Clinical Trials Register

Summary
EudraCT Number:	2017-002667-17
Sponsor's Protocol Code Number:	CVAY736X2207
National Competent Authority:	France - ANSM
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2018-02-20
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

France - ANSM

A.2

EudraCT number

2017-002667-17

A.3

Full title of the trial

A subject-, investigator-, and sponsor-blinded, randomized, placebo-controlled, multicenter study to investigate efficacy, safety, and tolerability of VAY736 in patients with idiopathic pulmonary fibrosis

Étude multicentrique, contrôlée par placebo, randomisée, pour laquelle les patients, les investigateurs et le promoteur sont en aveugle, visant à examiner l’efficacité, la sécurité d’emploi et la tolérance de VAY736 chez des patients atteints de fibrose pulmonaire interstitielle diffuse (FPID)

A.3.1

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

Study of pharmacodynamics, pharmacokinetics, safety and tolerability of VAY736 in patients with idiopathic pulmonary fibrosis

Étude de la pharmacodynamique, de la pharmacocinétique, de la sécurité d’emploi et de la tolérance de VAY736 chez des patients atteints de fibrose pulmonaire idiopathique

A.4.1

Sponsor's protocol code number

CVAY736X2207

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT03287414

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	Novartis Pharma AG
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	Novartis Pharma AG
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	Novartis Pharma S.A.S
B.5.2	Functional name of contact point	Information&Communication Médicales
B.5.3	Address:
B.5.3.1	Street Address	2 et 4 rue Lionel Terray
B.5.3.2	Town/ city	Rueil-Malmaison
B.5.3.3	Post code	92500
B.5.3.4	Country	France
B.5.4	Telephone number	+33 1 5547 6600
B.5.5	Fax number	+33 1 5547 6100
B.5.6	E-mail	icm.phfr@novartis.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.3.2	Product code	VAY736
D.3.4	Pharmaceutical form	Powder for solution for injection/infusion
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	not established
D.3.9.3	Other descriptive name	VAY736
D.3.9.4	EV Substance Code	SUB31641
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	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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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	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	Solution for injection/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

Idiopathic pulmonary fibrosis

fibrose pulmonaire idiopathique

E.1.1.1

Medical condition in easily understood language

Scar tissue in the lung that interferes with the ability to breathe

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	10021240
E.1.2	Term	Idiopathic pulmonary fibrosis
E.1.2	System Organ Class	10038738 - Respiratory, thoracic and mediastinal disorders

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To assess the efficacy of VAY736 in patients with idiopathic pulmonary fibrosis

Évaluer l’efficacité de VAY736 chez des patients atteints de fibrose pulmonaire idiopathique

E.2.2

Secondary objectives of the trial

Impact of VAY736 on survival and disease progression.

Other protocol-defined secondary objectives may apply

Évaluer l’impact de VAY736 sur la survie et la « progression de la maladie »

D'autres objectifs lies au protocole peuvent s'appliquer

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

•A diagnosis of definite or probable IPF within 5 years of the screening visit, as defined by the ATS/ERS/JRS/ALAT Diagnostic Guidelines (Raghu et al 2011)
•Seropositive at screening for at least one of the following auto-antibodies: RF, ANA, anti-dsDNA, anti-CCP, Scl-70, SSA (anti-Ro), SSB (anti-La), anti-RNP, anti-Smith, Jo-1, PL-7, PL-12, EJ, OJ, SRP, Ku, Mi-2, anti-PM/Scl;
OR
Presence of hilar/mediastinal adenopathy (>1cm in short-axis diameter), identified by screening HRCT scan of the chest
•FVC 50-90% predicted (inclusive)
•DLCO, corrected for hemoglobin, 30-79% predicted (inclusive)
•FEV1/FVC >70%
•Unlikely to die from cause other than IPF within the next 3 years, in the opinion of the investigator
•Unlikely to undergo lung transplantation during this trial

•Diagnostic de FPID certaine ou probable dans les 5 ans précédant la visite de sélection, défini par les directives de diagnostic ATS/ERS/JRS/ALAT
•Séropositivité à la sélection pour au moins un des auto-anticorps suivants : RF, ANA, anti-ADNdb, anti-CCP, Scl-70, SSA (anti-Ro), SSB (anti-La), anti-RNP, anti-Smith, Jo-1, PL-7, PL-12, EJ, OJ, SRP, Ku, Mi-2, anti- PM/Scl ; OU présence d’une adénopathie hilaire/médiastinale (> 1 cm dans le diamètre de l’axe court) identifiée par examen de TDMHR du thorax à la sélection
•CVF entre 50 et 90 % (inclus) de la valeur prédite
• Capacité de Diffusion Pulmonaire (CPD) corrigée pour l’hémoglobine entre 30 et 79 % (inclus) de la valeur prédite
•VEMS/CVF > 70 %
•Décès par une cause autre que la FPID peu probable au cours des 3 prochaines années, de l’avis de l’investigateur
•Greffe pulmonaire peu probable au cours de cet essai

E.4

Principal exclusion criteria

•Emphysema > fibrosis on screening HRCT (must be confirmed by central reader)
•History of major organ, hematopoietic stem cell or bone marrow transplant
•Findings on screening HRCT that are inconsistent with IPF, suggest the possibility of AEIPF
(new ground-glass opacities (GGO) or consolidation), or any new concerning
pulmonary nodules (central reader)
•Clinically diagnosed AE-IPF or other significant clinical worsening within 3 months of
randomization
•class III/IV Congestive Heart Failure (CHF), Ejection Fraction (EF) <25%
•Current smoker (must have negative cotinine test)
•Prior use of any B-cell depleting therapy (e.g., rituximab, ofatumumab, or other anti-CD20
mAb, anti-CD40, anti-CD19,anti-CD22 mAb, anti-CD52 mAb, or anti-BAFF mAb)

•Emphysème > fibrose à la TDMHR de sélection (doit être confirmé par un lecteur central)
•Antécédents de greffe d’un organe majeur, de cellules souches hématopoïétiques ou de moelle osseuse
•Observations sur la TDMHR de sélection non pertinentes avec une FPID, suggérant une possibilité d’exacerbation aiguë (EA) de la FPID (nouvelle opacité ou consolidation en verre dépoli) ou tout nouveau nodule pulmonaire inquiétant (lecteur central)
•Diagnostic clinique d’EA-FPID ou d’une autre aggravation clinique significative dans les 3 mois précédant la randomisation
•ICC de classe III/IV de la NYHA, FE < 25 %
•Fumeur actuel (doit être négatif au test de cotinine)
•Antécédents d’utilisation de tout traitement de déplétion des lymphocytes B (par ex., rituximab, ofatumumab ou autre AcM anti-CD20, anti-CD40, anti-CD19, AcM anti-CD22, AcM anti-CD52 ou AcM anti-BAFF)

E.5 End points

E.5.1

Primary end point(s)

Change from baseline to end of treatment epoch (48 weeks of treatment) in Forced Vital Capacity (FVC).

Variation entre la référence et la fin du traitement (48 semaine de traitement) de la capacité vitale forcée (CVF)

E.5.1.1

Timepoint(s) of evaluation of this end point

end of treatment epoch (48 weeks of treatment)

fin du traitement (48 semaine de traitement)

E.5.2

Secondary end point(s)

All-Cause mortality; Progression-free survival (PFS); Disease progression; Composite Endpoint; Change from baseline to end of treatment epoch (Week 48) in Diffusing Capacity of the Lungs (DLCO); Change from baseline to the end of treatment epoch (Week 48) in 6-minute walk test and in distance-saturation product; Change from baseline to the end of treatment epoch (Week 48) in resting oxygen saturation (on room air); Immunogenicity of VAY736; To assess the pharmacokinetics Cmin,ss of VAY736 after multiple s.c. doses; Idiopathic Pulmonary Fibrosis (IPF) -related Mortality; Change from baseline to end of treatment epoch (Week 48) in Total Lung Capacity (TLC)
Other protocol defined endpoints may apply

E.5.2.1

Timepoint(s) of evaluation of this end point

across the 48 weeks of treatment and at the end of study

pendant les 48 semaine de traitement et a la fin du traitement

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

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)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

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

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

Canada

France

Germany

Ireland

Italy

United Kingdom

United States

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

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:

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

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

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

2018-02-20

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-06-06

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

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