Clinical Trials Register

Summary
EudraCT Number:	2011-002415-28
Sponsor's Protocol Code Number:	CSL654_3001
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2011-10-28
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

Spain - AEMPS

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

Estudio de Fase II/III, abierto y multicéntrico, de la seguridad y la eficacia de una proteína de fusión del factor IX recombinante de la coagulación con la albúmina (rIX-FP) en sujetos con hemofilia 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

EStudio de la eficacia y seguridad del factor recombinante IX en pacientes con hemofilia B severa

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	+496421392930
B.5.5	Fax number	+496421393172
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	Proteína de fusión del factor IX recombinante de la coagulación con la albúmina humana (rIX-FP)
D.3.2	Product code	CSL654
D.3.4	Pharmaceutical form	Powder for concentrate 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 or rIX-FP
D.3.9.3	Other descriptive name	Proteína de fusión recombinante que une el factor de coagulación IX con albúmina humana.
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	Proteína de fusión del factor IX recombinante de la coagulación con la albúmina humana (rIX-FP)
D.3.2	Product code	CSL654
D.3.4	Pharmaceutical form	Powder for concentrate 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 or rIX-FP
D.3.9.3	Other descriptive name	Proteína recombinante de fusión que une el factor de coagulación IX con albúmina humana.
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	Proteína de fusión del factor IX recombinante de la coagulación con la albúmina humana (rIX-FP)
D.3.2	Product code	CSL654
D.3.4	Pharmaceutical form	Powder for concentrate 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 or rIX-FP
D.3.9.3	Other descriptive name	Proteína de fusión recombinante que une el factor de coagulación IX con albúmina humana.
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	Proteína de fusión del factor IX recombinante de la coagulación con la albúmina humana (rIX-FP)
D.3.2	Product code	CSL654
D.3.4	Pharmaceutical form	Powder for concentrate 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 or rIX-FP
D.3.9.3	Other descriptive name	Proteína de fusión recombinante que une el factor de coagulación IX con albúmina humana.
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).

Profilaxis y tratamiento de los episodios hemorrágicos en sujetos con deficiencia congénita del factor IX (FIX) (Hemofilia 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.

Estudio sobre la profilaxis y el tratamiento de un producto de fuente no-plasmática (recombinante) que sustituye la falta del factor de coagulación IX en pacientes con hemofilia severa 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	14.0
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

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.

El objetivo principal del estudio es evaluar la eficacia de la rIX-FP y la respuesta clínica en la prevención de los episodios hemorrágicos (profilaxis) en pacientes con hemofilia B severa.

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.

Los objetivos secundarios del estudio son:
?Evaluar la seguridad de la rIX-FP.
?Evaluar la respuesta clínica a la rIX-FP en el tratamiento de los episodios hemorrágicos en pacientes con hemofilia B severa.
?Evaluar la eficacia de la rIX-FP en la profilaxis quirúrgica.
?Evaluar la farmacocinética (FC) de una dosis única de rIX-FP.

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

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

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.

Podrán entrar en el estudio los sujetos que cumplan todos los criterios de inclusión siguientes:
?Varones de 12 a 65 años de edad.
?Hemofilia B severa documentada (actividad del FIX ? 2%), o confirmada en la Selección por el laboratorio central.
?Sujetos que han recibido productos de FIX (FIX derivado del plasma y/o recombinante) durante > 150 días de exposición (DE), confirmado por su médico.
?Ausencia de historia previa confirmada de formación de inhibidores del FIX (lo que se define como dos estudios positivos consecutivos ? que requieren un estudio confirmador en una segunda muestra extraída poco después de la positiva previa), ausencia confirmada de inhibidores detectables (lo que se define como < 0,6 unidades Bethesda [UB]) en la Selección por el laboratorio central, y ausencia de historia familiar de formación de inhibidores frente al FIX.
?Obtención del consentimiento informado por escrito para la participación en el estudio antes de la práctica de cualquier procedimiento específico del estudio.

SOLAMENTE en los sujetos a demanda:
?Sujetos que han presentado un promedio mínimo de 2 episodios hemorrágicos de origen no traumático al mes en los 3 a 6 últimos meses, que requirieron tratamiento sustitutivo con FIX y que se encuentran documentados en su historia médica.
?Sujetos que están dispuestos a cambiar a una pauta de profilaxis.

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/?L 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.

No podrán entrar en el estudio los sujetos que cumplan cualquiera de los siguientes criterios de exclusión:
?Hipersensibilidad conocida (reacción alérgica o anafilaxia) a cualquier producto de FIX o a las proteínas del hámster.
?Trastorno conocido de la coagulación, congénito o adquirido, distinto de la deficiencia congénita de FIX.
?En tratamiento actualmente con agentes inmunomoduladores IV, como inmunoglobulina, o en tratamiento crónico con corticosteroides sistémicos.
?Recuento de plaquetas < 100.000/µl en la Selección.
?Sujetos positivos para el VIH con un recuento de CD4 < 200/mm3. A criterio del investigador, un sujeto VIH positivo podrá participar en el estudio si recibe tratamiento antiviral.
?Concentración sérica de aspartato aminotransferasa (AST) o de alanina aminotransferasa (ALT) > 5 x LSN en la Selección.
?Concentración sérica de creatinina > 2 x LSN en la Selección.
?Evidencia de trombosis, incluidos trombosis venosa profunda, ictus, infarto de miocardio o émbolo arterial, en el plazo de los 4 meses previos a la administración del Día 1.
?Episodio hemorrágico con riesgo para la vida, como hemorragia en el sistema nervioso central, tracto gastrointestinal, cuello/garganta o episodio hemorrágico severo de origen traumático, o intervención quirúrgica mayor, en el plazo de los 4 meses previos a la administración del Día 1.
?Tratamiento con cualquier producto en investigación (PEI) distinto de la rIX-FP en las 4 semanas previas a la primera administración de la rIX-FP el Día 1.
?Enfermedad articular inflamatoria no hemofílica concurrente u otro proceso médico que, en opinión del Investigador, pudiera actuar como factor de confusión de los resultados del estudio.
?Sospecha de incapacidad (por ejemplo, problemas de lenguaje o trastorno mental) o de falta de deseos de cumplir con los procedimientos del estudio, o historia de incumplimiento del tratamiento.

SOLAMENTE en los sujetos a demanda:
?Sujetos con sinovitis activa.
?Sujetos que reciben habitualmente un infusión de factor IX antes de una actividad (por ejemplo, deporte) como medida preventiva más de 2 veces al mes.

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.

Tasas anualizadas de hemorragia espontánea durante el tratamiento a demanda comparado con las tasas anualizadas espontáneas durante el tratamiento profiláctico.

E.5.1.1

Timepoint(s) of evaluation of this end point

approximately 20 weeks

Aproximadamente 20 semanas

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)

?Número de episodios hemorrágicos espontáneos por sujeto después del comienzo del tratamiento.
?Porcentaje de episodios hemorrágicos que han precisado una o dos infusiones de rIX-FP para alcanzar la hemostasia.
?Evaluación clínica global por el Investigador de la eficacia hemostática en el tratamiento de los episodios hemorrágicos.
?Uso de rIX-FP en el tratamiento profiláctico.

E.5.2.1

Timepoint(s) of evaluation of this end point

168 hours

168 horas

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)

Yes

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)

E.8.2.2

Placebo

E.8.2.3

Other

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

Austria

Bulgaria

France

Germany

Israel

Italy

Japan

Russian Federation

Spain

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.

El fin de la participación en el estudio de un sujeto individual se produce con el final del período de seguimiento, que se define como la finalización de la última visita del estudio, tras el cual no más estudios relacionados con los procedimientos se llevarán a cabo. Los sujetos en profilaxis (grupo 1) y en a demanda (grupo 2) completarán la visita de fin de estudio aproximadamente a la semana 60. El final del ensayo está definido como la última visita del último paciente.

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

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

Pacientes que terminaron su participación en el ensayo pueden volver a la terapia estandar de acuerdo a su plan de tratamiento. Hay otros productos Factor recombinante IX disponibles en el mercado.

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

2011-12-23

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2011-12-01

P. End of Trial
P.	End of Trial Status	Completed

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IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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