Clinical Trials Register

Summary
EudraCT Number:	2015-002136-40
Sponsor's Protocol Code Number:	261203
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:	2015-10-15
Trial 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

2015-002136-40

A.3

Full title of the trial

Phase 3, prospective, multi-center, open label study to investigate
safety, immunogenicity, and hemostatic efficacy of PEGylated Factor VIII
(BAX 855) in previously untreated patients (PUPs) and minimally treated patients
(MTPs) < 6 years with severe hemophilia A (FVIII < 1%)

Estudio de fase III prospectivo, multicéntrico y abierto para investigar la seguridad,la inmunogenicidad y la eficacia hemostática del factor VIII PEGilado (BAX 855) en pacientes no tratados previamente (PNTP) y pacientes tratados mínimamente (PTM) < 6 años con hemofilia A grave (FVIII < 1 %)

A.3.1

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

A study of safety, effect on the immune system and blood clotting of BAX855 (factor VIII of blood clotting) in pediatric patients who have not received or received minimal treatment for their severe hemophilia A.

Estudio de seguridad, efecto sobre el sistema inmune y la coagulación de la sangre
de BAX855 (factor VIII de coagulación de la sangre) en pacientes pediátricos que
no han recibido o recibido un tratamiento mínimo para su hemofilia A grave.

A.3.2

Name or abbreviated title of the trial where available

BAX855 PUP study

Estudio BAX855 PUP

A.4.1

Sponsor's protocol code number

261203

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/072/2013

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Baxalta Innovations GmbH
B.1.3.4	Country	Austria
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	Baxalta Innovations GmbH
B.4.2	Country	Austria
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Maria Teresa Alvarez Roman
B.5.2	Functional name of contact point	Maria Teresa Alvarez Roman
B.5.3	Address:
B.5.3.1	Street Address	Hospital Universitario La Paz, Paseo de la Castellana 261
B.5.3.2	Town/ city	Madrid
B.5.3.3	Post code	28046
B.5.3.4	Country	Spain
B.5.4	Telephone number	+34912071945
B.5.6	E-mail	talvarezroman@gmail.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	PEGylated rFVIII
D.3.2	Product code	BAX855
D.3.4	Pharmaceutical form	Powder and solution for solution for injection
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	Not applicable
D.3.9.2	Current sponsor code	BAX855
D.3.9.3	Other descriptive name	BAX 855 (PEGYLATED RFVIII)
D.3.9.4	EV Substance Code	SUB90763
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.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Not applicable
D.3.9.2	Current sponsor code	BAX 855
D.3.9.3	Other descriptive name	BAX 855 (PEGYLATED RFVIII)
D.3.9.4	EV Substance Code	SUB90763
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.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Not applicable
D.3.9.2	Current sponsor code	BAX 855
D.3.9.3	Other descriptive name	BAX 855 (PEGYLATED RFVIII)
D.3.9.4	EV Substance Code	SUB90763
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.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Not applicable
D.3.9.2	Current sponsor code	BAX 855
D.3.9.3	Other descriptive name	BAX 855 (PEGYLATED RFVIII)
D.3.9.4	EV Substance Code	SUB90763
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	Yes
D.3.11.13.1	Other medicinal product type	Recombinant product with covalently bound polyethyleneglycol

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

Severe hemophilia A (FVIII <1%)

Hemofilia A Grave (FVIII <1%)

E.1.1.1

Medical condition in easily understood language

Hemophilia A

Hemofilia A

E.1.1.2

Therapeutic area

Diseases [C] - Blood and lymphatic diseases [C15]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	18.1
E.1.2	Level	LLT
E.1.2	Classification code	10060612
E.1.2	Term	Hemophilia A
E.1.2	System Organ Class	100000004850

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

To determine safety including immunogenicity of BAX 855 based on the
incidence of inhibitor development to FVIII (≥ 0.6 BU/mL using the Nijmegen modification of the Bethesda assay)

El objetivo principal es evaluar la seguridad, incluida la inmunogenicidad, de BAX
855 a partir de la incidencia del desarrollo de inhibidores del FVIII (≥0,6 unidades
de Bethesda [UB]/ml mediante la modificación de Nijmegen del ensayo Bethesda).

E.2.2

Secondary objectives of the trial

Safety
1. To determine the immunogenicity of BAX 855 in terms of binding IgG and IGM antibodies to FVIII, PEG-FVIII and PEG
2. To determine the safety of BAX 855 based on adverse events (AEs) and serious adverse events (SAEs)
Hemostatic Efficacy
3. To assess the efficacy of prophylactic treatment with BAX 855
4. To characterize the efficacy of BAX 855 in the control of bleeding episodes
5. To evaluate the efficacy of BAX 855 for perioperative management, if surgery is required
Pharmacokinetics
6. To determine the incremental recovery (IR) of BAX 855 at baseline and over time
7. To determine half-life of BAX 855 at baseline (optional)

Seguridad 1.-Determinar la inmunogenicidad de BAX 855 en cuanto a anticuerpos
de unión IgG e IgM al FVIII, al PEG-FVIII y al PEG. 2.-Determinar la seguridad de
BAX 855 a partir de los acontecimientos adversos (AA) y de los acontecimientos
adversos graves (AAG). 3.-Evaluar la eficacia del tratamiento profiláctico con BAX
855. 4.- Caracterizar la eficacia de BAX 855 en el control de los episodios
hemorrágicos. 5.- Evaluar la eficacia de BAX 855 para tratamientos perioperatorios
si es necesaria una intervención quirúrgica. 6.- Determinar la recuperación
incremental (RI) de BAX 855 al inicio y a lo largo del tiempo. 7.- Determinar la
semivida de BAX 855 al inicio (opcional).

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Subject is < 6 years old at the time of screening
2. Subject is previously untreated or minimally treated with ≤ 3 EDs to ADVATE or BAX 855 at any time prior to screening
3. Subject has severe hemophilia A (FVIII < 1%) as determined by the central laboratory, or a historical FVIII level < 1% as determined at any local laboratory and/or a FVIII gene mutation consistent with severe hemophilia A
4. Subject is immune competent with a CD4+ count > 200 cells/mm3, as confirmed by central laboratory at screening
5. Parent or legally authorized representative is willing and able to comply with the requirements of the protocol
Additional inclusion criteria for Part B (ITI):
6. Parent or legal representative has/have voluntarily provided signed informed consent for ITI portion
7. Subject has a confirmed inhibitor titer (≥ 0.6 BU) as determined by the central laboratory based on a second repeat blood sample and requires ITI therapy

1.-Sujeto < seis años de edad en el momento de la selección. 2.-Sujeto no tratado
previamente o mínimamente tratado con ? 3 DE de ADVATE o BAX 855 en algún
momento antes de la selección. 3.-Sujeto con hemofilia A grave (FVIII < 1 %),
según lo determinado por el laboratorio central, unas concentraciones históricas de
FVIII < 1 % según lo determinado por cualquier laboratorio local, o una mutación en
el gen FVIII compatible con la hemofilia A grave. 4.-Sujeto inmunocompetente con
una cifra de CD4+ > 200 células/mm3, según lo confirmado por el laboratorio
central en la selección. 5.-Progenitor o representante legal dispuestos y capaces de
cumplir los requisitos del protocolo Criterios de inclusión adicionales para la parte B
(ITI): 6.-Progenitor o representante legal que otorguen su consentimiento informado
firmado de forma voluntaria para la parte de ITI. 7.-Sujeto con un valor confirmado
de inhibidores (? 0,6 UB) según lo determinado por el laboratorio central a partir de
una segunda muestra de sangre repetida que requiera tratamiento de ITI.

E.4

Principal exclusion criteria

1. Subject has detectable FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening
2. Subject has a history of FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay or the Bethesda assay) at any time prior to screening
3. Subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand’s disease)
4. Subject has been previously treated with FFP, cryoprecipitate, any type of FVIII concentrate other than ADVATE or BAX 855v or was administered ADVATE or BAX 855 for > 3 EDs at any time prior to screening
5. Subject has received any kind of blood-transfusion such as PRBC, platelets or plasma prior to screening at any time prior to screening
6. The subject’s weight is < 5 kg
7. Subject’s platelet count is < 100,000/mL
8. Subject has known hypersensitivity towards mouse or hamster proteins, PEG or Tween 80
9. Subject has severe chronic hepatic dysfunction [eg, > 5 times upper limit of normal alanine aminotransferase (ALT), aspartate aminotransferase (AST), or a documented INR > 1.5] in his medical history or at the time of screening
10. Subject has severe renal impairment (serum creatinine > 1.5 times the upper limit of normal)
11. Subject has current or recent (< 30 days) use of other PEGylated drugs prior to study participation or is scheduled to use such drugs during study participation
12. Subject is scheduled to receive during the course of the study a systemic immunomodulating drug (e.g. corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day, or α-interferon) other than anti-retroviral chemotherapy
13. Subject has participated in another clinical study involving an IP or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study
14. Parent or legally authorized representative has a medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject safety or compliance
15. Parent, legally authorized representative or subject are a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (ie, children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study.
Additional exclusion criteria for Part B (ITI)
16. Spontaneous disappearance of the inhibitor prior to ITI
17. FVIII inhibitor titer ≥ 0.6 BU is not confirmed by a second new blood sample and determined at the central laboratory
18. Inability or unwillingness to comply with the protocol

1.-Sujeto con anticuerpos inhibidores del FVIII detectables (? 0,6 UB mediante la modificación de Nijmegen del ensayo Bethesda), según lo confirmado por el laboratorio central en la selección. 2.-Sujeto con antecedentes de anticuerpos inhibidores del FVIII (? 0,6 UB mediante la modificación de Nijmegen del ensayo Bethesda o el ensayo de Bethesda) en cualquier momento antes de la selección. 3.-Al sujeto se le ha diagnosticado un defecto hemostático heredado o adquirido diferente a la hemofilia A (como un defecto cualitativo de las plaquetas o la enfermedad de von Willebrand). 4.-Sujeto tratado previamente con plasma fresco congelado (PFC), crioprecipitado, cualquier tipo de concentrado de FVIII distinto de ADVATE o BAX 855 o al que se haya administrado ADVATE o BAX 855 durante >3 DE en algún momento antes de la selección. 5.-Sujeto que haya recibido cualquier tipo de transfusión de sangre, como concentrado de eritrocitos, trombocitos o plasma en cualquier momento antes de la selección. 6.-Peso del sujeto < 5 kg. 7.-Cifra de plaquetas del sujeto < 100 000/ml. 8.-El sujeto tiene una hipersensibilidad conocida a las proteínas de ratón o hámster, PEG o Tween 80.
9.-Sujeto con disfunción hepática crónica grave (p. ej., alanina aminotransferasa [ALT] o aspartato aminotransferasa [AST] > cinco veces el límite superior de la normalidad o INR documentado > 1,5) en sus antecedentes médicos o en el momento de la selección. 10.-Sujeto con insuficiencia renal grave (creatinina sérica > 1,5 veces el límite superior de la normalidad).11.-Sujeto que utilice o haya
utilizado recientemente (< 30 días) otros fármacos PEGilados antes de participar en el estudio o que tenga previsto utilizar estos fármacos durante su participación en el estudio. 12.-Sujeto que tenga previsto recibir durante el transcurso del estudio un fármaco inmunomodulador sistémico (como corticoesteroides en una dosis equivalente a hidrocortisona superior a 10 mg/día o ?-interferón) que no sea quimioterapia antirretrovírica . 13.-El sujeto ha participado en otro estudio clínico
que utilizó un PEI o un dispositivo en investigación durante los 30 días anteriores a la inscripción o está programado que participe en otro estudio clínico con un PEI o un dispositivo en investigación durante el transcurso de este estudio. 14.-Progenitor o representante legal que presente una enfermedad médica, psiquiátrica o cognitiva, o que consuma drogas o alcohol, de manera que, en opinión del investigador, pueda afectar a la seguridad o al cumplimiento del sujeto. 15.-Progenitor, representante legal o sujeto que sean miembros del equipo que realiza este estudio o que tengan una relación de dependencia con alguno de los miembros del equipo del estudio. Las relaciones de dependencia incluyen a parientes cercanos (es decir, hijos, pareja/cónyuge, hermanos, padres), así como los empleados del investigador o el personal del centro que realizan el estudio. Criterios de exclusión adicionales para la parte B (ITI): 16.-Desaparición espontánea del inhibidor antes de la ITI. 17.-Valor de inhibidores del FVIII ? 0,6 UB sin confirmar mediante una segunda muestra nueva de sangre según lo determinado en el laboratorio central. 18.-Incapacidad o falta de disposición para cumplir el protocolo.

E.5 End points

E.5.1

Primary end point(s)

Incidence of FVIII inhibitor development
The success rate of ITI therapy with BAX 855

Incidencia del desarrollo de inhibidores del FVIII. Tasa de éxito del tratamiento de
ITI con BAX 855 .

E.5.1.1

Timepoint(s) of evaluation of this end point

The set of subjects to be analyzed includes all subjects who developed an inhibitor (at any time) and all subjects who did not develop an inhibitor and had ≥ 50 EDs. An interim analysis is planned after 50 subjects have completed 50 EDs. The final CSR will also analyze subjects who develop an inhibitor and subjects who did not, but completed at least 100 EDs.

El conjunto de sujetos que se van a analizar incluye a todos los sujetos que hayan desarrollado un inhibidor (en cualquier momento) y a todos los sujetos que no hayan desarrollado un inhibidor y hayan tenido ? 50 DE. Está previsto efectuar un análisis intermedio cuando 50 sujetos hayan cumplido 50 DE. En el informe del estudio clínico (IEC) final también se analizarán los sujetos que hayan desarrollado un inhibidor y los que no, pero que hayan cumplido al menos 100 DE.

E.5.2

Secondary end point(s)

Binding IgG and IgM antibodies to FVIII, PEG-FVIII and PEG
AEs and SAEs
Clinically significant changes in vital signs and clinical laboratory parameters (hematology and clinical chemistry)
Efficacy:
 Annualized bleeding rate (ABR) for prophylactic and on-demand treatment
 Number of BAX 855 infusions per bleeding episode
 Overall hemostatic efficacy rating at 24 h after initiation of treatment and at resolution of bleed
 Weight-adjusted consumption of BAX 855 per month, per year and per event (prophylaxis, treatment of bleeding episode, surgery) and the number of infusions per month and per year
 Assessment of intra-, post- and perioperative hemostatic efficacy in case of surgery
 Intra- and postoperative blood loss in case of surgery
Pharmacokinetics:
 IR at baseline and over time
 Half-life at baseline (optional)
Additional Outcome Measures for ITI:
The rate of partial success and failure of ITI with BAX 855
ABR during ITI
Weight-adjusted consumption of BAX 855 per month and per year for each ITI regimen employed

Anticuerpos de unión IgG e IgM al FVIII, al PEG-FVIII y al PEG. AA y AAG. Cambios clínicamente significativos en las constantes vitales y en los parámetros clínicos analíticos (hematología y bioquímica clínica). Eficacia: Tasa anualizada de hemorragias (TAH) con el tratamiento profiláctico y a demanda. Número de infusiones de BAX 855 por episodio hemorrágico. Calificación global de la eficacia hemostática a las 24 horas de iniciar el tratamiento y en la resolución de la hemorragia. Consumo de BAX 855 ajustado por peso al mes, al año y por acontecimiento (profilaxis, tratamiento de episodios hemorrágicos e intervención
quirúrgica), y número de infusiones por mes y por año. Evaluación de la eficacia
hemostática intraoperatoria, posoperatoria y perioperatoria en caso de intervención
quirúrgica. Pérdida de sangre intraoperatoria y posoperatoria en caso de
intervención quirúrgica. Farmacocinética RI al inicio y a lo largo del tiempo.
Semivida al inicio (opcional). Esto se basa en un parámetro de FC abreviado
empleando dos puntos temporales posteriores a la infusión: 15-30 minutos y 24-48
horas. Criterios de valoración adicionales para ITI: Las tasas de éxito parcial y de
fracaso de la ITI con BAX 855. TAH durante la ITI. Consumo ajustado por peso de
BAX 855 por mes y por año de cada pauta de ITI utilizada.

E.5.2.1

Timepoint(s) of evaluation of this end point

Binding antibodies to FVIII, BAX 855 and PEG and all other secondary safety outcome measures will be analyzed descriptively.
The ABR will be analyzed by point and interval estimates derived from a negative binomial model with treatment regimen (on-demand vs. prophylaxis) as a covariate and the duration of the observation period as an offset. Other secondary efficacy outcome measures as well as IR over time will be analyzed descriptively.
Outcomes of ITI will be summarized descriptively.

Se analizarán descriptivamente los anticuerpos de unión al FVIII, BAX 855 y PEG,
así como todos los demás criterios de valoración secundarios de la seguridad. La TAH se analizará mediante estimaciones puntuales e interválicas derivadas de un modelo binomial negativo con la pauta de tratamiento (a demanda frente a profiláctico) como covariable y la duración del periodo de observación como variable de exposición. Se analizarán descriptivamente otros criterios de valoración de la eficacia secundarios, así como la RI a lo largo del tiempo. Los resultados de la ITI se resumirán de forma descriptiva.

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)

E.8.2.2

Placebo

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

E.8.5

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

Austria

Belgium

Bulgaria

Czech Republic

Denmark

Finland

France

Germany

Hungary

Italy

Korea, Republic of

Lithuania

Malaysia

Netherlands

New Zealand

Norway

Poland

Romania

Singapore

Spain

Sweden

Switzerland

Taiwan

Turkey

Ukraine

United Kingdom

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

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:

110

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

Yes

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

Pediatric patients under age of 6 years

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

110

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)

none

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2015-11-19

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2015-11-05

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

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