Clinical Trials Register

Summary
EudraCT Number:	2011-004730-34
Sponsor's Protocol Code Number:	361.11
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2012-03-02
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

Italy - Italian Medicines Agency

A.2

EudraCT number

2011-004730-34

A.3

Full title of the trial

A phase II multicentre, randomized, controlled open-label study on the use of anti-thymocyte globulin and rituximab for immunomodulation of graft-versus-host disease in allogeneic matched transplants for non malignancies

Studio di fase II multicentrico, randomizzato, controllato, open-label, sull'utilizzo di globuline anti-timocita e rituximab per la modulazione della malattia da trapianto verso l'ospite nel contesto del trapianto allogenico in patologie non maligne

A.3.1

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

T&B depletion non malignant

A.3.2

Name or abbreviated title of the trial where available

T&Bnm

A.4.1

Sponsor's protocol code number

361.11

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	OSPEDALE PEDIATRICO BAMBINO GESU' DI ROMA
B.1.3.4	Country	Italy
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Fresenius
B.4.2	Country	Germany
B.4.1	Name of organisation providing support	MEDAC
B.4.2	Country	Germany
B.4.1	Name of organisation providing support	Adienne
B.4.2	Country	Italy
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	University of Milano-Bicocca
B.5.2	Functional name of contact point	Trial Data Center
B.5.3	Address:
B.5.3.1	Street Address	Via Cadore 48
B.5.3.2	Town/ city	Monza
B.5.3.3	Post code	20052
B.5.3.4	Country	Italy
B.5.4	Telephone number	02-64488164
B.5.5	Fax number	02-64488262
B.5.6	E-mail	grazia.valsecchi@unimib.it

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	Yes
D.2.1.1.1	Trade name	ATG Fresenius S
D.2.1.1.2	Name of the Marketing Authorisation holder	Fresenius Biotech
D.2.1.2	Country which granted the Marketing Authorisation	Germany
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.3.4	Pharmaceutical form	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	NA
D.3.9.1	CAS number	NA
D.3.9.2	Current sponsor code	DB00098
D.3.9.3	Other descriptive name	NA
D.3.9.4	EV Substance Code	NA
D.3.10	Strength
D.3.10.1	Concentration unit	mg/kg milligram(s)/kilogram
D.3.10.2	Concentration type	range
D.3.10.3	Concentration number	10 to 15
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)	Yes
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
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	Yes
D.2.1.1.1	Trade name	MABTHERA*EV 2F 10ML 100MG
D.2.1.1.2	Name of the Marketing Authorisation holder	ROCHE SpA
D.2.1.2	Country which granted the Marketing Authorisation	Italy
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.3.4	Pharmaceutical form	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.3.8	INN - Proposed INN	RITUXIMAB
D.3.9.1	CAS number	174722-31-7
D.3.9.2	Current sponsor code	NA
D.3.9.3	Other descriptive name	NA
D.3.9.4	EV Substance Code	SUB12570MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg/m2 milligram(s)/square meter
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	200
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	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	No
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	Yes
D.2.1.1.1	Trade name	Treosulfan
D.2.1.1.2	Name of the Marketing Authorisation holder	Medac
D.2.1.2	Country which granted the Marketing Authorisation	Germany
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/04/186
D.3 Description of the IMP
D.3.4	Pharmaceutical form	Powder 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	TREOSULFAN
D.3.9.1	CAS number	299-75-2
D.3.9.2	Current sponsor code	9296
D.3.9.3	Other descriptive name	NA
D.3.9.4	EV Substance Code	SUB11235MIG
D.3.10	Strength
D.3.10.1	Concentration unit	gm/m2 gram(s)/square meter
D.3.10.2	Concentration type	range
D.3.10.3	Concentration number	3 to 8
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	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	No
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	Yes
D.2.1.2	Country which granted the Marketing Authorisation	Italy
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.3.4	Pharmaceutical form	Powder 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	FLUDARABINE PHOSPHATE
D.3.9.1	CAS number	75607-67-9
D.3.9.2	Current sponsor code	657237
D.3.9.3	Other descriptive name	NA
D.3.9.4	EV Substance Code	SUB13897MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg/m2 milligram(s)/square meter
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	25
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	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	No
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	Yes
D.2.1.1.1	Trade name	tepadina
D.2.1.1.2	Name of the Marketing Authorisation holder	Adienne S.r.l.
D.2.1.2	Country which granted the Marketing Authorisation	Germany
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	ev/0/06/424
D.3 Description of the IMP
D.3.4	Pharmaceutical form	Powder 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.3.8	INN - Proposed INN	THIOTEPA
D.3.9.1	CAS number	52-24-4
D.3.9.2	Current sponsor code	DB04572
D.3.9.4	EV Substance Code	SUB10985MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg/g milligram(s)/gram
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	100
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	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	No
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

graft-versus-host disease

malattia del trapianto contro l’ospite

E.1.1.1

Medical condition in easily understood language

allogeneic matched transplants for non malignancies

trapianto allogenico di cellule staminali emopoietiche da donatore HLA-compatibile in pazienti affetti da patologie non maligne

E.1.1.2

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	14.1
E.1.2	Level	SOC
E.1.2	Classification code	10021428
E.1.2	Term	Immune system disorders
E.1.2	System Organ Class	10021428 - Immune system 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

Assess in a randomized fashion the benefit on standard graft-versus-host disease (GVHD) prophylaxis of the addition of ATG-Fresenius S in transplants from matched related donors (MRD) and of anti-CD20 rituximab in transplants from matched unrelated donors (MUD)

Determinare il beneficio dell’aggiunta alla profilassi standard della malattia del trapianto contro l’ospite (GvHD) di siero antilinfocitario di coniglio (ATG-Fresenius S) nel trapianto allogenico di cellule staminali emopoietiche da donatore familiare HLA-compatibile (MRD) e dell’anticorpo monoclonale anti-CD20 rituximab nel trapianto allogenico di cellule staminali emopoietiche da donatore volontario HLA-compatibile (MUD).

E.2.2

Secondary objectives of the trial

For patients transplanted from a MUD

Reduced incidence and severity of cGVHD after transplantation using rituximab

valutare la sicurezza ed efficacia di un regime di condizionamento basato sull’impiego di treosulfano, fludarabina, tiotepa (Tepadina) e ATG-Fresenius S in pazienti affetti da malattie non maligne e sottoposti a trapianto allogenico di cellule staminali emopoietiche (HSCT).

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

 Availability of a matched related donor (MRD) or Matched Unrelated Donor (MUD)
 Lansky or Karnofsky Index ≥ 60
 Inherited metabolic disorders: DQ ≥70 (+ MRI Loes score ≤ 9 for adrenoleukodystrophy)
 Adequate cardiac, renal, hepatic and pulmonary functions as evidenced by:
 Serum creatinine ≤1.5 × upper limit of normal (ULN)
 Heart shortening fraction (left-ventricle) > 28 % or LVEF > 55%
 Serum bilirubin ≤1.5 × ULN (except for Wolman disease),
 AST and ALT ≤ 2.5 × ULN (except for thalassemic syndromes and Wolman disease)
 Pulmonary function: if cooperative: FEV1 and FVC on pulmonary function testing > 60 %; if non cooperative: pulse oximetry > 95 % in room air
 Availability of autologous back up marrow (> 2 x 108 TNC+ cells/kg or > 2 x 106 CD34+ cells/kg) for MUD
 Adequate contraception in female patients of child-bearing potential
 Signed informed consent

 Disponibilità di un donatore familiare HLA-compatibile (MRD) o di un donatore volontario HLA-compatibile (MUD)
 Score di Lansky o Karnofsky ≥ 60
 Per le malattie metaboliche ereditarie: DQ ≥70 (+ MRI Loes score ≤ 9 per l’adrenoleucodistropia)
 Adeguata funzione cardiaca, renale, epatica e polmonare valutata come:
 Creatinina sierica ≤1.5 × il limite superiore di normalità (ULN)
 Frazione di accorciamento del cuore (venticolo sinistro) > 28 % o LVEF > 55%
 Bilirubina sierica ≤1.5 × ULN (fatta eccezione per la malattia di Wolman),
 AST/GOT e ALT/GPT ≤ 2.5 × ULN (fatta eccezione per le sindromi talassemiche e la malattia di Wolman)
 Funzione polmonare: se collaborante: test di funzionalità respiratoria con FEV1 e FVC > 60 %; se non collaborante: saturazione dell’ossigeno > 95 % in aria-ambiente
 Disponibilità di un back-up autologo di midollo osseo (> 2 x 10e8 total nucleated cells (TNC)/kg o > 2 x 10e6 cellule CD34+/kg) per i trapianti da MUD
 Adeguata contraccezione nelle pazienti femmine in età fertile
 Consenso informato firmato

E.4

Principal exclusion criteria

 Any malignancy
 Liver cirrhosis evidenced on liver histology (performed in suspicious cases or in case of Wolman disease)
 HIV- positivity
 Clinically significant pleural effusion or ascites
 Pregnancy or lactation
 Known hypersensitivity to trial drugs
 Participation in another experimental drug trial in the 2 months preceding enrolment
 Non-cooperative behaviour or non-compliance
 Previous HSCT

 Malattia maligna di qualsiasi tipo
 Cirrosi epatica dimostrata alla biposia epatica (eseguita nei casi sospetti o in caso di malattia di Wolman)
 Positività per HIV
 Versamento pleurico o ascite clinicamente significative
 Gravidanza o allattamento
 Ipersensibilità nota ai farmaci in studio
 Participazione ad un altro studio sperimentale su farmaci nei 2 mesi precedenti l’arruolamento
 Comportamento non collaborante o non compliante
 Precedente HSCT

E.5 End points

E.5.1

Primary end point(s)

For patients transplanted from a MRD

The primary end-point is the cumulative incidence of a combined end-point defined as the time from randomization to:
- primary and secondary graft failure,
- aGVHD II-IV,
- cGVHD,
- death,
whichever occurs first.
For patients transplanted from a MUD

The primary end-point is the cumulative incidence of a combined end-point defined as the time from randomization to:
- aGVHD II-IV,
- EBV viremia,
whichever occurs first.

Per i pazienti trapiantati da MRD
L’end-point primario è l’incidenza cumulativa di un end-point combinato e definito come il tempo intercorrente tra la randomizzazione del paziente e:
- l’insorgenza di rigetto primaro e secondario,
- l’insorgenza di GVHD acuta di grado II-IV,
- l’insorgenza di GvHD cronica,
- la morte,
qualsiasi di questi eventi si verifichi per primo.
Per i pazienti trapiantati da MUD
L’end-point primario è l’incidenza cumulativa di un end-point combinato e definito come il tempo intercorrente tra la randomizzazione del paziente e:
- l’insorgenza di GVHD acuta di grado II-IV,
- la positivizzazione della viremia per EBV,
qualsiasi di questi eventi si verifichi per primo.

E.5.1.1

Timepoint(s) of evaluation of this end point

E.5.2

Secondary end point(s)

1. the cumulative incidence and severity of cGVHD after randomization
2. the incidence of TRM after randomization
3. the overall survival after randomization

1. l’incidenza e la severità della GvHD cronica dopo la randomizzazione
2. l’incidenza di TRM dopo la randomizzazione
1. la sopravvivenza globale dopo la randomizzazione

E.5.2.1

Timepoint(s) of evaluation of this end point

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

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

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)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

Yes

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

Yes

E.8.2.3.1

Comparator description

terapia standard senza aggiunta dell'IMP

standard therapy without the IMP

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

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

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

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

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:

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

Yes

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

minori

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

130

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)

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

2011-07-20

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2011-07-20

P. End of Trial
P.	End of Trial Status	Ongoing

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