Clinical Trials Register

Summary
EudraCT Number:	2013-003262-13
Sponsor's Protocol Code Number:	CSL627_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:	2014-06-09
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

2013-003262-13

A.3

Full title of the trial

A Phase III Open Label, Multicenter, Extension Study to Assess the Safety and Efficacy of Recombinant Coagulation Factor VIII (rVIII-SingleChain, CSL627) in Subjects with Severe Hemophilia A

Estudio de extensión de fase III, abierto, multicéntrico para evaluar la seguridad y eficacia del factor de la coagulación VIII recombinante (rVIII-cadena sencilla, CSL627) en pacientes con hemofilia A severa

A.3.1

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

An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A

Estudio abierto de seguridad y eficacia del factor FVIII recombinante en pacientes con hemofilia A severa

A.4.1

Sponsor's protocol code number

CSL627_3001

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

P/215/2012

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 GmbH
B.5.2	Functional name of contact point	Clin.Trial Registration Coordinator
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	3491708 86 00
B.5.5	Fax number	0496421394196
B.5.6	E-mail	clinicaltrials@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	rVIII-SingleChain
D.3.2	Product code	CSL627
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.3.8	INN - Proposed INN	Lonoctocog alfa
D.3.9.1	CAS number	1388129-63-2
D.3.9.2	Current sponsor code	CSL627
D.3.9.3	Other descriptive name	rVIII-SingleChain
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	rVIII-SingleChain
D.3.2	Product code	CSL627
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.3.8	INN - Proposed INN	Lonoctocog alfa
D.3.9.1	CAS number	1388129-63-2
D.3.9.2	Current sponsor code	CSL627
D.3.9.3	Other descriptive name	rVIII-SingleChain
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	rVIII-SingleChain
D.3.2	Product code	CSL627
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.3.8	INN - Proposed INN	Lonoctocog alfa
D.3.9.1	CAS number	1388129-63-2
D.3.9.2	Current sponsor code	CSL627
D.3.9.3	Other descriptive name	rVIII-SingleChain
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	rVIII-SingleChain
D.3.2	Product code	CSL627
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.3.8	INN - Proposed INN	Lonoctocog alfa
D.3.9.1	CAS number	1388129-63-2
D.3.9.2	Current sponsor code	CSL627
D.3.9.3	Other descriptive name	rVIII-SingleChain
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.IMP: 5
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	rVIII-SingleChain
D.3.2	Product code	CSL627
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.3.8	INN - Proposed INN	Lonoctocog alfa
D.3.9.1	CAS number	1388129-63-2
D.3.9.2	Current sponsor code	CSL627
D.3.9.3	Other descriptive name	rVIII-SingleChain
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	3000
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

Hemophilia A
Severe hemophilia A

Hemofilia A
Hemofilia A Severa

E.1.1.1

Medical condition in easily understood language

Hemophilia A is a rare but serious bleeding disorder which affects males and is characterized by a deficiency in the plasma protein known as coagulation Factor VIII.

Hemofilia A is una enfermedad rara pero seria de la sangre que afecta a varones y se caracteriza por una deficiencia en el plasma de una proteina conocida como Factor de coagulación VIII

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	17.0
E.1.2	Level	LLT
E.1.2	Classification code	10060613
E.1.2	Term	Hemophilia A (Factor VIII)
E.1.2	System Organ Class	100000004850

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 this study is to evaluate the safety of long term use of rVIII-SingleChain

El principal objetivo de este estudio es evaluar la seguridad del uso a largo plazo del factor r VIII de cadena sencilla.

E.2.2

Secondary objectives of the trial

The secondary objectives of the study are:
? To measure the incidence rate of inhibitor formation to FVIII after 10 EDs and after 50 EDs
? To collect and evaluate additional efficacy information on the prophylaxis and treatment of bleeding events
? To assess the hemostatic efficacy of rVIII-SingleChain for subjects who undergo surgery
? To characterize the safety profile of rVIII-SingleChain

Los objetivos secundarios del estudio son:
-Medir la tasa de incidencia de formación de inhibidores del factor VIII tras 10 DE y tras 50 DE.
-Recoger y evaluar información adicional sobre la eficacia de la profilaxis y el tratamiento de los episodios hemorrágicos.
-Evaluar la eficacia hemostática del factor rVIII - cadena sencilla en pacientes sometidos a cirugía.
-Calificar el perfil de seguridad del factor rVIII - cadena sencilla.

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

Subjects may participate in a surgical substudy in which rVIII-SingleChain may be administered before, during and after surgery. The sub-study objective is to assess the hemostatic efficacy of rVIII-SingleChain for subjects who undergo surgery.

Los pacientes pueden participar en un subestudio quirúrgico en el que rVIII-cadena sencilla pueda ser administrado antes, durante y después de la cirugía. El objetivo del subestudio es evaluar la eficacia hemostática de rVIII -cadena sencilla para pacientes que experimentaron la cirugía

E.3

Principal inclusion criteria

? Males of any age who have been diagnosed with severe hemophilia A (FVIII activity levels < 1%)
? Participated in a previous CSL-sponsored clinical study with rVIII-SingleChain. "

-Varones de cualquier edad que hayan sido diagnosticado de hemofilia A ( niveles de actividad FVIII<1%)

-Haber participado en un estudio de investigación previo con factor VIII recombinante de cadena simple patrocinado por CSL.

E.4

Principal exclusion criteria

? Known or suspected hypersensitivity to rVIII?SingleChain or to any excipients of rVIII?SingleChain or Chinese hamster ovary (CHO) proteins.
? Currently receiving a therapy not permitted during the study.

-Hipersensibilidad conocida o presunta al factor rVIII - cadena sencilla o a cualquiera de los excipientes del factor rVIII -cadena sencilla o a las proteínas de ovario de hámster chino (OHC).

-Estar recibiendo actualmente algún tratamiento no permitido durante el estudio

E.5 End points

E.5.1

Primary end point(s)

The primary outcome measure is the incidence rate of inhibitor formation to FVIII over 100 EDs.

El criterio de valoración principal es la tasa de incidencia de formación de inhibidores del factor VIII tras 100 DE.

E.5.1.1

Timepoint(s) of evaluation of this end point

At the closest visit after 100 EDs (up to three years).

En la visita más cercana después de 100 EDS (hasta tres años).

E.5.2

Secondary end point(s)

? Treatment success: Rate of treatment success where treatment success of a bleeding episode is defined as a rating of "excellent" or "good" on the investigator's clinical assessment of hemostatic efficacy 4-point scale "excellent, good, moderate or poor/no response".
? Annualized bleeding rate: The annualized bleeding rate for subjects taking prophylaxis and on-demand treatment regimens
? The proportion of bleeding episodes requiring 1, 2, 3, or > 3 infusions of rVIII-SingleChain to achieve hemostasis
? Consumption of rVIII-SingleChain - infusions: The number of rVIII-SingleChain infusions per month and per year
? Consumption of rVIII-SingleChain - IU/kg: rVIII-SingleChain consumption (IU/kg) per month and per year, and per event during prophylaxis, on-demand, and surgery.
? Hemostatic efficacy of rVIII-SingleChain for subjects who undergo surgery: The investigator will rate the efficacy of the rVIII-SingleChain treatment during surgery based on a hemostatic efficacy four point rating scale of "excellent, good, moderate or poor/no response".
? The incidence rate of inhibitor formation to FVIII

-La evaluación clínica del investigador sobre la eficacia hemostática en el tratamiento de episodios hemorrágicos según una escala ordinal de 4 puntos (excelente, buena, moderada, escasa/ninguna respuesta)
-Se resumirá la tasa anualizada de hemorragias (traumáticas y no traumáticas) durante el tratamiento profiláctico y a demanda.
-Se tabulará la proporción de episodios hemorrágicos que precisen 1, 2, 3 o más de 3 infusiones del factor rVIII - cadena sencilla para conseguir la hemostasia.
-Consumo de rVIII-cadena sencilla-infusión: número de infusiones de rVIII-cadena sencilla por mes y por año
-Consumo de rVIII-cadena sencilla-IU/Kg: consumo rVIII-cadena sencilla (IU/kg) por mes y por año y por evento durante profilaxis, o en demanda, y cirugía
-Eficacia hemostática de rVIII-cadena sencilla en pacientes sometidos a cirugía: el investigador tasará la eficacia del tratamiento de factor rVIII - cadena sencilla en pacientes sometidos a cirugía sobre la eficacia hemostática en el tratamiento de episodios hemorrágicos según una escala ordinal de 4 puntos (excelente, buena, moderada, escasa/ninguna respuesta).
-Tasa de incidencia de la formación del inhibidor FVIII

E.5.2.1

Timepoint(s) of evaluation of this end point

For secondary end points 1 to 5: Up to three years
For secondary end point 6: From the start of surgery through the post-operative recovery (generally up to 14 days after surgery)
For secondary end point 7: After 10 and after 50 exposure days

Criterios secundarios de valoración del 1-5: hasta 3 años
Criterios secundarios de valoración 6: desde el comienzo de la cirugía hasta la recuperación postoperatorio (generalmente hasta 14 días después de la cirugía)
Para el criterio secundario de valoración punto 7: después de 10 días y después de 50 días de exposición.

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

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.8.3

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

Chile

European Union

Georgia

Japan

Korea, Democratic People's Republic of

Lebanon

Malaysia

Philippines

Russian Federation

South Africa

Switzerland

Thailand

Turkey

Ukraine

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

UVUP

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)

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:

124

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

This clinical trial will be conducted in adults but also in children in different age groups, including children younger than 12 years of age.

Este estudio se llevará a cabo en adultos pero también en niños de diferentes grupos de edad, incluyendo niños menores de 12 años.

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

185

F.4.2.2

In the whole clinical trial

200

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)

Subjects who end their participation in the trial may continue treatment if the product is commercially available in their country or will return to standard of care treatment for hemophilia A as appropriate.

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

2014-06-30

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2014-06-05

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2021-01-19

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