Clinical Trials Register

Summary
EudraCT Number:	2012-002030-37
Sponsor's Protocol Code Number:	A7281009
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:	2013-04-02
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

2012-002030-37

A.3

Full title of the trial

A DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED, PARALLEL, DOSE-RANGING STUDY TO EVALUATE THE EFFICACY AND SAFETY OF PF-00547659 IN PATIENTS WITH MODERATE TO SEVERE ULCERATIVE COLITIS (TURANDOT)

UNO STUDIO DOSE-RANGING, IN DOPPIO CIECO, RANDOMIZZATO, CONTROLLATO CON PLACEBO, A GRUPPI PARALLELI, PER VALUTARE LA SICUREZZA E L’EFFICACIA DI PF-00547659 IN SOGGETTI CON COLITE ULCEROSA DI GRADO DA MODERATO A GRAVE (TURANDOT)

A.3.1

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

Study to test whether PF-00547659 is safe and improves symptoms in patients with UC

A.3.2

Name or abbreviated title of the trial where available

TURANDOT

A.4.1

Sponsor's protocol code number

A7281009

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	Pfizer Inc, 235 East 42nd Street, New York, NY10017, United States
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	Pfizer 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	Pfizer Inc
B.5.2	Functional name of contact point	Clinical Trials.gov Call Center
B.5.3	Address:
B.5.3.1	Street Address	235 East 42nd Street
B.5.3.2	Town/ city	New York
B.5.3.3	Post code	NY10017
B.5.3.4	Country	United States
B.5.4	Telephone number	0018007181021
B.5.5	Fax number	0013037391119
B.5.6	E-mail	clinicaltrials.gov_inquiries@pfizer.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.2	Product code	PF-00547659
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	Subcutaneous 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	PF-00547659
D.3.9.3	Other descriptive name	Anti-MAdCAM antibody
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	75
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	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	Solution for injection
D.8.4	Route of administration of the placebo	Subcutaneous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Ulcerative Colitis (UC)

Colite Ulcerosa (CU)

E.1.1.1

Medical condition in easily understood language

Ulcerative Colitis (UC)

Colite Ulcerosa

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	15.1
E.1.2	Level	LLT
E.1.2	Classification code	10045365
E.1.2	Term	Ulcerative colitis
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 characterize the dose-response and efficacy of PF-00547659 in inducing clinical remission based upon Mayo Score in subjects with moderate to severe ulcerative colitis.

Caratterizzare la relazione dose-risposta e l'efficacia del PF-
00547659 nell'indurre la remissione clinica in base al punteggio Mayo in soggetti con colite ulcerosa da moderata a grave.

E.2.2

Secondary objectives of the trial

Secondary Objectives
• To evaluate the induction of clinical response based upon Mayo Score in subjects with moderate to severe ulcerative colitis.
• To evaluate the safety and tolerability of PF-00547659 in subjects with moderate to severe ulcerative colitis.
• To assess the PF-00547659 concentration time course.
• To evaluate the effect of induction treatment of PF-00547659 on quality-of-life in subjects with moderately to severely active ulcerative colitis.

Exploratory Objective
To explore the relationship between PK, PD, and clinical endpoints.

Valutare l'induzione della risposta clinica in base al punteggio Mayo in soggetti
con colite ulcerosa da moderata a grave.
• Valutare la sicurezza e la tollerabilità del PF-00547659 in soggetti con colite
ulcerosa da moderata a grave.
• Valutare il profilo di concentrazione-tempo del PF-00547659.
• Valutare l'effetto del trattamento di induzione del PF-00547659 sulle misure di
qualità della vita in soggetti con colite ulcerosa da moderata a grave.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Evidence of a personally signed and dated informed consent document indicating that the subject (or a legal representative) has been informed of all pertinent aspects of the study.
2. Subjects are willing and able to participate in the study, complete subject assessments, attend scheduled clinic visits, and comply with all protocol requirements as evidenced by written informed consent.
3. Male and/or female subjects between the ages of ≥18 and ≤65 years at the time of informed consent.
4. A diagnosis of UC for ≥3 months. A biopsy report must be available to confirm the histological diagnosis in the subject’s source documentation. In addition, a report documenting disease duration and extent of disease (e.g., proctosigmoiditis, left-sided colitis and pancolitis) based upon prior colonoscopy must also be available in source documentation. NOTE: A colonoscopy with biopsy will need to be performed, if this documentation is not available.
5. Must have a flexible sigmoidoscopy (or colonoscopy, if preferred) indicative of active UC (Mayo endoscopic subscore of at least 2) during screening after all other inclusion criteria have been met.
6. Must have active disease beyond the rectum (>15 cm of active disease at the Screening endoscopy).
7. Must have active UC with a Total Mayo Score of 6 to 12 points and moderate to severe disease on endoscopy (Mayo endoscopic subscore of at least 2).
8. Must have failed or been intolerant of at least one conventional therapy such as mesalamine, steroids and immunosuppressants (AZA, 6-MP or MTX) or anti-TNF. Subjects will be stratified based upon prior experience with anti-TNFs (naïve or experienced). Documentation of the current and prior UC treatment (ie, steroids and immunosuppressants) will be captured for additional analyses and must be provided in the source documentation.
9. Male and female subjects of childbearing potential must agree to use a highly effective method of contraception (defined at the time of signing the informed consent) throughout the study and through the conclusion of subject participation. A subject is of childbearing potential if, in the opinion of the investigator, he/she is biologically capable of having children and is sexually active.
• Women of childbearing potential (WOCBP) must have a negative serum pregnancy test result at screening and a negative urine pregnancy test result at baseline. WOCBP are defined as women who are biologically capable of becoming pregnant, including women who are using contraceptives or whose sexual partners are either sterile or using contraceptives.
• Women of non-childbearing potential (WONCBP) do not require a serum and urine pregnancy test and must meet at least one of the following criteria:
• Have undergone hysterectomy or bilateral oophorectomy;
• Have medically confirmed ovarian failure or
• Are medically confirmed to be post-menopausal (cessation of regular menses for at least 12 consecutive months with no alternative pathological or physiological cause; laboratory confirmation of FSH level may be indicated if subject has history of amenorrhea for ≥ 52 weeks).

Evidenza di un consenso informato firmato personalmente che indichi che il
soggetto (o il suo rappresentante legale) sia stato informato di tutti gli aspetti
pertinenti dello studio.
2. I soggetti siano disposti ed in grado di partecipare allo studio, completino le
valutazioni dei soggetti, vadano alle visite previste in ospedale e siano
complianti con tutti i requisiti del protocollo come evidenziato dal consenso
informato scritto.
3. Uomini e/o donne con un’età ≥18 e ≤65 anni al momento della firma del
consenso informato.
4. Una diagnosi di CU ≥3 mesi. Nei documenti originali del soggetto deve
essere presente un referto di una biopsia per confermare una diagnosi istologica. In aggiunta, un referto sulla base di una prima colonscopia che
documenti la durata della patologia e la sua estensione (per es.
proctosigmoidite, colite del lato sinistro e pancolite) deve essere presente nei source
document del paziente. NOTA:Se questa documentazione non è disponibile,
dovrà essere effettuata una colonscopia con biopsia.
5. Deve avere una sigmoidoscopia flessibile (o colonscopia, se preferita)
indicativa di CU attiva (subpunteggio endoscopico di Mayo di almeno 2)
durante lo screening o dopo che tutti gli altri criteri di inclusione siano stati
rispettati.
6. Deve avere una patologia attiva al di là del retto (> 15 cm di patologia
attiva all’endoscopia di Screening)
7. Deve avere una CU con un punteggio Mayo Totale da 6 a 12 punti e una
patologia da moderata a severa all’endoscopia (subpunteggio endoscopico di
Mayo di almeno 2).
8. Deve aver fallito o essere stato intollerante ad almento una delle terapie
convenzionali come con mesalamina, steroidi e immunosoppressori (AZA, 6-
MP o MTX) o anti-TNF. I soggetti saranno stratificati sulla base della
precedente esperienza con anti-TNF (naïve o experienced). Nei documenti
originali del paziente deve essere fornita la documentazione dell’attuale e
precedente trattamento per la CU (per es. steroidi e immunosoppressori).
9. Soggetti maschi e femmine in grado di generare figli devono essere
d’accordo ad utilizzare un metodo di contraccezione altamente efficace
(definito al momento della firma del consenso informato) per tutta la durata
dello studio e alla conclusione della partecipazione del soggetto. Un soggetto
è definito in grado di avere figli se, nell’opinione dello sperimentatore,
egli/ella è biologicamente capace di avere bambini ed è sessualmente attivo.
- Le donne in grado di avere figli (WOCBP) devono avere un test di
gravidanza sul siero negativo allo screening e un test di gravidanza
sulle urine con risultato negativo al basale. WOCBP sono definite le
donne che sono biologicamente in grado di divenire gravide, incluse le donne che stanno usando contraccettivi o i cui partner sessuali sino o sterili o stiano usando contraccettivi.
- Le donne non potenzialmente in grado di avere figli non richiedono i
test sul siero e urine e devono avere almeno uno dei seguenti
criteri:
- essere state sottoposte a isterectomia o ovariectomia bilaterale;
- Avere conferma medica di insufficienza ovarica o
-Essere in menopausa confermata a livello medico (cessazione di un ciclo mensile regolare per almeno 12 mesi consecutivi con nessuna causa patologica o fisiologica; conferma di laboratorio di livelli di FSH potrebbe essere indicata se il soggetto ha una storia di amenorrea di ≥ 52 settimane).

E.4

Principal exclusion criteria

1. Subjects with a diagnosis of indeterminate colitis or Crohn’s disease. Subjects with clinical findings suggestive of Crohn’s disease, e.g., fistulae or granulomas on biopsy are also excluded.
2. Subjects with an imminent need for or planned surgery.
3. Subjects with colonic dysplasia or neoplasia.
4. Subjects with toxic megacolon.
5. Subjects with primary sclerosing cholangitis.
6. Subjects with known colonic stricture.
7. Subjects with a history of colonic or small bowel obstruction or resection.
8. Abnormal findings on the chest x-ray film performed routinely before initiating a new biologic therapy, such as presence of tuberculosis (TB), general infections, heart failure, or malignancy. (Chest x-ray examination may be performed up to 12 weeks prior to study entry (screening). Documentation of the official reading must be located and available in the source documentation).
9. Any history or current evidence of latent or active tuberculosis infection, evidence of prior or currently active tuberculosis by chest radiography, residing with or frequent close contact with individual(s) with active tuberculosis. Subjects who have a positive Mantoux (PPD) tuberculin skin test or a positive Interferon Gamma Release Assay (IGRA to be tested at the site’s local lab) during screening or within 12 weeks prior to randomization. The following are acceptable assays: QuantiFERON® - TB Gold test (QFT-G), QuantiFERON® - TB Gold In-Tube test (QFT-GIT) and T-SPOT®-TB test) during screening or within 12 weeks prior to screening.
10. Presence of active enteric infections (positive stool culture and sensitivity).
11. Pre-existing demyelinating disorder such as Multiple Sclerosis or new onset seizures, unexplained sensory motor, or cognitive behavioral, neurological deficits, or significant abnormalities noted during screening.
12. Known history of HIV based on documented history with positive serological test, or positive HIV serologic test at screening, tested at the site’s local lab. (Note: a documented negative HIV test within one year of screening is acceptable and does not need to be repeated).
13. Presence of transplanted organ. Skin grafts to treat pyoderma gangrenosum are allowed.
14. Significant concurrent medical condition at the time of screening or baseline visit, including, but not limited to, the following:
• Any major illness/condition or evidence of an unstable clinical condition that, in the investigator’s judgement will substantially increase the risk to the subject if he or she participates in the study.
• Cancer or history of cancer or lymphoproliferative disease within the previous 5 years (other than resected cutaneous basal cell or squamous cell carcinoma that has been treated with no evidence of recurrence).
• Right or left heart failure including symptomatic diastolic dysfunction or unexplained elevation of troponin I (>0.05 ng/mL). Subjects with screening or baseline value of NTproBNP >124 pg/mL must have an echocardiogram and cardiology consult that excludes right or left heart failure.
• Acute coronary syndrome and any history of significant cerebrovascular disease within 24 weeks before screening.
15. Any major elective surgery scheduled to occur during the study.
16. Prior evidence of liver injury or toxicity due to methotrexate.
17. Abnormality in hematology and/or chemistry profiles during screening:
18. IV or IM (parenteral) steroids are excluded. If receiving corticosteroid treatment orally, subjects must have been on a stable dose for at least 1 week prior to baseline and must be willing to maintain the stable dose regimen through Week 12.
19. If receiving 6-MP or AZA (≤2.5 mg/kg) or MTX subjects must have been on a stable dose for at least 8 weeks prior to screening.
20. If receiving 5-aminosalicylic acid (5-ASA) treatment orally, subjects must be on a stable dose for at least 2 weeks prior to Mayo Score screening procedures.
21. Other biologics including any anti-TNFs within 6 weeks from baseline/randomization.
22. Known use of prior therpay of natalizumab, vedolizumab, AMG-181 or rhuMAb b7.
23. Treatment with another investigational procedure, product (s) such as immunosuppressants used in transplantation or live (attenuated) vaccine within 30 days before the baseline visit.
24. Inability to complete any of the neurological assessments without a clear explanation.
25. Current or history within 2 years of serious psychiatric disease or alcohol or drug abuse.
26. Pregnant or breastfeeding women.
27. Subjects who are investigational site staff members or relatives of those site staff members or subjects who are Pfizer employees directly involved in the conduct of the trial.

1. Soggetti con una diagnosi di colite indeterminata o morbo di Crohn.
2. Soggetti con evidenza clinica suggestiva di morbo di Crohn, per es. anche
fistule e o granulomi siano esclusi alla biopsia.
2. Soggetti con imminente necessità o chirurgia programmata.
3. Soggetti con displasia o neoplasia del colon.
4. Soggetti con megacolon tossico
5. Soggetti con colangite primaria sclerosante
6. Soggetti con stenosi del colon nota
7. Soggetti con una storia di ostruzione o resezione del colon o del piccolo intestino
8. Anomalie polmonari ai raggi X effettuate routinariamente prima di iniziare
una terapia biologica, come presenza di tubercolosi (TB). Infezioni generali,
insufficienza cardiaca o malignità. (I raggi x al polmone potrebbero essere
effettuati fino a 12 settimane prima dell’entrata nello studio (screening). Nei
source document deve essere presente e disponibile documentazione della
lettura ufficiale.
9. Qualsiasi storia o evidenza corrente di infezione di tubercolosi latente o
attiva, evidenza, attraverso raggi X, di precedente o corrente tubercolosi
attiva, residente o con contatti frequenti con individui con tubercolosi attiva.
Soggetti che hanno un test cutaneo alla tubercolina, Mantoux (PPD) positivo o
un saggio di Rilascio di Interferone Gamma positivo (IGRA da testare presso il
laboratorio locale del centro) durante lo screening o entro 12 settimane prima
della randomizzazione. I seguenti saggi sono accettabili: QuantiFERON® - TBGold test (QFT-G), QuantiFERON® - TB Gold In-Tube test (QFT-GIT) e T-SPOT durante lo screeing o 12 settimane prima dello screening.
10. Presenza di infezione enterica attiva (cultura delle feci positiva e
sensibilità).
11. Disordini de-mielinanti pre-esistenti come Sclerosi Multipla o insorgenza di nuove crisi, inspiegabile deficit motorio sensoriale o comportamento cognitivo, neurologico o anomalie significative notate durante lo screening.
12. Storia di HIV nota sulla base della storia della documentazione con test sierologico positivo, o test HIV sierologico positivo allo screening, e stato ad un laboratorio locale del centro. (Nota: un test HIV negativo documentato entro un anno dallo screening è accettabile e non necessita di esser
ripetuto).

E.5 End points

E.5.1

Primary end point(s)

• Proportion of subjects in Clinical Remission at Week 12 as defined by a Total Mayo Score of 2 points or lower with no individual subscore exceeding 1 point and rectal bleed subscore of 0 or 1.

Proporzione di soggetti in
Remissione Clinica alla Settimana 12 come definita da un punteggio totale
Mayo inferiore o uguale a 2 punti, con nessun sottopunteggio individuale
maggiore di 1 punto, e un sottopunteggio di sanguinamento rettale pari a 0 o 1

E.5.1.1

Timepoint(s) of evaluation of this end point

Week 12

12 Settimane

E.5.2

Secondary end point(s)

Secondary Endpoints
• Proportion of subjects with a Clinical Response at Week 12 with a decrease from baseline in Total Mayo Score by at least 3 points and at least 30% decrease in subscore for rectal bleeding of at least 1 point or absolute subscore of 0 or 1.
• Proportion of subjects with mucosal healing at Week 12 (defined as absolute Mayo subscore for endoscopy of 0 or 1).
• Proportion of subjects with change from baseline in partial Mayo Score of ≤ 2 with no individual subscore >1 at Weeks 4, 8, 12.
• Change from baseline in Mayo Score and in individual Mayo subscores at Weeks 4, 8, and 12.
• Change from baseline in fecal calprotectin at Weeks 4, 8, 12.
• Change from baseline in hsCRP at Weeks 4, 8, 12.
• Change over time in the mean IBDQ domain and total scores from baseline to Week 12.
• Proportion of subjects with an IBDQ total score of ≥170 at Week 12.
• Safety and tolerability evaluated by the frequency of AEs, SAEs, AEs leading to discontinuation of study treatment.
• PF-00547659 concentration-time profile.

Exploratory Endpoints
• Change from baseline in Simple Clinical Colitis Activity Index (SCCAI) at Weeks 4, 8, 12.
• Percentage of subjects with a decrease in the SCCAI scores of ≥3 points at Weeks 4 and 12.

• Percentage of subjects with clinical remission at Weeks 4, 8, and 12 defined as a total SCCAI score of < 2 points.
• Laboratory tests, electrocardiograms (ECGs) and vital signs will be summarized.

Health Outcomes
• Proportion of subjects with a clinically meaningful change in the IBDQ total score (>/= 16 points) at Week 12.
• Changes over time in the European Quality of Life 5 Dimensions questionnaire (EQ 5D)(TM) and EQ 5D visual analog scale (VAS) scores from baseline to Week 12.

Proporzione di soggetti con Risposta Clinica alla Settimana 12 con una
diminuzione rispetto al basale del punteggio totale Mayo di almeno 3
punti e diminuzione di almeno il 30% del sottopunteggio per il
sanguinamento rettale di almeno 1 punto o sottopunteggio assoluto
pari a 0 o 1.
 Proporzione di soggetti con guarigione mucosale alla Settimana 12
(definita come sottopunteggio Mayo assoluto per l'endoscopia pari a 0 o
1).
 Proporzione di soggetti con un cambiamento rispetto al basale del
punteggio Mayo parziale di ≤ 2 con nessun sottopunteggio individuale >1 alle Settimane 4, 8, 12.
 Cambiamento rispetto al basale del punteggio Mayo e dei sottopunteggi
Mayo individuali alle Settimane 4, 8 e 12.
 Cambiamento rispetto al basale della calprotectina fecale alle Settimane
4, 8 e 12.
 Cambiamento rispetto al basale della hsCRP alle Settimane 4, 8, 12.
 Cambiamento nel tempo rispetto al basale dei punteggi IBDQ medi per
dominio e totali alla Settimana 12.
 Proporzione di soggetti con un punteggio IBDQ totale di 170 alla
Settimana 12.
 Sicurezza e tollerabilità valutate tramite la frequenza di AE, SAE e AE
che conducono all'interruzione del trattamento dello studio.
 Profilo concentrazione-tempo del PF-00547659.
Endpoint esplorativi
 Cambiamento rispetto al basale dell'indice semplice di attività clinica
della colite (Simple Clinical Colitis Activity Index, SCCAI) alle Settimane
4, 8, 12.
 Percentuale di soggetti con una diminuzione dei punteggi SCCAI di ≥ 3
punti alle Settimane 4 e 12.
 Percentuale di soggetti con remissione clinica alle Settimane 4, 8, e 12
definita come punteggio totale SCCAI di < 2 punti.
 Test di laboratorio, elettrocardiogrammi (ECG) e segni vitali saranno
riassunti.
Effetti sulla salute
 Proporzione di soggetti con un cambiamento clinicamente significativo
nel punteggio totale IBDQ (≥16 punti) alla Settimana 12.
 Cambiamenti nel tempo dei punteggi del questionario europeo sulla
qualità della vita in 5 dimensioni (European Quality of Life 5 Dimensions
questionnaire, EQ5D)™ e della Scala analogica visiva (VAS) dello EQ5D
rispetto al basale alla Settimana 12.

E.5.2.1

Timepoint(s) of evaluation of this end point

Weeks 4, 8, and 12

Settimane 4, 8, 12

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

Yes

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)

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

105

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Australia

Austria

Belgium

Brazil

Bulgaria

Canada

Croatia

Czech Republic

Denmark

France

Germany

Hungary

Israel

Italy

Korea, Republic of

Netherlands

New Zealand

Norway

Poland

Russian Federation

Serbia

Slovakia

South Africa

Spain

Sweden

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

Last subject last visit

LSLV

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:

300

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

170

F.4.2.2

In the whole clinical trial

300

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)

As per protocol

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

2013-04-20

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2013-04-03

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2016-02-04

Select Country:	Select Age Range:
Select Trial Status:	Select Trial Phase:
Select Gender:
Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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