Clinical Trials Register

Summary
EudraCT Number:	2016-000522-18
Sponsor's Protocol Code Number:	REDUCE-RISKincd-PBID-TRIAL
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2021-06-08
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

2016-000522-18

A.3

Full title of the trial

Risk-stratified randomized controlled trial in paediatric Crohn¿s Disease: Methotrexate versus Azathioprine or Adalimumab for mantaining remission in patients at low or at high risk for aggressive disease course, respectively ¿ a treatment strategy

Studio randomizzato controllato in pazienti pediatrici affetti da Malattia di Crohn stratificati per classi di rischio: Methotrexate a confronto con Azatioprina o Adalimumab per il mantenimento dello stato di remissione rispettivamente nei pazienti a basso o alto livello di rischio per decorso aggressiva della malattia.

A.3.1

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

Comparison of the effectiveness of treatment with an immunosuppressant medication over another or with respect to a biological drug in the maintenance of remission in children suffering from Crohn's Disease.

Confronto dell¿efficacia di una terapia con un farmaco immunosoppressore rispetto ad un altro o rispetto ad un farmaco biologico nel mantenimento della remissione nel bambino affetto da Malattia di Crohn.

A.3.2

Name or abbreviated title of the trial where available

REDUCE-RISKincd-PBID-TRIAL

A.4.1

Sponsor's protocol code number

REDUCE-RISKincd-PBID-TRIAL

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	UMBERTO I - POLICLINICO DI ROMA
B.1.3.4	Country	Italy
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	PIBDNet
B.4.2	Country	France
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Dipartimento di Pediatria
B.5.2	Functional name of contact point	Unit¿ funzionale del "contact-point
B.5.3	Address:
B.5.3.1	Street Address	viale Regina Elena 324
B.5.3.2	Town/ city	Roma
B.5.3.3	Post code	00161
B.5.3.4	Country	Italy
B.5.4	Telephone number	0649979324
B.5.5	Fax number	0649979324
B.5.6	E-mail	gastropediatria@uniroma1.it

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	METHOTREXATE - 20 MG/0.8 ML SOLUZIONE INIETTABILE 4 SIRINGHE PRERIEMPITE MONOUSO DA 1.25 ML
D.2.1.1.2	Name of the Marketing Authorisation holder	PFIZER 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	Methotrexate
D.3.2	Product code	N.A
D.3.4	Pharmaceutical form	Powder for solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intramuscular use
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.IMP: 2
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	HUMIRA - 40MG/0.8ML SOLUZ. INIETTABILE- USO SOTTOCUTANEO-FLACONCINO(VETRO) 0.8ML 2 ASTUCCI:1 FLACONCINO+1SIRINGA+1AGO+1ADATT.STERILE-2TAMPONI IMBEVUTI ALCOOL
D.2.1.1.2	Name of the Marketing Authorisation holder	ABBVIE LIMITED
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	Humira
D.3.2	Product code	N.A
D.3.4	Pharmaceutical form	Solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
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	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 3
D.1.2 and D.1.3	IMP Role	Comparator
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	AZATIOPRINA ASPEN - 50 COMPRESSE
D.2.1.1.2	Name of the Marketing Authorisation holder	ASPEN PHARMA TRADING LIMITED
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	Azatioprina
D.3.2	Product code	N.A
D.3.4	Pharmaceutical form	Film-coated tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.11 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.IMP: 4
D.1.2 and D.1.3	IMP Role	Comparator
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	PURINETHOL - 50 MG COMPRESSE25 COMPRESSE
D.2.1.1.2	Name of the Marketing Authorisation holder	ASPEN PHARMA TRADING LIMITED
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	Purinethol
D.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	Yes
D.3.7	Routes of administration for this IMP	Oral use
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

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Crohn Disease

Malattia di Crohn

E.1.1.1

Medical condition in easily understood language

Crohn's disease in children has a course more aggressive than in adults; therefore it requires equally aggressive therapy early.

La malattia di Crohn nel bambino ha un decorso pi¿ aggressivo che nell¿adulto; pertanto necessita di terapia altrettanto aggressive precocemente.

E.1.1.2

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

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	10042759
E.1.2	Term	Symptoms involving digestive system
E.1.2	System Organ Class	100000004856

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To compare the effectiveness of weekly subcutaneously administered MTX for maintaining relapse-free sustained steroid/EN-free 1-year
remission compared with:
¿ daily oral AZA/6MP in low risk paediatric CD
¿ subcutaneously administered adalimumab in high risk paediatric CD

Paragonare l¿efficacia della somministrazione settimanale sottocutanea del Methotrexate nel mantenimento dello stato di remissione libero da ricadute per almeno un anno senza impiego di steroidi o nutrizione enterale con:
-Somministrazione orale giornaliera di Azatioprina/6Mercaptopurina in pazienti pediatrici con Malatita di Crohn a basso rischio
-Somministrazione sottocutanea di Adalimumab in pazienti pediatrici con Malattia di Crohn ad alto rischio

E.2.2

Secondary objectives of the trial

Comparison between the two treatment arms per risk group (high risk or
low risk for aggressive disease evolution) (and inter-risk group analysis for MTX-
treated patients)

Paragonare i due bracci di trattamento stratificati per classi di rischio (alto rischio o basso rischio per evoluzione aggressiva di malattia)(ed analisi tra i gruppi di rischio per i pazienti trattati con Methotrexate).

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Children 6-17, with a new-onset CD diagnosed according to established criteria, requiring a steroid-based or EN based induction therapy
2. At initial diagnosis, wPCDAI >40
3. all wPCDAI scores (0-120) are possible at inclusion (patients in remission and patients with active disease)
4. Luminal active CD (B1) with or without B2 and/or B3 disease behavior
5. Initial exposure to 5-ASA and derivate is tolerated
6. Exposure to antibiotics is tolerated
7. No evidence of active or latent TB ie,
i) A negative QuantiFERON TB-Gold In-Tube test at screening, AND
ii) A chest radiograph (views as per local guidelines) taken at screening or within the 3 months prior to screening (with the report or films available for investigator review) without evidence of active or latent TB infection, AND
iii) No history of either untreated or inadequately treated latent or active TB infection
b) Previously treated for TB: i.e., if a subject has previously received an adequate course of therapy as per local standard of care for either latent TB or active TB. In these cases, no QuantiFERON TB-Gold In-Tube test (or equivalent assay) need be obtained, but a chest radiograph must be obtained if not done so within 3 months prior to screening.
8. If one of the following criteria is present, patients are allocated to the high risk group prior randomization:
• Complex fistulizing perianal disease
• Panenteric disease phenotype
• Severe growth impairment (height z-score <-2 percentiles) likely related to CD
• Significant hypoalbuminemia (12.5 despite 3 weeks of optimized induction therapy with steroids or EEN
• B2, B3 or B2B3 disease behavior
• Overall cumulative disease extend of =60 cm 8.
9. Informed and signed consent

- Bambini di età compresa tra 6 e 17 anni con nuova diagnosi di MC secondo i criteri riconosciuti, in trattamento con terapia di induzione con corticosteroidi o Nutrizione Enterale.
- Alla diagnosi malattia attiva (wPCDAI>40)
- Tutti i wPCDAI score (0-120) sono possibili al momento dell’inclusione
- MC e attiva a livello luminale con o senza presentazione di tipo B2 e/o B3.
- Concesso l’iniziale impiego di 5-ASA e derivati e l’esposizione ad antibiotici
- - Esclusa una infezione da Mycobacterium tubercolosis, ovvero:
a.Nessuna evidenza di tubercolosi attiva o latente,
b. Test QuantiFERON® TB Gold negativo al momento dello screening
c. Radiografia del torace alla visita di screening o entro i 3 mesi prima dello screening senza evidenza di infezione da TBC attiva o latente
d. Nessun precedente di una infezione da TB latente o attiva non trattata o non
adeguatamente trattata
- In caso di soggetti precedentemente trattati per la tubercolosi, oltre al test QuantiFERON® TB Gold negativo alla visita di screening, deve essere documentata una radiografia del torace negativa, entro non oltre 3 mesi prima dello screening
- In caso di pazienti nei quali viene riscontrata una TB latente durante lo screening (definita come un test QuantiFERON TB Gold in provetta [o test equivalente] positivo) in cui la tubercolosi attiva è stato esclusa, è possibile l’arruolamento solo se il trattamento profilattico per la TB è stato avviato per un minimo di 4 settimane prima della prima somministrazione del farmaco in studio.
Se uno dei seguenti criteri è presente il paziente viene allocato nel gruppo ad “alto rischio”:
1. Malattia perianale complessa
2. Malattia panenterica
3. Severo ritardo di crescita (height z-score <2) verosimilmente dovuto alla MC
4. Ipoalbuminemia significativa (<30g/L) elevata PCR (valori> ad almeno 2 volte la norma) o wPCDAI>12.5 nonostante 3 settimane di terapia di induzione ottimizzata con steroidi o nutrizione enterale
5. Malattia complicata (B2,B3 o B2B3)
6. estensione di malattia complessivamente >60 cm
- Accettazione della partecipazione allo studio con firma del consenso informato

E.4

Principal exclusion criteria

1. Patients with wPCDAI<42,5 at initial diagnosis
2. No induction therapy with steroids or enteral nutrition
3. Previous therapy with any IBD-related medications other than induction therapy
as detailed in this protocol (except 5-ASA).
4. Pregnancy or refusal to use contraceptives during the study period in pubertal
patients (both boys and girls) unless absolute abstinence (no sexual activity) is
confirmed at each study visit. Positive pregnancy testing throughout the study will
trigger prompt withdrawal of the patient from the study.
5. Lactating mothers
6. Children with perianal fistulising disease who require surgical therapy (drainage,
seton placement)
7. Patients homozygous for TPMT or those with TPMT activity <6 nmol/h/ml
erythrocytes or <9nmol 6MTG/g Hb/h), unless they qualify as high risk patients
8. Evidence of un-drained and un-controlled abscess/phlegmon
9. Contraindication to any drugs used in the trial (thiopurines, methotrexate or adalimumab)
10. Current or previous malignancy
11. Serious comorbidities (such as renal insufficiency, hepatitis, respiratory
insufficiency)
12. patients with suspected of sepsis, cytomegalovirus infection, listeriosis and other opportunistic infections
12. Infection with mycobacterium tuberculosis, hepatitis B or C, HIV
13. Moderate to severe heart failure (NYHA class III/IV)
14. Oral anticoagulant therapy, anti-malaria therapy
15. Live vaccines exposure (including yellow fever) less than 3 weeks prior inclusion

-Pazienti con wPCDAI<42,5 alla diagnosi
- Pazienti non trattati con steroidi o nutrizione enterale per l’induzione della remissione
- Precedente terapia con altri farmaci impiegati per IBD diversi da quelli precedenti (accetto 5-ASA)
- Gravidanza o rifiuto di usare i contraccettivi durante il periodo di studio in pazienti in età puberale (maschi e femmine) a meno che l'astinenza assoluta (nessuna attività sessuale) sia confermata ad ogni visita di studio. Un test di gravidanza positivo nel corso dello studio determinerà il ritiro del paziente dallo studio.
- Donne in allattamento
- Bambini con malattia anale fistolizzante che richiede prevalentemente trattamento della malattia perianale e malattia non luminale
- Pazienti in omozigosi per TPMT (o con attività TPMT molto bassa: <6 nmol/h/ml eritrociti o <9nmol 6MTG/g Hb/h)
- Evidenza di ascessi o flemmoni non drenati
Controindicazioni per uno dei farmaci usati nel trial (adalimumab, methotrexate o azatioprina)
- Attuali o precedenti tumori
- - Serie comorbidità (come insufficienza renale, epatite, insufficienza respiratoria) che possano interferire con la terapia medica
- Infezione attiva da Mycobacterium tubercolosis
- Pazienti con sospetta sepsi, infezione da cytomegalovirus, listeriosi o altre infezioni da agenti opportunisti
- Pazienti HIV, HCV e HBV
- Insufficienza cardiaca moderata-severa (classe NYHA III/IV)
- Terapia anticoagulante orale o anti-amalaria
- Esposizione a vaccini vivi nelle 3 settimane precedenti all’ingresso nello studio

E.5 End points

E.5.1

Primary end point(s)

Comparing the two treatment arms per group
for sustained steroid/EN-free remission at Month 12, where sustained remission is defined as wPCDAI S12.5 and CRP S1,5-fold upper limit
without a relapse or need for EEN/steroids since week 12.

Paragonare l’efficacia dei due gruppi di trattamento per gruppo nel mantenimento di una remissione prolungata a 12 mesi senza ricorso a terapia steroidea e/o nutrizione enterale, dove per remissione prolungata si intende un wPCDAI=12.5 e una PCR=1,5 volte il limite massimo senza recidive o bisogno di steroidi/nutrizione enterale dalla settimana 12

E.5.1.1

Timepoint(s) of evaluation of this end point

At months 12

12 mesi

E.5.2

Secondary end point(s)

Comparing the 2 treatment arms per risk group and comparing methotrexate treatment between high and low risk group

Paragonare i due gruppi di trattamento stratificati per rischio di malattia severa e valutare l¿efficacia del methotrexate nei pazienti ad ad alto e basso rischio di malattia severa.

E.5.2.1

Timepoint(s) of evaluation of this end point

12 months

12 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

Yes

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

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

Yes

E.8.1.7

Other

Yes

E.8.1.7.1

Other trial design description

Assicurare il cieco in un secondo medico che valuter¿ il wPCDAI, PCDAI e PGA ad ogni visita

To ensure blinding of a 2nd physician who scores wPCDAI, PCDAI & PGA at each visit

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

Belgium

France

Germany

Hungary

Italy

Netherlands

Poland

United Kingdom

Czechia

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

At the end of enrolement, treatment and monitoring

Fine del periodo di arruolamento, trattamento e monitoraggio

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)

F.1.1.5

Children (2-11years)

Yes

F.1.1.5.1

Number of subjects for this age range:

156

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

156

F.1.2

Adults (18-64 years)

F.1.3

Elderly (>=65 years)

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

300

F.4.2.2

In the whole clinical trial

312

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 will be followed as per the regular treatment of their disease.

I pazienti continueranno ad essere seguiti regolarmente per le esigenze legate alla loro patologia

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

2017-02-24

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2017-02-15

P. End of Trial
P.	End of Trial Status	Ongoing

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