Clinical Trials Register

Summary
EudraCT Number:	2019-001978-28
Sponsor's Protocol Code Number:	J2G-MC-JZJB
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2019-12-20
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

Spain - AEMPS

A.2

EudraCT number

2019-001978-28

A.3

Full title of the trial

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

Un ensayo multicéntrico, aleatorizado y abierto de fase 3 que compara selpercatinib con los medicamentos cabozantinib o vandetanib, preferidos por los médicos, en pacientes con cáncer medular de tiroides progresivo y avanzado con RET mutante y sin tratamiento anterior con inhibidores de quinasa (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 Selpercatinib to Cabozantinib or Vandetanib in Patients with RET-Mutant Medullary Thyroid Cancer

Un ensayo de fase 3 que compara selpercatinib con cabozantinib o vandetanib en pacientes con cáncer medular de tiroides

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	Lilly S.A.
B.1.3.4	Country	Spain
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	Island House 11, Eastgate Road, Eastgate Business Park
B.5.3.2	Town/ city	Little Island, Co. Cork
B.5.3.3	Post code	T45 KD39
B.5.3.4	Country	Ireland
B.5.4	Telephone number	0034918362958
B.5.6	E-mail	ensayosclinicos@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	LY3527723
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	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.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	LY3527723
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	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.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.3.4	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	CABOZANTINIB
D.3.9.1	CAS number	849217-68-1
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	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.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 film-coated tablets
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.3.4	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.3.9.3	Other descriptive name	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	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.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.3.4	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	CABOZANTINIB
D.3.9.1	CAS number	849217-68-1
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	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

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

Pacientes de ambos sexos con cáncer medular de tiroides progresivo y avanzado con RET mutante y sin tratamiento anterior con inhibidores de quinasa

E.1.1.1

Medical condition in easily understood language

Medullary Thyroid Cancer

Cáncer medular de tiroides

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 selpercatinib versus cabozantinib or vandetanib.

Comparar la SSFT de pacientes con CMT avanzado y progresivo con RET mutante y sin tratamiento anterior con inhibidores de quinasa tratados con selpercatinib con la SSFT de aquellos tratados con 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 selpercatinib versus cabozantinib or vandetanib

- To evaluate the safety and tolerability of selpercatinib compared to cabozantinib or vandetanib

- To compare the tolerability of selpercatinib versus cabozantinib or vandetanib

- To assess/evaluate performance of local RET laboratory tests compared to a single, entral test.

- To assess the PK of selpercatinib in the patient population.

- Comparar otros resultados de eficacia según los criterios RECIST 1.1 observados en pacientes con CMT avanzado y progresivo con RET mutante y sin tratamiento anterior con inhibidores de quinasa tratados con selpercatinib con los otros resultados de aquellos tratados con cabozantinib o vandetanib.
- Evaluar la seguridad y la tolerabilidad de selpercatinib en comparación con el cabozantinib o el vandetanib.
- Comparar la tolerabilidad de selpercatinib con la del cabozantinib o la del vandetanib.
- Evaluar el rendimiento de las pruebas de laboratorio RET locales en comparación con una sola prueba central.
- Evaluar la PK de selpercatinib en la población de pacientes.

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 3 months after
- Written informed consent

-Edad: todos los pac.de 12 a de edad y mayores, después de dar su consentimiento / consentimiento por escrito del representante / representante legalmente designado. -CMT metastásico, confirmado histológicamente. - Presentar durante la selección enfermedad mensurable y progresiva desde un punto de vista radiológico y de acuerdo con una BICR, en comparación con una imagen anterior que se hubiera tomado en el transcurso de los 14 meses anteriores, según el criterio del investigador. - Presentar una mutación en el gen RET en el tumor, el ADN genómico o la sangre.
- Funciones hematológica, hepática y renal aceptables. - Los pacientes de ambos sexos con capacidad de procrear deben estar dispuestos a utilizar métodos anticonceptivos convencionales y eficaces durante el tratamiento y los 3 meses posteriores. - Consentimiento informado por escrito

E.4

Principal exclusion criteria

- Additional validated oncogenic driver in MTC if known
- Prior systemic treatment with kinase inhibitor(s)
- Major surgery (excluding placement of vascular access) within 4 weeks prior to planned start of study treatment
- Radiotherapy within 1 week of the first dose of study treatment (within 4 weeks if >30% 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 vandetanib or cabozantinib
- Pregnancy or lactation
- Active second malignancy

- Presencia de otra mutación oncoiniciadora validada en el CMT (si se conoce).
- Haber recibido anteriormente un tratamiento sistémico con inhibidores de quinasas.- Haberse sometido a una intervención de cirugía mayor (salvo la colocación quirúrgica de un dispositivo de acceso vascular) en el transcurso de las 4 semanas anteriores a la fecha prevista de inicio del tratamiento del estudio.
- Haber recibido radioterapia en el transcurso de la semana anterior a la primera dosis del tratamiento del estudio (o de las 4 semanas anteriores, si se ha irradiado más del 30 % de la médula ósea).
- Presencia de un tumor primario, metástasis o carcinomatosis leptomeníngea sintomáticos en el sistema nervioso central (SNC), o compresión de la médula espinal sin tratar.
- Presencia de enfermedad cardiovascular activa clínicamente importante o antecedentes de infarto de miocardio en el transcurso de los 6 meses anteriores a la fecha prevista de inicio del tratamiento del estudio, o prolongación del intervalo QT corregido en función de la frecuencia cardíaca de acuerdo con la fórmula de Fridericia (QTcF) > 470 ms.
- Presencia de infección fúngica, vírica o bacteriana sistémica, activa y sin controlar, o enfermedad concomitante grave en curso, como hipertensión o diabetes, a pesar de recibir tratamiento óptimo. No es necesario realizar pruebas de detección de enfermedades crónicas.
- Presencia de síndrome de hipoabsorción activo y clínicamente importante, o de cualquier otra enfermedad que posiblemente afecte la absorción intestinal del medicamento del estudio.
- Recibir en la actualidad tratamiento con inhibidores o inductores potentes del citocromo P450 3A4 (CYP3A4).
- Recibir en la actualidad tratamiento con inhibidores de la bomba de protones (IBP).
- Presentar hipersensibilidad a cualquiera de los excipientes de vandetanib a cabozantinib.
- Estar embarazada o en período de lactancia.
- Presentar una segunda neoplasia maligna.

E.5 End points

E.5.1

Primary end point(s)

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

Supervivencia sin fracaso del tratamiento (SSFT) de acuerdo con la revisión de un comité independiente que desconocerá la asignación del tratamiento (BICR, por sus siglas en inglés).
SSFT de acuerdo con una 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)

Intervalo de tiempo: Desde el período inicial hasta la progresión de la enfermedad, la presencia de toxicidad inaceptable o la muerte por cualquier causa (se estima que la duración máxima será de 30 meses).

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)
6. PFS2 by Investigator
7. Comparative Tolerability: Patient-Reported Outcomes (Functional Assessment of Cancer Therapy-Side Effects [FACT-GP5])
8. RET mutation status
9. Predose plasma concentrations at Day 8 of Cycle 1, and at Day 1 of Cycles 2 through 6.

1. Supervivencia sin progresión (SSP) según la BICR (SSP según la BICR).
2.TGR: Porcentaje de participantes con respuesta completa (RC) o parcial (RP) según la BICR.
3.Duración de la respuesta (DdR) según la BICR.
4.Supervivencia global (SG).
6.Supervivencia sin progresión 2 (SSP2) según el criterio del investigador.
7.Tolerabilidad comparativa: Resultados comunicados por los pacientes (evaluación funcional de los efectos secundarios del tratamiento para el cáncer [FACT-GP5]).
8. Estado de mutación RET
9. Predosificar las concentraciones plasmáticas en el día 8 del ciclo 1 y en el día 1 de los ciclos 2 a 6.

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. Desde el período ini. hasta la progresión de la enf. o la muerte por cualquier causa (se estima que la duración máxima será de 30 m).
2.Desde el período ini.hasta la progresión de la enf.o la muerte (se estima que la duración máx. será de 30 m).
3.Desde la fecha de la RC o la RP hasta la fecha de la progresión de la enf. o la muerte por cualquier causa (se estima que la dur. máx. será de 30 m). 4.Desde el período ini.hasta la muerte por cualquier causa (se estima que la duración máx. será de 60 m).
5.Desde el período inicial hasta la segunda progresión de la enf. o la muerte por cualquier causa (se estima que la duración máx será de 48 m). 6.Desde el período inicial hasta la progresión de la enfermedad, la presencia de tox. inaceptable o la muerte por cualquier causa (30 meses).

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

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

Czech Republic

France

Germany

India

Israel

Italy

Japan

Korea, Republic of

Netherlands

Poland

Russian Federation

Spain

United Kingdom

United States

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

Última vista ultimo paciente

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

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1

Number of subjects for this age range:

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

Ninguno

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

2020-02-25

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-01-28

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

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Select Trial Status:	Select Trial Phase:
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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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