Clinical Trials Register

Summary
EudraCT Number:	2011-005409-65
Sponsor's Protocol Code Number:	AI452-021
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:	2012-12-11
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

2011-005409-65

A.3

Full title of the trial

A Phase 3 Evaluation of Daclatasvir in Combination with Peginterferon Lambda-1a and Ribavirin (RBV) or Telaprevir in Combination with Peginterferon Alfa-2a and RBV in Patients with Chronic Hepatitis C Genotype 1b who are Treatment Naïve or Prior Relapsers to Alfa/RBV Therapy.
Revised Protocol Number 01, incorporating amendment 02

Studio di Fase 3 per la valutazione di Daclatasvir in combinazione con Peginterferone Lambda-1a e Ribavirina (RBV) o Telaprevir in combinazione con Peginterferon alfa-2a e RBV in pazienti con Epatite C Cronica Genotipo 1b naive al trattamento o precedentemente ricaduti dopo terapia con alfa/RBV.
protocollo revisionato 01, che include l'emendamento 02.

A.3.1

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

Efficacy and safety evaluation of a regimen consisting of peginterferon lambda-1a + ribavirin + daclatasvir (Lambda + RBV + DCV) in HCV genotype 1b treatment naïve patients or prior relapsers to peginterferon alfa + ribavirin (alfa + RBV) therapy

Valutazione dell’efficacia e sicurezza di un regime costituito da peginterferon lambda-1a + ribavirina + daclatasvir (Lambda-RBV+DCV) nel trattamento di pazienti naive in pazienti con HCV Cronica Genotipo 1b naive al trattamento o precedentemente ricaduti dopo terapia con alfa/RBV

A.3.2

Name or abbreviated title of the trial where available

The STRUCTURE Study

Studio STRUCTURE

A.4.1

Sponsor's protocol code number

AI452-021

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	BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
B.1.3.4	Country	Belgium
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	Bristol-Myers Squibb International Corporation
B.4.2	Country	Belgium
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Bristol-Myers Squibb International Corporation
B.5.2	Functional name of contact point	EU Study Start-Up Unit
B.5.3	Address:
B.5.3.1	Street Address	Parc de l'Alliance - Avenue de Finlande, 8
B.5.3.2	Town/ city	Braine-l'Alleud
B.5.3.3	Post code	1420
B.5.3.4	Country	Belgium
B.5.6	E-mail	clinical.trials@bms.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 Interferon Lambda
D.3.2	Product code	BMS-914143
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	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	PEGINTERFERON LAMBDA-1a
D.3.9.1	CAS number	914617-98-4
D.3.9.2	Current sponsor code	BMS-914143 / PEG IFN-λ1
D.3.10	Strength
D.3.10.1	Concentration unit	µg/ml microgram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	400
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	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	Daclatasvir
D.3.2	Product code	BMS-790052
D.3.4	Pharmaceutical form	Film-coated tablet
D.3.4.1	Specific paediatric formulation	No
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	daclatasvir
D.3.9.2	Current sponsor code	BMS-790052
D.3.9.3	Other descriptive name	HCV NS5A Replication Co-Factor Inhibitor
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	60
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	Comparator
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	Incivo
D.2.1.1.2	Name of the Marketing Authorisation holder	Janssen Cilag International NV
D.2.1.2	Country which granted the Marketing Authorisation	United Kingdom
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	Film-coated tablet
D.3.4.1	Specific paediatric formulation	No
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	TELAPREVIR
D.3.9.1	CAS number	402957-28-2
D.3.9.4	EV Substance Code	SUB31651
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	375
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	Comparator
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	Pegasys
D.2.1.1.2	Name of the Marketing Authorisation holder	Roche Registration Limited
D.2.1.2	Country which granted the Marketing Authorisation	United Kingdom
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 injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	PEGINTERFERON ALFA-2A
D.3.9.1	CAS number	198153-51-4
D.3.9.4	EV Substance Code	SUB16452MIG
D.3.10	Strength
D.3.10.1	Concentration unit	µg/ml microgram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	360
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	No
D.IMP: 5
D.1.2 and D.1.3	IMP Role	Comparator
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	RIBASPHERE
D.2.1.1.2	Name of the Marketing Authorisation holder	Three Rivers Pharmaceuticals,
D.2.1.2	Country which granted the Marketing Authorisation	United States
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	Film-coated tablet
D.3.4.1	Specific paediatric formulation	No
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	RIBAVIRIN
D.3.9.1	CAS number	36791-04-5
D.3.9.4	EV Substance Code	SUB10297MIG
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	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.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

Chronic Hepatitis C Virus Infection (Genotype 1b)

Epatite Cronica C genotipo 1b

E.1.1.1

Medical condition in easily understood language

Chronic Hepatitis C

Epatite Cronica C

E.1.1.2

Therapeutic area

Diseases [C] - Virus Diseases [C02]

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	10008912
E.1.2	Term	Chronic hepatitis C
E.1.2	System Organ Class	10021881 - Infections and infestations

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 compare the efficacy of Lambda/RBV/DCV to alfa-2a/RBV/TVR in subjects with chronic infection with HCV GT-1b, measured as the proportion of subjects who achieve SVR12 at post-treatment follow-up Week 12.

Confrontare l’efficacia di Lambda/RBV/DCV rispetto ad alfa-2a/RBV/TVR in soggetti con infezione cronica da HCV GT 1b, misurata come la proporzione di soggetti che ottengono una SVR12 alla settimana 12 di follow-up post trattamento.

E.2.2

Secondary objectives of the trial

•Compare the proportion of subjects who achieve SVR12 in treatment-naive subjects at post-treatment follow-up Week12 •Compare the proportion of subjects with rash related dermatologic events of special interest reported during the first 12 weeks of treatment •Compare the proportion of subjects who develop treatment emergent cytopenic abnormalities (anemia as defined by Hb <10 g/dL, and/or neutropenia as defined by ANC <750/mm³, and/or thrombocytopenia as defined by platelets <50,000/mm³) through EOT (max. 48 weeks). See Protocol sections 1.3.2 & 1.3.3 for additional objectives.

•Confrontare la percentuale di soggetti ottengono SVR12 soggetti naive al trattamento nel periodo di follow-up di 12 sett post-tratt •Confrontare l’insorgenza di eventi cutanei tipo rash in particolare nelle prime 12 sett di trattam •Confrontare la percentuale di soggetti che sviluppano anomalie all’emocromo di tipo citopenico: anemia (definita da valori di emoglobina<10 g/dL), neutropenia (definita dalla riduzione del numero dei neutrofili < 750/mm3), trombocitopenia (definita dalla riduzione del numero delle piastrine < 50,000/mm3), per tutta la durata del trattamento (massimo 48 settimane). Vedi Protocollo sezione 1.3.2 & 1.3.3 per gli ulteriori obiettivi.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

• Patients chronically infected with HCV GT-1b • Naïve to prior treatment or documented evidence of relapse after 48 wks of treatment with alfa/RBV • HCV RNA viral load ≥ 100,000 copies/mL at screening • Patients with compensated cirrhosis are permitted

• Soggetti con infezione cronica da HCV Genotipo GT-1b • Soggetti naive a precedente trattamento o evidenza documentata di ricaduta di malattia dopo 48 settimane di trattamento con alfa/RBV • HCV RNA ≥ 100,000 copie/mL al momento dello screening • Sono permessi pazienti con cirrosi compensata

E.4

Principal exclusion criteria

• Infection with HCV other than GT-1b • Positive HBsAg or HIV-1/HIV-2 antibody test at screening • Evidence of chronic liver disease caused by diseases other than chronic HCV infection • Current evidence of or history of variceal bleeding, hepatic encephalopathy, or ascites requiring diuretics or paracentesis or evidence of any of these findings on physical examination performed at screening • Current or known history of cancer (except adequately treated in situ carcinoma of the cervix, or basal or squamous cell carcinoma of the skin) within 5 years prior to screening • Evidence of decompensated cirrhosis based on radiologic criteria or biopsy results and clinical criteria • Laboratory values: hemoglobin < 12.0 g/dL (males) or < 11.0 g/dL (females), platelets <90,000/mm³, total serum bilirubin ≥ 2 mg/dL (unless due to Gilbert's disease).

• Soggetti con infezione da HCV diversa da Genotipo-1b • Soggetti positive allo screening per gli anticorpi di HBsAg o HIV-1/HIV-2 • Evidenza di malattia epatica cronica causata da altre malattie diverse da malattia cronica da HCV • L'evidenza o anamnesi di sanguinamento da varici, encefalopatia epatica, ascite che richiede l’uso di diuretici o paracentesi o evidenza di uno qualsiasi di quest’ultimii su esame fisico effettuato al momento dello screening. • La storia attuale o nota di malattia tumorale (eccetto carcinoma in situ della cervice, o carcinoma a cellule basali o squamose della pelle adeguatamente trattati) entro 5 anni prima dello screening. • Evidenza di cirrosi scompensata in base a criteri radiologici o ai risultati della biopsia e ai criteri clinici • Valori di laboratorio: emoglobina < 12.0 g/dL (maschi) o < 11.0 g/dL (femmine), piastrine <90,000/mm³, bilirubina totale sierica ≥ 2 mg/dL (a meno che a causa della Malattia di Gilbert).

E.5 End points

E.5.1

Primary end point(s)

To compare the efficacy of Lambda/RBV/DCV to alfa-2a/RBV/TVR in subjects with chronic infection with HCV GT-1b, measured as the proportion of subjects with SVR12.

Confrontare l’efficacia di Lambda/RBV/DCV verso alfa-2a/RBV/TVR in suggetti con infezione cronica da HCV GT-1b, misurata con la proportzione di suggetti con SVR12.

E.5.1.1

Timepoint(s) of evaluation of this end point

The Week 12 post-treatment follow-up visit. An additional analysis will be conducted when all subjects complete the Week 24 post-treatment follow-up visit.

L'analisi dell'endpoint primario sarà effettuata dopo che tutti i soggetti raggiungeranno la visita della settimana 12 di follow-up post-trattamento. Un'ulteriore analisi sarà effettuata quando tutti i soggetti completeranno la visita della settimana 24 follow-up post-trattamento.

E.5.2

Secondary end point(s)

Proportion of subjects with • SVR12 in naive • dermatologic events • treatment emergent cytopenic abnormalities • on-treatment IFN associated flu-like/musculoskeletal symptoms • SVR24

Proporzione di soggetti con • SVR12 nein naive • eventi dermatologici • trattamento delle anomalie citopeniche emergenti • on-treatment IFN associato a sindrome simil-influenzale /sindrome musculoscheletrica • SVR24

E.5.2.1

Timepoint(s) of evaluation of this end point

• After all subjects reach the Week 12 post-treatment follow-up visit • After all subjects complete the first 12 wks of treatment • After all subjects complete treatment • After all subjects complete treatment • After all subjects reach the Week 24 post-treatment follow-up visit.

• Dopo che tutti i soggetti hanno raggiunto la visita della Settimana Week 12 follow-up post-trattamento • Dopo che tutti i soggetti hanno completato le prime 12 settimane di trattamento • Dopo che tutti i soggetti hanno completato il trattamento • Dopo che tutti i soggetti hanno raggiunto la visita della Settimana 24 di follow-up post-trattamento.

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

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

Yes

E.6.11

Pharmacogenomic

Yes

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

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

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)

Yes

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

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

Israel

Japan

Korea, Democratic People's Republic of

Russian Federation

Taiwan

United States

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
Analysis of primary endpoint will be conducted after all subjects reach
the Week 12 posttreatment follow-up visit. An additional analysis will be conducted when all subjects
complete the Week 24 post-treatment follow-up visit.

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:

527

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

235

F.4.2.2

In the whole clinical trial

585

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)

Patients could be enrolled in a long-term follow-up study (AI452-016). For subjects who do not wish or are ineligible to participate in the
rollover study, the investigator should ensure that the subject receives
appropriate standard of care to treat the condition under the study.

I pazienti potranno entare in uno studio a lungo termine di Follow-up (AI452-016).
Per i soggetti che non vogliono o non sono ammissibili a partecipare allo studio di rollover, lo sperimentatore deve assicurare che il soggetto riceva
un’adeguata terapia per trattare la condizione clinica in studio.

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

2013-01-08

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2012-11-26

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2014-10-09

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Select Date Range: to
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IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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