Clinical Trials Register

Summary
EudraCT Number:	2017-000373-36
Sponsor's Protocol Code Number:	997HA402
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:	2021-01-22
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

2017-000373-36

A.3

Full title of the trial

A Non-controlled, Open-Label, Multicenter, Study of Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Subjects with Inhibitors Undergoing the First ITI Treatment

Studio multicentrico, in aperto, non controllato sull'efficacia di rFVIIIFc per l'induzione della tolleranza immunologica (ITI) in soggetti affetti da emofilia A grave con inibitori, che si sottopongono al Primo trattamento ITI.

A.3.1

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

A clinical study exploring the use of rFVIIIFc in patients with severe Haemophilia A who are undergoing Immune Tolerance Induction (ITI) for the first time

Uno studio clinico che esplora l¿uso di rFVIIIFc in pazienti con grave emofilia A, che vengono sottoposti per la prima volta a induzione della tolleranza immunologica (ITI)

A.3.2

Name or abbreviated title of the trial where available

A clinical study exploring the use of rFVIIIFc in patients with severe Haemophilia A who are undergo

Uno studio clinico che esplora l¿uso di un fattore, chiamato rFVIIIFc, in pazienti con grave emofili

A.4.1

Sponsor's protocol code number

997HA402

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	BIOVERATIV THERAPEUTICS 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	Bioverativ Therapeutics 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	Bioverativ Therapeutics Inc
B.5.2	Functional name of contact point	na
B.5.3	Address:
B.5.3.1	Street Address	225 Second Avenue
B.5.3.2	Town/ city	Waltham, MA
B.5.3.3	Post code	02451
B.5.3.4	Country	United States
B.5.4	Telephone number	0000000
B.5.5	Fax number	00000000
B.5.6	E-mail	clinicaltrials@bioverativ.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	ELOCTA
D.2.1.1.2	Name of the Marketing Authorisation holder	Swedish Orphan Biovitrum AB (publ)
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	recombinant Factor VIII Fc
D.3.2	Product code	rFVIIIFc
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.3.8	INN - Proposed INN	na
D.3.9.2	Current sponsor code	BIIB031
D.3.9.3	Other descriptive name	rFVIIIFc, recombinant Factor FVIII Fc
D.3.9.4	EV Substance Code	SUB168409
D.3.10	Strength
D.3.10.1	Concentration unit	IU international unit(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	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

Hemophilia A

Emofilia A

E.1.1.1

Medical condition in easily understood language

Hemophilia A is a genetic disorder that impairs the body's ability to control blood clotting or coagulation, which is used to stop bleeding.

L¿emofilia A ¿ un disturbo genetico che riduce la capacit¿ dell¿organismo di
controllare la coagulazione del sangue, che a sua volta viene utilizzata per bloccare le emorragie

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	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10060613
E.1.2	Term	Hemophilia A (Factor VIII)
E.1.2	System Organ Class	100000004850

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10053751
E.1.2	Term	Hemophilia A with anti factor VIII
E.1.2	System Organ Class	100000004850

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

The primary objective of the study is to describe the time to tolerance (i.e., ITI succes) with rFVIIIFc in subjects within a maximum of 48 weeks (12 months) of ITI treatment.

L¿obiettivo principale dello studio ¿ quello di descrivere il tempo richiesto all¿insorgenza della tolleranza (per es il trattamento ITI) con rFVIIIFc in soggetti che hanno ricevuto un periodo massimo di 48 settimane (12 mesi) di trattamento ITI.

E.2.2

Secondary objectives of the trial

¿To describe the outcome of ITI treatment
¿To describe the relapse rate over the 48-week (12 mesi) period following successful ITI performed with rFVIIIFc
¿To describe the intercurrent bleeding during ITI and during the 48-week (12 months) period after successful ITI performed with rFVIIIFc
¿To describe the safety and tolerability of rFVIIIFc when used for ITI
¿To evaluate the impact of ITI treatment with rFVIIIFc on health economics and adherence

¿ Descrivere l¿esito del trattamento ITI
¿ Descrivere il tasso di ricaduta nel periodo di 48 settimane (12 mesi) successivo
a una ITI eseguita con rFVIIIFc con esito positivo
¿ Descrivere l¿emorragia intercorrente durante l¿ITI e durante il periodo di 48 settimane (12 mesi) successivo a una ITI eseguita con rFVIIIFc con esito positivo
¿ Descrivere la sicurezza e la tollerabilit¿ di rFVIIIFc quando usato per ITI
¿ Valutare l¿impatto del trattamento ITI con rFVIIIFc sull¿economia e la compliance sanitaria

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1.Ability of the subject or his legally authorized representative (e.g., parent or legal guardian) to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information in accordance with national and local subject privacy regulations.
2.Male subjects of any age diagnosed with severe hemophilia A (as confirmed from the medical record).
3.Currently diagnosed with high titer inhibitors (historical peak =5 BU/mL, according to medical records)
4.Previously treated with any plasma-derived or recombinant conventional or Extended Half-Life FVIII.

1.Abilità del soggetto o o del suo rappresentante legalmente autorizzato (ad esempio genitore o tutore legale) per comprendere lo scopo ei rischi dello studio e fornire consenso informato firmato e datato e l’autorizzazione per utilizzare le informazioni sanitarie protette in conformità alle normative nazionali e locali in materia di privacy.
2. Soggetti di sesso maschile di qualsiasi età con diagnosi di emofilia A grave (come
confermato dalle cartelle cliniche).
3. Pazienti attualmente diagnosticati con inibitori ad alta titolazione (picco storico =5 BU/ml,
secondo le cartelle cliniche)
4. Pazienti precedentemente trattati con FVIII derivati dal plasma o ricombinanti
convenzionali o con emivita estesa.

E.4

Principal exclusion criteria

1. Other coagulation disorder(s) in addition to hemophilia A.
2. Previous ITI
3. History of hypersensitivity or anaphylaxis associated with any rFVIIIFc administration.
4. Planned major surgery scheduled during the study unless deferred until after study completion (minor surgery such as tooth extraction or insertion/replacement of central venous access device is allowed)
5. Abnormal renal function (serum creatinine > 1.5 mg/dl or 2x upper limit of normal (ULN) for subject age based on local laboratory range) as assessed by local laboratory.
6. Serum alanine aminotransferase or aspartate aminotransferase >5 × upper limit of normal (ULN) as assessed by local laboratory.
7. Serum total bilirubin >3 × ULN as assessed by local laboratory
8. Current enrollment or a plan to enroll in any interventional clinical study in which an investigational treatment or approved therapy, other than rFVIIIFc, for investigational use is administered within 30 days (or 5 half-lives of the agent, whichever is longer) prior to the Baseline Visit.
9. Inability to comply with study requirements.
10. Presence of any medical or psychological condition or laboratory result that in the opinion of the Investigator can interfere with the subject’s ability to comply with the protocol requirements or makes the subject not appropriate for inclusion to the study and treatment with Elocta/Eloctate (rFVIIIFc).
11. Other unspecified reasons that, in the opinion of the Investigator or Bioverativ, make the subject unsuitable for enrollment.
12. Unwillingness or inability to comply with the requirements of the protocol, including the presence of any condition (physical, mental, or social) that prevents the subject from participating in visits as scheduled or entering required information into the EDP in a timely manner
13. Concurrent systemic treatment with immunosuppressive drugs within 12 weeks (3 mesi) prior to Screening. Exceptions to this include: ribavirin for treatment of hepatitis C virus (HCV), and/or systemic steroids (a total of 2 courses of pulse treatments lasting no more than 7 days within 12 weeks (3 mesi) prior to Day 1) and/or inhaled steroids
14. High risk of cardiovascular, cerebrovascular, or other thromboembolic events, as judged by the Investigator. The following criteria refer to tests performed within 26 weeks (6.5 months) prior to Screening. If not available, a new test will be drawn at the Screening visit.
15. Cluster of differentiation 4 (CD4) lymphocytes < 200 mm3 at Screening, if known as human immunodeficiency virus (HIV) antibody positive based on medical history or HIV testing
16. Viral load of >400 copies/mL at Screening, if known HIV antibody positive, based on medical history.

1. Altro(i) disturbo(i) di coagulazione in aggiunta all’emofilia A.
2. Precedente ITI
3. Anamnesi con reazioni di ipersensibilità associate alla somministrazione
di rFVIIIFc
4. Interventi chirurgici importanti programmati durante lo studio a meno che dovranno essere posticipati a dopo il completamento dello studio (sono consentiti interventi minori come l’estrazione di un dente o l’inserimento/sostituzione di cateteri venosi centrali)
5. Funzione renale anomala (creatinina serica >1,5 mg/dl o 2x limite superiore del normale (ULN) per soggetto in base al range di laboratorio locale) valutata da un laboratorio locale
6. Alanina aminotransferasi (ALT) o aspartato aminotransferasi (AST) nel siero >5 volte superiore al limite della norma (ULN), valutato da un laboratorio locale
7. Bilirubina totale nel siero >3 volte l’ULN, valutata da laboratorio locale
8. Partecipazione o intenzione di partecipare a uno studio clinico interventistico in cui un trattamento sperimentale o una terapia approvata, diverse rispetto a rFVIIIFc, per l'impiego della sperimentazione viene somministrato entro 30 giorni (o 5 mesi di vita dell'agente, a seconda di quale è più lungo) prima della visita di base.
9. Incapacità di rispettare i requisiti di studio.
10. Presenza di condizioni mediche o psicologiche o esami di laboratorio che, a giudizio dello sperimentatore, possono interferire con la
capacità del paziente di rispettare i requisiti del protocollo o che rendono il paziente non adatto per l’inclusione nello studio e nel trattamento con r Elocta/Eloctate (rFVIIIFc)
11. Altre ragioni non specificate che, a giudizio dell'Investigatore o di Bioverativ, rendono il soggetto inadatto all’arruolamento
12. Rifiuto o incapacità di conformarsi ai requisiti del protocollo, inclusa la presenza di qualsiasi condizione (fisica, mentale o sociale) che impedisce al soggetto di partecipare alle visite come previsto o di inserire in tempi ragionevoli le informazioni richieste nell’EPD
13. Contemporaneo trattamento sistemico con farmaci immunosoppressori nelle 12 settimane (3 mesi) prima dello screening. Le eccezioni includono: ribavirina per
il trattamento del virus dell’epatite C (HCV) e/o steroidi sistemici (un totale di 2 cicli di trattamenti del battito cardiaco che non durino più di 7 giorni entro le 12 settimane (3 mesi) precedenti il giorno 1) e/o steroidi per inalazione
14. Elevato rischio di eventi cardiovascolari, cerebrovascolari o altri eventi tromboembolitici, come stabilito dal medico di studio.
I seguenti criteri si riferiscono a test effettuati entro 26 settimane (6,5 mesi) prima dello screening. Se non disponibili, un nuovo test deve essere effettuato alla visita di screening.
15 Cluster di differenziazione 4 (CD4) nei linfociti < 200 mm3, se noto come
anticorpo del virus dell'immunodeficienza umana (HIV) positivo sulla base della storia medica o del test HIV
16. Carica virale di > 400 copie/ml allo screening, se un anticorpo noto dell’HIV risulta positivo sulla base della storia medica

E.5 End points

E.5.1

Primary end point(s)

The primary endpoint of this study is time to tolerization with a
maximum of 48 weeks (12 months) ITI treatment offered in the study.
(Tolerization defined as inhibitor titer <0.6BU/ml, rFVIIIFc incremental
recovery [IR] = 66% of the expected IR, and t1/2 of =7hrs)

L'endpoint primario di questo studio è il tempo alla tolleranza con un trattamento ITI di massimo di 48 settimane (12 mesi) offerto nello studio. (Tolleranza definita come titolazione dellì inibitore <0.6BU / ml, recupero rFVIIIFc recupero incrementale (IR) > 66% dell’IR atteso e t1 / 2 di =7hrs)

E.5.1.1

Timepoint(s) of evaluation of this end point

The primary endpoint of time to tolerization with a maximum of 12
months (48 weeks) of ITI treatment, will be summarized descriptively using minimum, mean, standard deviation, median, 25th and 75th
quartiles, and maximum. A Kaplan-Meier plot showing percentage of
subjects who are tolerized against time from the first ITI infusion will
also be generated. Subjects not achieving ITI success during the 12
month (48-week) ITI period will be censored at the last observed time
for this analysis. The ITIFAS will be used for this analysis

Verrà fornito un riepilogo dell’endpoint primario del tempo alla tolleranza con un massimo di 12 mesi (48 settimane) di trattamento ITI in maniera descrittiva usando la deviazione minima, media e standard, i quartili mediano, 25º e 75° e il massimo. Verrà anche generato un tracciato di Kaplan-Meier con la percentuale di soggetti tolleranti rispetto al tempo dalla prima infusione dell’ITI. I soggetti che non avranno ottenuto esito positivo nella ITI durante i 12 mesi (48 settimane) del periodo di ITI saranno censurati all’ultima osservazione per questa analisi. Per questa analisi verrà utilizzato l’insieme di analisi ITIFAS

E.5.2

Secondary end point(s)

¿ITI success
oConfirmed negative titers consisting of 2 consecutive negative inhibitor
assessments within 2 weeks (+/-3 days) based on local laboratory
results <0.6 BU/mL by the Nijmegen-modified Bethesda assay;
determination of negative titer based on the Bethesda assay is
acceptable at sites that have not yet adopted the Nijmegen-modified
Bethesda assay oIR) =1.32 IU/dL per IU/kg in 2 consecutive
assessments representing =66% of the expected IR 2 IU/dL per IU/kg
(SmPC;USPI).
o t1/2 =7 hours
¿Occurrence of relapse( defined as confirmed positive inhibitor titer =
0.6 BU/mL),
abnormal recovery after tolerance is achieved, and t¿ <7 hours) during
the Tapering or Follow-Up Periods
¿Number of bleeding episodes during ITI and during the 48-week (12-
month)period after successful ITI performed with rFVIIIFc
¿Adverse events (AEs) and serious adverse events (SAEs)
¿Number of days away from work or school
¿Number of hospitalization days
¿Adherence (defined as percentage of administered doses versus
planned doses)
¿Consumption of rFVIIIFc (measured in total rFVIIIFc use)

ITI con esito positivo o Titolazione negativa consistente di due valutazioni negative per l¿inibitore entro 2 settimane (+/- 3 giorni) in base ai risultati del laboratorio locale (<0,6 BU/ml con il test di Bethesda modificato di Nijmegen); la determinazione del titolo negativo basato sul dosaggio Bethesda ¿ accettabile nei siti che non hanno ancora adottato il test di Nijmegen- Bethesda modificato o IR) = 1,32 UI / dL per UI / kg in 2 determinazioni consecutive rappresentanti il 66% dell¿RI atteso di 2 UI/dl per UI/kg (Smpc;USPI)
t1/2 di FVIII =7 ore
Evento di ricaduta (definito come titolazione positiva dell¿inibitore >0,6 BU/ml); recupero anomalo dopo il raggiungimento della tolleranza e t1/2 < 7 ore durante i periodi di tapering o di follow-up
Numero di episodi di sanguinamento durante l¿ITI e durante il periodo di 48 settimane (12 mesi) successivo alla ITI con rFVIIIFc eseguita con esito positivo
Eventi avversi emergenti dal trattamento (TEAE) ed eventi avversi gravi emergenti dal trattamento (TESAE)
Numero di giorni persi di lavoro o scuola
Numero di giorni di ricovero
Compliance (definita come percentuale di dosi somministrate rispetto alle dosi programmate)
Consumo di rFVIIIFc (misurato come uso totale di rFVIIIFc)

E.5.2.1

Timepoint(s) of evaluation of this end point

After Baseline Visit which is the start of ITI treatment, subjects will be
seen at Week 2 and every fourth week until subjects acheive a negative
inhibitor titer (<0.6 BU/mL). Starting at time of first negative inhibitor
titer until ITI success OR 48 weeks (12 months) of treatment, subjects
will be seen every 2nd week. With ITI success (3 success criteria met)
and continuing for 16 weeks (Tapering period), subjects will have
assessment visits after 2 weeks, 4 weeks and then every 4th week
thereafter. Follow-up Period constitutes the time period in which subject
returns to prophylactic dosing regimen and will be approximately 32
weeks (8 months) in duration.

Dopo la visita basale, che l inizio del trattamento ITI, i soggetti saranno visti alla settimana 2 e ad ogni quarta settimana fino a quando i soggetti raggiungeranno una titolazione negativa dell¿inibitore (<0,6 BU/ml). A partire dalla prima titolazione negativa dell¿inibitore fino a ITI con esito positivo O alle 48 sett (12 mesi) di trattamento, i soggetti saranno vistati a sett alterne. In caso di ITI con esito positivo (i 3 criteri per l¿esito positivo sono stati soddisfatti) e continuando per 16 sett (periodo di riduzione graduale), i soggetti effettueranno visite di valutazione dopo 2 sett, 4 sett e successivamente ogni 4 set. Il periodo di f-up costituisce il periodo di tempo in cui il soggetto ritorna alla posologia profilattica, che avr¿ una durata di circa 32 settimane (8 mesi).

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

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)

E.7.4

Therapeutic use (Phase IV)

Yes

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

E.8.2.4

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

Canada

Japan

United States

Belgium

Bulgaria

France

Germany

Italy

Spain

United Kingdom

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

The end of study is last subject, last visit for final collection of data.

La fine dello studio ¿ l'ultima visita dell'ultimo soggetto per la raccolta finale dei dati.

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.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

Yes

F.1.1.4.1

Number of subjects for this age range:

F.1.1.5

Children (2-11years)

Yes

F.1.1.5.1

Number of subjects for this age range:

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)

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

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Parents/legal guardians of subjects must be provided with the informed consent information prior to the Screening Visit to allow adequate time for review and an opportunity to discuss the study with the Investigator/designee.

I genitori / tutori legali dei soggetti devono essere forniti delle informazioni contenute nel consenso informato prima della visita di screening per consentire un adeguato tempo di revisione e con l¿opportunit¿ di discutere lo studio con l'investiga

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

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

NONE

nessuno

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

2018-01-02

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2017-10-10

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

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