Clinical Trials Register

Summary
EudraCT Number:	2018-000183-28
Sponsor's Protocol Code Number:	VX17-445-102
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:	2021-02-09
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

2018-000183-28

A.3

Full title of the trial

A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

Studio di fase 3 randomizzato, controllato, in doppio cieco, volto a valutare la sicurezza e l’efficacia della terapia combinata VX-445 in soggetti con fibrosi cistica eterozigoti per la mutazione F508del e una mutazione con funzione minima (F/MF)

A.3.1

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

A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

Studio di fase 3 della terapia combinata VX-445 in soggetti con fibrosi cistica eterozigoti per la mutazione F508del e una mutazione con funzione minima (F/MF)

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

VX17-445-102

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	VERTEX PHARMACEUTICALS INCORPORATED
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	Vertex Pharmaceuticals Incorporated
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	Vertex Pharmaceuticals Incorporated
B.5.2	Functional name of contact point	Clinical Trials and Medical Info
B.5.3	Address:
B.5.3.1	Street Address	50 Northern Avenue
B.5.3.2	Town/ city	Boston
B.5.3.3	Post code	MA 02210-1862
B.5.3.4	Country	United States
B.5.4	Telephone number	0018776348789
B.5.5	Fax number	0015105958183
B.5.6	E-mail	medicalinfo@vrtx.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	100-mg VX-445/50-mg TEZ/75-mg IVA FDC
D.3.2	Product code	[VX-445/TEZ/IVA]
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	Tezacaftor
D.3.9.1	CAS number	1152311-62-0
D.3.9.2	Current sponsor code	VX-661
D.3.9.3	Other descriptive name	VX-661
D.3.9.4	EV Substance Code	SUB33135
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	50
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Ivacaftor
D.3.9.1	CAS number	873054-44-5
D.3.9.2	Current sponsor code	VX-770
D.3.9.4	EV Substance Code	SUB33103
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	75
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	VX-445
D.3.9.1	CAS number	2216712-66-0
D.3.9.2	Current sponsor code	VX-445
D.3.9.3	Other descriptive name	VX-445
D.3.9.4	EV Substance Code	SUB185183
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	100
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	Kalydeco 150 mg film-coated tablets
D.2.1.1.2	Name of the Marketing Authorisation holder	Vertex Pharmaceuticals (Europe) Limited. MA n.: EU/1/12/782/001-002
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	Yes
D.2.5.1	Orphan drug designation number	EU/3/08/556
D.3 Description of the IMP
D.3.1	Product name	Ivacaftor
D.3.2	Product code	[VX-770]
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	Ivacaftor
D.3.9.1	CAS number	873054-44-5
D.3.9.2	Current sponsor code	VX-770
D.3.9.4	EV Substance Code	SUB33103
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	150
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
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

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Cystic Fibrosis

Fibrosi Cistica

E.1.1.1

Medical condition in easily understood language

Cystic Fibrosis

Fibrosi Cistica

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	20.0
E.1.2	Level	PT
E.1.2	Classification code	10011762
E.1.2	Term	Cystic fibrosis
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic 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 evaluate the efficacy of VX-445 in Triple Combination (TC) with TEZ and IVA in subjects with CF who are heterozygous for F508del and a minimal function mutation (F/MF subjects)

Valutare l’efficacia di VX-445 in tripla combinazione (TC) con tezacaftor (TEZ) e ivacaftor (IVA) in soggetti con fibrosi cistica (FC) eterozigoti per F508del e una mutazione con funzione minima (soggetti F/MF)

E.2.2

Secondary objectives of the trial

• To evaluate the safety of VX-445 in TC with TEZ and IVA
• To evaluate the pharmacodynamics (PD) of VX-445 in TC with TEZ and IVA
• To evaluate the pharmacokinetics (PK) of VX-445, TEZ, and IVA when administered in TC

• Valutare la sicurezza di VX-445 in TC con TEZ e IVA
• Valutare la farmacodinamica (PD) di VX-445 in TC con TEZ e IVA
• Valutare la farmacocinetica (PK) di VX-445, TEZ e IVA durante la somministrazione in TC

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Subject (or his or her legally appointed and authorized representative) will sign and date an informed consent form (ICF), and, when appropriate, an assent form.
2. Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.
3. Age 12 years or older, on the date of informed consent.
4. Confirmed diagnosis of CF as determined by the investigator.
5. Heterozygous for F508del and an MF mutation (F/MF genotypes, see Appendix A for eligible MF mutations). If the screening CFTR genotype result is not received before randomization, a previous CFTR genotype laboratory report may be used to establish eligibility. Subjects who have been randomized and whose screening genotype does not confirm study eligibility must be discontinued from the study (Section 9.9).
6. Forced expiratory volume in 1 second (FEV1) value =40% and =90% of predicted mean for age, sex, and height (equations of the Global Lung Function Initiative [GLI]) at the Screening Visit. Spirometry measurements must meet American Thoracic Society/European Respiratory Society criteria for acceptability and repeatability.
7. Stable CF disease as judged by the investigator.
8. Willing to remain on a stable CF treatment regimen (as defined in Section 9.5) through completion of study participation.

1. Il soggetto (o il suo rappresentante legalmente nominato e autorizzato) apporrà la propria firma e la data su un modulo di consenso informato (MCI) e, ove opportuno, su un modulo di assenso.
2. Il soggetto è disposto e in grado di presentarsi alle visite programmate, di osservare il piano terapeutico e le restrizioni previste per lo studio, di sottoporsi a test di laboratorio, di osservare le linee guida in materia di contraccezione e le altre procedure dello studio.
3. L'età del soggetto deve essere di almeno 12 anni alla data del consenso informato.
4. Il soggetto deve avere una diagnosi confermata di fibrosi cistica come stabilito dallo sperimentatore.
5. Il soggetto risulta eterozigote per F508del con mutazione MF (genotipi F/MF, si veda l'Appendice A per un elenco delle mutazioni MF ammissibili). Qualora i risultati relativi al genotipo CFTR allo screening non siano disponibili prima della randomizzazione, potrà essere utilizzato un rapporto di laboratorio precedente relativo al genotipo CFTR per stabilire l'idoneità del soggetto. I soggetti che sono stati randomizzati e il cui genotipo allo screening non conferma l'idoneità a partecipare allo studio dovranno interrompere lo studio (Sezione 9.9).
6. Il soggetto presenta un valore di volume espiratorio massimo nel primo secondo (FEV1) =40% e =90% del valore medio predetto per età, sesso e altezza (in base alle equazioni della Global Lung Function Initiative [GLI]) alla visita di screening. Le misure spirometriche devono soddisfare i criteri della American Thoracic Society/European Respiratory Society in termini di accettabilità e ripetibilità.
7. Il soggetto deve essere affetto da fibrosi cistica stabile a giudizio dello sperimentatore.
8. Il soggetto deve essere disposto a seguire un regime terapeutico stabile per la fibrosi cistica (come definito nella Sezione 9.5) fino al completamento della sua partecipazione allo studio.

E.4

Principal exclusion criteria

1. History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug(s) to the subject. This includes, but is not limited to, the following:
• Clinically significant cirrhosis with or without portal hypertension
• Solid organ or hematological transplantation.
• Alcohol or drug abuse in the past year, including, but not limited to, cannabis, cocaine, and opiates, as deemed by the investigator.
• Cancer, except for squamous cell skin cancer, basal cell skin cancer, and Stage 0 cervical carcinoma in situ (all 3 with no recurrence for the last 5 years)
2. History of hemolysis.
3. Glucose-6-phosphate dehydrogenase (G6PD) deficiency, defined as G6PD activity less than the lower limit of normal (LLN) or 70% of the mean of the LLN and the upper limit of normal (ULN), whichever is greater. A documented historical quantitative G6PD activity value measured in an established laboratory that runs the assay routinely and obtained during a prior Vertex study or as part of routine clinical care may be used to establish eligibility.
4. Any of the following abnormal laboratory values at screening:
• Hemoglobin <10 g/dL
• Total bilirubin =2 × ULN
• Aspartate transaminase (AST), alanine transaminase (ALT), gamma-glutamyl transferase (GGT), or alkaline phosphatase (ALP) =3 × ULN
• Abnormal renal function defined as glomerular filtration rate =50 mL/min/1.73 m2 (calculated by the Modification of Diet in Renal Disease Study Equation) for subjects =18 years of age and =45 mL/min/1.73 m2 (calculated by the Counahan-Barratt equation) for subjects aged 12 to 17 years (inclusive)
5. An acute upper or lower respiratory infection, PEx, or changes in therapy (including antibiotics) for sinopulmonary disease within 28 days before the first dose of study drug (Day 1).
6. Lung infection with organisms associated with a more rapid decline in pulmonary status (including, but not limited to, Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus). For subjects who have had a history of a positive culture, the investigator will apply the following criteria to establish whether the subject is free of infection with such organisms:
• The subject has not had a respiratory tract culture positive for these organisms within the 12 months before the date of informed consent.
• The subject has had at least 2 respiratory tract cultures negative for such organisms within the 12 months before the date of informed consent, with the first and last of these separated by at least 3 months, and the most recent one within the 6 months before the date of informed consent.
7. An acute illness not related to CF (e.g., gastroenteritis) within 14 days before the first dose of study drug (Day 1).
8. Ongoing or prior participation in a study of an investigational treatment within 28 days or 5 terminal half-lives (whichever is longer) before screening. The duration of the elapsed time may be longer if required by local regulations.
9. Use of prohibited medications as defined in Table 9-2, within the specified window before the first dose of study drug (Day 1).
10. Pregnant or nursing females. Females of childbearing potential must have a negative pregnancy test at screening (serum test) and Day 1 (urine test).
11. The subject or a close relative of the subject is the investigator or a subinvestigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study at that site. However, an adult (aged 18 years or older) who is a relative of a study staff member may be randomized in the study provided that
• the adult lives independently of and does not reside with the study staff member, and
• the adult participates in the study at a site other than the site at which the family member is employed.

1. Storia di qualsiasi malattia o condizione clinica che, secondo lo sperimentatore, potrebbe confondere i risultati dello studio o rappresentare un ulteriore rischio per la somministrazione del(i) farmaco(i) dello studio al soggetto. Ciò include, a titolo esemplificativo, le seguenti malattie o condizioni cliniche:
• Cirrosi clinicamente significativa con o senza ipertensione portale
• Trapianto di organo solido o ematologico
• Abuso di sostanze alcoliche o stupefacenti nell'ultimo anno inclusi, a titolo esemplificativo, cannabis, cocaina e oppiacei, a giudizio dello sperimentatore.
• Cancro, fatta eccezione per il carcinoma della pelle a cellule squamose, per il carcinoma della pelle a cellule basali e per il carcinoma cervicale in situ in Stadio 0 (tutti 3 con nessuna ricorrenza osservata negli ultimi 5 anni)
2. Storia di emolisi.
3. Deficit di glucosio-6-fosfato deidrogenasi (G6PD), definito come attività di G6PD inferiore al limite inferiore della norma (LLN) o 70% della media di LLN e del limite superiore della norma (ULN), a seconda di quale è maggiore. Per stabilire l'idoneità del soggetto, potrebbe essere utilizzato un valore quantitativo documentato storico dell'attività di G6PD, misurato in un laboratorio di comprovata esperienza che esegue regolarmente il test e ottenuto durante un precedente studio di Vertex o nel quadro di cure cliniche ordinarie.
4. Uno qualsiasi dei seguenti valori di laboratorio anomali allo screening:
• Emoglobina <10 g/dl
• Bilirubina totale =2 × ULN
• Aspartato aminotransferasi (AST), alanina aminotransferasi (ALT), gamma glutamil transpeptidasi (GGT) o fosfatasi alcalina (ALP) =3 × ULN
• Funzione renale anomala intesa come velocità di filtrazione glomerulare =50 ml/min/1,73 m2 (calcolata in base all'equazione dello studio Modification of Diet in Renal Disease) per i soggetti di età =18 anni e =45 ml/min/1,73 m2 (calcolata in base all'equazione Counahan-Barratt) per i soggetti di età compresa fra 12 e 17 anni
5. Un’infezione acuta delle vie respiratorie superiori o inferiori, esacerbazione polmonare o variazioni della terapia (inclusi antibiotici) per malattia sinopolmonare nei 28 giorni antecedenti la prima dose del farmaco dello studio (Giorno 1).
6. Infezione polmonare a causa di organismi associati a un deterioramento più rapido dello stato polmonare (inclusi, a titolo esemplificativo, Burkholderia cenocepacia, Burkholderia dolosa e Mycobacterium abscessus). Per i soggetti con una storia di coltura positiva, lo sperimentatore applicherà i seguenti criteri per stabilire se il soggetto ha superato l'infezione da questi organismi:
• Il soggetto non ha avuto una coltura del tratto respiratorio positiva per questi organismi nei 12 mesi antecedenti la data del consenso informato.
• Il soggetto ha avuto almeno 2 colture del tratto respiratorio negative per questi organismi nei 12 mesi antecedenti la data del consenso informato; la prima e l'ultima coltura sono state effettuate ad almeno 3 mesi di distanza l'una dall'altra e la più recente è stata effettuata entro i 6 mesi antecedenti la data del consenso informato.
7. Una malattia acuta non correlata alla fibrosi cistica (es. gastroenterite) nei 14 giorni antecedenti la prima dose del farmaco dello studio (Giorno 1).
8. Partecipazione in corso o precedente a uno studio su un trattamento sperimentale entro 28 giorni o 5 emivite terminali (a seconda di quale periodo è più lungo) prima dello screening. La durata del suddetto periodo di tempo potrebbe essere maggiore se richiesto dalle normative locali.
9. Utilizzo di farmaci limitati secondo la definizione riportata nella Tabella 9-2, entro la finestra specificata prima della prima dose del farmaco dello studio (Giorno 1).
10. Donne in gravidanza o in allattamento. Le donne in età fertile devono risultare negative al test di gravidanza allo screening (esame del siero) e al Giorno 1 (esame delle urine).
11. vedere ENG

E.5 End points

E.5.1

Primary end point(s)

Absolute change in ppFEV1 from baseline at Week 4

Cambiamento assoluto della percentuale del valore previsto di volume espiratorio forzato in 1 secondo (ppFEV1) dal basale alla Settimana 4

E.5.1.1

Timepoint(s) of evaluation of this end point

Week 4

Settimana 4

E.5.2

Secondary end point(s)

Key Secondary Endpoints ¿ Absolute change in ppFEV1 from baseline through Week 24 ¿ Number of pulmonary exacerbations (PEx) through Week 24 ¿ Absolute change in SwCl from baseline through Week 24 ¿ Absolute change in CFQ-R respiratory domain score from baseline through Week 24 ¿ Absolute change in body mass index (BMI) from baseline at Week 24 ¿ Absolute change in SwCl from baseline at Week 4 ¿ Absolute change in CFQ-R respiratory domain score from baseline at Week 4 Other Secondary Endpoints ¿ Time-to-first PEx through Week 24 ¿ Absolute change in BMI z-score from baseline at Week 24 Absolute change in body weight from baseline at Week 24 ¿ Safety and tolerability assessments based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry ¿ PK parameters of VX-445, TEZ, M1-TEZ, and IVA

Endpoint secondari chiave
• Cambiamento assoluto del ppFEV1 dal basale alla Settimana 24
• Numero di riacutizzazioni polmonari (PEx) fino alla Settimana 24
• Cambiamento assoluto del cloruro contenuto nel sudore (SwCl) dal basale alla Settimana 24
• Cambiamento assoluto del punteggio relativo al dominio respiratorio del questionario revisionato per la FC (CFQ-R) dal basale alla Settimana 24
• Cambiamento assoluto dell’indice di massa corporea (IMC) dal basale alla Settimana 24
• Cambiamento assoluto di SwCl dal basale alla Settimana 4
• Cambiamento assoluto del punteggio relativo al dominio respiratorio del CFQ-R dal basale alla Settimana 4
Altri endpoint secondari
• Tempo alla prima PEx fino alla Settimana 24
• Cambiamento assoluto del punteggio z dell’IMC dal basale alla Settimana 24
• Cambiamento assoluto del peso corporeo dal basale alla Settimana 24
• Valutazioni della sicurezza e della tollerabilità basate su eventi avversi (AE), valori clinici di laboratorio, ECG, segni vitali e pulsossimetria
• Parametri PK di VX-445, TEZ, M1-TEZ e IVA

E.5.2.1

Timepoint(s) of evaluation of this end point

Week 4;
Baseline through Week 24;
At Week 24

Settimana 4;
Dal basale alla Settimana 24;
Alla Settimana 24

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

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

Yes

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)

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

Canada

United States

Austria

Belgium

France

Germany

Greece

Italy

Netherlands

Sweden

United Kingdom

Czechia

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

LSLV

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:

290

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

F.3.3.7

Others

Yes

F.3.3.7.1

Details of other specific vulnerable populations

adolescents 12-17 years

adolescenti 12-17 anni

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

134

F.4.2.2

In the whole clinical trial

360

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)

An open-label study will be available for subjects who complete the Week 24 Visit and are eligible. The Safety Follow-up Visit is not required for subjects who complete the Week 24 Visit and enroll in an open-label study within 28 days after the last dose of study drug.

Uno studio in aperto sarà disponibile per i soggetti che completano la visita della settimana 24 e risultano eleggibili. La visita di follow-up della sicurezza non è richiesta per i soggetti che hanno completato la visita della settimana 24 e vengono arruolati nello studio in aperto entro 28 giorni dopo l'ultima dose del farmaco in studio.

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

2018-09-17

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-07-03

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

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IMP with orphan designation in the indication
Orphan Designation Number:
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