Clinical Trials Register

Summary
EudraCT Number:	2018-002823-41
Sponsor's Protocol Code Number:	AC-065B302
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2021-06-14
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

2018-002823-41

A.3

Full title of the trial

A multicenter, randomized, double-blind, placebo-controlled, parallelgroup,group-sequential, adaptive, Phase 3 study with open-label extension period to assess the efficacy and safety of selexipag as an addon
to standard of care therapy in subjects with inoperable or persistent/recurrent after surgical and/or interventional treatment ChronicThromboembolic Pulmonary Hypertension.

Studio di Fase 3, adattativo, a gruppi paralleli e coorti sequenziali, controllato con placebo, in doppio cieco, randomizzato, multicentrico, con periodo di estensione in aperto, per valutare l’efficacia e la sicurezza di selexipag in aggiunta alla terapia con standard di cura, in soggetti con ipertensione polmonare cronica tromboembolica inoperabile o persistente/recidivante dopo trattamento chirurgico e/o interventistico.

A.3.1

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

A clinical study to assess the efficacy and safety of selexipag as an add-on to standard of care therapy in subjects with inoperable or persistent/recurrent, after surgical treatment and/or Chronic
Thromboembolic Pulmonary Hypertension (CTEPH).

Studio clinico per valutare l'efficacia e la sicurezza di selexipag in aggiunta alla terapia con standard di cura in soggetti con ipertensione polmonare cronica tromboembolica inoperabile o persistente/recidivante dopo trattamento chirurgico e/o interventistico

A.3.2

Name or abbreviated title of the trial where available

SELECT

A.4.1

Sponsor's protocol code number

AC-065B302

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	ACTELION PHARMACEUTICALS LTD
B.1.3.4	Country	Switzerland
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	Actelion Pharmaceuticals Ltd
B.4.2	Country	Switzerland
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Actelion Pharmaceuticals Ltd
B.5.2	Functional name of contact point	Clinical Trial Disclosure Desk
B.5.3	Address:
B.5.3.1	Street Address	Gewerbestrasse, 16
B.5.3.2	Town/ city	Allschwil
B.5.3.3	Post code	4123
B.5.3.4	Country	Switzerland
B.5.6	E-mail	clinical-trials-disclosure@its.jnj.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	Uptravi
D.2.1.1.2	Name of the Marketing Authorisation holder	Janssen-Cilag International NV
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	SELEXIPAG
D.3.2	Product code	[ACT-293987]
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	SELEXIPAG
D.3.9.2	Current sponsor code	ACT-293987
D.3.9.4	EV Substance Code	SUB130805
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	200
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	Film-coated tablet
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

Chronic Thromboembolic Pulmonary Hypertension, inoperable or persistent/recurrent

Ipertensione polmonare cronica tromboembolica inoperabile o persistente/recidivante

E.1.1.1

Medical condition in easily understood language

Chronic Thromboembolic Pulmonary Hypertension (CTEPH) is a disease associated with abnormally high pressure in the blood vessels of the lungs, caused by the formation of blood clots.

L'ipertensione polmonare cronica tromboembolica è una malattia associata a una pressione anormalmente elevata nei vasi sanguigni dei polmoni, causata dalla formazione di coaguli di sangue.

E.1.1.2

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

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	10068739
E.1.2	Term	Chronic thromboembolic pulmonary hypertension
E.1.2	System Organ Class	100000004855

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

The primary objective of the study is to evaluate the effect of selexipag on pulmonary vascular resistance (PVR) versus placebo in subjects with inoperable CTEPH (i.e., technically non-operable) and persistent/recurrent CTEPH after surgical (pulmonary endarterectomy [PEA]) and/or interventional (balloon pulmonary angioplasty [BPA]) treatment at Week 20.

Valutare l’effetto di selexipag sulla resistenza vascolare polmonare (PVR) rispetto al placebo in soggetti con CTEPH inoperabile (cioè, tecnicamente non operabile) e CTEPH persistente/recidivante dopo trattamento chirurgico (endoarteriectomia polmonare [PEA]) e/o trattamento interventistico (angioplastica polmonare a palloncino [BPA]) alla Settimana 20.

E.2.2

Secondary objectives of the trial

The secondary objectives of the study are to evaluate the effects of
selexipag versus placebo on:
• Exercise capacity.
• Time to clinical worsening (TTCW).
• All-cause death or hospitalizations related to PH worsening.
• WHO FC.
• Patient-reported outcomes (PROs).
• Dyspnea.
• N-terminal pro b-type natriuretic peptide (NT-proBNP).

Valutare gli effetti di selexipag rispetto al placebo su:
• Capacità di esercizio fisico.
• Tempo al peggioramento clinico
• Tutte le cause di decesso od ospedalizzazioni correlate al peggioramento dell’ipertensione polmonare (PH).
• Classe funzionale OMS (WHO-FC).
• Esiti riferiti dal paziente.
• Dispnea.
• Pro-peptide natriuretico di tipo B N-terminale (NT-proBNP).

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Signed and dated ICF.
2. Male and female subjects >or=18 (or the legal age of consent in the jurisdiction in which the study is taking place) < or= 85 years old at Screening (Visit 1).
3. Subjects with diagnosis of CTEPH and inoperability confirmed by the corresponding adjudication
committee (AC; country-specific adjudication committee [CSAC] or central adjudication committee [CAC] (see Section 3.4.3), defined as one of the following options:
a) Inoperable CTEPH (ie., technically non-operable) with:
– Diagnosis of CTEPH based on at least two of the following assessments
performed in the 14-month period prior to randomization (Visit 2):
ventilation/perfusion (V/Q) scan; pulmonary angiography (PA);
computed tomography pulmonary angiogram (CTPA) and/or magnetic
resonance angiography (MRA).
– RHC (and LHC, if needed) 1 performed at least 90 days after start of full anticoagulation, showing:
o PVR at rest >or=400 dyn.sec/cm5 or =5 Wood units for the hemodynamic cohort and PVR at rest >or=300 dyn.sec/cm5 or >or=3.75 Wood units for the
non-hemodynamic cohort,
o Mean pulmonary arterial pressure (mPAP) >or=25 mmHg;
Pulmonary arterial wedge pressure (PAWP) = 15 mmHg or, if not available or unreliable, a left ventricular end diastolic pressure (LVEDP) = 15 mmHg.
b) Persistent/recurrent CTEPH after BPA and deemed inoperable with:
– Diagnosis of CTEPH based on at least one of the following assessments performed in the 14-month period prior to randomization (Visit 2) and after last interventional (BPA) treatment: V/Q scan, PA, CTPA or MRA.
– RHC (and LHC, if needed)1 performed at least 90 days after last interventional (BPA) treatment and at least 90 days after start of full anticoagulation, showing: PVR at rest >or =400 dyn.sec/cm5 or >oe =5 Wood units for the hemodynamic cohort and PVR at rest >or=300 dyn.sec/cm5 or >or=3.75 Wood units for the non-hemodynamic cohort, mPAP >or= 25 mmHg; PAWP <or= 15 mmHg, or, if not available or unreliable, an LVEDP =
15 mmHg.
c) Persistent/recurrent CTEPH after PEA (including PEA followed by BPA) with: – Diagnosis of CTEPH based on at least one of the following assessments performed in the 14-month period prior to randomization (Visit 2) and after last surgical (PEA) or interventional (BPA) treatment: V/Q scan,PA, CTPA or MRA.
-RHC(and LHC, if needed)1 performed at least 90 days after last surgical (PEA) or interventional (BPA) treatment and at least 90 days after start of full anticoagulation, showing: PVR at rest >or= 400 dyn.sec/cm5 or 5 Wood units for the hemodynamic cohort and PVR at rest =300 dyn.sec/cm5 or =3.75 Wood units for the non-hemodynamic cohort,
o mPAP >or=25 mmHg,
o PAWP <or=15 mmHg, or, if not available or unreliable, an LVEDP <or=15 mmHg.
4. PH in WHO FC I–IV.
5. Subject able to perform the 6MWT with a minimum distance of 100 m and a maximum distance of 450 m at screening visit (Visit 1).
6. A woman of childbearing potential [see definition in Section 4.5.1] is eligible only if all the following applies:
a. Negative serum pregnancy test at Screening and a negative urine pregnancy test at randomization.
b. Agreement to undertake monthly urine pregnancy tests during the study and up to at least 30 days after study treatment discontinuation.
c. Agreement to use one of the methods of birth control described in Section 4.5 from Screening visit up to at least 30 days after study treatment discontinuation.

1. Modulo di consenso informato firmato e datato.
2. Soggetti di sesso maschile e femminile di età >o=18 (o che abbiano raggiunto la maggiore età, considerata tale, nei Paesi in cui si svolge lo studio) e <=85 anni anni allo screening (Visit1).
3. Soggetti con diagnosi di CTEPH, e inoperabilità confermate dal corrispondente comitato di revisione (AC; Comitato di revisione specifico per paese [CSAC] o dal Comitato di revisione centrale [CAC]) [vedere Sezione 3.4.3], definita come una delle seguenti opzioni:
a. CTEPH inoperabile (cioè, tecnicamente non operabile) con:
-Diagnosi di CTEPH basata su almeno due delle seguenti valutazioni eseguite nel periodo di 14 mesi precedente la randomizzazione (Visita 2):
oScintigrafia ventilatoria/perfusoria (scansione V/Q).
o Angiografia polmonare (PA), angiogramma polmonare con tomografia computerizzata (CTPA) e/o
o Angiografia con risonanza magnetica (MRA).
-RHC(e LHC, se necessario) 1 eseguito almeno 90 giorni dopo l’inizio della terapia anticoagulante completa, che evidenzia:
o PVR a riposo >o=400 dyn.s/cm5 o >o=5 Wood units per la coorte emodinamica e PVR a riposo >o=300 dyn.sec/cm5 o >o=3.75 Wood units per la coorte non-emodinamica..
o Pressione arteriosa polmonare media mPAP >o=25 mmHg.
o Pressione di incuneamento dell’arteria polmonare (PAWP) <o=15 mmHg o, se il valore non è disponibile o non affidabile, pressione telediastolica ventricolare sinistra (LVEDP) <o=15 mmHg.
b. CTEPH persistente/recidivante dopo BPA, e diventato inoperabile con:
-Diagnosi di CTEPH basata su almeno una delle seguenti valutazioni effettuate nel periodo di 14 mesi precedente la randomizzazione (Visita 2) e dopo l'ultimo trattamento interventistico (BPA): Scintigrafia ventilatoria/perfusoria (scansione V/Q), PA, CTPA o MRA.
-RHC1 (e LHC, se necessario)eseguito almeno 90 giorni dopo dopo l'ultimo trattamento interventistico (BPA): e almeno 90 giorni dopo l’inizio della terapia anticoagulante completa, che evidenzia:
o PVR a riposo >0=400 dyn.s/cm5 o >0= 5 Wood units per la coorte emodinamica e PVR a riposo >o=300 dyn.sec/cm5 o >o=3.75 Wood unitàs per la coorte non-emodinamica
o mPAP >o=25 mmHg.
o PAWP <o=15 mmHg o, se non disponibile o non affidabile, LVEDP <o=15 mmHg.
c. CTEPH persistente/recidivante dopo PEA (inclusa PEA seguita da BPA) con:
-Diagnosi di CTEPH basata su almeno una delle seguenti valutazioni eseguite nel periodo di 14 mesi prima della randomizzazione (Visita 2) e dopo l'ultimo trattamento chirurgico (PEA) o interventistico (BPA): scansione V/Q, PA, CTPA o MRA.
-RHC(e LHC, se necessario)1 eseguito almeno 90 giorni dopo l'ultimo trattamento chirurgico (PEA) o interventistico (BPA) e almeno 90 giorni dopo l’inizio della terapia anticoagulante completa, che evidenzia:
o PVR a riposo >0=400 dyn.s/cm5 o >0= 5 Wood units per la coorte emodinamica e PVR a riposo >o=300 dyn.sec/cm5 o >o=3.75 Wood units per la coorte non-emodinamica
o mPAP >o=25 mmHg.
o PAWP <o=15 mmHg o, se non disponibile o non affidabile, LVEDP =15 mmHg.
4. PH in FC OMS I-IV.
5. Soggetto capace di eseguire il 6MWT per una distanza minima di 100 m e una distanza massima di 450 m alla visita di screening (Visita 1).

E.4

Principal exclusion criteria

General exclusion criteria:
1. Planned or current treatment with another investigational treatment up to 3 months prior to randomization.
2. Any known factor or disease that might interfere with treatment compliance, study conduct, or interpretation of the results, such as drug or alcohol dependence or psychiatric disease.
3. Known concomitant life-threatening disease with a life expectancy < 12 months.
Exclusion criteria related to the disease:
4. Planned BPA within 26 weeks after randomization.
5. Change in dose or initiation of new PH-specific therapy within 90 days prior to the baseline RHC (and LHC, if needed) qualifying for enrollment for the hemodynamic cohort and within 90 days prior to randomization (Visit 2) for the non-hemodynamic cohort.
6. Treatment with prostacyclin (epoprostenol), prostacyclin analogs (ie, treprostinil, iloprost, beraprost) or prostacyclin receptor agonists (ie, selexipag/Uptravi®) within 90 days prior to randomization (Visit 2), except those given at vasodilator testing during RHC.
7. Change in dose or initiation of new diuretics and/or calcium channel blockers within 1 week prior to baseline RHC (and LHC, if needed).
Exclusion criteria related to comorbidities:
8. Any co-morbid condition that may influence the ability to perform a reliable and reproducible 6MWT, including use of walking aids (cane, walker, etc).
9. Other as per selexipag Summary of Product Characteristics (SmPC).
Exclusion criteria related to selexipag use:
10. As per selexipag Summary of Product Characteristics (SmPC).

Criteri di esclusione generali
1. Trattamento pianificato o in atto con un’altra terapia sperimentale fino a 3 mesi prima della randomizzazione.
2. Qualsiasi fattore o disturbo noto che potrebbe interferire con la compliance al trattamento, la conduzione dello studio o l’interpretazione dei risultati, come ad esempio la dipendenza da droghe o alcol o una malattia psichiatrica.
3. Concomitanza nota di malattia potenzialmente letale con un’aspettativa di vita <12 mesi.
Criteri di esclusione legati alla malattia
4. BPA pianificata entro 26 settimane dalla randomizzazione.
5. Modifica della dose o inizio di una nuova terapia specifica per la PH entro 90 giorni dall’ RHC eseguita alla visita di baseline (e LHC, se necessario) per l’arruolamento nella corte emodinamica ed entro i 90 giorni prima della randomizzazione (Visit 2) per la corte non-emodinamica .
6. Trattamento con prostaciclina (epoprostenolo), analoghi della prostaciclina (ad es. treprostinil, iloprost, beraprost) o agonisti del recettore della prostaciclina (cioè selexipag/Uptravi) entro 90 giorni dalla randomizzazione (Visita 2) eccetto per quelli somministrati nel test vasodilatazione durante l’RHC.
7. Modifica della dose o inizio di una nuova terapia con diuretici e/o bloccanti i canali del calcio entro 1 settimana prima della RHC basale (e LHC, se necessario).
Criteri di esclusione relativi alle comorbilità
8. Qualsiasi malattia concomitante che può influire sulla capacità di eseguire un test 6MWT affidabile e riproducibile, compreso l’utilizzo di ausili per camminare (bastone, deambulatore e così via).
9. Altro come da SmPC del Selexipag
Criteri di esclusione relativi all’uso di selexipag
10. Come da SmPC del Selexipag

E.5 End points

E.5.1

Primary end point(s)

The primary efficacy endpoint of this study is the pulmonary vascular
resistance (PVR) at Week 20, assessed at rest, within 2–5 hours postdose,
expressed as percent of baseline PVR.

L'endpoint primario di efficacia di questo studio è la resistenza vascolare polmonare
(PVR) alla settimana 20, valutata a riposo, entro 2-5 ore dopo la dose,
espressa come percentuale del PVR basale.

E.5.1.1

Timepoint(s) of evaluation of this end point

Assessed at rest in screening period (Day -60 to Day -14; baseline) and
assessed at rest within 2–5 hours post-dose at Week 20 during
treatment period.

Valutata a riposo nel periodo di screening (dal giorno -60 al giorno -14, basale) e
valutata a riposo entro 2-5 ore dopo la dose alla settimana 20 durante il
periodo di trattamento.

E.5.2

Secondary end point(s)

1. Change from baseline in 6-minute walk distance (6MWD) to Week 26 (key secondary endpoint).
2. TTCW (key secondary endpoint), where clinical worsening is defined (adapted from the CHMP definition [EMEA 2008]) as at least one of the following components confirmed by the CEC, when applicable:
– All-cause death
– Non-planned PH-related hospitalization
– PH-related deterioration identified by at least one of the following:
• Increase from baseline in WHO FC4;
• Deterioration from baseline by at least 15% in exercise capacity, as measured by the 6MWD4;
New or worsening signs or symptoms of right heart failure,
defined as a reported AE with one of the preferred terms "CTEPH", "pulmonary hypertension", "right ventricular failure", "right ventricular dysfunction" and/or "acute right ventricular failure".
3. All-cause death or hospitalizations related to PH worsening.
4. Improvement in WHO FC from baseline
4. Proportion of subjects with improvement in WHO FC from baseline to Week 26.
5. Change from baseline to Week 26 in Pulmonary arterial hypertensionsymptoms
and impact questionnaire (PAH-SYMPACT®) cardiopulmonary symptoms domain and cardiovascular symptoms domain.
6. Change from baseline to Week 26 in Borg dyspnea index (BDI)/Borg
CR10®.
7. Change from baseline to Week 26 in NT-proBNP.

1. Modifica dal basale nel 6MWD alla Settimana 26 (endpoint secondario chiave).
2.Tempo al peggioramento clinico (endpoint secondario chiave) dove il peggioramente clinico è definito (adattato dal Committee for Medicinal Products for Human Use (CHMP)] come almeno uno dei seguenti elementi, confermati dal CEC, quando applicabile:
¿ Decesso per qualsiasi causa.
¿ Ospedalizzazione non programmata correlata alla PH.
¿ Peggioramento correlato alla PH identificato mediante almeno uno dei seguenti:
o Aumento, rispetto al basale, della classe funzionale OMS (WHO-FC);
o Peggioramento, rispetto al basale, di almeno il 15% della capacità di esercizio fisico, misurato attraverso il 6MWD4;
Nuovo o peggioramento dei segni o sintomi di insufficienza cardiaca destra definiti come AE segnalati con uno dei seguenti termini preferiti: «CTEPH», «ipertensione polmonare», «insufficienza ventricolare destra», «disfunzione ventricolare destra» e «insufficienza ventricolare destra acuta».
3.Tutte le cause di morte o ospedalizzazione correlate al peggioramento della PH.
•4.Aumento della FC OMS dal basale alla Settimana 26.
•5. Variazione dal basale alla Settimana 26 dei punteggi del questionario sui sintomi e impatto dell'ipertensione polmonare arteriosa (PAH-SYMPACT™®), nell’ambito dei sintomi cardio polmonari e cardio vascolari.
6.Variazione dal basale alla Settimana 26 nell’indice di dispnea di Borg / Borg CR10®.
7.Variazione dal basale alla Settimana 26 nell’NTproBNP.

E.5.2.1

Timepoint(s) of evaluation of this end point

1. Week 26
2. end of double blind treatment period
3. end of double blind treatment period
4. Week 26
5. Week 26
6. Week 26
7. Week 26

1. Settimana 26
2. alla fine del periodo in doppio cieco
3. alla fine del periodo in doppio cieco
4. Settimana 26
5.Settimana 26
6. Settimana 26
7. Settimana 26

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

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)

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

Yes

E.8.1.7.1

Other trial design description

gruppo sequenziale

group sequential

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

Brazil

Canada

China

Hong Kong

Israel

Korea, Democratic People's Republic of

Mexico

Russian Federation

Singapore

South Africa

Taiwan

Thailand

Turkey

Ukraine

United States

Austria

Belgium

Bulgaria

Denmark

Estonia

Finland

Germany

Greece

Hungary

Italy

Latvia

Lithuania

Netherlands

Poland

Portugal

Slovakia

Spain

Sweden

Switzerland

United Kingdom

Czechia

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

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

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:

182

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

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

F.4.2.2

In the whole clinical trial

280

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)

After the subject's study completion, the investigator/delegate will
explain to subjects what treatment(s) / medical care is necessary and
available according to local regulations. If indicated, the Sponsor will
provide subjects who completed the study and did not prematurely
discontinue study treatment with OL study treatment until access to commercial
selexipag is possible in this indication in the subject's country of
residence, according to local regulatory requirements.

Dopo il completamento dello studio del soggetto, lo sperimentatore / delegato spiegherà ai soggetti quale trattamento / cura medica sono necessari e disponibili secondo le normative locali. Se indicato, Actelion fornirà ai soggetti, che hanno completato lo studio e che non hanno sopseso prematuramente il trattamento di studio, il trattamento in aperto (OL) fino alla commercializzazione, per l'indicazione in studio, nel paese di residenza del soggetto, in linea con i requisiti normativi locali.

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

2019-07-03

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-05-07

P. End of Trial
P.	End of Trial Status	Prematurely Ended

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