Clinical Trials Register

Summary
EudraCT Number:	2011-006032-23
Sponsor's Protocol Code Number:	CSL654_3002
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:	2012-09-27
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

2011-006032-23

A.3

Full title of the trial

A Phase III Open-label, Multicenter, Pharmacokinetics, Safety, and Efficacy Study of a Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP) in Previously Treated Children with Hemophilia B

Studio di fase 3, in aperto, multicentrico, sulla farmacocinetica, sulla sicurezza e sull efficacia di una proteina di fusione ricombinante che lega il fattore IX della coagulazione con albumina (rIX-FP) in bambini con emofilia B precedentemente trattati

A.3.1

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

A safety and efficacy Study of a recombinant Factor IX in pediatric patients with severe Hemophilia B

Studio sulla sicurezza e sull’efficacia del fattore IX ricombinante in pazienti pediatrici con grave emofilia B

A.4.1

Sponsor's protocol code number

CSL654_3002

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/251/2011

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	CSL BEHRING GMBH
B.1.3.4	Country	Germany
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	CSL Behring GmbH
B.4.2	Country	Germany
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	PAREXEL International
B.5.2	Functional name of contact point	Project Leader
B.5.3	Address:
B.5.3.1	Street Address	Kartauserstr. 47
B.5.3.2	Town/ city	Freiburg
B.5.3.3	Post code	79102
B.5.3.4	Country	Germany
B.5.4	Telephone number	+49 761 28280 11
B.5.5	Fax number	+49 30306857203
B.5.6	E-mail	ilja.weber@parexel.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	EMEA/OD/117/09
D.3 Description of the IMP
D.3.1	Product name	proteina di fusione ricombinante che lega il fattore IX di coagulazione, con albumina (rIX-FP)
D.3.2	Product code	CSL654
D.3.4	Pharmaceutical form	Powder and solvent for solution for injection/infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.9.2	Current sponsor code	CSL654 or rIX-FP
D.3.10	Strength
D.3.10.1	Concentration unit	IU international unit(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	250
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.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	EMEA/OD/117/09
D.3 Description of the IMP
D.3.1	Product name	proteina di fusione ricombinante che lega il fattore IX di coagulazione, con albumina (rIX-FP)
D.3.2	Product code	CSL654
D.3.4	Pharmaceutical form	Powder and solvent for solution for injection/infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.9.2	Current sponsor code	CSL654 or rIX-FP
D.3.10	Strength
D.3.10.1	Concentration unit	IU international unit(s)
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	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.IMP: 3
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	EMEA/OD/117/09
D.3 Description of the IMP
D.3.1	Product name	proteina di fusione ricombinante che lega il fattore IX di coagulazione, con albumina (rIX-FP)
D.3.2	Product code	CSL654
D.3.4	Pharmaceutical form	Powder and solvent for solution for injection/infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.9.2	Current sponsor code	CSL654 or rIX-FP
D.3.10	Strength
D.3.10.1	Concentration unit	IU international unit(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	1000
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.IMP: 4
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	EMEA/OD/117/09
D.3 Description of the IMP
D.3.1	Product name	proteina di fusione ricombinante che lega il fattore IX di coagulazione, con albumina (rIX-FP)
D.3.2	Product code	CSL654
D.3.4	Pharmaceutical form	Powder and solvent for solution for injection/infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.9.2	Current sponsor code	CSL654 or rIX-FP
D.3.10	Strength
D.3.10.1	Concentration unit	IU international unit(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	2000
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

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Prophylaxis and treatment of bleeding episodes in previously treated children with congenital FIX deficiency (hemophilia B)

Profilassi e trattamento degli episodi di sanguinamento in bambini con deficit congenito di fattore IX (FIX) (emofilia B)

E.1.1.1

Medical condition in easily understood language

A study on prophylaxis and treatment of a non-plasma source (recombinant) product that replaces the missing clotting factor IX in previously treated children with sever Hemophilia B.

Uno studio sulla profilassi ed il trattamento di un prodotto non derivato dal plasma (ricombinante)che sostituisce la mancanza del fattore IX di coagulazione in bambini già trattati....

E.1.1.2

Therapeutic area

Body processes [G] - Genetic Phenomena [G05]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	15.0
E.1.2	Level	LLT
E.1.2	Classification code	10060614
E.1.2	Term	Hemophilia B (Factor IX)
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 PK of a single dose of rIX-FP. •To evaluate the safety of rIX-FP with respect to the development of inhibitors to FIX in patients with severe hemophilia B (FIX: ≤ 2%).

-valutare la farmacocinetica (PK) di una singola dose; -valutare la sicurezza di rIX-FP rispetto allo sviluppo di inibitori del FIX in pazienti pediatrici affetti da una forma grave di emofilia B (FIX:<=2%).

E.2.2

Secondary objectives of the trial

•To evaluate the safety of rIX-FP, based on AEs and the development of antibodies to rIX-FP. •To evaluate the clinical response of rIX-FP for the prevention of bleeding episodes. •To evaluate the clinical response of rIX-FP in treatment bleeding episodes.

-valutare la sicurezza del rIX-FP in base agli AE correlati ed allo sviluppo di anticorpi al rIX-FP; -valutare la risposta clinica al rIX-FP nella prevenzione degli episodi di sanguinamento; -valutare la risposta clinica del rIX-FP del trattamento degli episodi di sanguinamento.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

•Male subjects, aged younger than 12 years (Prior to their 12th birthday at Day 1), and body weight ≥ 10 kg at the time of screening visit •Documented severe hemophilia B (FIX activity of ≤ 2%) •Subjects who are currently receiving routine FIX replacement therapy and have received FIX products for > 150 EDs (6 to < 12 years of age) or > 50 EDs (< 6 years of age), confirmed by their treating physician. •No confirmed prior history of FIX inhibitor formation (defined as two consecutive positive tests –requiring a confirmatory test on a second separately drawn blood sample shortly after the previous positive test), no confirmed detectable inhibitors (defined as < 0.6 Bethesda Units [BU]) at Screening by the central laboratory, and no family history ofinhibitor formation against FIX. •Written informed consent for study participation obtained before undergoing any study specific procedures. •Ability of subject/caregiver to assess a bleeding episode, and document treatment with an electronic diary.

• Soggetti di sesso maschile di età <12 anni (il Giorno 1 deve precedere il loro 12° compleanno) e con peso corporeo >=10 kg alla visita di screening; • Emofilia B grave documentata (attività di FIX <=2%); • Soggetti in trattamento corrente con terapia profilattica sostitutiva del FIX e che abbiano ricevuto prodotti a base di FIX per >150 ED (da 6 a <12 anni) o >50 ED (<6 anni), come confermato dal medico curante; • Nessuna storia precedente confermata di formazione di inibitori del FIX (definita come due test positivi consecutivi – è necessario un test di conferma su un secondo campione di sangue prelevato separatamente a breve distanza dal precedente test positivo), nessuna conferma da parte del laboratorio centrale della presenza di inibitori rilevabili (definiti come <0,6 unità Bethesda [BU]) allo Screening e nessuna familiarità per la formazione di inibitori del FIX; • Consenso informato scritto alla partecipazione allo studio ottenuto prima di sottoporre il soggetto a qualsiasi procedura specifica dello studio; • Capacità del soggetto/caregiver di valutare un episodio di sanguinamento e di documentare il trattamento su un diario elettronico.

E.4

Principal exclusion criteria

•Known hypersensitivity (allergic reaction or anaphylaxis) to any FIX product or hamster protein. •Known congenital or acquired coagulation disorder other than congenital FIX deficiency. •Currently receiving IV immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment. •Platelet count < 100,000/μL at Screening. •Documented HIV positive subjects with a CD4 count < 200/mm3 •Serum aspartate aminotransferase (AST) or serum alanine aminotransferase (ALT) concentration > 5 x upper limit of normal (ULN) at Screening. •Serum creatinine concentration > 2 x ULN at Screening. •Experienced a life-threatening bleeding episode or had major surgical intervention within 4 months prior to dosing on Day 1. •Evidence of thrombosis, including deep vein thrombosis, stroke, myocardial infarction or arterial embolus within 4 months prior to dosing on Day 1. •Planning to have major surgical procedure during the study period. •Use of any Investigational Medicinal Product (IMP) other than rIX-FP within 4 weeks prior to the first day of rIX-FP administration. •Participated in any extended half-life Investigational FIX product clinical study other than for rIX-FP. •Concurrent inflammatory joint disease or other medical condition that could confound study results. •Suspected inability or unwillingness of study subjects and/or caregiver to comply with study procedures or history of noncompliance.

• Ipersensibilità nota (reazione allergica o anafilassi) a qualsiasi prodotto a base di FIX o alle proteine di criceto; • Disturbo della coagulazione congenito o acquisito noto diverso dal deficit congenito di FIX; • Terapia corrente con agenti immunomodulatori per via EV come immunoglobulina o trattamento cronico con corticosteroidi per via sistemica; • Conta piastrinica <100.000/µL allo Screening; • Soggetti con positività documentata all’HIV con conta delle cellule CD4 <200/mm3; • Concentrazioni sieriche di aspartato aminotransferasi (AST) o alanina aminotransferasi (ALT) >5 x il limite superiore della norma (ULN) allo Screening; • Concentrazioni di creatinina sierica >2 x ULN allo Screening; • Episodio di sanguinamento potenzialmente fatale o intervento di chirurgia maggiore nei 4 mesi precedenti la somministrazione di rIX-FP al Giorno 1; • Evidenze di trombosi, inclusa trombosi venosa profonda, ictus, infarto del miocardio ed embolo arterioso nei 4 mesi precedenti la somministrazione di rIX-FP al Giorno 1; • Intervento di chirurgia maggiore pianificato durante il periodo di studio; • Uso di eventuali prodotti medicinali sperimentali (IMP) diversi da rIX-FP nelle 4 settimane precedenti il primo giorno di somministrazione di rIX-FP; • Precedente partecipazione a eventuali studi clinici su un prodotto sperimentale a base di FIX con emivita lunga diverso da rIX-FP; • Patologia articolare infiammatoria o altra affezione concomitante che potrebbe confondere i risultati dello studio; • Sospetta incapacità o mancata disponibilità dei soggetti di studio e/o caregiver a rispettare le procedure di studio o storia di mancata compliance.

E.5 End points

E.5.1

Primary end point(s)

•Incremental recovery (IU/ml/IU/kg) of 50 IU/kg rIX-FP. •Half-life (t1/2) of a single dose of 50 IU/kg rIX-FP. •AUC of the last sample with quantifiable drug concentration (AUC0-t) of a single dose of 50 IU/kg rIX-FP. •Clearance of a single dose of 50 IU/kg rIX-FP. •The number of subjects with FIX inhibitors.

• Recupero incrementale (IU/mL/IU/kg) di rIX-FP 50 IU/kg; • Emivita (t1/2) di una dose singola di rIX-FP 50 IU/kg; • AUC dell’ultimo campione con concentrazioni di farmaco quantificabili (AUC-t) di una dose singola di rIX-FP 50 IU/kg; • Clearance di una dose singola di rIX-FP 50 IU/kg; • Numero di soggetti con inibitori del FIX.

E.5.1.1

Timepoint(s) of evaluation of this end point

10-14 days for the PK assessment. approximately 12 months for the inhibitor assessment. (throughout the study)

10-14 giorni per la valutazione farmacocinetica (PK). Circa 12 mesi per la valutazione degli inibitori (per l’intera durata dello studio)

E.5.2

Secondary end point(s)

•The frequency of related AEs to rIX-FP over the course of the study. •The number of subjects who developed antibodies against rIX-FP. •The consumption of rIX-FP, as expressed as number of infusion and IU/kg per month and per year, as well as IU/kg per event. •Proportion of bleeding episodes requiring one, two or more than two infusions of rIX-FP to achieve hemostasis.

• Frequenza degli AE correlati a rIX-FP nel corso dello studio; • Numero di soggetti che sviluppano anticorpi anti rIX-FP; • Consumo di rIX-FP, espresso come numero di infusioni e IU/kg al mese e all’anno, nonché come IU/kg per evento; • Percentuale di episodi di sanguinamento che necessitano di una, due o più infusioni di rIX-FP per raggiungere l’emostasi.

E.5.2.1

Timepoint(s) of evaluation of this end point

approximately 12 months (throughout the study)

circa 12 mesi (per l’intera durata dello studio)

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

Yes

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

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

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

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)

Information not present in EudraCT

E.8.2.2

Placebo

Information not present in EudraCT

E.8.2.3

Other

Information not present in EudraCT

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

Australia

Canada

Russian Federation

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

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)

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

F.3.3.2

Women of child-bearing potential using contraception

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)

Patients who end their participation in the trial can be enrolled in the planned extension study or return to standard therapy according to their health care scheme. Patients who has received rIX-FP and discontinue participation in the study prematurely can return to standard therapy according to their health care scheme.

I pazienti che completano la partecipazione allo studio possono essere arruolati nello studio di estensione previsto o tornare alla terapia standard in base al loro piano di cure. I pazienti che hanno ricevuto rIX-FP e interrompono anticipatamente la partecipazione allo studio possono tornare alla terapia standard in base al loro piano di cure.

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

2012-08-16

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2012-09-18

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2014-10-05

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