Clinical Trials Register

Summary
EudraCT Number:	2017-003158-18
Sponsor's Protocol Code Number:	203PF203
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:	2020-11-05
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

2017-003158-18

A.3

Full title of the trial

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of BG00011 in Patients With Idiopathic Pulmonary Fibrosis

Studio randomizzato, in doppio cieco, controllato con placebo per valutare l’efficacia e la sicurezza di BG00011 in pazienti affetti da fibrosi polmonare idiopatica

A.3.1

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

Evaluating the Efficacy and Safety of BG00011 in Participants with Idiopathic Pulmonary Fibrosis

Valutazione l’efficacia e la sicurezza di BG00011 in pazienti affetti da fibrosi polmonare

A.3.2

Name or abbreviated title of the trial where available

Evaluating the Efficacy and Safety of BG00011 in Participants with Idiopathic Pulmonary Fibrosis

Valutazione dell’efficacia e la sicurezza di BG00011 in pazienti affetti da fibrosi polmonare

A.4.1

Sponsor's protocol code number

203PF203

A.5.4

Other Identifiers

Name:

Number:

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	BIOGEN IDEC RESEARCH LIMITED
B.1.3.4	Country	United Kingdom
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	Biogen Idec Research Limited
B.4.2	Country	United Kingdom
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Biogen Idec Research Limited
B.5.2	Functional name of contact point	Medical
B.5.3	Address:
B.5.3.1	Street Address	Innovation House, 70 Norden Road
B.5.3.2	Town/ city	Maidenhead
B.5.3.3	Post code	SL6 4AY
B.5.3.4	Country	United Kingdom
B.5.4	Telephone number	000000000000
B.5.5	Fax number	0000000000000000
B.5.6	E-mail	clinicaltrials@biogen.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.2.1.1.1	Trade name	NA
D.2.1.1.2	Name of the Marketing Authorisation holder	NA
D.2.1.2	Country which granted the Marketing Authorisation	Italy
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	humanized anti-alpha v beta 6 mAb
D.3.2	Product code	[BG00011]
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Not yet assigned
D.3.9.2	Current sponsor code	BG00011
D.3.9.4	EV Substance Code	SUB191995
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	56
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	Yes
D.3.11.13.1	Other medicinal product type	humanized monoclonal antibody

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
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

mild to moderate Idiopathic Pulmonary Fibrosis

fibrosi polmonare idiopatica (FPI) da lieve a moderata

E.1.1.1

Medical condition in easily understood language

mild to moderate scarring of lung tissue

cicatrici da lievi a moderate del tessuto polmonare

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	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

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

To evaluate the efficacy of BG00011 compared with placebo in subjects with Idiopathic pulmonary fibrosis (IPF).

valutare l’efficacia di BG00011 rispetto al placebo in soggetti affetti da FPI.

E.2.2

Secondary objectives of the trial

To evaluate
-The efficacy of BG00011 compared with placebo in subjects with IPF as determined by change in percent predicted FVC
-The safety and tolerability of BG00011; and to evaluate the serum concentration of BG00011
To assess
-Progression-free survival in subjects who receive BG00011 compared with placebo.
-The occurrence of IPF exacerbation in participants who receive BG00011 compared with placebo
-The incidence of absolute decline in FVC =10% in subjects who receive BG00011 compared with placebo.
-The time to death or lung transplantation in subjects who receive BG00011 compared with placebo, and the transplant-free survival rate at Week 26 and Week 52
-The time to nonselective hospitalizations in subjects who receive BG00011 compared with placebo.
-Additional PFT findings in subjects who receive BG00011 compared with placebo
- Performance on the 6MWT in participants who receive BG00011 compared with placebo
Please refer to protocol for further secondary objectives

Per valutare
- l’efficacia di BG00011 rispetto al placebo in soggetti affetti da FPI, come determinato dal cambiamento di FVC predetto in percentuale.
- la sicurezza e la tollerabilità di BG00011; e di Valutare la concentrazione sierica di BG00011
- la sopravvivenza libera da progressione in soggetti che ricevono BG00011 rispetto al placebo.
- Valutare l’occorrenza dell’esacerbazione della FPI in soggetti che ricevono BG00011 rispetto al placebo.
- l’incidenza del declino assoluto di FVC >=10% in soggetti che ricevono BG00011 rispetto al placebo.
- il tempo al decesso o al trapianto del polmone in soggetti che ricevono BG00011 rispetto al placebo e il tasso di sopravvivenza libera da trapianto alla Settimana 26 e alla Settimana 52.
- il tempo di ricovero obbligatorio in soggetti che ricevono BG00011 rispetto al placebo.
- i risultati degli ulteriori test di funzionalità polmonare (PFT) in soggetti che ricevono BG00011 rispetto al placebo.
- la prestazione del test del camm

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Female subjects must be surgically sterile,
postmenopausal (minimum 1 year without menses), or
agree to use 1 or more forms of highly effective
contraception from the time of signing of the informed
consent form (ICF) until 3 months after the last injection
of study medication. Male subjects must also agree to
use 1 or more forms of highly effective contraception for
either themselves or their partners from signing of ICF
until 4 months after last injection of study medication.
- Diagnosed with Idiopathic pulmonary fibrosis (IPF).
- Combination of high-resolution computed tomography
(HRCT) pattern and, if one has been obtained, surgical
lung biopsy pattern, consistent with diagnosis of IPF.
- Carbon monoxide diffusion capacity (DLco) (corrected
for hemoglobin): 30% to 79% predicted of normal at
Screening, with no clinically significant deterioration
between the Screening Visit and randomization, as
determined by the Investigator.
- Forced (expiratory) vital capacity (FVC) >=50%
predicted of normal at Screening, with no clinically
significant deterioration between the Screening Visit and
randomization, as determined by the Investigator.
- If a subject is taking nintedanib or pirfenidone, they
must be on a stable dose for at least 8 weeks prior to
randomization.
NOTE: Other protocol defined inclusion criteria may apply.

- I soggetti di sesso femminile devono essere chirurgicamente sterili, in postmenopausa (minimo 1 anno senza mestruazioni) oppure devono accettare di utilizzare 1 o più metodi di contraccezione altamente efficaci dal momento della firma del modulo di consenso informato (ICF) fino a 3 mesi dopo l’ultima iniezione del farmaco dello studio. Anche i soggetti di sesso maschile devono accettare di utilizzare 1 o più metodi di contraccezione altamente efficaci per se stessi o per le loro partner dalla firma dell’ICF fino a 4 mesi dopo l’ultima iniezione del farmaco dello studio.
- Diagnosi di fibrosi polmonare idiopatica (FPI).
- Combinazione di un modello di tomografia computerizzata ad alta risoluzione (HRCT) e, se è stato acquisito, di un modello di biopsia polmonare chirurgica, coerenti con la diagnosi di FPI.
- Capacità di diffusione del monossido di carbonio (DLco) (corretta per l’emoglobina): prevista essere dal 30% al 79% del valore normale allo screening, senza deterioramento clinicamente significativo tra la visita di screening e la randomizzazione, come stabilito dallo sperimentatore.
- Capacità vitale forzata (espiratoria) (CVF) prevista essere >=50% del valore normale allo screening, senza deterioramento clinicamente significativo tra la visita di screening e la randomizzazione, come stabilito dallo sperimentatore.
- Se un soggetto sta assumendo nintedanib o pirfenidone, questi devono essere a dose stabile per almeno 8 settimane prima della randomizzazione.

NOTA: possono essere applicati altri criteri di inclusione definiti dal protocollo.

E.4

Principal exclusion criteria

- Unable to perform pulmonary functional tests (PFTs) or
undergo HRCT procedure.
- Peripheral capillary oxygen saturation (SpO2) <90% at
rest (if on oxygen supplementation, must be =2 L/min at
rest).
- Airway obstruction (i.e., prebronchodilator FEV1/FVC
<0.7) or evidence of a bronchodilator response as
defined by an absolute increase of =12% and an
increase of =200 milliliters (mL) in FEV1 or FVC, or both,
after bronchodilator use, compared with the values
before bronchodilator use at Screening.
- End-stage fibrotic disease likely requiring organ
transplantation within 12 months, or if the subject has
initiated active evaluation for organ transplantation.
- The extent of emphysema in the lungs exceeds
fibrosis, based on central review of HRCT
scans.
- Body weight <60 kg at Screening.
- History of or ongoing malignant disease, including solid
tumors and hematologic malignancies, with the
exception of basal cell carcinomas, squamous cell
carcinomas, and carcinoma in situ of the cervix that
have been completely excised and considered cured >2
years prior to Screening.
- Significant cardiac disease (e.g., New York Heart
Association Class 3 or 4; myocardial
infarction within the past 6 months; unstable angina;
coronary angioplasty or coronary artery bypass graft
within the past 6 months; uncontrolled atrial or
ventricular cardiac arrhythmias; or pulmonary
hypertension requiring pharmacologic treatment).
- Clinical diagnosis of any connective tissue disease
(including but not limited to scleroderma,
polymyositis/dermatomyositis, systemic lupus
erythematosus, and rheumatoid arthritis) or a diagnosis
of interstitial pneumonia with autoimmune features as
determined by the Investigator.
- Other disease that may interfere with testing
procedures or, in the judgment of the Investigator, may
interfere with study participation or may put the patient
at risk when participating in this study.
- Other unspecified reasons that, in the opinion of the
Investigator or Biogen, make the subject unsuitable for
enrollment.
NOTE: Other protocol defined exclusion criteria may apply.

- Soggetto non in grado di eseguire i test di funzionalità polmonare (PFT) o di sottoporsi alla procedura di HRCT.
- Saturazione dell’ossigeno a livello capillare periferico (SpO2) <90% a riposo (se in ossigenoterapia, deve essere <=2 l/min a riposo).
- Ostruzione delle vie aeree (per es., volume espiratorio massimo in 1 secondo [VEMS]/CVF pre-broncodilatatore <0,7) o evidenza di risposta del broncodilatatore definita come un aumento assoluto >=12% e un aumento >=200 millilitri (ml) in VEMS o CVF, o entrambi, dopo l’utilizzo del broncodilatatore, rispetto ai valori precedenti all’utilizzo del broncodilatatore allo screening.
- Malattia fibrotica in fase terminale che può richiedere un trapianto di organi entro i 12 mesi, oppure nel caso in cui il soggetto abbia iniziato una valutazione attiva per il trapianto di organi.
- L’estensione dell’enfisema nei polmoni supera quello della fibrosi, sulla base di un esame centrale delle scansioni HRCT.
- Peso corporeo <60 kg allo screening.
- Anamnesi di o malattia maligna in corso, compresi tumori solidi e neoplasie ematologiche maligne, ad esclusione del carcinoma a cellule basali, del carcinoma a cellule squamose e del carcinoma in situ della cervice che sono stati completamente escissi e sono considerati guariti per >2 anni prima dello screening.
- Malattia cardiaca significativa (per es., classe 3 o 4 secondo la New York Heart Association [Associazione cardiologica di New York]; infarto miocardico negli ultimi 6 mesi; angina instabile; angioplastica coronarica o bypass aorto-coronarico negli ultimi 6 mesi; aritmie cardiache atriali o ventricolari non controllate; oppure ipertensione polmonare che richiede un trattamento farmacologico).
- Diagnosi clinica di una qualsiasi malattia del tessuto connettivo (tra cui, senza limitazioni, sclerodermia, polimiosite/dermatomiosite, lupus eritematoso sistemico e artrite reumatoide) oppure diagnosi di polmonite interstiziale con caratteristiche autoimmuni, come stabilito dallo sperimentatore.
- Altre malattie che possono interferire con le procedure dei test o che, a giudizio dello sperimentatore, possono interferire con la partecipazione allo studio o che possono mettere il paziente a rischio durante la partecipazione allo studio.
- Altri motivi non specificati, secondo il parere dello sperimentatore

E.5 End points

E.5.1

Primary end point(s)

Yearly Rate of Change in Forced (Expiratory) Vital Capacity (FVC)

Tasso di variazione annuale della capacità vitale forzata (espiratoria) (CVF)

E.5.1.1

Timepoint(s) of evaluation of this end point

various time point assessments throughout the study

varie valutazioni del punto di rilevazione durante lo studio

E.5.2

Secondary end point(s)

Percent Predicted Yearly Rate of Change in FVC - Time to Progression - Time to First Acute Exacerbation - Percentage of Participants with at least 1 Acute Exacerbation - Number of Exacerbations - Number of Participants with Absolute Decline of 10% Predicted in FVC - Time to Death or Lung Transplantation - Time to All Non-Elective Hospitalizations - Time to All Non-Elective Respiratory Hospitalizations - Change from Baseline in Absolute Predicted FVC - Change from Baseline in Percent Predicted FVC - Change from Baseline in Absolute Predicted Carbon Monoxide Diffusion Capacity (DLco) - Change from Baseline in Percent Predicted Carbon Monoxide Diffusion Capacity (DLco) - Change from Baseline in Absolute Predicted Total Lung Capacity - Change from Baseline in Percent Predicted Total Lung Capacity - Change from Baseline in 6 Minute Walk Test (6MWT) Parameters - Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) - Percentage of Participants With Anti-BG00011 Antibodies in the Serum - Concentration of BG00011 in the Serum

- Tasso di variazione annuale percentuale previsto nella CVF
- Tempo alla progressione
- Tempo alla prima riacutizzazione acuta
- Percentuale di partecipanti con almeno 1 riacutizzazione acuta
- Numero di riacutizzazioni
- Numero di partecipanti con una diminuzione assoluta prevista del 10% nella CVF
- Tempo al decesso o al trapianto polmonare
- Tempo a tutti i ricoveri d’urgenza
- Tempo a tutti i ricoveri d’urgenza per problemi respiratori
- Variazione dal basale nella CVF assoluta prevista
- Variazione dal basale nella CVF percentuale prevista
- Variazione dal basale nella capacità di diffusione del monossido di carbonio (DLco)
assoluta prevista
- Variazione dal basale nella capacità di diffusione del monossido di carbonio (DLco)
percentuale prevista
- Variazione dal basale nella capacità polmonare totale assoluta prevista
- Variazione dal basale nella capacità polmonare totale percentuale prevista
- Variazione dal basale nei parametri del test del cammino in 6 minuti (6MWT)
- Percentuale dei partecipanti con eventi avversi (AE) ed eventi
avversi gravi (SAE)
- Percentuale di partecipanti con anticorpi anti-BG00011 nel siero
- Concentrazione sierica di BG00011

E.5.2.1

Timepoint(s) of evaluation of this end point

Change to baseline, weeks 26 and 52

Modifica al baseline, settimana 26 e 52

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

Yes

E.6.7

Pharmacodynamic

Yes

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)

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

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

Argentina

Austria

Belgium

Canada

Czechia

Denmark

France

Germany

Greece

Israel

Italy

Korea, Republic of

Netherlands

Poland

Russian Federation

Spain

Turkey

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

The end of study is last subject, last visit.

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:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

203

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

290

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)

Normal treatment

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

2018-09-03

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-06-18

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

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IMP with orphan designation in the indication
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