Clinical Trials Register

Summary
EudraCT Number:	2011-005210-11
Sponsor's Protocol Code Number:	BAY94-9027/13024
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:	2012-09-28
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

2011-005210-11

A.3

Full title of the trial

A Phase II/III, multicenter, partially randomized, open label trial investigating safety and efficacy of on-demand and prophylactic treatment with BAY 94-9027 in Severe Hemophilia A

Studio in aperto, di Fase II/III, multicentrico, parzialmente randomizzato per la valutazione della sicurezza e dell'efficacia di Bay 94-9027 nel trattamento domanda o in profilassi con BAY 94-9027 d'Emofilia A grave

A.3.1

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

A clinical trial investigating safety and efficacy of a long-acting factor VIII in severe hemophila A

Studio clinico per valutare sicurezza ed efficacia di un fattore VIII a lunga emivita nell'emofilia A grave

A.3.2

Name or abbreviated title of the trial where available

PROTECT VIII

A.4.1

Sponsor's protocol code number

BAY94-9027/13024

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	BAYER HEALTHCARE AG
B.1.3.4	Country	Germany
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	Bayer HealthCare AG
B.4.2	Country	Germany
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Bayer HealthCare AG
B.5.2	Functional name of contact point	Bayer Clin. Trials Contact CTP Team
B.5.3	Address:
B.5.3.1	Street Address	Bayer Pharma AG, S102, Level2, Room 156
B.5.3.2	Town/ city	BERLINO
B.5.3.3	Post code	13342
B.5.3.4	Country	Germany
B.5.4	Telephone number	NA
B.5.5	Fax number	NA
B.5.6	E-mail	clinical-trials-contact@bayerhealthcare.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	Yes
D.2.5.1	Orphan drug designation number	EU/3/10/847
D.3 Description of the IMP
D.3.1	Product name	BAY: 94-9027 (500 IU/vial)
D.3.2	Product code	NA
D.3.4	Pharmaceutical form	Powder and solvent for solution 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.3.9.2	Current sponsor code	BAY 94-9027
D.3.9.3	Other descriptive name	PEGylated B-Domain Deleted Recombinant Human Antihemophilic Factor VIII
D.3.10	Strength
D.3.10.1	Concentration unit	IU/ml international unit(s)/millilitre
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	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	Yes
D.2.5.1	Orphan drug designation number	EU/3/10/847
D.3 Description of the IMP
D.3.1	Product name	BAY 94-9027 (1000 IU/vial)
D.3.2	Product code	B02BD02
D.3.4	Pharmaceutical form	Powder and solvent for solution 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.3.9.2	Current sponsor code	BAY 94-9027
D.3.10	Strength
D.3.10.1	Concentration unit	IU/ml international unit(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: 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	Yes
D.2.5.1	Orphan drug designation number	EU/3/10/847
D.3 Description of the IMP
D.3.1	Product name	BAY 94-9027 (3000 IU/vial)
D.3.2	Product code	NA
D.3.4	Pharmaceutical form	Powder and solvent for solution 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.3.9.2	Current sponsor code	BAY 94-9027
D.3.10	Strength
D.3.10.1	Concentration unit	IU/ml international unit(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	1200
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.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

Severe Hemophilia A

Emofilia A grave

E.1.1.1

Medical condition in easily understood language

Severe blood clotting factor VIII deficiency

Carenza grave di fattore VIII della coagulazione

E.1.1.2

Therapeutic area

Diseases [C] - Blood and lymphatic diseases [C15]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	15.0
E.1.2	Level	LLT
E.1.2	Classification code	10053753
E.1.2	Term	Hemophilia A without inhibitors
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

• To assess the efficacy of BAY 94-9027 in prevention and treatment of bleeding at different infusion schedules Part B: Major surgery using BAY 94-9027 • To assess the safety and efficacy of BAY 94-9027 in the prevention of bleeding during major surgical

PARTE A: Valutare l’efficacia di BAY 94-9027, somministrato secondo diversi schemi di infusione, nella prevenzione e nel trattamento delle emorragie. PARTE B: Valutare la sicurezza e l’efficacia di BAY 94-9027 nella prevenzione delle emorragie durante gli interventi di chirurgia maggiore

E.2.2

Secondary objectives of the trial

• To evaluate the subject’s assessment of response to treatment • To demonstrate the safety and tolerability of BAY 94-9027 when used in both the on-demand and prophylaxis settings • To assess frequency of inhibitor development • To assess pharmacokinetics and incremental recovery following administration of BAY 94-9027 • To assess treatment satisfaction with BAY 94-9027 and its impact on quality-of-life, work productivity and pain as reported by the subjects

Analizzare la valutazione del soggetto alla risposta al trattamento. Dimostrare la sicurezza e la tollerabilità di BAY 94-9027 utilizzato sia nel trattamento "a domanda" sia nel trattamento in profilassi Valutare la frequenza dello sviluppo di inibitori Valutare la farmacocinetica e il recupero (recovery) dopo somministrazione di BAY 94-9027 Analizzare la soddisfazione del soggetto al trattamento con BAY 94-9027 ed il suo impatto sulla qualità della vita, sulla produttività al lavoro e sul dolore.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

• Male; 12 to 65 years of age (or Male 18-65 years of age in countries where enrollment of minors is not permitted) • Subjects with severe hemophilia A (baseline FVIII activity FVIII:C <1%) determined by measurement at the time of screening or from reliable prior documentation (eg, measurement in other clinical trials, result from approved clinical laboratory) • Previously treated with FVIII concentrate(s) (plasma derived or recombinant) for a minimum of 150 ED • Immunocompetent. If HIV positive, CD4+ lymphocyte count >200/mm3

•Maschi di età compresa tra i 12 e i 65 anni (o Maschi di età compresa tra i 18 e i 65 anni, dove non è permessa l’inclusione di pazienti minori) •Pazienti con Emofilia A grave (al basale, concentrazione di fattore VIII, FVIII:C ≤1%) documentata da un esame effettuato allo screening o da un esame storico (ad esempio misurazione effettuata durante la partecipazione in un precedente studio clinico, oppure risultato ottenuto presso un laboratorio clinico accreditato) •Pazienti precedentemente trattati con qualsiasi tipo di concentrato di FVIII (plasma derivato o ricombinante) per un minino di 150 giorni di esposizione (ED) •Pazienti Immunocompetenti. Se positivi al test dell’HIV, con conta linfocitaria CD4+ >200/mm3 •Volontà e capacità da parte del paziente e/o del genitore di esercitarsi ad apprendere l’uso del diario elettronico per la registrazione di tutti i sanguinamenti e le infusioni effettuati nel corso dello studio •Consenso informato scritto del genitore o del rappresentante legale. Quando ritenuto appropriato può essere richiesto anche il consenso del paziente.

E.4

Principal exclusion criteria

• Current evidence of inhibitor to FVIII with a titer ≥ 0.6 BU/mL, measured by the Nijmegen modified Bethesda assay at the time of screening (central laboratory) (BU: Bethesda unit) • History of inhibitor to FVIII with a titer ≥ 0.6 BU, or clinical history suggestive of inhibitor requiring modification of treatment. Subjects with a maximum historical titer of 1.0 BU on a single measurement but with at least 3 subsequent successive negative results (< 0.6 BU) thereafter are eligible • Any other inherited or acquired bleeding disorder in addition to Hemophilia A • Platelet count < 100,000/mm3 • Creatinine > 2x upper limit of normal • AST or ALT > 5x upper limit of normal (AST: aspartate aminotransferase; ALT: alanine aminotransferase) • The subject is currently participating in another investigational drug study, or has participated in a clinical study involving an investigational drug within 30 days of study entry. Subjects who are currently participating in an investigational study in which they are treated with a currently marketed FVIII concentrate are not excluded. Subjects currently treated with BAY 81-8973, and who have received at least 100 ED with the investigational product, may continue treatment with the product up to the start of Visit 1. • Any subject who is receiving chemotherapy, immune modulatory drugs other than anti-retroviral chemotherapy, or chronic use of oral or intravenous (IV) corticosteroids within the last 3 months. Brief courses of prednisone/methyprednisolone (< 14 days) for treatment of disorders such as synovitis, asthma, etc are allowed at the discretion of the treating physician

•Evidenza allo Screening (ovvero all’esame del laboratorio centrale) della presenza di anticorpi inibitori del FVIII con un titolo > 0,6 UB/mL, misurata secondo il metodo Bethesda modificato secondo Nijmegen (UB: Unità Bethesda) •Storia di sviluppo di alloanticorpi inibitori del FVIII con un titolo > 0,6 UB/mL, o storia medica suggestiva per la presenza di inibitori che hanno richiesto una modificazione del regime terapeutico. Sono comunque eleggibili quei soggetti con riscontro di titolo storico massimo di inibitori pari a 1.0 UB in un’occasione, ma con almeno tre risultati successivi negativi (< 0,6 UB). •Presenza di qualsiasi altra malattia emorragica, ereditata o acquisita, diversa dall’emofilia A •Conta piastrinica < 100,000/mm3 •Valore di Creatinina due volte maggiore rispetto al limite superiore di normalità o valori di ALT/AST cinque volte superiori al limite di normalità •Nota ipersensibilità al principio attivo, o a qualunque dei suoi componenti (es. proteine del topo o criceto) •Paziente che sta già partecipando ad uno studio sperimentale con altro principio attivo , o che ha partecipato ad un trial clinico nei 30 giorni precedenti l’inclusione in questo. Non sono invece esclusi i soggetti che stanno prendendo parte ad uno studio clinico con concentrati di FVIII già in commercio. . Pazienti che sono in trattamento con BAY 81-8973, e che hanno superato i 100 ED a questo farmaco sperimentale, possono proseguire la terapia con il farmaco sperimentale fino alla Visita 1 di questo studio •Soggetti sottoposti a chemioterapia, in trattamento con agenti immunomodulatori diversi dalla terapia anti-retrovirale, o in trattamento cronico con corticosteroidi per via orale o per via endovenosa almeno negli ultimi 3 mesi. Sono permessi brevi cicli di prednisone/metilprednisolone (< 14 giorni) per il trattamento di patologie concomitanti quali sinoviti, asma ecc. sono permessi a discrezione del trattamento medico. •Qualunque soggetto che, a giudizio dello Sperimentatore, risulti incapace o non disponibile a seguire le procedure previste dallo studio

E.5 End points

E.5.1

Primary end point(s)

Efficacy Prophylaxis & Treatment of bleeds: Annualized number of bleeds Pharmacokinetics: Pharmacokinetic data and incremental recovery of FVIII activity

Risposta al trattamento definita come numero “annualizzato” di eventi emorragici. Valutazione della farmacocinetica e del recupero (recovery) dopo somministrazione di BAY 94-9027

E.5.1.1

Timepoint(s) of evaluation of this end point

Prophylaxis & Treatment of bleeds: • End of Treatment Visit (after 36 weeks of treatment) Pharmacokinetics: • Baseline Visit = PK 1 Visit • End of Treatment Visit = PK 2 Visit

Profilassi e Trattamento dei sanguinamenti: • Visita ''End of Treatment'' (dopo 36 settimane di trattamento) - Farmacocinetica: Visita basale = Visita PK 1 • Visita ''End of Treatment'' = Visita PK 2

E.5.2

Secondary end point(s)

Efficacy Prophylaxis & Treatment of bleeds: Description of bleeding according to location and frequency of total bleeds (spontaneous and trauma), joint bleeds, trauma, spontaneous bleeds and all bleeds Treatment of bleeds: Number of infusions required to control a bleed -Subject or investigator assessment of response to treatment of a bleed, as excellent, good, moderate, poor -When counting bleeds, any infusion for a new spontaneous bleed reported within 72 h of a previous bleed reported for the same site should be considered a follow-up infusion and not a new bleed. Prophylaxis -Proportion of prophylaxis infusions that were followed by a bleed within 48, 72 and 96 h -Number of patients requiring dose escalation - Patient reported outcomes: -Patient satisfaction and burden with BAY 94-9027 -Change in overall pain severity and interference due to pain -Change in quality of life ( as assessed by Haemo-QoL/Haemo-QoL-A and HUI) -Change in work productivity and activity impairment Minor surgery: -Assessment of adequacy of hemostasis during minor surgical interventions including blood loss, transfusion, and/or hemostatic-related surgical complications. Part B: Major surgery -Change or drop in hemoglobin/hematocrit -Blood loss -Need for additional hemostatic medication, including blood products -Units of blood transfused -Hemostatic-related surgical complications -Surgeon’s assessment of hemostasis as excellent, good, moderate or poor -Investigator’s assessment of response to treatment as excellent, good, moderate or poor -rFVIII usage expressed as number of infusions and IU/kg per year, as well as IU/kg per event (prophylaxis, bleed treatment, and surgery) -Specific diagnostic evaluations (eg. Imaging to follow size of a hematoma) Safety -Vital signs (pulse, blood pressure, respiratory rate, temperature) -Physical examination -12-lead ECG -Health assessment -Standard laboratory assessment (CBC, chemistry, routine urinalysis) -Development of antibodies to BAY 94-9027, PEG and/or FVIII -Quantitative PEG measurement

Profilassi di efficacia e trattamento dei sanguinamenti: • Descrizione dei sanguinamenti in base alla localizzazione e frequenza dei sanguinamenti totali (spontanei e traumatici), emartri, trauma, sanguinamenti spontanei e tutti i tipi di sanguinamento
Trattamento dei sanguinamenti: • Numero delle infusioni richieste per controllare un sanguinamento • Valutazione del Paziente o dello Sperimentatore della risposta al trattamento di un sanguinamento come “eccellente, “buono”, “moderato”, “scarso” • Nella conta dei sanguinamenti, qualsiasi infusione per un nuovo sanguinamento spontaneo riportato entro 72 ore da un sanguinamento precedente avvenuto nello stesso sito di localizzazione dovrebbe essere considerato una infusione di follow-up anziché un nuovo sanguinamento.
Profilassi:
• Entità delle infusioni di profilassi seguite da un sanguinamento entro 48, 72 e 96 ore • Numero dei Pazienti che richiedono un aumento della dose
- Esiti riferiti dai Pazienti: • soddisfazione e disagio con BAY 94-9027 • Variazioni nella classificazione globale della gravità del dolore ed interferenze dovute al dolore • Variazioni nella qualità della vita (secondo scale Haemo-QoL/Haemo-QoL-A e HUI) • Variazioni nella produttività lavorativa e nello svolgimento delle normali attività - chirurgia minore che impediscono la produttività: • Valutazione dell’emostasi durante interventi di chirurgia minore, perdite di sangue, trasfusioni e/o complicazioni chirurgiche di tipo emostatico.
-Parte B: -Chirurgia maggiore • Variazione o calo di emoglobina/ematocrito • perdita di sangue • Necessità di terapia emostatica aggiuntiva inclusi prodotti plasmaderivati • Unità di sangue trasfuso • Complicazioni chirurgiche di tipo emostatico • Valutazione da parte del chirurgo della emostasi come “eccellente, “buono”, “moderato”, “scarso” • Valutazione dello Sperimentatore della risposta al trattamento di un sanguinamento come “eccellente, “buono”, “moderato”, “scarso” • • uso di rFVIII espresso come numero di infusioni e IU/kg all’anno, IU/kg per evento (profilassi, trattamento dei sanguinamenti, chirurgia) • Valutazioni diagnostiche specifiche (es. Imaging per monitorare la dimensione dell’ematoma)
-Sicurezza • Segni vitali (pulsazioni, pressione sanguigna, frequenza respiratoria, temperatura) • Esame fisico • ECG 12 lead• -Stato di salute• Valutazioni Standard di laboratorio (CBC, chimica, esami unrine di routine) • Sviluppo di anticorpi verso il BAY 94-9027, PEG e/o FVIII • Misurazione quantitativa PEG

E.5.2.1

Timepoint(s) of evaluation of this end point

Prophylaxis & Treatment of bleeds: • End of Treatment Visit (after 36 weeks of treatment) -Part B: Major surgery • Post-operative Visit

Profilassi e Trattamento dei sanguinamenti: • Visita ''End of Treatment'' (dopo 36 settimane di trattamento)
PARTE B: Chirurgia maggiore • Visita Post-operatoria

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

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

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

Yes

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

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

Yes

E.8.1.7.1

Other trial design description

parzialmente randomizzato

partially randomized

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

Information not present in EudraCT

E.8.2.2

Placebo

Information not present in EudraCT

E.8.2.3

Other

Information not present in EudraCT

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

Australia

Canada

Colombia

Israel

Japan

Korea, Republic of

Mexico

Singapore

Taiwan

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

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)

F.1.1.5

Children (2-11years)

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:

100

F.1.3

Elderly (>=65 years)

F.2 Gender

F.2.1

Female

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

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

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

140

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

2012-09-14

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2012-09-14

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2019-11-21

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