Clinical Trials Register

Summary
EudraCT Number:	2015-002586-39
Sponsor's Protocol Code Number:	MC-TER.2/SSc
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Temporarily Halted
Date on which this record was first entered in the EudraCT database:	2018-02-06
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

2015-002586-39

A.3

Full title of the trial

Randomized, Multicenter, Double-Blind, Placebo-Controlled Trial to
Evaluate the Efficacy and Safety of Terguride Plus Symptomatic Therapy
in Subjects With Diffuse Cutaneous Systemic Sclerosis

Studio randomizzato, multicentrico, in doppio cieco, controllato con placebo per valutare la sicurezza e l’efficacia di Terguride in aggiunta a terapia sintomatica in soggetti affetti da sclerosi sistemica cutanea diffusa (TERGISS)

A.3.1

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

Terguride Plus Symptomatic Therapy in Patients with Diffuse Cutaneous Systemic Sclerosis

Terguride in aggiunta a terapia sintomatica in soggetti affetti da sclerosi sistemica cutanea diffusa

A.3.2

Name or abbreviated title of the trial where available

Terguride Plus Symptomatic Therapy in Patients with Diffuse Cutaneous Systemic Sclerosis

Terguride in aggiunta a terapia sintomatica in soggetti affetti da sclerosi sistemica cutanea diffus

A.4.1

Sponsor's protocol code number

MC-TER.2/SSc

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	MEDAC GMBH
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	medac GmbH
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	medac GmbH
B.5.2	Functional name of contact point	Project Manager Clinical Research
B.5.3	Address:
B.5.3.1	Street Address	Theaterstrasse 6
B.5.3.2	Town/ city	Wedel
B.5.3.3	Post code	D 22880
B.5.3.4	Country	Germany
B.5.4	Telephone number	4941038006434
B.5.5	Fax number	4941038006422
B.5.6	E-mail	c.guimbal-schmolck@medac.de

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	EMA/OD/153/12
D.3 Description of the IMP
D.3.1	Product name	Terguride
D.3.2	Product code	/
D.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	TERGURIDE HYDROGENMALEATE
D.3.9.1	CAS number	37686-85-4
D.3.9.2	Current sponsor code	/
D.3.9.4	EV Substance Code	SUB04725MIG
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	0.25
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	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	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	Yes
D.3.11.13.1	Other medicinal product type	Alcaloide dell'ergot semisintetico
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	EMA/OD/153/12
D.3 Description of the IMP
D.3.1	Product name	Terguride
D.3.2	Product code	/
D.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	TERGURIDE HYDROGENMALEATE
D.3.9.1	CAS number	37686-85-4
D.3.9.2	Current sponsor code	/
D.3.9.4	EV Substance Code	SUB04725MIG
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	1
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	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	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	Yes
D.3.11.13.1	Other medicinal product type	Alcaloide dell’ergot semisintetico
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	Yes
D.2.1.1.1	Trade name	METOCLOPRAMIDE ACCORD - 10 MG COMPRESSE 28 COMPRESSE IN BLISTER PVC/PVDC/AL
D.2.1.1.2	Name of the Marketing Authorisation holder	ACCORD HEALTHCARE ITALIA S.R.L.
D.2.1.2	Country which granted the Marketing Authorisation	Italy
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	Metoclopramide
D.3.2	Product code	/
D.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	METOCLOPRAMIDE
D.3.9.1	CAS number	364-62-5
D.3.9.2	Current sponsor code	/
D.3.9.3	Other descriptive name	METOCLOPRAMIDE
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	10
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	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	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
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Tablet
D.8 Placebo: 2
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Tablet

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Diffuse cutaneous systemic sclerosis (dcSSc)

Sclerosi sistemica cutanea diffusa (dcSSc)

E.1.1.1

Medical condition in easily understood language

systemic sclerosis

sclerosi sistemica

E.1.1.2

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

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	10042953
E.1.2	Term	Systemic sclerosis
E.1.2	System Organ Class	100000004859

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 is to assess the efficacy of terguride compared to
placebo in terms of absolute change in the mRSS during the double-blind
treatment phase in subjects with dcSSc.

L’obiettivo primario consiste nel valutare l’efficacia di terguride rispetto al placebo in termini di variazione assoluta dello Skin Score secondo Rodman modificato (mRSS) durante la fase di trattamento in doppio cieco in soggetti affetti da dcSSc.

E.2.2

Secondary objectives of the trial

The secondary and other objectives include the following:
• To assess the efficacy of terguride compared to placebo in terms of
impact on skin thickening, visceral involvement, functional status,
quality of life, and pharmacodynamic parameters during the double-blind
treatment phase.
• To assess the safety of terguride compared to placebo in a 52-week
double-blind treatment phase.
• To assess the efficacy and safety of terguride in the open-label, 52-
week open-label extension phase.
• To assess the prognostic value of CYP2D6 polymorphism with regard to
the HIwT terguride dosage.

Gli obiettivi secondari e gli altri obiettivi includono quanto segue:
• Valutare l’efficacia di terguride rispetto al placebo in termini di impatto su ispessimento cutaneo, coinvolgimento viscerale, stato funzionale, qualità della vita e parametri farmacodinamici durante la fase di trattamento in doppio cieco.
• Valutare la sicurezza di terguride rispetto al placebo in una fase di trattamento in doppio cieco di 52 settimane.
• Valutare l’efficacia e la sicurezza di terguride nella fase di estensione in aperto di 52 settimane.
• Valutare il valore prognostico del polimorfismo del citocromo P450 (CYP) 2D6 in riferimento al più alto dosaggio di terguride ben tollerato a livello individuale (HIwT).

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Each subject must meet all of the following criteria to be enrolled in this
study (criterion #7 must also be met for inclusion in the open-label
extension phase of the study):
1. Is a male or female ≥18 years of age.
2. Fulfills the ACR/EULAR 2013 criteria for classification of SSc
3. Has diffuse cutaneous involvement (defined by skin fibrosis in at least
1 of the following sites: upper arms, thorax, abdomen, or thighs)
according to scleroderma classification criteria (LeRoy et al 1988),
confirmed independently by a second investigator who is unaware of the
assessment of the first investigator.
4. Has a baseline mRSS between 10 and 25 units, inclusive.
5. Has had onset of the first non-Raynaud's manifestation of SSc within
36 months of screening.
6. Is willing and able to comply with all aspects of the study; in
particular, has the ability to attend study visits and assessments at least
until the end of the double-blind treatment phase.
7. If a male subject is capable of reproduction or a female subject is of childbearing potential, he/she consents to practice sexual abstinence (Sexual abstinence refers to refraining from heterosexual intercourse during the entire period of risk associated with the study treatment. The
reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the subject.) or use another highly effective method of contraception (Pearl index < 1), such as combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation, progestogen only hormonal contraception associated with inhibition of ovulation, intrauterine device, intrauterine hormone releasing system, bilateral tubal occlusion or vasectomy in combination with a second
method of contraception, such as a condom or a cervical cap/diaphragm with spermicide, during the trial and for at least 6 months thereafter.
8. Has provided written informed consent.

Ogni soggetto deve soddisfare tutti i seguenti criteri per poter essere arruolato in questo studio (il criterio #7 deve essere soddisfatto anche per l’inclusione nella fase di estensione in aperto dello studio):
1. Essere un uomo o una donna di almeno 18 anni.
2. Soddisfare i criteri del 2013 per la classificazione della sclerosi sistemica (SSc) dell’American College of Rheumatology/European League Against Rheumatism.
3. Presentare coinvolgimento cutaneo diffuso (definito con fibrosi cutanea in almeno 1 dei seguenti siti: arti superiori, torace, addome o cosce) in base ai criteri di classificazione della sclerodermia (LeRoy 1988), confermato in maniera indipendente da un secondo sperimentatore che è all’oscuro della valutazione del primo sperimentatore.
4. Presentare un mRSS al basale compreso tra 10 e 25 unità (incluso).
5. Aver avuto l’insorgenza della prima manifestazione di sclerosi sistemica (SSc) non riconducibile al fenomeno di Raynaud nei 36 mesi antecedenti lo screening.
6. Essere disposto e in grado di rispettare tutti gli aspetti dello studio, in particolare potersi sottoporre alle visite e alle valutazioni dello studio almeno fino alla fine della fase di trattamento in doppio cieco.
7. Per i soggetti di sesso maschile in grado di procreare o per i soggetti di sesso femminile in età fertile, è necessario acconsentire alla pratica dell’astinenza sessuale (per astinenza sessuale si intende l’astensione dai rapporti eterosessuali durante l’intero periodo di rischio associato al trattamento in studio. L’affidabilità dell’astinenza sessuale deve essere valutata in relazione alla durata dello studio clinico e allo stile di vita abituale e preferito del soggetto) o all’utilizzo di un altro metodo contraccettivo altamente efficace (Indice di Pearl <1), come la contraccezione ormonale combinata (contenente estrogeno e progestinico) associata all’inibizione dell’ovulazione, la contraccezione ormonale a base di solo progestinico associata all’inibizione dell’ovulazione, un dispositivo intrauterino, un sistema intrauterino a rilascio ormonale, l’occlusione tubarica bilaterale o la vasectomia in combinazione con un secondo metodo contraccettivo, come un preservativo o un diaframma/cappuccio cervicale con spermicida durante lo studio e per almeno 6 mesi successivi.
8. Aver fornito consenso informato scritto.

E.4

Principal exclusion criteria

Subjects meeting any of the following criteria will be excluded from the
study:
1. Has abnormal hematological values prior to baseline (Visit 2).
2. Has abnormal liver function tests prior to baseline (Visit 2).
3. Has abnormal renal function tests prior to baseline (Visit 2).
4. Has any known renal crisis history.
5. Has cardiac abnormalities at screening (Visit 1).
6. Has any of the following lung criteria at screening (Visit 1):
• Forced vital capacity (FVC) <40% predicted
• Diffusing capacity of the lung for carbon monoxide (DLCO) <30%
predicted.
7. Has a previous diagnosis of severe pulmonary arterial hypertension
(PAH).
8. Has malabsorption requiring parenteral nutrition at screening (Visit
1).
9. Has had treatment with putative disease-modifying agents within 8
weeks of screening.
10. Has had treatment with cyclophosphamide within 26 weeks of
screening (Visit 1).
11. Has had treatment with rituximab within 52 weeks of screening
(Visit 1).
12. Has a known acute or uncontrolled chronic infection (eg, hepatitis B
or C, tuberculosis, or human immunodeficiency virus [HIV]).
13. History of hematopoietic stem cell transplantation or planned
hematopoietic stem cell transplantation within the next 12 months, or
total lymphoid irradiation.
14. Has any concurrent medical condition(s) that, in the opinion of the
investigator, may confound the results of the study.
15. Is pregnant or lactating.

I soggetti che soddisfano uno dei criteri seguenti saranno esclusi dallo studio:
1. Presentare valori ematici anomali prima del basale (Visita 2)
2. Presentare valori della funzionalità epatica anomali prima del basale (Visita 2).
3. Presentare valori della funzionalità renale anomali prima del basale (Visita 2).
4. Presentare una storia nota di crisi renali.
5. Presentare un' anomalia cardiaca allo screening (Visita 1).
6. Presentare uno dei seguenti criteri polmonari allo screening
(Visita 1):
• Capacità vitale forzata (FVC) <40% del valore previsto
• Capacità di diffusione del monossido di carbonio
(DLCO) <30% del valore previsto (emoglobina corretta)
7. Presentare una precedente diagnosi di ipertensione arteriosa polmonare grave (PAH).
8. Presentare malassorbimento richiedente nutrizione parenterale allo screening (Visita 1).
9. Essersi sottoposto a trattamento con possibili agenti modificanti la malattia nelle 8 settimane antecedenti lo screening.
10. Essersi sottoposto a trattamento con ciclofosfamide nelle 26 settimane antecedenti lo screening (Visita 1).
11. Essersi sottoposto a trattamento con rituximab nelle 52 settimane antecedenti lo screening (Visita 1).
12. Presentare una infezione cronica nota acuta o non controllata (ad esempio, epatite B o C, tubercolosi o virus dell’immunodeficienza umana [HIV]).
13. Storia di trapianto di cellule staminali emopoietiche o trapianto di cellule staminali emopoietiche programmato nei successivi 12 mesi, oppure irradiazione linfoide totale.
14. Presentare condizioni mediche che, secondo lo sperimentatore, possono alterare i risultati dello studio.
15. In caso di gravidanza o allattamento.

E.5 End points

E.5.1

Primary end point(s)

The primary efficacy endpoint is the absolute change in mRSS between baseline and Week 52 of the double-blind treatment phase.

L’endpoint primario di efficacia è rappresentato dalla variazione assoluta dell’mRSS tra il basale e la Settimana 52 della fase di trattamento in doppio cieco.

E.5.1.1

Timepoint(s) of evaluation of this end point

Settimana 52 della fase in doppio cieco.

Week 52 of double blind phase

E.5.2

Secondary end point(s)

• The absolute change in total score of the HAQ-DI between baseline and Week 52 of the double-blind treatment phase.
• The binary mRSS response defined as improvement of mRSS by at least 30% (relative) or 5 points (absolute) between baseline and Week 52 of the double-blind treatment phase.

• La variazione assoluta nel punteggio totale dell’indice di disabilità ottenuto attraverso il questionario di valutazione dello stato di salute (Health Assessment Questionnaire Disability Index, HAQ-DI) tra il basale e la Settimana 52 della fase di trattamento in doppio cieco.
• La risposta binaria dell’mRSS definita come miglioramento dell’mRSS per almeno il 30% (relativo) o 5 punti (assoluto) tra il basale e la Settimana 52 della fase di trattamento in doppio cieco.

E.5.2.1

Timepoint(s) of evaluation of this end point

Week 52 of double blind phase

Settimana 52 della fase in doppio cieco.

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

Yes

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

Quality of life

Qualità di vita.

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

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

Belgium

France

Germany

Hungary

Italy

Netherlands

Poland

Portugal

Romania

Switzerland

United Kingdom

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 is defined as the date on which all subjects have completed, or have had the opportunity to complete, the safety visit (Visit 13).

Viene definita fine dello studio la data in cui tutti i soggetti hanno completato o hanno avuto la possibilità di completare la visita di sicurezza (Visita 13).

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

Information not present in EudraCT

F.1.1.2

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

Information not present in EudraCT

F.1.1.3

Newborns (0-27 days)

Information not present in EudraCT

F.1.1.4

Infants and toddlers (28 days-23 months)

Information not present in EudraCT

F.1.1.5

Children (2-11years)

Information not present in EudraCT

F.1.1.6

Adolescents (12-17 years)

Information not present in EudraCT

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

134

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

Yes

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

100

F.4.2.2

In the whole clinical trial

148

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)

Patient's care after end of trial participation will be left at the doctor's discretion. Subjects who would like to remain on protocol-specified therapy after the open-label extension phase has been completed will
be permitted to continue treatment at the investigator's discretion, as provided for by the local country's regulatory mechanisms. Data collection during this phase will be limited to administration of protocol-specified therapy and SAE reporting.

L’assistenza al paziente dopo la partecipazione allo studio clinico è lasciata a discrezione del medico. I soggetti che vorranno proseguire la terapia indicata dal protocollo in seguito al completamento della fase di estensione in aperto potranno continuare il trattamento a discrezione dello sperimentatore, come previsto dalla normativa locale del Paese. La raccolta dei dati in questa fase sarà limitata alla somministrazione della terapia indicata dal protocollo e alla segnalazione dei SAE.

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

2016-07-27

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2016-07-19

P. End of Trial
P.	End of Trial Status	Temporarily Halted

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