Clinical Trials Register

Summary
EudraCT Number:	2014-004048-36
Sponsor's Protocol Code Number:	CRFB002H2301E1
National Competent Authority:	France - ANSM
Clinical Trial Type:	EEA CTA
Trial Status:
Date on which this record was first entered in the EudraCT database:	2016-10-27
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

France - ANSM

A.2

EudraCT number

2014-004048-36

A.3

Full title of the trial

RAINBOW extension study: an extension study to evaluate the long term efficacy and safety of RAnibizumab compared with laser therapy for the treatment of INfants BOrn prematurely With retinopathy of prematurity

Extension de l’étude RAINBOW: Une étude d’extension pour comparer l’efficacité et la tolérance à long terme du ranibizumab à celles du traitement par laser chez les nourrissons prématurés atteints de rétinopathie du prématuré (ROP)

A.3.1

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

This study is to determine if ranibizumab injected into the eye is superior to laser therapy in the treatment of retinopathy of prematurity (ROP). The study will assess the ability of these treatments to lead to regression of active ROP and prevent the development of ocular complications that are associated with poor visual outcome.

Cette étude consiste à déterminer si l'injection de ranibizumab dans l'oeil est supérieure à la thérapie au laser dans le traitement de la rétinopathie de la prématuré (ROP). L'étude évaluera la capacité de ces traitements de conduire à une régression de ROP active et de prévenir le développement des complications oculaires qui sont associés à des résultats visuels pauvres.

A.3.2

Name or abbreviated title of the trial where available

RAINBOW Extension study

Extension de l’étude RAINBOW

A.4.1

Sponsor's protocol code number

CRFB002H2301E1

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/186/2014

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Novartis Pharma Services AG
B.1.3.4	Country	Switzerland
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	Novartis Pharma Services AG
B.4.2	Country	Switzerland
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Novartis Pharma S.A.S
B.5.2	Functional name of contact point	Information&Communication Médicales
B.5.3	Address:
B.5.3.1	Street Address	2 et 4 rue Lionel Terray
B.5.3.2	Town/ city	Rueil-Malmaison
B.5.3.3	Post code	92500
B.5.3.4	Country	France
B.5.4	Telephone number	+3315547 6600
B.5.5	Fax number	+3315547 6100
B.5.6	E-mail	icm.phfr@novartis.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	Yes
D.2.1.1.1	Trade name	Lucentis
D.2.1.1.2	Name of the Marketing Authorisation holder	Novartis Europharm Limited
D.2.1.2	Country which granted the Marketing Authorisation	European Union
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	Lucentis
D.3.2	Product code	RFB002
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravitreal use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	RANIBIZUMAB
D.3.9.1	CAS number	347396-82-1
D.3.9.2	Current sponsor code	RFB002
D.3.9.4	EV Substance Code	SUB22314
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	10
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	Yes
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

Retinopathy of Prematurity (ROP)

Rétinopathie du prématuré (ROP)

E.1.1.1

Medical condition in easily understood language

Retinopathy of prematurity (ROP) is a pathologic process that occurs in the developing retina of low birth-weight preterm neonates.

La rétinopathie du prématuré (ROP) est un un processus pathologique qui se produit dans la rétine en développement chez les nouveau-nés prématurés ayant un faible poids à la naissance.

E.1.1.2

Therapeutic area

Diseases [C] - Eye Diseases [C11]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	19.0
E.1.2	Level	PT
E.1.2	Classification code	10038933
E.1.2	Term	Retinopathy of prematurity
E.1.2	System Organ Class	10015919 - Eye 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

To assess visual function of the better seeing eye

Évaluer la fonction visuelle de l’œil qui voit le mieux

E.2.2

Secondary objectives of the trial

To assess:
- number of patients having any ocular Adverse Event
- number of patients having any systemic Adverse Event
- visual function of the worse seeing eye
- absence of active Retinopathy of Prematurity
- absence of ocular structural abnormalities
- recurrence of ROP
- number of Lucentis administrations
- refraction in each eye
- standing/sitting height and leg length
- weight
- head circumference
- respiratory function
- hearing function
- duration of hospitalization
- weight at first hospital discharge

Évaluer:
- le nombre de patients ayant un événement indésirable oculaire
- le nombre de patients ayant un événement indésirable systémique
- la fonction visuelle de l'œil qui voit le moins bien
- l'absence de ROP active
- l'absence d'anomalies structurelles de l'œil
- la récidive de la ROP
- le nombre d'injections de Lucentis
- la réfraction dans chaque œil

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

The patient successfully completed the core study CRFB002H2301
The patient received study treatment in both eyes at baseline of study CRFB002H2301

Le patient a été au terme de l'étude principale CRFB002H2301
Le patient a reçu le traitement de l'étude dans les deux yeux au début de l'étude CRFB002H2301

E.4

Principal exclusion criteria

Patient has a medical condition or personal circumstance which precludes study participation or compliance with study procedures, as assessed by the Investigator
Patient has been discontinued from the core study CRFB002H2301 at any time

Other protocol-defined inclusion/exclusion criteria may apply

Le patient présente une maladie ou se trouve dans une circonstance personnelle qui empêche sa participation à l'étude ou le respect des procédures de l'étude, d'après l'investigateur
La participation du patient à l'étude principale CRFB002H2301 a été arrêtée à un moment quelconque.

D'autres critères d'inclusion / exclusion définis par le protocole peuvent être appliquées

E.5 End points

E.5.1

Primary end point(s)

Visual acuity (VA) in the better-seeing eye

L’acuité visuelle (AV) de l’œil qui voit le mieux

E.5.1.1

Timepoint(s) of evaluation of this end point

VA is being assessed at the patient's fifth birthday.

L’acuité visuelle est évaluée au cinquième anniversaire du patient.

E.5.2

Secondary end point(s)

Ocular Adverse Events
Systemic Adverse Events
VA of the worse-seeing eye
Active ROP
Ocular structural abnormalities
Recurrence of ROP
Lucentis administrations
Refraction
Height/leg length
Weight
Head circumference
Respiratory function
Hearing function
Hospitalization (duration)
Weight (discharge)

Les événements indésirables oculaires
Les événements indésirables systémiques
La fonction visuelle de l'œil qui voit le moins bien
ROP active
D'autres anomalies structurelles de l'œil
La récidive de la ROP
Les injections de Lucentis
La refraction
La taille/la longueur de la jambe
Le poids
La circonférence de la tête
La fonction respiratoire
La fonction d'audition
Hospitalisation (durée)
Poids (décharge)

E.5.2.1

Timepoint(s) of evaluation of this end point

At the patient's fifth birthday
At the patient's fifth birthday
At the patient's fifth birthday
40 and 52 weeks after initial treatment
40 and 52 weeks after initial treatment, at patient's two years corrected age and fifth birthday
40 and 52 weeks after initial treatment
40 weeks after initial treatment
At patient's two years corrected age and fifth birthday
At patient's two years corrected age and fifth birthday
At patient's two years corrected age and fifth birthday
at patient's two years corrected age
At patient's two years corrected age and fifth birthday
At patient's two years corrected age and fifth birthday
At patient's two years corrected age and fifth birthday
At patient's two years corrected age and fifth birthday

Au cinquième anniversaire du patient.
Au cinquième anniversaire du patient.
Au cinquième anniversaire du patient.
40 et 52 semaines après le traitement initial
40 et 52 semaines après le traitement initial, à l'âge corrigé de 2 ans et au cinquième anniversaire du patient
40 et 52 semaines après le traitement initial
40 semaines après le traitement initial
À l'âge corrigé de 2 ans et au cinquième anniversaire du patient
À l'âge corrigé de 2 ans et au cinquième anniversaire du patient
À l'âge corrigé de 2 ans et au cinquième anniversaire du patient

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

Yes

E.6.13.1

Other scope of the trial description

extension study to CRBF002H2301 (RAINBOW study)

Extension de l’étude CRBF002H2301 (RAINBOW)

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.8.3

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

Belgium

Canada

Chile

Colombia

Croatia

Czech Republic

Denmark

Egypt

Estonia

Finland

France

Germany

Greece

Hungary

India

Italy

Japan

Lithuania

Malaysia

Mexico

Poland

Romania

Russian Federation

Saudi Arabia

Slovakia

Sweden

Taiwan

Turkey

United Arab Emirates

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

La dernière visite du dernier sujet

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

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:

300

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:

300

F.1.1.5

Children (2-11years)

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

The patient population consists of preterm born infants, therefore an inclusion criteria is the written informed consent from a parent or legal guardian

La population de patients se compose de nourrissons prématurés , donc un critère d'inclusion est le recueil du formulaire de consentement éclairé signé par le(s) parent(s) ou tuteur(s) légal (légaux).

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

300

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

Aucun

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

2016-06-15

Ethics Committee Opinion of the trial application

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

P. End of Trial
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IMP with orphan designation in the indication
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