Clinical Trials Register

Summary
EudraCT Number:	2012-004867-38
Sponsor's Protocol Code Number:	NN7999-3895
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:	2018-02-15
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

2012-004867-38

A.3

Full title of the trial

Safety and efficacy of nonacog beta pegol (N9-GP) in Previously Untreated Patients with Haemophila B.

An open-label single-arm multicentre non-controlled phase 3a trial investigating safety and efficacy
of nonacog beta pegol (N9-GP) in prophylaxis and treatment of bleeding episodes in previously
untreated patients with haemophilia B (FIX activity ≤2%)

Sicurezza ed efficacia di nonacog beta pegol (N9-GP) in pazienti con Emofilia B precedentemente non trattati.

Studio di fase 3a, multicentrico in aperto, a singolo braccio non controllato, su efficacia e sicurezza di nonacog beta pegol (N9-GP) in profilassi e nel trattamento degli episodi di sanguinamento in pazienti con Emofilia B precedentemente non trattati (attività del FIX ≤2%).

A.3.1

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

Safety and efficacy of nonacog beta pegol (N9-GP) in Previously Untreated Patients with Haemophila B

Sicurezza ed efficacia di nonacog beta pegol (N9-GP) in pazienti con Emofilia B precedentemente non trattati

A.3.2

Name or abbreviated title of the trial where available

An open-label single-arm multicentre non-controlled phase 3a trial investigating safety and efficacy

Sicurezza ed efficacia di nonacog beta pegol (N9-GP) in pazienti con Emofilia B precedentemente non

A.4.1

Sponsor's protocol code number

NN7999-3895

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/731/2009

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	NOVO NORDISK. S.P.A.
B.1.3.4	Country	Italy
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	Novo Nordisk A/S
B.4.2	Country	Denmark
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Novo Nordisk A/S
B.5.2	Functional name of contact point	Global Clinical Registry
B.5.3	Address:
B.5.3.1	Street Address	Novo Allé
B.5.3.2	Town/ city	Bagsvaerd
B.5.3.3	Post code	2880
B.5.3.4	Country	Denmark
B.5.4	Telephone number	.
B.5.5	Fax number	.
B.5.6	E-mail	clinicaltrials@novonordisk.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	Yes
D.2.5.1	Orphan drug designation number	EU/3/09/640
D.3 Description of the IMP
D.3.1	Product name	N9-GP 2000IU /fiala
D.3.4	Pharmaceutical form	Powder and solvent for solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	NONACOG BETA PEGOL
D.3.9.2	Current sponsor code	NNC0156-009
D.3.9.4	EV Substance Code	SUB33409
D.3.10	Strength
D.3.10.1	Concentration unit	IU/ml international unit(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	500
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	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	Yes
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	Yes
D.2.5.1	Orphan drug designation number	EU/3/09/640
D.3 Description of the IMP
D.3.1	Product name	N9-GP 500IU/ fiala
D.3.4	Pharmaceutical form	Powder and solvent for solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	NONACOG BETA PEGOL
D.3.9.2	Current sponsor code	NNC0156-009
D.3.9.4	EV Substance Code	SUB33409
D.3.10	Strength
D.3.10.1	Concentration unit	IU/ml international unit(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	125
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	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

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Haemophilia B

Emofilia B

E.1.1.1

Medical condition in easily understood language

Bleeding disorder, inherited deficiency in clotting factor IX

Disordine ereditario della coagulazione

E.1.1.2

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10018939
E.1.2	Term	Haemophilia B (Factor IX)
E.1.2	System Organ Class	100000004850

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 immunogenicity of N9-GP

Valutare l'immunogenicità di N9-GP

E.2.2

Secondary objectives of the trial

- To evaluate safety of N9-GP (nonacog beta pegol)
- To evaluate efficacy of N9-GP (nonacog beta pegol)
o in long-term prophylaxis treatment
o in the treatment of bleeding episodes
o through the surrogate marker: FIX activity
o through monitoring of number of doses and consumption of N9-GP

1.Valutare la sicurezza di N9-GP
2.Valutare l'efficacia di N9-GP:
- nel trattamento di profilassi a lungo termine
-nel trattamento degli episodi di sanguinamento
-attraverso un marker surrogato: attività del FIX
-attraverso il monitoraggio del numero delle dosi necessarie ed il consumo di N9-GP

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Informed consent obtained before any trial-related activities. Trialrelated
activities are any procedures that are carried out as part of the
trial, including activities to determine suitability for the trial
- Male, < 6 years of age at the time of signing informed consent
- Patients with the diagnosis of haemophilia B (FIX activity level ≤ 2%)
based on medical records or central laboratory results
- Previously untreated or exposed to FIX containing products less than
or equal to 3 exposure days (5 previous exposure days to blood
components are acceptable)

-Consenso Informato ottenuto prima di qualsiasi attività specifica dello studio
-Soggetto maschio di età inferiore ai 6 anni di età al momento della firma del consenso informato
-Diagnosi di Emofilia B (Livello di attività del FIX ≤ 2%)
-Pazienti precedentemente non trattati o con un massimo di 3 giorni di esposizione a prodotti ricombinanti contenenti FIX o di 5 giorni per prodotti plasma derivati

E.4

Principal exclusion criteria

- Any history of FIX inhibitors (defined by medical records)
- Known or suspected hypersensitivity to trial product or related
products
- Previous participation in this trial. Participation is defined as first dose
administered of trial product
- Receipt of any investigational medicinal product within 30 days before
screening
- Congenital or acquired coagulation disorder other than haemophilia B
- Any chronic disorder or severe disease which, in the opinion of the
Investigator, might jeopardise patient's safety or compliance with the
protocol
- Patient's parent(s)/LAR(s) mental incapacity, unwillingness to
cooperate, or a language barrier precluding adequate understanding and
cooperation

-Anamnesi di inibitori al FIX ( da cartella medica)
-Sospetta o riconosciuta ipersensività al prodotto sperimentale o a prodotti correlati
-Trattamento con altri prodotti sperimentali fino a 30 giorni precedenti allo screening
-Altre patologie della coagulazione oltre l'Emofilia B congenite od acquisite
-Qualsiasi disordine cronico o malattia grave che nell'opinione dello sperimentatore potrebbe mettere a rischio la sicurezza o la compliance del protocollo del paziente

E.5 End points

E.5.1

Primary end point(s)

Incidence of inhibitory antibodies against FIX

Incidenza di Anticorpi inibitori contro il Fattore IX

E.5.1.1

Timepoint(s) of evaluation of this end point

All objectives/endpoints will be evaluated when the first 20 PUPs have
reached at least 50 EDs, when the first 40 PUPs have reached 100 EDs,
and at end of trial. End of trial will be up to 4 years after the patient has
reached 100 EDs.

Tutti gli obiettivi/endpoints verranno valutati quando i primi 20 pazienti PUP raggiungeranno un minimo di 50 giorni di esposizione, quando i primi 40 pazienti PUP raggiungeranno 100 giorni di esposizione e al termine della sperimentazione.

E.5.2

Secondary end point(s)

- Number and frequency of adverse events, serious adverse events, and
Medical Events of Special interest
- Number of breakthrough bleeding episodes during prophylaxis
(annualised bleeding rate)
- Haemostatic effect by 4-point haemostatic response scale ("excellent",
"good", "moderate" and "poor")

-Numero e frequenza degli eventi avversi, degli eventi avversi seri e MESI (Medical Event of Special Interest)
-Numero degli episodi di sanguinamento durante trattamento in profilassi
-Valutazione dell'effetto emostatico secondo una scala a 4 punti

E.5.2.1

Timepoint(s) of evaluation of this end point

All objectives/endpoints will be evaluated when the first 20 PUPs have reached at least 50 EDs, when the first 40 PUPs have reached 100 EDs, and at end of trial. End of trial will be up to 4 years after the patient has reached 100 EDs.

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

Immunogenicity

Immunogenicità

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

E.8.1.1

Randomised

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

Information not present in EudraCT

E.8.1.6

Cross over

Information not present in EudraCT

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

E.8.2.3

Other

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

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

France

Germany

Israel

Italy

Japan

Malaysia

Spain

Thailand

United States

E.8.7

Trial has a data monitoring committee

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

Number of subjects for this age range:

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)

Yes

F.1.1.3.1

Number of subjects for this age range:

F.1.1.4

Infants and toddlers (28 days-23 months)

Yes

F.1.1.4.1

Number of subjects for this age range:

F.1.1.5

Children (2-11years)

Yes

F.1.1.5.1

Number of subjects for this age range:

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

F.1.3

Elderly (>=65 years)

F.2 Gender

F.2.1

Female

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

F.3.3.1

Women of childbearing potential not using contraception

Information not present in EudraCT

F.3.3.2

Women of child-bearing potential using contraception

Information not present in EudraCT

F.3.3.3

Pregnant women

Information not present in EudraCT

F.3.3.4

Nursing women

Information not present in EudraCT

F.3.3.5

Emergency situation

Information not present in EudraCT

F.3.3.6

Subjects incapable of giving consent personally

Information not present in EudraCT

F.3.3.7

Others

Information not present in EudraCT

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)

N/A

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

2015-10-07

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2015-11-12

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

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