Clinical Trials Register

Summary
EudraCT Number:	2021-000694-85
Sponsor's Protocol Code Number:	VX20-121-103
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:	2021-10-15
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

2021-000694-85

A.3

Full title of the trial

A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-121 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive CFTR Mutation and No F508del Mutation

Studio di fase 3, randomizzato, in doppio cieco, controllato, per la valutazione della sicurezza ed efficacia della terapia combinata con VX-121 nei soggetti con fibrosi cistica omozigoti per F508del,eterozigoti per F508dele con una mutazione di gating (F/G) o con funzione residua (F/RF)oppure che abbianoalmeno 1 altra mutazione di CFTRresponsiva allatripla combinazione e nessuna mutazioneF508del

A.3.1

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

A Phase 3 Study of VX-121 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive CFTR Mutation and No F508del Mutation

Studio di fase 3 della terapia combinata con VX-121 in soggetti con fibrosi cistica che sono omozigoti per F508del,eterozigoti per F508del e con una mutazione di gating (F/G) o con funzione residua (F/RF), oppure che abbianoalmeno 1 altra mutazione CFTR responsiva alla tripla combinazione e nessuna mutazione F508del

A.3.2

Name or abbreviated title of the trial where available

A Phase 3 Study of VX-121 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous fo

Studio di fase 3 della terapia combinata con VX-121 in soggetti con fibrosi cistica che sono omozigo

A.4.1

Sponsor's protocol code number

VX20-121-103

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	+18776348789
B.5.5	Fax number	+15105958183
B.5.6	E-mail	medicalinfo@vrtx.com

D. IMP Identification
D.IMP: 1
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	Kalydeco
D.2.1.1.2	Name of the Marketing Authorisation holder	Vertex Pharmaceuticals (Ireland) Limited
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.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	VX-121/tezacaftor/deutivacaftor
D.3.2	Product code	[VX-121/TEZ/D-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.3.9.4	EV Substance Code	SUB188271
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	deutivacaftor
D.3.9.1	CAS number	1413431-07-8
D.3.9.2	Current sponsor code	VX-561
D.3.9.4	EV Substance Code	SUB194586
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	125
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.9.1	CAS number	2374124-50-0
D.3.9.2	Current sponsor code	VX-121
D.3.9.4	EV Substance Code	SUB191626
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: 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	Kaftrio
D.2.1.1.2	Name of the Marketing Authorisation holder	Vertex Pharmaceuticals (Ireland) Limited
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/3/18/2116
D.3 Description of the IMP
D.3.1	Product name	elexacaftor/tezacaftor/ivacaftor
D.3.2	Product code	[ELX/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	elexacaftor
D.3.9.1	CAS number	2216712-66-0
D.3.9.2	Current sponsor code	VX-445
D.3.9.4	EV Substance Code	SUB193216
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.3.8 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.3.9.4	EV Substance Code	SUB188271
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.3.11 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
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

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-121/TEZ/D-IVA in CF subjects who are homozygous for F508del, heterozygous for F508del and a gating (F/G) or residual function (F/RF) mutation, or have at least 1 other triple combination responsive (TCR) CFTR mutation and no F508del mutation

Valutare l’efficacia di VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in soggetti con fibrosi cistica (FC) omozigoti per F508del, eterozigoti per F508del e con una mutazione di gating (F/G) o con funzione residua (F/RF), o con almeno 1 altra mutazione CFTRresponsiva allatripla combinazione (TCR) e nessuna mutazione F508del

E.2.2

Secondary objectives of the trial

• To evaluate the safety of VX-121/TEZ/D-IVA
• To evaluate the pharmacokinetics (PK) of VX-121/TEZ/D-IVA

• Valutare la sicurezza di VX-121/TEZ/D-IVA
• Valutare la farmacocinetica (PK) di VX-121/TEZ/D-IVA

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Subject will sign and date an informed consent form, 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. Subjects aged 12 years or older, on the date of informed consent
4. Confirmed diagnosis of CF as determined by the investigator
5. Subject has one of the following genotypes: 1) homozygous for F508del; 2) heterozygous for F508del and a gating (F/G) mutation; 3) heterozygous for F508del and a residual function (F/RF) mutation; 4) at least 1 other TCR CFTR mutation identified as responsive to ELX/TEZ/IVA and no F508del mutation.
6. For subjects currently receiving Vertex CFTR modulator therapy, FEV1 value =40% and =90% of predicted mean for age, sex, and height at the Screening Visit. All subjects not currently receiving Vertex CFTR modulator therapy must have an FEV1 value =40% and =80% of predicted mean.
7. Stable CF disease as judged by the investigator.
8. Willing to remain on a stable CF treatment regimen through completion of study participation.

1. Il soggetto firmerà e daterà un modulo di consenso informato e un modulo di assenso, ove opportuno.
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. I soggetti devono avere un'età pari o superiore ai 12 anni alla data del consenso informato.
4. Diagnosi confermata di fibrosi cistica come stabilito dallo sperimentatore.
5. Il soggetto ha uno dei seguenti genotipi: 1) omozigote per F508del; 2) eterozigote per F508del e mutazione di gating (F/G); 3) eterozigote per F508del e mutazione della funzione residuale (F/RF); 4) almeno un'altra mutazione TCR CFTR identificata come responsiva a ELX/TEZ/IVA e nessuna mutazione F508del.
6. Per i soggetti che attualmente ricevono la terapia con modulatore Vertex CFTR, valore FEV1 =40% e =90% della media prevista per età, sesso e altezza alla visita di screening. Tutti i soggetti che attualmente non ricevono la terapia con modulatore Vertex CFTR devono avere un valore FEV1 =40% e =80% della media prevista.
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 fino al completamento della sua partecipazione allo studio.

E.4

Principal exclusion criteria

1. History of any comorbidity 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.
2. History of intolerance to study drug that would pose an additional risk to the subject in the opinion of the investigator.
3. Any of the following abnormal laboratory values at screening:
• Hemoglobin <10 g/dL
• Total bilirubin =2 × upper limit of normal
• Aspartate transaminase, alanine transaminase, gamma-glutamyl transferase, or alkaline phosphatase =3 × ULN
• Abnormal renal function defined as glomerular filtration rate =50 mL/min/1.73 m2 for subjects =18 years of age and =45 mL/min/1.73 m2 for subjects aged 12 to 17 years.
4. An acute upper or lower respiratory infection, PEx, or changes in therapy for sinopulmonary disease within 28 days before the first dose of ELX/TEZ/IVA in the Run-in Period.
5. Lung infection with organisms associated with a more rapid decline in pulmonary status. 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.
6. An acute illness not related to CF within 14 days before the first dose of ELX/TEZ/IVA in the Run-in Period.
7. Ongoing or prior participation in a study of an investigational treatment other than a Vertex CFTR modulator within 28 days or 5 terminal half-lives before screening, or participation in an interventional study of a non-investigational treatment from screening through end of study participation. The duration of the elapsed time may be longer if required by local regulations.
8. Use of prohibited medications within the specified window before the first dose of ELX/TEZ/IVA in the Run-in Period.
9. Pregnant or breast-feeding females. Female subjects must have a negative pregnancy test at screening and Run-in Period/Day -28.
10. The subject or a close relative of the subject is the investigator or a sub investigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study at that site. However, an adult who is a relative of a study staff member may be enrolled 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. Anamnesi di qualsiasi comorbilità che, secondo lo sperimentatore, potrebbe confondere i risultati dello studio o rappresentare un ulteriore rischio per la somministrazione del farmaco/dei farmaci dello studio al soggetto.
2. Anamnesi di intolleranza al farmaco dello studio che, secondo lo sperimentatore, potrebbe rappresentare un ulteriore rischio per il soggetto.
3. Uno qualsiasi dei seguenti valori di laboratorio anomali allo screening:
• Emoglobina <10 g/dl
• Bilirubina totale =2 x limite superiore della norma
• Aspartato aminotransferasi, alanina aminotransferasi, gamma-glutamiltransferasi o fosfatasi alcalina =3 × ULN
• Funzionalità renale anomala definita come velocità di filtrazione glomerulare =50 ml/min/1,73 m2 per i soggetti di età =18 anni e =45 ml/min/1,73 m2 per i soggetti di età compresa tra 12 e 17 anni.
4. Un'infezione acuta delle vie respiratorie superiori o inferiori, PEx o cambiamenti nella terapia per la malattia sinopolmonare entro 28 giorni prima della prima dose di ELX/TEZ/IVA nel periodo di run-in.
5. Infezione polmonare con organismi associati a un più rapido declino dello stato polmonare. Per i soggetti con un'anamnesi 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.
6. Una malattia acuta non correlata alla FC nei 14 giorni precedenti la prima dose di ELX/TEZ/IVA nel periodo di run-in.
7. Partecipazione in corso o precedente a uno studio su un trattamento sperimentale diverso da un modulatore Vertex CFTR entro 28 giorni o 5 emivite terminali prima dello screening, o partecipazione a uno studio interventistico su un trattamento non sperimentale dallo screening fino alla fine della partecipazione allo studio. La durata del suddetto periodo di tempo potrebbe essere maggiore se richiesto dalle normative locali.
8. Uso di farmaci proibiti entro la finestra specificata prima della prima dose di ELX/TEZ/IVA nel periodo di run-in.
9. Donne in gravidanza o che allattano al seno. I soggetti di sesso femminile devono avere un test di gravidanza negativo allo screening e al periodo di run-in/giorno -28.
10. Il soggetto o un parente stretto del soggetto è lo sperimentatore o uno sperimentatore secondario, un assistente di ricerca, un farmacista, un coordinatore dello studio o altro personale direttamente coinvolto nella conduzione dello studio in quel centro. Tuttavia, un adulto imparentato con un membro del personale dello studio può essere arruolato nello studio a condizione che
• l'adulto abbia una condizione abitativa indipendente e non risieda con il membro del personale dello studio e
• l'adulto partecipi allo studio in un centro diverso da quello in cui lavora il suo familiare.

E.5 End points

E.5.1

Primary end point(s)

Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1) through Week 24.

Cambiamento assoluto dal basale della percentuale del volume espiratorio forzato previsto in 1 secondo (ppFEV1) alla Settimana24

E.5.1.1

Timepoint(s) of evaluation of this end point

Baseline through 24 weeks.

Dal basale per 24 settimane.

E.5.2

Secondary end point(s)

• Absolute change from baseline in sweat chloride (SwCl) through Week 24
• Proportion of subjects with SwCl <60 mmol/L through Week 24 (pooled with data from VX20-121-102)
• Proportion of subjects with SwCl <30 mmol/L through Week 24 (pooled with data from VX20-121-102)

• Cambiamento assoluto del cloruro contenuto nel sudore (SwCl) dal basale alla Settimana 24
• Percentuale di soggetti con SwCl <60mmol/l fino alla Settimana24 (aggregata con dati dello Studio VX20-121-102)
• Percentuale di soggetti con SwCl <30mmol/l fino alla Settimana24 (aggregata con dati dello Studio VX20-121-102)

E.5.2.1

Timepoint(s) of evaluation of this end point

Baseline through 24 weeks.

Dal basale per 24 settimane.

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

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

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

Israel

United States

Austria

Belgium

Denmark

France

Germany

Ireland

Italy

Netherlands

Norway

Poland

Sweden

Switzerland

United Kingdom

Greece

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

462

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

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

175

F.4.2.2

In the whole clinical trial

550

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)

none

nessuno

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

2021-12-21

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-11-03

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

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Orphan Designation Number:
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