Clinical Trials Register

Summary
EudraCT Number:	2019-004630-42
Sponsor's Protocol Code Number:	TAS-120-301
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2021-06-04
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

2019-004630-42

A.3

Full title of the trial

A Phase 3, Open-Label, Randomized Study of Futibatinib Versus
Gemcitabine-Cisplatin Chemotherapy as First-Line Treatment of Patients
with Advanced Cholangiocarcinoma Harboring FGFR2 Gene
Rearrangements

Studio di fase 3, in aperto, randomizzato su futibatinib rispetto a chemioterapia con gemcitabina-cisplatino come trattamento di prima linea per pazienti con colangiocarcinoma avanzato che presenta riarrangiamenti del gene FGFR2

A.3.1

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

A.3.2

Name or abbreviated title of the trial where available

FOENIX-CCA3

A.4.1

Sponsor's protocol code number

TAS-120-301

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT04093362

A.5.4

Other Identifiers

Name:

IND Number

Number:

121062

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	TAIHO ONCOLOGY 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	Taiho Oncology 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	Taiho Oncology, Inc
B.5.2	Functional name of contact point	Senior Study Manager
B.5.3	Address:
B.5.3.1	Street Address	101 Carnegie Center, Suite 101
B.5.3.2	Town/ city	Princeton
B.5.3.3	Post code	NJ 08540
B.5.3.4	Country	United States
B.5.4	Telephone number	+16092507333
B.5.5	Fax number	+16097505300
B.5.6	E-mail	jdean@taihooncology.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Comparator
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	Cisplatin 1 mg/mL Concentrate for Solution for Infusion
D.2.1.1.2	Name of the Marketing Authorisation holder	Accord Healthcare Limited
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	Cisplatin 1 mg/mL Concentrate for Solution for Infusion (or generic equivalent)
D.3.2	Product code	[NA]
D.3.4	Pharmaceutical form	Concentrate for solution for injection/infusion
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	Cisplatin
D.3.9.1	CAS number	15663-27-1
D.3.9.2	Current sponsor code	NA
D.3.9.3	Other descriptive name	Cisplatin
D.3.9.4	EV Substance Code	SUB07483MIG
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	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	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	Gemcitabine 38 mg/ml Concentrate for Solution for Infusion
D.2.1.1.2	Name of the Marketing Authorisation holder	STADApharm GmbH
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	Gemcitabine 38 mg/ml Concentrate for Solution for Infusion (or generic equivalent)
D.3.2	Product code	[NA]
D.3.4	Pharmaceutical form	Concentrate for solution for injection/infusion
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	Gemcitabine
D.3.9.1	CAS number	95058-81-4
D.3.9.2	Current sponsor code	NA
D.3.9.4	EV Substance Code	SUB07892MIG
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	38
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: 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	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	EU/3/19/2146
D.3 Description of the IMP
D.3.1	Product name	Futibatinib
D.3.2	Product code	[TAS-120]
D.3.4	Pharmaceutical form	Coated tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	FUTIBATINIB
D.3.9.1	CAS number	1448169-71-8
D.3.9.2	Current sponsor code	TAS-120, TAS-06-02985
D.3.9.3	Other descriptive name	FUTIBATINIB
D.3.9.4	EV Substance Code	SUB194664
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	4
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

Advanced solid tumors

Tumori solidi avanzati

E.1.1.1

Medical condition in easily understood language

Advanced solid tumors

Tumori solidi avanzati

E.1.1.2

Therapeutic area

Diseases [C] - Cancer [C04]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	LLT
E.1.2	Classification code	10065147
E.1.2	Term	Malignant solid tumor
E.1.2	System Organ Class	100000004864

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

Progression-free survival (PFS) by independent central review (ICR), based on Response Evaluation Criteria in Solid Tumors, Version 1.1 (RECIST 1.1)

Sopravvivenza libera da progressione (PFS) mediante revisione centrale indipendente (ICR), basata sui criteri di valutazione della risposta nei tumori solidi, versione 1.1 (RECIST 1.1)

E.2.2

Secondary objectives of the trial

• Objective response rate (ORR)
• Disease control rate (DCR)
• Overall survival (OS)
• PFS according to Investigator assessment of radiologic images
• Safety and tolerability

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Provide written informed consent.
2. Is = 18 years of age (or meets the country's regulatory definition for legal adult age).
3. The patient has histologically confirmed, locally advanced, or metastatic, or recurrent unresectable iCCA harboring FGFR2 gene rearrangements based on testing performed by the designated central laboratory.
4. Patient has radiographically measurable disease per RECIST 1.1.
5. Patients who have received treatment for locally advanced disease (for example, trans-arterial chemoembolization, selective internal radiation therapy, external beam radiation) must have evidence of radiographic progression with measurable disease outside the previously-treated lesions.
6. Eastern Cooperative Oncology Group performance status (ECOG PS) 0 or 1.
7. Adequate organ function as defined by the following criteria:
• Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) = 3.0 × upper limit of normal (ULN); if liver function abnormalities are due to underlying liver metastasis, AST and ALT = 5 × ULN.
• Total bilirubin = 1.5 × ULN, or = 3.0 × ULN for patients with Gilbert's syndrome.
• White blood cell (WBC) count = 2000/mm3 (= 2.0 × 109/L)
• Absolute neutrophil count (ANC) = 1000/mm3 (ie, = 1.0 × 109/L)
• Platelet count = 100,000/mm3 (= 100 × 109/L)
• Hemoglobin = 9.0 g/dL
• Phosphorus = 1.5 × ULN
• Creatinine clearance = 60 mL/min
8. Women of child-bearing potential (WOCBP) must have a negative serum pregnancy test within 7 days prior to administration of the first dose of study medication. Female patients are not considered to be of child bearing potential if they have a history of hysterectomy or are post menopausal defined as no menses for 12 months without an alternative medical cause. Both males and females of reproductive potential must agree to use effective birth control during the study prior to the first dose and for 3 months after the last dose of study therapy, or according to the local label for each study therapy (whichever is longer).
9. Willing and able to comply with scheduled visits and study procedures.

1. Fornire il consenso informato per iscritto.
2. Avere un’età = 18 anni (o soddisfare la definizione normativa del Paese per l’età adulta legale).
3. Essere affetto da iCCA non resecabile, localmente avanzato, metastatico o ricorrente, confermato istologicamente, che presenta riarrangiamenti del gene FGFR2 sulla base di test eseguiti dal laboratorio centrale designato.
4. Avere una malattia misurabile radiograficamente secondo RECIST 1.1.
5. I pazienti che hanno ricevuto un trattamento per la malattia localmente avanzata (ad es. chemioembolizzazione trans-arteriosa, radioterapia interna selettiva, radioterapia a fasci esterni) devono presentare evidenza di progressione radiografica con malattia misurabile al di fuori delle lesioni precedentemente trattate.
6. Stato di validità secondo l’Eastern Cooperative Oncology Group (ECOG-PS) da 0 a 1.
7. Adeguata funzionalità d’organo, come definita dai seguenti criteri:
• Aspartato aminotransferasi (AST) e alanina aminotransferasi (ALT) = 3,0 volte il limite superiore di normalità (ULN); se le anomalie della funzionalità epatica sono dovute a metastasi epatiche di base, AST e ALT = 5 volte l'ULN.
• Bilirubina totale = 1,5 volte l'ULN, o = 3,0 volte l'ULN per i pazienti con sindrome di Gilbert.
• Conta dei leucociti (WBC) = 2000/mm3 (= 2,0 × 109/L)
• Conta assoluta dei neutrofili (ANC) = 1000/mm3 (ossia = 1,0 × 109/L in unità internazionali [UI])
• Conta piastrinica = 100.000/mm3 (UI: = 100 × 109/L)
• Emoglobina = 9,0 g/dL
• Fosforo = 1,5 volte l'ULN
• Clearance della creatinina = 60 mL/min
8. Le donne in grado di procreare devono presentare un test di gravidanza su siero negativo nei 7 giorni prima della somministrazione della prima dose del farmaco di studio. Le donne non vengono considerate in grado di procreare se sono state sottoposte a una precedente isterectomia o sono in post-menopausa, definita come assenza di mestruazioni da 12 mesi senza una causa medica alternativa. Sia gli uomini sia le donne in grado di procreare devono acconsentire a utilizzare un metodo contraccettivo efficace durante lo studio prima dell’assunzione della prima dose e per 3 mesi dopo l’assunzione dell’ultima dose della terapia, o secondo l'etichetta locale per ogni terapia di studio (se più lunga) .
9. Disponibilità e capacità di rispettare le visite programmate e le procedure dello studio.

E.4

Principal exclusion criteria

1. Patient has received previous systemic anticancer therapy
• Patients receiving adjuvant or neoadjuvant treatment and completed = 6 months prior to randomization are eligible
2. Patient has mixed hepatocellular carcinoma – iCCA disease.
3. History and/or current evidence of any of the following disorders:
• Non-tumor related alteration of calcium-phosphorus homeostasis that is clinically significant in the opinion of the Investigator.
• Ectopic mineralization/calcification, including but not limited to soft tissue, kidneys, intestine, or myocardia and lung, considered clinically significant in the opinion of the Investigator.
• Retinal disorder confirmed by retinal examination and considered clinically significant in the opinion of the ophthalmologist.
4. History or current evidence of uncontrolled ventricular arrhythmias
5. Fridericia's corrected QT interval (QTcF) > 470 ms on ECG conducted during Screening.
6. Treatment with any of the following within the specified time frame prior to the first dose of study therapy, or failure to recover from side effects of these prior therapies:
• Major surgery within the previous 4 weeks (the surgical incision should be fully healed prior to the first dose of study therapy).
• Radiotherapy (any dose) for extended field within 4 weeks or limited field radiotherapy within 2 weeks, and/or has not recovered from acute impact of radiotherapy.
• Patients with locoregional therapy, e.g. transarterial chemoembolization, selective internal radiotherapy, or ablation within 4 weeks.
• Any history of liver transplant.
7. A serious illness or medical condition(s) including, but not limited to, the following:
• Brain metastases that are untreated or clinically or radiologically unstable (that is, have been stable for <1 month).
• Known acute systemic infection.
• Myocardial infarction, severe/unstable angina, or symptomatic congestive heart failure within the previous 6 months.
• Chronic nausea, vomiting, or diarrhea considered to be clinically significant in the opinion of the Investigator.
• Congenital long QT syndrome, or any known history of torsade de pointes, or family history of unexplained sudden death.
• Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that in the judgment of the Investigator would make the patient inappropriate for entry into this study.
8. Patients with a history of another primary malignancy whose natural history or treatment has the potential to interfere with the safety or efficacy assessment of the investigational regimen in the opinion of the Investigator.
9. Pregnant or breast-feeding female
10. The patient is unable to take oral medication.

1. Paziente sottoposto a precedente terapia antitumorale sistemica.
• I pazienti sottoposti a trattamento adiuvante o neoadiuvante e che l’abbiano completato = 6 mesi prima della randomizzazione sono idonei.
2. Paziente con carcinoma epatocellulare misto – iCCA.
3. Evidenza pregressa e/o attuale di uno dei seguenti disturbi:
• Alterazione non tumorale dell’omeostasi calcio-fosforo clinicamente significativa secondo il parere dello sperimentatore.
• Mineralizzazione/calcificazione ectopica, inclusi a titolo esemplificativo e non esaustivo tessuti molli, reni, intestino o miocardio e polmone, considerata clinicamente significativa secondo il parere dello sperimentatore.
• Disturbo retinico confermato dall’esame della retina e considerato clinicamente significativo secondo il parere dell’oculista.
4. Aritmie ventricolari non controllate, pregresse o in corso.
5. Intervallo QT corretto secondo Fridericia (QTcF) > 470 ms evidenziato da elettrocardiogramma (ECG) condotto durante lo screening.
6. Trattamento con una qualsiasi delle seguenti terapie entro il periodo di tempo specificato prima della prima dose della terapia in studio o mancato recupero dagli effetti collaterali di queste precedenti terapie:
• Intervento chirurgico maggiore nelle 4 settimane precedenti (l’incisione chirurgica deve essere completamente guarita prima della prima dose della terapia in studio).
• Radioterapia (qualsiasi dose) a campo esteso entro 4 settimane o radioterapia a campo limitato entro 2 settimane e/o mancato recupero dall’impatto acuto della radioterapia.
• Pazienti con terapia locoregionale, ad es. chemioembolizzazione trans-arteriosa (TACE), radioterapia interna selettiva (SIRT) o ablazione entro 4 settimane.
• Trascorsi di trapianto di fegato.
7. Malattia o condizione medica grave, tra cui, a titolo esemplificativo, le seguenti:
• Metastasi cerebrali non trattate o clinicamente o radiologicamente instabili (ossia rimaste stabili per <1 mese).
• Infezione sistemica acuta nota.
• Infarto miocardico, angina grave/instabile o insufficienza cardiaca congestizia sintomatica nei 6 mesi precedenti.
• Nausea, vomito o diarrea cronici considerati clinicamente significativi secondo il parere dello sperimentatore.
• Sindrome congenita del QT lungo o trascorsi noti di torsione di punta o anamnesi familiare di morte improvvisa inspiegabile.
• Altre gravi condizioni mediche o psichiatriche acute o croniche o anomalie di laboratorio che, a giudizio dello sperimentatore, renderebbero il paziente inappropriato per l’ammissione a questo studio.
8. Pazienti con anamnesi di un’altra neoplasia primaria la cui storia naturale o il trattamento della quale ha il potenziale di interferire con la valutazione della sicurezza o dell’efficacia del regime sperimentale secondo il parere dello sperimentatore.
9. Donna gestante o che allatta al seno.
10. Paziente incapace di assumere farmaci per via orale.

E.5 End points

E.5.1

Primary end point(s)

Progression Free Survival (PFS) per ICR central assessment

Sopravvivenza libera da progressione (PFS) mediante revisione centrale indipendente (ICR),

E.5.1.1

Timepoint(s) of evaluation of this end point

At the end of every 2 cycles (± 7 days) up to Cycle 4. Thereafter, every 3 cycles (± 7 days), or as clinically indicated, until radiologic progressive disease (PD) or initiation of new anticancer therapy (whichever occurs first).

Alla fine di ogni 2 cicli (±7 giorni) fino al ciclo 4. Dopo il ciclo 4, ogni 3 cicli (±7 giorni) o, come clinicamente indicato, fino a documentazione radiologica della progressione della malattia (PD) o dall'inizio di una nuova terapia anti-cancro (a seconda di quale circostanza si verifichi per prima).

E.5.2

Secondary end point(s)

Objective response rate (ORR)
• Disease control rate (DCR)
• Overall survival (OS)
• PFS according to Investigator assessment of radiologic images
• Treatment-emergent adverse events (TEAEs), including serious
adverse events (SAEs), clinical laboratory tests, vital signs,
ophthalmological exams, and 12-lead electrocardiogram (ECG).

• Tasso di risposta globale (ORR)
• Tasso di controllo della malattia (DCR)
• Sopravvivenza globale (OS)
• PFS secondo la valutazione delle immagini radiologiche da parte dello sperimentatore
• Eventi avversi associati al trattamento (TEAE), di eventi avversi gravi (SAE), esami clinici di laboratorio, parametri vitali ed ECG a 12 derivazioni.

E.5.2.1

Timepoint(s) of evaluation of this end point

Bullet point 1-4: At the end of every 2 cycles (± 7 days) up to Cycle 4. Thereafter, every 3 cycles (± 7 days), or as clinically indicated, until radiologic progressive disease (PD) or initiation of new anticancer therapy (whichever occurs first).

Bullet point 5: All throughout the study, from the time main informed
consent is signed through 30 days after administration of the last dose
of study therapy or until the start of new anticancer therapy, whichever
is earlier.

Punti 1-4: Alla fine di ogni 2 cicli (±7 giorni) fino al ciclo 4. Dopo il ciclo 4, ogni 3 cicli (±7 giorni) o, come clinicamente indicato, fino a documentazione radiologica della progressione della malattia (PD) o dall'inizio di una nuova terapia anti-cancro (a seconda di quale circostanza si verifichi per prima).

Punto 5: per tutta la durata dello studio, dalla firma del consenso informato fino ai 30 giorni successivi all'ultima dose del farmaco dello o fino all'inizio di una nuova terapia anti-cancro (a seconda di quale circostanza si verifichi per prima).

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

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

Tolerability

Tollerabilità

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

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

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

Australia

Brazil

Canada

Hong Kong

Japan

Korea, Republic of

Mexico

Peru

Taiwan

Thailand

United States

Belgium

France

Germany

Italy

Netherlands

Poland

Portugal

Spain

United Kingdom

Argentina

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 trial occurs after study completion, and is defined as the time when the last patient has discontinued study treatment (including patients in the Study Extension phase, if applicable) and completed all visits and scheduled procedures.

La fine dello studio si verifica dopo il completamento dello studio, ed è definito come il momento in cui l'ultimo paziente ha interrotto il trattamento di studio (compresi i pazienti nella fase di estensione dello studio, se applicabile) e completato tutte le visite e le procedure programmate.

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:

162

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

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

F.4.2.2

In the whole clinical trial

216

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)

The investigator remains responsible for following through an appropriate health care option for AEs that are considered related to the study or that caused the patient to discontinue the study. The patient should be followed up until the event is resolved or explained. Frequency of follow-up is left to the discretion of the investigator.

Lo sperimentatore rimane responsabile di seguire un'appropriata opzione di assistenza sanitaria per gli eventi avversi considerati correlati allo studio o che hanno causato l'interruzione dello studio da parte del paziente. Il paziente deve essere seguito fino a quando l'evento è risolto o spiegato. La frequenza di follow-up è lasciata alla discrezione dello Sperimentatore.

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

2021-03-15

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-05-11

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

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