Clinical Trials Register

Summary
EudraCT Number:	2019-001978-28
Sponsor's Protocol Code Number:	J2G-MC-JZJB
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:	2021-06-15
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

2019-001978-28

A.3

Full title of the trial

A Multicenter, Randomized, Open-label, Phase 3 Trial Comparing LOXO-292 to Physicians Choice of Cabozantinib or Vandetanib in Patients with Progressive, Advanced, Kinase Inhibitor Naïve, RET-Mutant Medullary Thyroid Cancer (LIBRETTO-531)

Studio di Fase III, multicentrico, randomizzato, in aperto, atto a comparare LOXO-292 e il trattamento scelto a discrezione del medico tra Cabozantinib o Vandetanib, in pazienti affetti da carcinoma midollare della tiroide, avanzato, in progressione, naïve all’inibitore della chinasi, con mutazione del gene RET (LIBRETTO-531)

A.3.1

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

A Phase 3 Trial Comparing LOXO-292 to Cabozantinib or Vandetanib in Patients with RET-Mutant Medullary Thyroid Cancer

Studio di Fase III atto a comparare LOXO-292 e Cabozantinib o Vandetanib, in pazienti affetti da carcinoma midollare della tiroide con mutazione del gene RET

A.3.2

Name or abbreviated title of the trial where available

LIBRETTO-531

A.4.1

Sponsor's protocol code number

J2G-MC-JZJB

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	ELI LILLY & COMPANY, LILLY CORPORATE CENTER
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	Eli Lilly and Company
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	Eli Lilly
B.5.2	Functional name of contact point	Clinical Trial Registry Office
B.5.3	Address:
B.5.3.1	Street Address	Lilly Corporate Center, DC 1526
B.5.3.2	Town/ city	Indianapolis
B.5.3.3	Post code	46285
B.5.3.4	Country	United States
B.5.4	Telephone number	000000
B.5.5	Fax number	000000
B.5.6	E-mail	EU_Lilly_Clinical_Trials@lilly.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	Yes
D.2.5.1	Orphan drug designation number	EU/3/05/344
D.3 Description of the IMP
D.3.1	Product name	SELPERCATINIB
D.3.2	Product code	[LY35277723]
D.3.4	Pharmaceutical form	Capsule, hard
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	Selpercatinib
D.3.9.2	Current sponsor code	LOXO-292
D.3.9.4	EV Substance Code	SUB193120
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	40
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	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/3/05/344
D.3 Description of the IMP
D.3.1	Product name	SELPERCATINIB
D.3.2	Product code	[LY35277723]
D.3.4	Pharmaceutical form	Capsule, hard
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	Selpercatinib
D.3.9.2	Current sponsor code	LOXO-292
D.3.9.4	EV Substance Code	SUB193120
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	80
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	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	Cometriq 140 mg daily-dose carton
D.2.1.1.2	Name of the Marketing Authorisation holder	Exelixis Inc.
D.2.1.2	Country which granted the Marketing Authorisation	United States
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/3/08/610
D.3 Description of the IMP
D.3.1	Product name	Cabozantinib
D.3.2	Product code	[Cabozantinib]
D.3.4	Pharmaceutical form	Capsule, hard
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.3.9.1	CAS number	849217-68-1
D.3.9.2	Current sponsor code	CABOZANTINIB
D.3.9.4	EV Substance Code	SUB93452
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	20
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	Caprelsa 100 mg compresse rivestite da film
D.2.1.1.2	Name of the Marketing Authorisation holder	Genzyme Europe B.V.
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	Vandetanib
D.3.2	Product code	[Vandetanib]
D.3.4	Pharmaceutical form	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	vandetanib
D.3.9.2	Current sponsor code	VANDETANIB
D.3.9.4	EV Substance Code	SUB29174
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	100
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: 5
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	Cometriq 140 mg daily-dose carton
D.2.1.1.2	Name of the Marketing Authorisation holder	Exelixis Inc.
D.2.1.2	Country which granted the Marketing Authorisation	United States
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/3/08/610
D.3 Description of the IMP
D.3.1	Product name	Cabozantinib
D.3.2	Product code	[Cabozantinib]
D.3.4	Pharmaceutical form	Capsule, hard
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.3.9.1	CAS number	849217-68-1
D.3.9.2	Current sponsor code	CABOZANTINIB
D.3.9.4	EV Substance Code	SUB93452
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	80
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

Male or female patients with progressive, advanced, Kinase Inhibitor Naïve, RET-Mutant Medullary Thyroid Cancer

Pazienti di sesso maschile o femminile affetti da carcinoma midollare della tiroide, avanzato, in progressione, naïve all’inibitore della chinasi, con mutazione del gene RET

E.1.1.1

Medical condition in easily understood language

Medullary Thyroid Cancer

carcinoma midollare della tiroide

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	PT
E.1.2	Classification code	10027105
E.1.2	Term	Medullary thyroid cancer
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

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

To compare TFFS of patients with progressive, advanced, kinase inhibitor naïve, RET-mutant MTC treated with LOXO-292 versus cabozantinib or vandetanib

Confrontare la TFFS di pazienti con CMT avanzato, in progressione, con mutazione di RET e naïve agli inibitori delle chinasi trattati con LOXO-292 rispetto a cabozantinib o vandetanib.

E.2.2

Secondary objectives of the trial

- To compare other efficacy outcomes, based on RECIST 1.1 criteria, observed in patients with progressive, advanced, kinase inhibitor naïve, RET-mutant MTC treated with LOXO-292 versus cabozantinib or vandetanib
- To evaluate the safety and tolerability of LOXO-292 compared to cabozantinib or vandetanib
- To compare the tolerability of LOXO-292 versus cabozantinib or vandetanib
-To assess/evaluate performance of local RET laboratory tests compared to a single, central test.
-To assess the PK of LOXO-292 in patients receiving LOXO-292

- Confrontare altri outcome di efficacia, sulla base dei criteri RECIST 1.1, osservati in pazienti con CMT avanzato, in progressione, con mutazione di RET e naïve agli inibitori delle chinasi trattati con LOXO-292 rispetto a cabozantinib o vandetanib
- Valutare la sicurezza e la tollerabilità di LOXO- 292 rispetto a cabozantinib o vandetanib
- Confrontare la tollerabilità di LOXO-292 rispetto a cabozantinib o vandetanib
-Analizzare/valutare l’esecuzione di test di laboratorio RET a livello locale in confronto ad un singolo test a livello centrale.
-Valutare la farmacocinetica di LOXO-292 in pazienti che ricevono LOXO-292.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Age: All patients of 12 years of age and older, after giving assent / Legally designated representative/ participant written consent.
- Histologically confirmed, metastatic MTC
- Radiographic progressive, measurable disease per BIRC at screening compared with a previous image taken within the prior 14 months as assessed by the investigator
- A RET gene alteration in tumor, genomic DNA or blood.
- Adequate hematologic, hepatic and renal function
- Willingness of men and women of reproductive potential to observe conventional and effective birth control for the duration of treatment and for 6 months after the last dose of study drug.
- Written informed consent

- Età: Tutti i pazienti di età uguale o superiore a 12 anni, dopo aver ottenuto il loro assenso o il consenso scritto del partecipante/ legale rappresentante
- MTC metastatico confermato istologicamente
- Malattia in progressione radiografica e misurabile per BIRC allo screening rispetto a un'immagine precedente presa entro i 14 mesi precedenti, secondo la valutazione dello sperimentatore
- Un'alterazione del gene RET nel tumore, nel DNA genomico o nel sangue.
- Funzionalità ematologica, epatica e renale adeguata
- Disponibilità di uomini e donne in età fertile ad adottare un sistema contraccettivo convenzionale ed efficace per la durata del trattamento e per i 6 mesi successivi all’ultima dose di farmaco in studio.
- Consenso informato scritto

E.4

Principal exclusion criteria

- Additional validated oncogenic driver in MTC if known
- Prior systemic treatment with kinase inhibitor(s)
- Major surgery (excluding biopsy and placement of vascular access) within 4 weeks prior to planned start of study treatment
- Radiotherapy within 2 week of the first dose of study treatment (within 4 weeks if >25% bone marrow irradiated)
- Symptomatic primary CNS tumor, metastases, leptomeningeal carcinomatosis, or untreated spinal cord compression.
- Clinically significant active cardiovascular disease or history of myocardial infarction within 6 months prior to planned start of study treatment or prolongation of the QT interval corrected for heart rate using Fridericia's formula (QTcF) > 470 msec
- Active uncontrolled systemic bacterial, viral, or fungal infection or serious ongoing intercurrent illness, such as hypertension or diabetes, despite optimal treatment. Screening for chronic conditions is not required
- Clinically significant active malabsorption syndrome or other condition likely to affect gastrointestinal absorption of the study drug
- Current treatment with strong cytochrome P450 3A4 (CYP3A4) inhibitors or inducers.
- Current treatment with proton pump inhibitors (PPIs)
- Known hypersensitivity to any of the excipients of LOXO-292 or cabozantinib
- Pregnancy or breastfeeding
- Other malignancy unless nonmelanoma skin cancer, carcinoma in situ of the cervix or malignancy diagnosed =or >2 years previousl and not currently active

- Driver oncogenico addizionale validato nel MTC, se noto
- Precedenti trattamenti sistemici con inibitori della chinasi
- Chirurgia maggiore (escludendo la biopsia e il posizionamento dell'accesso vascolare) entro 4 settimane prima dell'inizio pianificato del trattamento in studio
- Radioterapia entro 2 settimane dalla prima dose del trattamento in studio (entro 4 settimane se> 25% di midollo osseo irradiato)
- Tumore primitivo sintomatico del SNC, metastasi, carcinomatosi leptomeningea o compressione del midollo spinale non trattata.
- Malattia cardiovascolare attiva clinicamente significativa o storia di infarto del miocardio entro 6 mesi prima dell'inizio pianificato del trattamento di studio o prolungamento dell'intervallo QT corretti per la frequenza cardiaca usando la formula di
Fridericia (QTcF)> 470 msec
- Infezione batterica, virale o batterica sistemica incontrollata attiva o grave malattia intercorrente in corso, come ipertensione o diabete, nonostante un trattamento ottimale. Lo screening per condizioni croniche non è richiesto
- Sindrome da malassorbimento attivo clinicamente significativo o altre condizioni che possono influenzare l'assorbimento gastrointestinale del farmaco in studio
- Trattamento attuale con potenti inibitori o induttori del citocromo P450 3A4 (CYP3A4).
- Trattamento attuale con inibitori della pompa protonica (PPI)
- Ipersensibilità nota a uno qualsiasi degli eccipienti di LOXO-292 o cabozantinib
- Gravidanza o allattamento al seno
- Altre malignita’ tranne il tumore della pelle non-melanoma, il carcinoma della cervice in situ oppure malignita’ diagnosticate 2 o piu’ anni prima e attualmente non attive.

E.5 End points

E.5.1

Primary end point(s)

Treatment Failure-Free Survival (TFFS) by Blinded Independent Committee Review (BICR)

Sopravvivenza senza fallimento del trattamento (TFFS) valutata dal Blinded Independent Committee Review (BICR)

E.5.1.1

Timepoint(s) of evaluation of this end point

Time Frame: Baseline to Progressive Disease, Unacceptable Toxicity or Death from Any Cause (Estimated at up to 30 Months)

Arco di tempo: dal Baseline fino alla progressione della malattia, insorgenza di valori di tossicità inaccetttabili o morte per qualsiasi causa (stimata fino a 30 mesi)

E.5.2

Secondary end point(s)

1. Progression Free Survival (PFS) by BICR (PFS by BICR)
2. ORR: Percentage of Participants with Complete Response (CR) or Partial Response (PR) by BICR
3. Duration of Response (DoR) by BICR
4. Overall Survival (OS)
5. PFS2 by Investigator
6. Comparative Tolerability: Patient-Reported Outcomes (Functional Assessment of Cancer Therapy-Side Effects [FACT-GP5])

1. PFS valutata da BICR
2. ORR valutato dallo sperimentatore e dal BICR
3. DoR valutata dallo sperimentatore e dal BICR
4. OS
5. PFS2 valutata dallo sperimentatore
6. Tollerabilità comparativa: Outcomes riferiti dai pazienti (Valutazione funzionale degli Effetti Indesiderati della Terapia Antitumorale [FACT-GP5])

E.5.2.1

Timepoint(s) of evaluation of this end point

1. Baseline to Progressive Disease or Death from Any Cause (Estimated at up to 30 Months)
2. Baseline through Disease Progression or Death (Estimated at up to 30 Months)
3. Date of CR or PR to Date of Disease Progression or Death Due to Any Cause (Estimated at up to 30 Months)
4. Baseline to Date of Death from Any Cause (Estimated at up to 60 Months)
5. Baseline to Second Disease Progression or Death from Any Cause (Estimated at up to 48 Months)
6. Baseline to Progressive Disease, Unacceptable Toxicity or Death from Any Cause (30 months)

1. dal Baseline fino alla progressione della malattia, o morte per qualsiasi causa (stimata fino a 30 mesi)
2. dal Baseline fino alla progressione della malattia o alla morte (stimata fino a 30 mesi)
3. Data della CR o PR fino alla data della progressione della malattia o della morte per qualsiasi causa (stimata fino a 30 mesi)
4. dal Baseline fino alla data della morte per qualsiasi causa (stimata fino a 60 mesi)
5. dal Baseline fino alla progressione della seconda malattia o alla morte per qualsiasi causa (stimata fino a 48mesi)
6. dal Baseline fino alla progressione della malattia, insorgenza di valori di tossicità inaccetttabili o morte per qualsiasi causa (stimata fino a 30 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

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

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)

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

China

India

Israel

Japan

Korea, Republic of

Russian Federation

United States

France

Germany

Italy

Netherlands

Poland

Spain

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

LVLS

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)

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

310

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

137

F.4.2.2

In the whole clinical trial

400

F.5

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

None

Nessuno

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2020-01-22

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-02-27

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

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