Clinical Trials Register

Summary
EudraCT Number:	2020-005269-15
Sponsor's Protocol Code Number:	CO42865
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2021-12-23
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

2020-005269-15

A.3

Full title of the trial

A PHASE III, RANDOMIZED, OPEN-LABEL STUDY OF PRALSETINIB VERSUS STANDARD OF CARE FOR TREATMENT OF RET-MUTATED MEDULLARY THYROID CANCER

ESTUDIO ALEATORIZADO, ABIERTO DE FASE III, PARA COMPARAR PRALSETINIB CON EL TRATAMIENTO ESTÁNDAR DEL CÁNCER MEDULAR DE TIROIDES CON MUTACIÓN EN RET

A.3.1

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

A Study of Pralsetinib versus Standard of Care for Treatment of RET-Mutated Medullary Thyroid Cancer

Estudio de pralsetinib versus atención estándar para el tratamiento del cáncer de tiroides medular con mutación de RET

A.4.1

Sponsor's protocol code number

CO42865

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/271/2021

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	F. Hoffmann-La Roche Ltd
B.1.3.4	Country	Switzerland
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	F.Hoffmann-La Roche Ltd.
B.4.2	Country	Switzerland
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	F.Hoffmann-La Roche Ltd.
B.5.2	Functional name of contact point	Trial Information Support Line-TISL
B.5.3	Address:
B.5.3.1	Street Address	Grenzacherstrasse 124
B.5.3.2	Town/ city	Basel
B.5.3.3	Post code	CH-4070
B.5.3.4	Country	Switzerland
B.5.4	Telephone number	+34 637 32 02 90
B.5.5	Fax number	+34 913 248 196
B.5.6	E-mail	spain.start_up_unit@roche.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.3.1	Product name	Pralsetinib
D.3.2	Product code	RO 749-9790/F02-02
D.3.4	Pharmaceutical form	Capsule
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	PRALSETINIB
D.3.9.1	CAS number	2097132-94-8
D.3.9.2	Current sponsor code	RO 749-9790/F02-02
D.3.9.3	Other descriptive name	BLU-667, BLU123244, BLU3244, X581238, C683, SEE, 72C683
D.3.9.4	EV Substance Code	SUB196574
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: 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	Cometriq
D.2.1.1.2	Name of the Marketing Authorisation holder	Ipsen Pharma
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	Cometriq 20mg/80mg capsules from Ipsen Pharma
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.3.8 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.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
D.2.1.1.2	Name of the Marketing Authorisation holder	Exeliris
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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Cometriq 80mg/20mg capsules from Exelixis
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.3.8 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.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 100mg
D.2.1.1.2	Name of the Marketing Authorisation holder	Genzyme Europe
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	Caprelsa 100mg
D.3.4	Pharmaceutical form	Film-coated tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Vandetanib
D.3.9.1	CAS number	443913-073-3
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	Caprelsa 300mg
D.2.1.1.2	Name of the Marketing Authorisation holder	Genzyme Europe
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	Caprelsa 300mg
D.3.4	Pharmaceutical form	Film-coated tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Vandetanib
D.3.9.1	CAS number	443913-073-3
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	300
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

RET-mutated Medullary Thyroid Cancer (MTC)

Cáncer de tiroides medular con mutación en RET (MTC)

E.1.1.1

Medical condition in easily understood language

RET-Mutated Medullary Thyroid Cancer (MTC) is a form of thyroid carcinoma which originates from parafollicular cells and which produce the hormone calcitonin.

El cáncer de tiroides medular mutado con RET (MTC) es una forma de carcinoma de tiroides que se origina a partir de células parafoliculares y que produce la hormona calcitonina.

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

E.2 Objective of the trial

E.2.1

Main objective of the trial

• To evaluate the efficacy of pralsetinib compared with investigator’s choice of standard of care (SOC) multikinase inhibitor (MKI) therapy for patients with RET-mutated MTC

• Evaluar la eficacia de pralsetinib en comparación con el tratamiento de referencia con MKI elegido por el investigador en pacientes con CMT con mutación RET.

E.2.2

Secondary objectives of the trial

• To evaluate the efficacy of pralsetinib compared with investigator’s choice of SOC MKI therapy
• To evaluate objective response rate
• To evaluate overall survival
• To further characterize the safety and tolerability profile of pralsetinib
• To evaluate additional measures of anticancer activity, including duration of response, disease control rate, and clinical benefit rate
• To evaluate the efficacy of pralsetinib versus SOC treatment through patient-reported endpoints
• To evaluate the acceptability and palatability of pralsetinib capsules

- Evaluar la eficacia de pralsetinib en comparación con el tratamiento de referencia con MKI elegido por el investigador
- Evaluar la TRO
- Evaluar la SG
- Caracterizar mejor el perfil de seguridad y tolerabilidad de pralsetinib.
- Evaluar otras medidas de la actividad antineoplásica, como DR, TCE y TBC.
- Evaluar la eficacia de pralsetinib en comparación con el tratamiento de referencia mediante los criterios de valoración comunicados por los pacientes.
- Evaluar la aceptabilidad y la palatabilidad de pralsetinib en cápsulas.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

• Age >= 18 years (participants as young as 12 years of age will be allowed if permitted by local regulatory authorities)
• Histologically confirmed unresectable locally advanced or metastatic MTC and candidate for systemic therapy with SOC MKI
• No prior systemic anticancer treatment with MKI therapies for advanced or metastatic MTC
• Radiologically confirmed progressive disease within the last 14 months
• Confirmed RET mutation
• Able to swallow an oral medication
• Eastern Cooperative Oncology Group (ECOG)Performance Status of 0-2
• Men and women of childbearing potential must agree to use a highly effective contraceptive method during treatment period and for 14 days after the final dose of pralsetinib and for 120 days after the final dose of cabozantinib or vandetanib

- Edad mínima de 18 años en el momento de firmar el consentimiento informado o
asentimiento.
- Deben tener CMT metastásico o localmente avanzado irresecable confirmado
histológicamente y ser tributarios de tratamiento sistémico con un MKI de
referencia.
- Ausencia de tratamiento antineoplásico sistémico previo con MKI para el CMT
avanzado o metastásico.
- Progresión de la enfermedad confirmada radiológicamente en los últimos 14 meses
- Mutación confirmada de RET determinada localmente
- Capacidad de tragar un medicamento oral.
- Estado funcional del ECOG de 0-2
- Los hombres y mujeres en edad fértil deben aceptar utilizar un método anticonceptivo altamente eficaz durante el período de tratamiento y durante 14 días después de la dosis final de pralsetinib y durante 120 días después de la dosis final de cabozantinib o vandetanib.

E.4

Principal exclusion criteria

• Previously treated with any systemic kinase inhibitor therapy regimens, including a selective RET inhibitor, given for recurrent and/or metastatic disease
• Any additional known primary driver alterations other than RET
• History of pneumonitis within last 12 months
• Ongoing treatment with chronic immunosuppressants or systemic steroids >10 mg/day of prednisone
• Symptomatic CNS metastases or untreated spinal cord compression
• Clinically significant, uncontrolled, cardiovascular disease
• Other malignancy diagnosed or treated within the past 2 years
• Active, uncontrolled infection
• Have received organ or allogenic bone marrow or peripheral blood stem cell transplant

- Tratamiento previo con cualquier régimen sistémico con inhibidores de cinasas,
incluido un inhibidor selectivo de RET, administrado por enfermedad recurrente o
metastásica.
-Cualquier alteración adicional conocida del controlador principal que no sea RET
- Historia de neumonitis en los últimos 12 meses
- Tratamiento en curso con inmunodepresores crónicos o esteroides sistémicos que superen los 10 mg/día de prednisona
- Presencia de metástasis en el SNC o compresión de la médula espinal no tratada
- Enfermedad cardiovascular no controlada y de importancia clínica
- Antecedentes de otra neoplasia maligna primaria diagnosticada o que haya
precisado tratamiento en los 2 años previos a la aleatorización
- Presencia de infección activa no controlada
- Recepción de un trasplante de órgano o alotrasplante de médula ósea o de células
madre de sangre periférica.

E.5 End points

E.5.1

Primary end point(s)

Progression free survival by Blinded Independent Central Review (BICR)

Supervivencia libre de progresión según la revisión central independiente ciega (BICR)

E.5.1.1

Timepoint(s) of evaluation of this end point

Up to 13 years

Hasta 13 años

E.5.2

Secondary end point(s)

1. Time-to-treatment failure
2. Objective response rate
3. Overall survival
4. Incidence and severity of adverse events, with severity, as determined according to the National Cancer Institute Common Toxicity Criteria for Adverse Events version 5.0
5. Change from baseline in targeted clinical laboratory test results
6. Change from baseline in ECG results
7. Change from baseline in ECOG Performance Status
8. Duration of response
9. Disease control rate
10. Clinical benefit rate
11. Time to deterioration of function and quality of life
12. Acceptability Survey scores on Day 1 of Cycle 1 and Crossover Cycle 1

1. Fracaso en el tiempo transcurrido hasta el tratamiento
2. Tasa de respuesta objetiva
3. Supervivencia general
4. Incidencia y gravedad de los eventos adversos, con gravedad, según lo determinado de acuerdo con los Criterios de toxicidad comunes para eventos adversos del Instituto Nacional del Cáncer versión 5.0
5. Cambio con respecto al valor inicial en los resultados de las pruebas de laboratorio clínico específicas
6. Cambio con respecto al valor inicial en los resultados de ECG
7. Cambio con respecto a la línea de base en el estado de desempeño ECOG
8. Duración de la respuesta
9. Tasa de control de enfermedades
10. Tasa de beneficio clínico
11. Tiempo hasta el deterioro de la función y la calidad de vida.
12. Puntuaciones de la encuesta de aceptabilidad en el día 1 del ciclo 1 y el ciclo cruzado 1

E.5.2.1

Timepoint(s) of evaluation of this end point

1-4. Up to 13 years
5-6. From baseline (Day -28 to -1) to 13 years
7. From baseline to Day 1 of Cycle 1-5, and end of treatment
8-11. Up to 13 years
12. Day 1 of Cycle 1 and Crossover Cycle 1

1-4. Hasta 13 años
5-6. Desde el inicio (día -28 a -1) hasta los 13 años
7. Desde el inicio hasta el día 1 del ciclo 1-5 y el final del tratamiento
8-11. Hasta 13 años
12. Día 1 del ciclo 1 y ciclo cruzado 1

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

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

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

Mexico

Philippines

Thailand

Argentina

Austria

Belgium

Brazil

Bulgaria

Canada

Croatia

Denmark

Germany

Greece

Hungary

India

Israel

Italy

Korea, Republic of

Netherlands

Poland

Portugal

Romania

Russian Federation

Spain

Sweden

Ukraine

United Kingdom

United States

France

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 this study is defined as the date when the last patient, last visit occurs or when the required number of deaths for the final analysis of overall survival has been observed, whichever occurs last. The expected duration of the study is approximately, but not limited to 13 years.

El final de este estudio se define como la fecha en que se produce el último paciente, la última visita o cuando se ha observado el número requerido de muertes para el análisis final de supervivencia global, lo que ocurra en último lugar. La duración esperada del estudio es aproximadamente, pero no se limita a 13 años.

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

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:

138

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

102

F.4.2.2

In the whole clinical trial

198

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)

Please refer to section 6.6 of the protocol

Consulte la sección 6.6 del protocolo.

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

2022-03-18

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-03-14

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

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Orphan Designation Number:
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