ICH GCP - EU Clinical trials Registry - 2010-020047-12 (FR)

Kliniske forsøg Nct side

Summary
EudraCT Number:	2010-020047-12
Sponsor's Protocol Code Number:	P090202
National Competent Authority:	France - ANSM
Clinical Trial Type:	EEA CTA
Trial Status:
Date on which this record was first entered in the EudraCT database:	2011-04-01
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

A. Protocol Information

A.1

Member State Concerned

France - ANSM

A.2

EudraCT number

2010-020047-12

A.3

Full title of the trial

Etude de l'effet d'un traitement préventif par Nebivolol sur l'apparition et la progression de la dysfonction cardiaque chez les enfants atteints de dystrophie de Duchenne

A.3.1

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

Effet d’un traitement préventif par Nebivolol chez les enfants atteints de dystrophie de Duchenne

A.3.2

Name or abbreviated title of the trial where available

NEBIDYS

A.4.1

Sponsor's protocol code number

P090202

A.7

Trial is part of a Paediatric Investigation Plan

Information not present in EudraCT

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
B.1.3.4	Country	France
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	Association Naturalia et Biologia
B.4.2	Country	France
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	AP-HP ― Département de la Recherche Clinique et du Développement (DIRC Ile-de-France)
B.5.2	Functional name of contact point	Dr Henri-Marc BECANE
B.5.3	Address:
B.5.3.1	Street Address	Hôpital TROUSSEAU
B.5.3.2	Town/ city	PARIS
B.5.3.3	Post code	75012
B.5.3.4	Country	France
B.5.6	E-mail	henri-marc.becane@croix-rouge.fr

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	Temerit
D.2.1.1.2	Name of the Marketing Authorisation holder	Menarini
D.2.1.2	Country which granted the Marketing Authorisation	France
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	Temerit
D.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	Information not present in EudraCT
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Nébivolol
D.3.9.1	CAS number	118457-15-1
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	5
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
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Tablet
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Enfants atteints de dystrophie de Duchenne

E.1.1.2

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	13.1
E.1.2	Level	PT
E.1.2	Classification code	10013801
E.1.2	Term	Duchenne muscular dystrophy
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic disorders

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

Comparer l'effet du nébivolol à un placebo équivalent sur l'apparition de la dysfonction Ventriculaire Gauche.

E.2.2

Secondary objectives of the trial

Etudier secondairement :
- L'évolution de la FEVG dans chaque groupe.
- La tolérance du nébivolol chez ces jeunes enfants.
- L'effet des traitements sur le dosage du NT-ProBNP plasmatique
- La FEVD à l'état basal et sous traitement
- L'effet du traitement sur les sous-groupes traités et non traités par stéroïdes.
- L'effet du traitement sur les sous-groupes traités et non traités par IEC.
- A la fin de la période de 60 mois, la poursuite en ouvert du traitement par nébivolol sera proposée à chaque patient et la mortalité évaluée après une période de suivi de 60 mois supplémentaires.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Enfant atteints de dystrophie de Duchenne génétiquement prouvée,
- Agés de 10 à 15 ans
- Indemne de dysfonction VG L'absence de dysfonction VG sera définie par une FEVG?50% (ventriculographie isotopique datant de moins d'un mois).
- Consentement libre et éclairé des titulaires de l'autorité parentale et de l'enfant s'il est apte à comprendre
- Les enfants inclus auront une pression artérielle ? 80mmHg (décubitus dorsal) ou ? 70mmHg (position assise).
- En rythme sinusal sur ECG datant de moins de 3 mois
- Bilan hématologique et biochimique de moins de 3 mois attestant de l'absence d'anémie, d'insuffisance rénale ou hépatique sévère
- Affiliation à un régime de sécurité social

E.4

Principal exclusion criteria

- Les enfants qui ont une insuffisance rénale (définie par une urée>7mmol/l),
- Une atteinte hépatique (élévation des transaminases >3N)
- Bradycardie sinusale <50cpm
- Troubles conductifs de haut degré
- Une indication à un traitement B-bloquant
- Les enfants traités par tout agent cardio-vasculaire à l'exception des IEC.
- Les enfants ayant participé à un essai clinique dans les trois mois précédant l'étude, suspects de non compliance ou de non coopération ne seront pas inclus.

E.5 End points

E.5.1

Primary end point(s)

L'efficacité du traitement sera jugée sur la fraction d'éjection du ventricule gauche (FEVG), mesurée par ventriculographie isotopique. Une FEVG<45 % au cours du suivi du patient sera considéré comme un échec au traitement.

E.5.1.1

Timepoint(s) of evaluation of this end point

5 ans

E.5.2

Secondary end point(s)

- L’évolution de la FEVG dans chaque groupe.
- La tolérance du nébivolol.
- L’effet des traitements sur le dosage du NT-ProBNP plasmatique
- La FEVD à l’état basal et sous traitement
- L’effet du traitement sur les sous-groupes traités et non traités par stéroïdes.
- L’effet du traitement sur les sous-groupes traités et non traités par IEC.
- A la fin de la période de 60 mois, la poursuite en ouvert du traitement par nébivolol sera proposée à chaque patient et la mortalité évaluée après 60 mois supplémentaires.

E.5.2.1

Timepoint(s) of evaluation of this end point

5 ans

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

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)

Information not present in EudraCT

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

Information not present in EudraCT

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

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

Yes

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

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

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.1.1.1	In Utero	No
F.1.1.2	Preterm newborn infants (up to gestational age < 37 weeks)	No
F.1.1.3	Newborns (0-27 days)	No
F.1.1.4	Infants and toddlers (28 days-23 months)	No
F.1.1.5	Children (2-11years)	Yes
F.1.1.6	Adolescents (12-17 years)	Yes
F.1.2	Adults (18-64 years)	No
F.1.3	Elderly (>=65 years)	No
F.2 Gender
F.2.1	Female	No
F.2.2	Male	Yes
F.3 Group of trial subjects
F.3.1	Healthy volunteers	No
F.3.2	Patients	Yes
F.3.3	Specific vulnerable populations	No
F.3.3.1	Women of childbearing potential not using contraception	Information not present in EudraCT
F.3.3.2	Women of child-bearing potential using contraception	Information not present in EudraCT
F.3.3.3	Pregnant women	Information not present in EudraCT
F.3.3.4	Nursing women	Information not present in EudraCT
F.3.3.5	Emergency situation	Information not present in EudraCT
F.3.3.6	Subjects incapable of giving consent personally	Information not present in EudraCT
F.3.3.7	Others	Information not present in EudraCT
F.4 Planned number of subjects to be included
F.4.1	In the member state	70

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

2011-07-01

Ethics Committee Opinion of the trial application

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

P. End of Trial
P.	End of Trial Status

Kliniske forsøg Nct side

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