Clinical Trials Register

Summary
EudraCT Number:	2018-003428-35
Sponsor's Protocol Code Number:	KSI-CL-102
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2021-05-24
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2018-003428-35

A.3

Full title of the trial

A Phase 2, Prospective, Randomised, Double-masked, Active Comparator-controlled, Multi-center Study to Investigate the Efficacy and Safety of Repeated Intravitreal Administration of KSI-301 in Subjects with Neovascular (Wet) Age-related Macular Degeneration.

Studio di Fase 2, prospettico, randomizzato, in doppio cieco, controllato con farmaco di confronto attivo, multicentrico per determinare l’efficacia e la sicurezza della somministrazione intravitreale ripetuta di KSI-301 in soggetti con Degenerazione maculare neovascolare correlata all'età (DMS umida).

A.3.1

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

Study of Intravitreal KSI-301 in Patients With Neovascular (Wet) Agerelated Macular Degeneration

Studio di KSI-301 intravitreale in pazienti con degenerazione maculare neovascolare (umida) legate all'età.

A.3.2

Name or abbreviated title of the trial where available

Study of Intravitreal KSI-301 in Patients With Neovascular (Wet) Agerelated Macular Degeneration

Studio di KSI-301 intravitreale in pazienti con degenerazione maculare neovascolare (umida) legate a

A.4.1

Sponsor's protocol code number

KSI-CL-102

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT04049266

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	Kodiak Sciences Inc.
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	Kodiak Science Inc
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	Kodiak Science Inc
B.5.2	Functional name of contact point	KSI-CL-102 Trial Information
B.5.3	Address:
B.5.3.1	Street Address	2631 Hanover Street
B.5.3.2	Town/ city	Palo, Alto, California
B.5.3.3	Post code	94304
B.5.3.4	Country	United States
B.5.4	Telephone number	0000000
B.5.5	Fax number	0000000
B.5.6	E-mail	KSI-CL-102@kodiak.com

D. IMP Identification
D.IMP: 1
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	Eylea
D.2.1.1.2	Name of the Marketing Authorisation holder	Bayer AG
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	Aflibercept
D.3.2	Product code	[N/A]
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	AFLIBERCEPT
D.3.9.1	CAS number	862111-32-8
D.3.9.2	Current sponsor code	-
D.3.9.3	Other descriptive name	Aflibercept
D.3.9.4	EV Substance Code	SUB26987
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	40
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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
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	KSI-301 Drug Product
D.3.2	Product code	[KSI-301]
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.3.9.2	Current sponsor code	OG1953
D.3.9.3	Other descriptive name	KSI-301
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	50
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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
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

Neovascular (Wet) Age-related Macular Degeneration

Degenerazione Maculare Neovascolare (umida) legata all'età

E.1.1.1

Medical condition in easily understood language

Neovascular (Wet) Age-related Macular Degeneration

Degenerazione Maculare Neovascolare (umida) legata all'età

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	20.1
E.1.2	Level	LLT
E.1.2	Classification code	10067791
E.1.2	Term	Wet macular degeneration
E.1.2	System Organ Class	10015919 - Eye disorders

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	SOC
E.1.2	Classification code	10015919
E.1.2	Term	Eye disorders
E.1.2	System Organ Class	10015919 - Eye disorders

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10071129
E.1.2	Term	Neovascular age-related macular degeneration
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 demonstrate that KSI-301 5 mg is non-inferior to aflibercept 2 mg with respect to mean change in BCVA from Day 1 to Year 1. Year 1 is defined as the mean of the Week 48 and 52 measurements.

Dimostrare che il KSI-301 5 mg non è inferiore a aflibercept 2 mg rispetto alla variazione media della BCVA dal Giorno 1 all’Anno 1. L'Anno 1 è definito come la media delle misurazioni della Settimana 48 e 52.

E.2.2

Secondary objectives of the trial

•To evaluate the efficacy of KSI-301 5 mg compared to aflibercept 2 mg over the study duration of 96 weeks by assessing visual and anatomical parameters.
•To evaluate the proportion of subjects maintained on a Q12W, Q16W, or Q20W dosing regimen of KSI-301 over the duration of the first 52 weeks.
•To evaluate the safety and tolerability of KSI-301 5 mg compared to aflibercept 2 mg.
•To assess the systemic pharmacokinetics and immunogenicity of KSI-301.

• Valutare l'efficacia di KSI-301 5 mg rispetto ad aflibercept 2 mg nell'arco dello studio di 96 settimane valutando i parametri visivi e anatomici.
• Valutare la percentuale di soggetti mantenuti a un regime di dosaggio Q12W, Q16W o Q20W di KSI-301 nell'arco delle prime 52 settimane.
• Stabilire la sicurezza e la tollerabilità di KSI-301 5 mg rispetto ad aflibercept 2 mg.
• Valutare la farmacocinetica sistemica e l'immunogenicità di KSI-301.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1.Active, treatment-naïve choroidal neovascularization (CNV) secondary to AMD, including subfoveal, juxtafoveal and extrafoveal lesions or retinal angiomatous proliferation (RAP) lesions with a CNV component that affect the central subfield as evidenced by FA or OCT in the Study Eye at Screening.
2.The CNV area in the Study Eye must be at least 50% of total lesion size at Screening.
3.A lesion area <30 mm2 (12 disc areas) of any CNV lesion subtype in the Study Eye.
4.Intra and/or subretinal fluid and/or SHRM (subretinal hyperreflective material) affecting the central subfield of the Study Eye on OCT at Screening.
5.BCVA ETDRS score between 80 and 25 letters (20/25 to 20/320 Snellen equivalent), inclusive, in the Study Eye at Screening and reconfirmed at Day 1.
6.Decrease in vision in the Study Eye determined by the Investigator to be primarily the result of wet AMD.
7.Only one eye per subject is eligible to participate in the study.

1. Neovascolarizzazione coroideale (CNV) attiva, naïve al trattamento, secondaria alla DMS, comprese lesioni subfoveali, iuxtafoveali ed extrafoveali o lesioni da proliferazione angiomatosa retinica (RAP) con componente CNV che interessa il settore centrale come evidenziato da FA o OCT dell'Occhio oggetto dello studio allo Screening.
2. L'area di CNV nell'Occhio oggetto dello studio deve essere almeno il 50% della dimensione totale della lesione allo Screening.
3. Un'area della lesione < 30 mm2 (12 aree del disco) di qualsiasi sottotipo di lesione da CNV nell'Occhio oggetto dello studio.
4. Liquido intraretinico e/o sottoretinico e/o SHRM (materiale iperriflettente sottoretinico) che interessa il settore centrale dell'Occhio oggetto dello studio sulla OCT allo Screening.
5. Punteggio BCVA ETDRS tra 80 e 25 lettere (da 20/25 a 20/320 equivalente Snellen), comprese, nell'Occhio oggetto dello studio allo Screening e riconfermato al Giorno 1.
6. La diminuzione della vista nell'Occhio oggetto dello studio stabilita dallo sperimentatore che è principalmente conseguenza della DMS umida.
7. Solo un occhio per soggetto è eleggibile per la partecipazione allo studio

E.4

Principal exclusion criteria

1.BCVA of hand motion or worse in the non-Study Eye or non-physical presence of a non-Study Eye (i.e., monocular).
2.Active ocular or periocular infection or inflammation in either eye at Day 1.
3.CNV secondary to other causes in the Study Eye, including pathologic myopia, angioid streaks, prior trauma, ocular histoplasmosis, or others.
4.Any history of macular pathology unrelated to AMD but affecting vision or contributing to subretinal or intraretinal fluid, such as central serous chorioretinopathy.
5.Fibrosis or atrophy of >50% of the lesion size and/or involving the foveal center of the Study Eye at Screening
6.Subretinal blood affecting the foveal center of the Study Eye and/or more than 50% of the lesion size at Screening.
7.Any approved or investigational treatment for neovascular AMD (other than oral vitamin supplements) in the Study Eye at any time.
8.Prior macular laser (e.g., thermal laser or photodynamic therapy laser) in the Study Eye.

1. BCVA del movimento della mano o peggiore nell'Occhio non oggetto dello studio o mancata presenza fisica di un Occhio non oggetto dello studio (ovvero, monoculare).
2. Infezione o infiammazione oculare o perioculare attiva a livello di uno degli occhi al Giorno 1.
3. CNV secondaria ad altre cause nell'Occhio oggetto dello studio, tra cui miopia patologica, strie angioidi, trauma precedente, istoplasmosi oculare o altro.
4. Anamnesi di patologia maculare non correlata alla DMS ma che influisce sulla vista o contribuisce al liquido sottoretinico o intraretinico, come la corioretinopatia sierosa centrale.
5. Fibrosi o atrofia di > 50% della dimensione della lesione e/o con coinvolgimento del centro foveale dell'Occhio oggetto dello studio allo Screening
6. Sangue sottoretinico che interessa il centro foveale dell'Occhio oggetto dello studio e/o più del 50% della dimensione della lesione allo Screening.
7. Qualsiasi trattamento approvato o sperimentale per la DMS neovascolare (diverso dagli integratori di vitamine per via orale) nell'Occhio oggetto dello studio in qualsiasi momento.
8. Precedente laser maculare (ad es. laser termico o laser per terapia fotodinamica) nell'Occhio oggetto dello studio.

E.5 End points

E.5.1

Primary end point(s)

The primary endpoint of this study will be the mean change in BCVA from Day 1 to Year 1, comparing subjects receiving KSI-301 to aflibercept at a non-inferiority margin of 4 letters. Year 1 is defined as the mean BCVA at Weeks 48 and 52.

L'endpoint primario di questo studio sarà la variazione media del BCVA dal Giorno1 all'Anno 1, confrontando i soggetti che ricevono KSI-301 con un margine di non inferiorità di 4 lettere. L'Anno 1 è definito come il BCVA medio alle Settimane 48 e 52.

E.5.1.1

Timepoint(s) of evaluation of this end point

weeks 48 and 52

settimane 48 e 52

E.5.2

Secondary end point(s)

At Year 2:
•Proportion of subjects who gain = 5, =10 and =15 letters from baseline over time
•Proportion of subjects who lose = 5, =10 and =15 letters from baseline over time
•Mean change from baseline in BCVA from baseline over time
•Proportion of subjects with BCVA Snellen equivalent of 20/40 or better from baseline over time
•Proportion of subjects with BCVA Snellen equivalent of 20/200 or worse from baseline over time
•Mean change in OCT central subfield retinal thickness (CST) from baseline over time
•Mean change in OCT intraretinal fluid volume from baseline to Week 12, Week 16, and over time
•Mean change in OCT subretinal fluid volume from baseline to Week 12, Week 16, and over time
•Proportion of subjects without intraretinal fluid on OCT from baseline over time
•Proportion of subjects without subretinal fluid on OCT from baseline over time
•Proportion of subjects without pigment epithelial detachments on OCT from baseline over time
•Mean change in CNV total lesion area on FA from baseline at Year 2
•Mean chance in area of leakage on FA from baseline at Year 2

For BCVA and OCT, Year 1 is defined as the average measurement at Weeks 48 and 52 and Year 2 is defined as the average measurement at Weeks 92 and 96.

A full list of secondary endpoints, including the delineation of key versus additional secondary endpoints, will be outlined in the SAP.; At Year 1:
•Proportion of subjects on a Q12W, Q16W or Q20W dosing regimen of KSI-301 at 52 weeks
•Proportion of subjects who gain = 5, =10 and =15 letters from baseline over time
•Proportion of subjects who lose = 5, =10 and =15 letters from baseline over time
•Mean change from baseline in BCVA from baseline to Week 12, Week 16, and over time
•Proportion of subjects with BCVA Snellen equivalent of 20/40 or better from baseline over time
•Proportion of subjects with BCVA Snellen equivalent of 20/200 or worse from baseline over time
•Mean change in OCT central subfield retinal thickness (CST) from baseline to Week 12, Week 16, and over time
•Mean change in OCT intraretinal fluid volume from baseline to Week 12, Week 16, and over time
•Mean change in OCT subretinal fluid volume from baseline to Week 12,Week 16, and over time
•Proportion of subjects without intraretinal fluid on OCT from baseline to Week 12, Week 16 and over time
•Proportion of subjects without subretinal fluid on OCT from baseline to Week 12, Week 16 and over time
•Proportion of subjects without pigment epithelial detachments on OCT from baseline over time
•Mean change in CNV total lesion area on FA from baseline at Year 1
•Mean chance in area of leakage on FA from baseline at Year 1
•Number of study drug injections received in the KSI-301 and aflibercept groups through; Year 1
•For the KSI-301 group, the predictability of being on Q12W, Q16W, or Q20W dosing at the end of Year 1 on the basis of the first set of disease activity assessments (at weeks 20 and 24).

All'anno 2:
- Percentuale di soggetti che nel tempo acquisiscono > 5, >10 e >15 lettere rispetto alla baseline
- Percentuale di soggetti che nel tempo perdono > 5, >10 e >15 lettere rispetto alla baseline
- Variazione media nel BCVA dalla baseline nel tempo.
-Percentuale di soggetti con BCVA equivalente Snellen di 20/40 o meglio dalla baseline nel tempo
-Percentuale di soggetti con BCVA equivalente Snellen di 20/200 o peggio dalla baseline nel tempo
- Variazione media dello spessore della retina del sottocampo centrale misurata con tomografia a coerenza ottica (OCT) dalla baseline nel tempo.
- Variazione media del volume del liquido intraretinico misurata con tomografia a coerenza ottica (OCT) dalla baseline alla Settimana 12, alla Settimana 16 e nel tempo.
- Variazione media del volume del fluido sottoretinico misurata con tomografia a coerenza ottica (OCT) dalla baseline alla Settimana 12, alla Settimana 16 e nel tempo.
-Percentuale di soggetti senza liquido intraretinico misurato con tomografia a coerenza ottica (OCT) dalla baseline nel tempo.
-Percentuale di soggetti senza fluido sottoretinico misurata con tomografia a coerenza ottica (OCT) dalla baseline nel tempo.
-Proporzione di soggetti senza distacchi epiteliali di pigmento misurata con tomografia a coerenza ottica (OCT) dalla baseline nel tempo.
- Variazione media dell'area della lesione totale della neovascolarizzazione coroideale (CNV) rilevata da angiografia con fluoresceina (FA) rispetto alla baseline all'Anno 2
- Variazione media nell'area di fuoriuscita rilevata da angiografia con fluoresceina (FA) rispetto alla baseline all'Anno 2

Per BCVA e OCT, l'Anno 1 è definito come la misura media alle Settimane 48 e 52 e l'Anno 2 è definito come la misura media alle Settimane 92 e 96.

Un elenco completo degli endpoint secondari, compresa la definizione degli endpoint chiave rispetto a quelli secondari aggiuntivi, sarà delineato nel Piano di Analisi Statistica (SAP).; All’Anno 1:
- Proporzione dei soggetti su un regime di dosaggio Q12W, Q16W o Q20W di KSI-301 a 52 settimane.
- Percentuale di soggetti che nel tempo acquisiscono > 5, >10 e >15 lettere rispetto alla baseline
-Percentuale di soggetti che nel tempo perdono > 5, >10 e >15 lettere rispetto alla baseline
-Variazione media nel BCVA dalla baseline alla Settimana 12, alla Settimana 16 e nel tempo.
-Percentuale di soggetti con BCVA equivalente Snellen di 20/40 o meglio dalla baseline nel tempo
-Percentuale di soggetti con BCVA equivalente Snellen di 20/200 o peggio dalla baseline nel tempo
- Variazione media dello spessore della retina del sottocampo centrale misurata con tomografia a coerenza ottica (OCT) dalla baseline alla Settimana 12, alla Settimana 16 e nel tempo.
- Variazione media del volume del liquido intraretinico misurata con tomografia a coerenza ottica (OCT) dalla baseline alla Settimana 12, alla Settimana 16 e nel tempo.
- Variazione media del volume del fluido sottoretinico misurata con tomografia a coerenza ottica (OCT) dalla baseline alla Settimana 12, alla Settimana 16 e nel tempo.
-Percentuale di soggetti senza liquido intraretinico misurato con tomografia a coerenza ottica (OCT) dalla baseline alla Settimana 12, alla Settimana 16 e nel tempo.
-Percentuale di soggetti senza fluido sottoretinico misurata con tomografia a coerenza ottica (OCT) dalla baseline alla Settimana 12, alla Settimana 16 e nel tempo.
-Proporzione di soggetti senza distacchi epiteliali di pigmento misurata con tomografia a coerenza ottica (OCT) dalla baseline alla Settimana 12, alla Settimana 16 e nel tempo.
- Variazione media dell'area della lesione totale della neovascolarizzazione coroideale (CNV) rilevata da angiografia con fluoresceina (FA) rispetto alla baseline all'Anno 1
- Variazione media nell'area di fuoriuscita rilevata da angiografia con fluoresceina (FA) rispetto alla baseline all'anno 1
- Numero di iniezioni di farmaco in studio ricevute nei gruppi KSI-301 e aflibercept durante l’Anno 1; Anno 1:
-Per il gruppo KSI-301, la prevedibilità di un dosaggio Q12W, Q16W o Q20W alla fine dell'Anno 1 sulla base della prima serie di valutazioni dell'attività della malattia (alle settimane 20 e 24).

E.5.2.1

Timepoint(s) of evaluation of this end point

year 2; year 1; Year 1

Anno 2; Anno 1; Anno 1

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

Yes

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)

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

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)

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

Information not present in EudraCT

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

Israel

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

The end of the study according to the protocol is defined as the last follow-up visit of the last subject enrolled (that is, the last subject enrolled's Week 96 visit) or a Sponsor decision to terminate the study, whichever comes first. Study data may be collected beyond the end of the study.

La fine dello studio secondo il protocollo è definita come l'ultima visita di follow-up dell'ultimo soggetto arruolato (cioè, l'ultima visita dell'ultimo soggetto arruolato alla Settimana 96) o una decisione dello Sponsor di terminare lo studio, a seconda di quale dei due eventi si verifica per primo. I dati dello studio possono essere raccolti oltre la fine dello studio.

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.1.1.1

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:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

331

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

181

F.4.2.2

In the whole clinical trial

368

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)

After subjects complete their final follow-up visit or discontinue from the study prematurely, all subjects will return to standard of care treatment at the discretion of their treating physician. The Sponsor will not provide continued access to study treatment following the end of the study or the end of each subject's study treatment period.

Dopo che i soggetti completano la visita di follow-up finale o interrompono prematuramente lo studio, tutti i soggetti torneranno al trattamento standard di cura a discrezione del proprio medico curante. Lo Sponsor non fornirà l'accesso continuato al trattamento in studio dopo la fine dello studio o la fine del periodo di trattamento di studio di ciascun soggetto.

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

2020-02-27

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-11-07

P. End of Trial
P.	End of Trial Status	Prematurely Ended

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