Clinical Trials Register

Summary
EudraCT Number:	2011-001595-19
Sponsor's Protocol Code Number:	32-007
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2012-03-13
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

Italy - Italian Medicines Agency

A.2

EudraCT number

2011-001595-19

A.3

Full title of the trial

A Phase III, Multinational, Multicenter, Randomized, Double-Masked, Study Assessing the Safety and Efficacy of Intravitreal Injections of DE- 109 (three doses) for the Treatment of active, Non-Infectious Uveitis of the Posterior Segment of the eye.

Studio di fase III, multinazionale, multicentrico, randomizzato, in doppio cieco, per valutare la sicurezza e l`™efficacia di iniezioni intravitreali di DE-109 (tre dosi) per il trattamento della uveite attiva non infettiva del segmento posteriore dell`™occhio.

A.3.1

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

A study to see how safe and how well an investigational study drug will work to treat active non-infectious posterior, intermediate or panuveitis.

Studio per valutare la sicurezza e l'efficacia del prodotto in sperimentazione nel trattamento della uveite attiva non infettiva posteriore, intermedia o panuveite.

A.4.1

Sponsor's protocol code number

32-007

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	SANTEN INCORPORATED
B.1.3.4	Country	United States
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	SANTEN INCORPORATED
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	SANTEN INCORPORATED
B.5.2	Functional name of contact point	AJ ACKER
B.5.3	Address:
B.5.3.1	Street Address	555 GATEWAY DRIVE
B.5.3.2	Town/ city	NAPA
B.5.3.3	Post code	CA, 94558
B.5.3.4	Country	United States
B.5.4	Telephone number	+1 707 256-2407
B.5.5	Fax number	+1 707 254-1755
B.5.6	E-mail	aacker@Santeninc.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	DE-109
D.3.2	Product code	DE-109
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	rapamicina
D.3.9.1	CAS number	53123-88-9
D.3.9.2	Current sponsor code	DE-109
D.3.9.3	Other descriptive name	RAPA
D.3.9.4	EV Substance Code	NA
D.3.10	Strength
D.3.10.1	Concentration unit	% (W/W) percent weight/weight
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	.2
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: 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	DE-109
D.3.2	Product code	DE-109
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	rapamicina
D.3.9.1	CAS number	53123-88-9
D.3.9.2	Current sponsor code	DE-109
D.3.9.3	Other descriptive name	RAPA
D.3.9.4	EV Substance Code	NA
D.3.10	Strength
D.3.10.1	Concentration unit	% (W/W) percent weight/weight
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	2
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	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	DE-109
D.3.2	Product code	DE-109
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	rapamicina
D.3.9.1	CAS number	53123-88-9
D.3.9.2	Current sponsor code	DE-109
D.3.9.3	Other descriptive name	RAPA
D.3.9.4	EV Substance Code	NA
D.3.10	Strength
D.3.10.1	Concentration unit	% (W/W) percent weight/weight
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	4
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

Uveitis of the Posterior Segment of the Eye.

Uveite del segmento posteriore dell'occhio

E.1.1.1

Medical condition in easily understood language

Uveite active non-infectious posterior, intermediate or pan-uveitis.

Uveite attiva non infettiva posteriore, intermedia o panuveite.

E.1.1.2

Therapeutic area

Body processes [G] - Ocular Physiological Phenomena [G14]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	14.1
E.1.2	Level	PT
E.1.2	Classification code	10046851
E.1.2	Term	Uveitis
E.1.2	System Organ Class	10015919 - Eye 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

To assess the efficacy of DE-109 by comparing the proportion of subjects with vitreous haze score of 0 at Month 5 (SUN scale).

Valutazione dell'efficacia di DE-109 mediante comparazione della proporzione di soggetti con punteggio di opacità vitrosa 0 al mese 5 (Nomenclatura standard uveite, Standardized Uveitis Nomenclature [SUN] scala fotografica).

E.2.2

Secondary objectives of the trial

To evaluate the efficacy of DE-109 by comparing the: - Proportion of subjects with 2 or more unit change from baseline in vitreous haze score at Month 5 on SUN scale - Time to reach a vitreous haze score of 0 on SUN scale - Mean change from Baseline in vitreous haze score at Month 5 - Proportion of subjects on <5 mg/day of prednisone at Month 5 - Mean change from Baseline in Early Treatment Diabetic Retinopathy Study (ETDRS) best-corrected visual acuity (BCVA) at Month 5 - Mean change from Baseline in central foveal thickness as measured by optical coherence tomography (OCT) at Month 5 - Change from Baseline in the National Eye Institute (NEI) Visual Functioning Questionnaire-25 (VFQ-25)

Valutazione dell'efficacia di DE-109 mediante: • Proporzione di soggetti con un miglioramento di 2 unità nel punteggio dell’opacità vitrosa (scala SUN) al mese 5 • Tempo per raggiungere un punteggio di opacità vitrosa 0 (scala SUN) • Modifica media dalla baseline nel punteggio di opacità vitrosa al mese 5 (scala SUN) • Proporzione di soggetti su <5 mg/giorno di prednisone al mese 5 • Modifica media dalla baseline della acuità visiva meglio corretta (best-corrected visual acuity (BCVA)) nello studio della retinopatia diabetica con trattamento precoce (Early Treatment Diabetic Retinopathy Study (ETDRS)) al mese 5 • Modifica media dalla baseline dello spessore foveale centrale misurato con tomografia a coerenza ottica (optical coherence tomography (OCT)) al mese 5 • Modifica dalla baseline nel Visual Functioning Questionnaire-25 (VFQ-25) del National Eye Institute (NEI)

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Ability to give informed consent and attend all study visits 2. Males or females greater than or equal to 18 years of age 3. Have diagnosis of active uveitis determined by the Investigator to be noninfectious based on the subject's medical history, history of present illness, ocular examination, review of systems, physical examination, and any relevant, pertinent laboratory evaluations. If an anterior component is present, it must be less than the posterior component 4. Have active uveitis defined as having >1+ (excluding 1+) vitreous haze score (SUN scale) 5. Best-corrected ETDRS visual acuity letter score of 19 letters or more (20/400 Snellen equivalent or better) in study eye

Criteri di inclusione: 1. Capacità di dare il consenso informato e di essere presente a tutte le visite dello studio. 2. Maschi o femmine di almeno 18 anni di età. 3. Avere una diagnosi di uveite acuta che l’investigatore stabilisce essere non infettiva sulla base dell’anamnesi del soggetto, della storia della presente malattia, dell’esame oculare, della revisione del sistema, dell’esame fisico e di qualunque rilevante e pertinente esame di laboratorio. Se è presente una componente anteriore deve essere inferiore alla componente posteriore. 4. Avere uveite acuta definita come avere un punteggio di > 1+ (escludendo 1+) di opacità vitrosa (scala SUN). 5. Punteggio di lettere dell’acuità visiva meglio corretta ETDRS di almeno 19 lettere (20/400 Snellen equivalente) o migliore all’occhio dello studio.

E.4

Principal exclusion criteria

Ocular: 1. Active infectious uveitis. However, if the uveitis is the consequence of previous infectious disease, such as Tuberculosis, as long as the previous infectious disease is no longer active may be enrolled 2. Clinically suspected or confirmed central nervous system or ocular lymphoma 3. Primary diagnosis of anterior uveitis 4. Uncontrolled glaucoma, evidenced by an intraocular pressure of > 21 mmHg while on medical therapy, or chronic hypotony (<6 mmHg) 5. Any implantable corticosteroid-eluting device (e.g. Retisert, Ozurdex, I-vation, triamcinolone acetonide [TA] intravitreal implant): a. If the investigator confirms the removal of such device more than 90 days prior to Day 1, the subject will be eligible; b. If a subject received a Medidur implant, it should have been implanted no less than 3 years and 90 days prior to Day 1 6. Any significant ocular disease that could compromise vision in the study eye. These include, but are not limited to: a. Diabetic retinopathy: proliferative diabetic retinopathy (PDR) or nonproliferative diabetic retinopathy (NPDR) that compromise vision. Subjects with NPDR or PDR that does not compromise vision are not excluded from the study; b. Wet age-related macular degeneration; c. Myopic degeneration with active subfoveal choroidal neovascularization 7. Lens opacities or obscured ocular media other than vitreous haze upon enrollment such that reliable evaluations and grading of the posterior segment cannot be performed 8. Intraocular surgery within 90 days prior to Day 1 in the study eye 9. Capsulotomy within 30 days prior to Day 1 in the study eye 10. Any of the following treatments within 90 days prior to Day 1 or anticipated use of any of the following treatments to the study eye: a. Intravitreal injections (including but not limited to steroids or antivascular endothelial growth factors); b. Posterior subtenon steroids 11. Ocular or periocular infection in either eye 12. Pupillary dilation inadequate for quality stereoscopic fundus photography in the study eye 13. Media opacity that would limit clinical visualization, intravenous fluorescein angiography (IVFA), or OCT evaluation in the study eye 14. History of herpetic infection in the study eye or adnexa 15. Presence of known active, inactive toxoplasmosis or toxoplasmosis scar in either eye 16. Presence of any form of ocular malignancy in either eye including choroidal melanoma. Non-Ocular: 1. Allergy or hypersensitivity to study drug product or fluorescein dye 2. Participation in other investigational drug or device clinical trials within 30 days prior to Day 1, or planning to participate in other investigational drug or device clinical trials within 150 days following Day 1. This includes both ocular and non-ocular clinical trials. 3. Treatment with a monoclonal antibody or any other biologic therapy within the previous 30 days, or with alemtuzumab within the previous 12 months from Day 1 4. Immunosuppressive therapy (e.g., methotrexate, cyclosporine, cyclophosphamide, chlorambucil, mycophenolate mofetil, tacrolimus or azathioprine) other than prednisone or other corticosteroids for the treatment of uveitis within 30 days of the first study drug administration (Day 1) 5. Any recent systemic infection within 30 days of Screening 6. Known to be immunocompromised 7. History of cytomegalovirus infection or clinical evidence of active cytomegalovirus infection at Screening and/or Day 1 8. History of other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease condition that contraindicates the use of an investigational drug, might affect the interpretation of the results of the study, or renders the subject at high risk for treatment complications.

Criteri di esclusione: Oculare: 1)Uveite infettiva attiva. Ad ogni modo, se l’uveite compare in un soggetto come conseguenza di una precedente infezione, come ad esempio la tubercolosi, che tuttavia non è più attiva, tale soggetto potrà essere reclutato.2)Linfoma clinicamente sospetto o confermato del sistema nervoso centrale o oculare 3)Diagnosi primaria di uveite anteriore 4)Glaucoma non controllato, evidenziato da pressione intraoculare di > 21 mmHg durante terapia medica o ipotonia cronica (<6 mmHg) 5)Qualunque dispositivo corticosteroide-eluente impiantabile (per es. impianto intravitreale Retisert, Ozurdex, I-vation, triamcinolone acetonide [TA]): a) se l’investigatore conferma che la rimozione di tale dispositivo è avvenuta oltre 90 giorni prima del giorno 1, il soggetto sarà eleggibile; b) se un soggetto ha ricevuto un impianto Medidur, esso deve essere stato eseguito non meno di 3 anni e 90 giorni prima del giorno 1 6)Qualunque malattia oculare significativa che potrebbe compromettere la visione all’occhio dello studio. Queste comprendono, ma non sono limitate esclusivamente a: a)Retinopatia diabetica: retinopatia diabetica proliferativa (PDR) o retinopatia diabetica non proliferativa (NPDR) che comprometta la visione. Soggetti con NPDR o PDR che non compromettono la visione non sono esclusi dallo studio; b)Degenerazione maculare senile essudativa; c)Degenerazione miopica con neovascolarizzazione coroidale subfoveale. 7)Opacità del cristallino o media oculari, diversi dall’opacità vitrea, oscurati al momento del reclutamento tale che non possano essere eseguite valutazioni e gradazioni affidabili del segmento posteriore. 8)Chirurgia intraoculare entro 90 giorni prima del giorno 1 all’occhio dello studio. 9)Capsulotomia entro 30 giorni prima del giorno 1 all’occhio dello studio.10)Qualunque dei seguenti trattamenti entro 90 giorni prima del giorno 1 o uso previsto dei seguenti trattamenti all’occhio dello studio: a)Iniezioni intravitreali (includenti ma non limitate a steroidi o fattori della crescita dell’endotelio anti-vascolare) b)Steroidi posteriori del subtenon 11)Infezione oculare o perioculare a uno o l’altro occhio 12)Dilatazione della papilla inadeguata alla qualità della fotografia stereoscopica del fundus dell’occhio dello studio 13)Opacità dei mezzi che limiterebbero la visualizzazione clinica, angiografia endovenosa della fluorescina (IVFA), o valutazione OCT all’occhio dello studio 14)Storia di infezione erpetica all’occhio dello studio o agli annessi 15)Presenza di toxoplasmosi attiva, inattiva o cicatrice da toxoplasmosi all’uno o l’altro occhio 16)Presenza di qualsiasi forma di tumore maligno oculare all’uno o all’altro occhio incluso melanoma coroidale. Non oculare: 1)Allergia o ipersensibilità al farmaco dello studio o al colorante fluoresceina 2) Partecipazione a sperimentazioni cliniche di altro farmaco investigativo o di dispositivi entro 30 giorni prima del giorno 1 o progettazione di partecipare ad altre sperimentazioni cliniche di farmaco investigativo o di dispositivi entro 150 giorni dopo il giorno 1. Questo include sia sperimentazioni cliniche oculari che non oculari. 3)Trattamento con anticorpo monoclonale o altra terapia biologica entro i 30 giorni precedenti o con alemtuzumab entro 12 mesi precedenti al giorno 1. 4)Terapia immunosoppressiva (per es., metotrexate, ciclosporina, ciclofosfamide, clorambucile, micofenolato mofetil, tacrolimus o azatioprina) diversa da prednisone o altri corticosteroidi per il trattamento di uveite entro 30 giorni dalla prima somministrazione del farmaco dello studio (giorno 1) 5)Qualunque recente infezione sistemica entro 30 giorni di screening 6) Immunosoppressione accertata 7)Storia di infezione da citomegalovirus o evidenza clinica di infezione da citomegalovirus attiva allo screening e/o al giorno 1 8) Storia di altre malattie, disfunzione metabolica, risultati

E.5 End points

E.5.1

Primary end point(s)

The primary endpoint is the proportion of responders with vitreous haze score of 0 at Month 5 (SUN scale).

Misurazione end point primario: • Proporzione di soggetti con punteggio di opacità vitrosa 0 al mese 5 (Nomenclatura standard uveite, Standardized Uveitis Nomenclature [SUN] scala fotografica)

E.5.1.1

Timepoint(s) of evaluation of this end point

Month 5

Mese 5

E.5.2

Secondary end point(s)

The secondary efficacy variables will include: - Proportion of subjects with 2 or more unit change from baseline in vitreous haze score at Month 5 - Time to achieve a vitreous haze score of 0 - Mean change from Baseline in vitreous haze score at Month 5 - Proportion of subjects on <5 mg/day of prednisone at Month 5 - Mean change from Baseline in ETDRS BCVA at Month 5 - Mean change from Baseline in central foveal thickness as measured by OCT at Month 5 - Change from Baseline in the NEI VFQ-25

Misurazione end point secondario: Valutare l’efficacia di DE-109 confrontando: • Proporzione di soggetti con un miglioramento di 2 unità nel punteggio dell’opacità vitrosa (scala SUN) al mese 5 • Tempo per raggiungere un punteggio di opacità vitrosa 0 (scala SUN) • Modifica media dalla baseline nel punteggio di opacità vitrosa al mese 5 (scala SUN) • Proporzione di soggetti su <5 mg/giorno di prednisone al mese 5 • Modifica media dalla baseline della acuità visiva meglio corretta (best-corrected visual acuity (BCVA)) nello studio della retinopatia diabetica con trattamento precoce (Early Treatment Diabetic Retinopathy Study (ETDRS)) al mese 5 • Modifica media dalla baseline dello spessore foveale centrale misurato con tomografia a coerenza ottica (optical coherence tomography (OCT)) al mese 5 • Modifica dalla baseline nel Visual Functioning Questionnaire-25 (VFQ-25) del National Eye Institute (NEI)

E.5.2.1

Timepoint(s) of evaluation of this end point

Month 5

Mese 5

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

Yes

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)

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

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

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

- Stesso farmaco ad altro dosaggio

- same IMP used at different dosage

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

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

Argentina

Brazil

Chile

Colombia

Dominican Republic

India

Israel

Peru

Turkey

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

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)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

425

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

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

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

335

F.4.2.2

In the whole clinical trial

500

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)

Not different from standard of care

Non differente dalla terapia standard

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

2011-08-04

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2011-07-19

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

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