA)
6. Proportion of subjects with resolution of enthesitis (LEI = 0)
7. ACR 20 response rate (non-inferiority of ABT-494 vs adalimumab);
8. Change from baseline in SF-36 PCS
9. Change from baseline in FACIT-Fatigue Questionnaire at Week 12;
10. ACR 20 response rate (superiority of ABT-494 vs adalimumab);
11. Proportion of subjects with resolution of dactylitis (LDI = 0)
12. Change from baseline in Patient's Assessment of Pain NRS
(superiority of ABT-494 vs. adalimumab);
13. Change from baseline in HAQ-DI (superiority of ABT-494 vs.
adalimumab);
14. Change from baseline in Self-Assessment of Psoriasis Symptoms
(SAPS) Questionnaire
15. ACR 50/70 response rate
16. ACR 20 response rate

1. Variazione rispetto al baseline del punteggio HAQ-DI
2. Percentuale di soggetti che ottengono un punteggio sIGA (static Investigator Global Assessment) relativo alla Psoriasi pari a 0 o 1 ed un miglioramento di almeno 2 punti rispetto al baseline
3. Risposta PASI 75 (Psoriasis Area Severity Index) per soggetti con interessamento psoriasico = 3% del BSA al baseline
4. Variazione rispetto al baseline del punteggio SHS
5. Percentuale di soggetti che ottengono la risposta MDA
6. Variazione rispetto al baseline del punteggio LEI
7. Tasso di risposta ACR 20 alla Settimana 12 (non inferiorità di ABT-494 rispetto ad adalimumab);
8. Variazione rispetto al baseline del punteggio SF-36 PCS rilevato alla Settimana 12;
9. Variazione rispetto al baseline del FACIT-Fatigue questionnaire rispetto alla settimana 12;
10. Tasso di risposta ACR 20 alla Settimana 12 (superiorità di ABT-494 rispetto ad adalimumab);
11. Percentuale di soggetti con risoluzione della dattilite (LDI=0) alla settimana 24:
12. Variazione rispetto al baseline della valutazione del dolore da parte del paziente utilizzando la scala numerica NRS alla Settimana 12 (superiorità di ABT-494 rispetto ad adalimumab);
13. Variazione rispetto al baseline del punteggio HAQ-DI rilevato alla Settimana 12 (superiorità di ABT-494 rispetto ad adalimumab);
14. Variazione rispetto al baseline del punteggio SAPS (Self-Assessment of Psoriasis Symptoms) alla Settimana 16.
15. Risposta ACR50/70;
16. Risposta ACR20;

E.5.2.1

Timepoint(s) of evaluation of this end point

1. Week 12
2. Week 16
3. Week 16
4. Week 24
5. Week 24
6. Week 24
7. Week 12
8. Week 12
9. Week 12
10. Week 12
11. Week 24
12. Week 12
13. Week 12
14. Week 16
15. Week 12
16. Week 2; 1. alla settimana 12
2. alla settimana 16
3. alla settimana 16
4. alla settimana 24
5. alla settimana 24
6. alla settimana 24
7. alla settimana 24
8. alla settimana 12
9. alla settimana 12
10. alla settimana 12
11.alla settimana 24
12. alla settimana 12
13. alla settimana 12
14. alla settimana 16
15. alla settimana 12
16. alla settimana 2

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

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

Yes

E.6.10

Pharmacogenetic

Yes

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)

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)

Yes

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

Belarus

Bosnia and Herzegovina

Brazil

Canada

Chile

China

Colombia

Egypt

Hong Kong

Israel

Japan

Korea, Democratic People's Republic of

Korea, Republic of

Malaysia

Mexico

New Zealand

Puerto Rico

Russian Federation

Serbia

Singapore

South Africa

Taiwan

Turkey

Ukraine

United States

Switzerland

Argentina

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

Last subjects' last visit or the actual date of follow-up contact, whichever is later.

LVLS o la data attuale del contatto di follow-up, quale avvenga dopo

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

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)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

1468

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

237

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

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

661

F.4.2.2

In the whole clinical trial

1705

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)

Subjects who complete the last visit will have a follow-up visit approximately 30 days after the last dose of oral study drug. A followup phone call will also occur 70 days after the last administration of
injectable study drug. Subjects completing the study on open label (OL) therapy will only be required to have the 30 day follow-up visit if on oral study drug and the 70 day follow-up phone call if on injectable study drug.

I soggetti che completano l'ultima visita avranno una visita di follow-up circa 30 giorni dopo l'ultima dose di farmaco per via orale. Una chiamata telefonica di follow-up avverr¿ anche 70 giorni dopo l'ultima somministrazione di farmaco iniettabile. I soggetti che completano lo studio sulla terapia in aperto (OL) riceveranno la visita di follow up dopo 30 giorni in caso di somministrazione del farmaco per via orale e riceveranno la chiamata di follow up dopo 70 giorni in caso di somministra

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

2017-09-19

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2017-11-15

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

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IMP with orphan designation in the indication
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