Clinical Trials Register

Summary
EudraCT Number:	2011-002415-28
Sponsor's Protocol Code Number:	CSL654_3001
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:	2011-12-27
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

2011-002415-28

A.3

Full title of the trial

A Phase II/III Open-label, Multicenter, Safety and Efficacy Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects with Hemophilia B

Studio di fase II/III, multicentrico, in aperto sulla sicurezza e sull'™efficacia di una proteina di fusione albumina-fattore IX di coagulazione ricombinante (rIX-FP) in soggetti con Emofilia B

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 patients with severe Hemophilia B

Studio di efficacia e di sicurezza del Fattore IX ricombinante in pazienti con grave Emofilia B

A.4.1

Sponsor's protocol code number

CSL654_3001

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	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	CSL Behring
B.5.2	Functional name of contact point	Sr. Clinical Program Manager
B.5.3	Address:
B.5.3.1	Street Address	Emil-von-Behring-Str. 76
B.5.3.2	Town/ city	Marburg
B.5.3.3	Post code	35041
B.5.3.4	Country	Germany
B.5.4	Telephone number	+49 6421392930
B.5.5	Fax number	+49 6421393172
B.5.6	E-mail	kerstin.jung@cslbehring.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.3.1	Product name	Recombinant Fusion Protein Linking Human Coagulation Factor IX with Human Albumin (rIX-FP)
D.3.2	Product code	CSL654
D.3.4	Pharmaceutical form	Powder for solution for 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 o 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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Recombinant Fusion Protein Linking Human Coagulation Factor IX with Human Albumin (rIX-FP)
D.3.2	Product code	CSL654
D.3.4	Pharmaceutical form	Powder for solution for 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 o 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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Recombinant Fusion Protein Linking Human Coagulation Factor IX with Human Albumin (rIX-FP)
D.3.2	Product code	CSL654
D.3.4	Pharmaceutical form	Powder for solution for 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 o 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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Recombinant Fusion Protein Linking Human Coagulation Factor IX with Human Albumin (rIX-FP)
D.3.2	Product code	CSL654
D.3.4	Pharmaceutical form	Powder for solution for 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 o 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 subjects with congenital Factor IX (FIX) deficiency (Hemophilia B).

Profilassi e trattamento degli episodi di sanguinamento in soggetti 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 patients with sever Hemophilia B.

uno studio sulla profilssi ed il trattamento di un prodotto (ricombinante) non derivato dal plasma che supplisce il deficit di fattore IX di coagulazione in pazienti con grave emofilia B

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	14.1
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

E.2 Objective of the trial

E.2.1

Main objective of the trial

The primary objective of the study is to evaluate the efficacy of rIX-FP and the clinical response in preventing bleeding episodes (prophylaxis) in patients with severe hemophilia B.

L’obiettivo primario dello studio è valutare l’efficacia di rIX-FP e la risposta clinica nella prevenzione degli episodi di sanguinamento (profilassi) in pazienti con emofilia B grave.

E.2.2

Secondary objectives of the trial

The secondary objectives of the study are: •To evaluate the safety of rIX-FP. •To evaluate the clinical response to rIX-FP for the treatment of bleeding episodes in patients with severe hemophilia B. •To evaluate the efficacy of rIX-FP in surgical prophylaxis. •To evaluate the pharmacokinetics (PK) of a single dose of rIX-FP.

Gli obiettivi secondari dello studio sono tesi a: - valutare la sicurezza di rIX-FP - valutare la risposta clinica a rIX-FP per il trattamento degli episodi di sanguinamento in pazienti con grave emofilia B - valutare e l’efficacia di rIX-FP nella profilassi chirurgica - valutare la farmacocinetica (PK)

E.2.3

Trial contains a sub-study

Yes

E.2.3.1

Full title, date and version of each sub-study and their related objectives

OTHER SUBSTUDIES:
A Phase II/III Open-label, Multicenter, Safety and Efficacy Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects with Hemophilia B—Surgical Substudy

ALTRI SOTTOSTUDI:
Studio di fase II/III, multicentrico, in aperto, di valutazione della sicurezza ed efficacia di una proteina di fusione ricombinante fattore IX di coagulazione - albumina (rIX-FP) in soggetti con emo

E.3

Principal inclusion criteria

Male subjects, 12 to 65 years of age. - Documented severe hemophilia B (FIX activity of ≤ 2%), or confirmed at Screening by the central laboratory. - Subjects who have received FIX products (plasma-derived and/or recombinant FIX) for > 150 exposure days (EDs), 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 of inhibitor formation against FIX. - Written informed consent for study participation obtained before undergoing any study specific procedures. For on-demand subjects ONLY: - Subjects who have experienced a minimum average of 2 non-trauma induced bleeding episodes per month in the past 3 to 6 months, which required FIX replacement therapy and are documented in their medical records. - Subjects who are willing to switch to a prophylaxis regimen.

- Soggetti di sesso maschile, di età compresa tra 12 e 65 anni. - Grave emofilia B (attività di FIX <= 2%) documentata o confermata allo Screening dal laboratorio centrale. - Soggetti che hanno ricevuto prodotti contenenti FIX (plasmaderivati e/o FIX ricombinante) per > 150 giorni di esposizione, come confermato dal medico curante. - Nessuna anamnesi pregressa confermata di formazione di inibitori di FIX (definita come due test consecutivi positivi – è necessario un test di conferma su un secondo campione prelevato separatamente poco dopo il precedente test positivo), assenza di inibitori rilevabili (definita come < 0,6 unità Bethesda [BU]) confermata allo Screening dal laboratorio centrale e nessuna anamnesi familiare di formazione di inibitori contro FIX. - Consenso informato scritto alla partecipazione allo studio ottenuto prima di sottoporsi a qualsiasi procedura specifica per lo studio. SOLO per i soggetti in terapia al bisogno: - Soggetti che hanno evidenziato una media minima di 2 episodi di sanguinamento non indotti da trauma al mese negli ultimi 3 - 6 mesi, che hanno richiesto terapia sostitutiva con FIX e sono documentati nella cartella clinica. - Soggetti disposti a passare a un regime di profilassi.

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. - HIV positive subjects with a CD4 count < 200/mm3. An HIV-positive subject may participate in the study and receive antiviral therapy at the discretion of the Investigator. - Serum aspartate aminotransferase (AST) or serum alanine aminotransferase (ALT) concentration > 5 x ULN at Screening. - Serum creatinine concentration > 2 x ULN at Screening. - Evidence of thrombosis, including deep vein thrombosis, stroke, myocardial infarction or arterial embolus within 4 months prior to dosing on Day 1. - Experienced a life-threatening bleeding episode, including bleeding in the central nervous system, gastrointestinal tract, neck/throat or severe trauma-induced bleeding episode, or had major surgical intervention within 4 months prior to dosing on Day 1. - Use of any Investigational Medicine Product (IMP) other than rIX-FP within 4 weeks prior to the first rIX-FP administration on Day 1. - Concurrent non-hemophiliac inflammatory joint disease or other medical condition that, in the Investigator's judgment, could confound study results. - Suspected inability (e.g., language problem or mental condition) or unwillingness to comply with study procedures or history of noncompliance. - Subjects who have active synovitis. - Subject routinely receives factor IX infusion prior to activity (ie, sports) as a preventative measure more than 2 times per month.

- Nota ipersensibilità (reazione allergica o anafilassi) a qualsiasi prodotto contenente FIX o proteina di criceto. - Noto disturbo della coagulazione congenito o acquisito a parte il deficit congenito di FIX. - Terapia attuale con agenti immunomodulanti per via endovenosa, quali immunoglobuline o corticosteroidi sistemici cronici. - Conta piastrinica < 100.000/μl allo Screening. - Soggetti HIV positivi con una conta dei CD4 < 200/mm3. Un soggetto HIV positivo può partecipare allo studio e ricevere terapia antivirale a discrezione dello sperimentatore. - Concentrazione sierica di aspartato aminotransferasi (AST) o alanina aminostransferasi (ALT) > 5 volte il limite superiore della norma (ULN) allo Screening. - Concentrazione sierica di creatinina > 2 volte ULN allo Screening. - Evidenze di trombosi, compresa trombosi venosa profonda, ictus, infarto miocardico o embolia arteriosa, nei 4 mesi precedenti la somministrazione il Giorno 1. - Soggetti che hanno subito un episodio di sanguinamento pericoloso per la vita, compresi emorragia del sistema nervoso centrale, del tratto gastrointestinale, del collo/della gola o grave episodio emorragico indotto da trauma, o che hanno subito un intervento chirurgico maggiore negli ultimi 4 mesi prima della somministrazione il Giorno 1. - Uso di qualsiasi medicinale sperimentale a parte rIX-FP nelle 4 settimane precedenti la prima somministrazione di rIX-FP il Giorno 1. - Concomitante patologia infiammatoria articolare non emofilica o altra condizione medica che, a giudizio dello sperimentatore, potrebbe confondere i risultati dello studio. - Sospetta incapacità (es. problemi di linguaggio o condizione mentale) o mancata disponibilità a rispettare le procedure di studio o anamnesi di mancata compliance. - Soggetti con sinovite attiva. - Soggetti che ricevono abitualmente infusioni di fattore IX prima di attività (es. sport) come misura preventiva più di 2 volte al mese.

E.5 End points

E.5.1

Primary end point(s)

Annualized spontaneous bleeding events during the on-demand treatment period compared to the annualized spontaneous bleeding events during the routine prophylaxis treatment period.

Sanguinamenti spontanei annualizzati durante il periodo di trattamento al bisogno rispetto ai sanguinamenti annualizzati durante il periodo di trattamento profilattico di routine.

E.5.1.1

Timepoint(s) of evaluation of this end point

approximately 20 weeks

approssimativamente 20 settimane

E.5.2

Secondary end point(s)

- The frequency of related Adverse Events (AEs) to rIX-FP over the course of the study. - The number of subjects with FIX inhibitors. - The number of subjects with antibodies against rIX-FP. - Proportion of bleeding episodes requiring one or ≤ two infusions of rIX-FP to achieve hemostasis. - Investigator's overall clinical assessment of hemostatic efficacy for treatment of bleeding episodes, based on a four point ordinal scales (excellent, good, moderate, poor/ none). - rIX-FP consumed per month while maintaining assigned prophylactic treatment interval during routine prophylaxis. - Incremental recovery (IU/mL/IU/kg) at 30 minutes following infusion of 50 IU/kg rIX-FP. - Half-life (t1/2) of a single dose of 50 IU/kg rIX-FP. - AUC to 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. - Investigator's (or surgeon's) overall clinical assessment of hemostatic efficacy for surgical prophylaxis, based on a four point ordinal scale (excellent, good, moderate, poor/ none)

- Frequenza degli eventi avversi (AE) correlati a rIX-FP nel corso dello studio. - Numero di soggetti che evidenziano inibitori del FIX. - Numero di soggetti che evidenziano anticorpi contro rIX-FP. - Percentuale di episodi di sanguinamento che richiedono una o <= due infusioni di rIX-FP per raggiungere l'emostasi. - Valutazione clinica globale da parte dello sperimentatore dell'efficacia emostatica nel trattamento di episodi di sanguinamento, in base a una scala ordinale a quattro punti (eccellente, buona, moderata, scarsa/assente). - rIX-FP consumata al mese mantenendo l'intervallo terapeutico profilattico assegnato durante la profilassi di routine. - Recupero incrementale (UI/ml/UI/kg) 30 minuti dopo l'infusione di 50 UI/kg di rIX-FP. - Emivita (t1/2) di una singola dose di 50 UI/kg di rIX-FP. - AUC fino all'ultimo campione con concentrazione misurabile di farmaco (AUC0-t) di una singola dose di 50 UI/kg di rIX-FP. - Clearance di una singola dose di 50 UI/kg di rIX-FP. - Valutazione clinica globale da parte dello sperimentatore (o del chirurgo) dell'efficacia emostatica nella profilassi chirurgica, in base a una scala ordinale a quattro punti (eccellente, buona, moderata, scarsa/assente).

E.5.2.1

Timepoint(s) of evaluation of this end point

168 hours

168 ore

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

Yes

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

Yes

E.8.1.7.1

Other trial design description

nessun confronto,profilassi ed alla necessità

no comparator, prophylaxis and on demand treatment

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

Israel

Japan

Russian Federation

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 participation for an individual subject occurs with the end of the follow-up period, which is defined as completion of the final study visit, after which no further study-related procedures will be performed. Prophylaxis subjects (Arm 1) and On-demand subjects (Arm 2) will complete the end-of-study visit on approximately Week 60. The end of the trial is defined as the last-patient-last-visit.

La conclusione della partecipazione allo studio di un particolare soggetto avviene al termine del periodo di follow-up, definito come il completamento della visita di studio finale, dopo la quale non saranno eseguite altre procedure per lo studio...

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)

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:

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

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

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

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

minors between 12 and 17 years

minori tra 12 e 17 anni

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 return to standard therapy according to their health care scheme. There are several other recombinant Factor IX products available on the market.

I pazienti che concluderanno la loro partecipazione allo studio potranno ritornare alla terapia standard secondo quanto previsto dal Sistema Sanitario Nazionale.Ci sono diversi altri prodotti con Fattore IX ricombinante disponibili sul mercato.

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

2012-02-07

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2011-11-15

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2014-07-21

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IMP with orphan designation in the indication
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