Clinical Trials Register

Summary
EudraCT Number:	2021-006196-42
Sponsor's Protocol Code Number:	CJDQ443E12101
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:	2022-06-21
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

Expand All Collapse All

A. Protocol Information

A.1

Member State Concerned

Spain - AEMPS

A.2

EudraCT number

2021-006196-42

A.3

Full title of the trial

KontRASt-03: A Phase Ib/II, multicenter, open-label platform study of JDQ443 with select combinations in patients with advanced solid tumors harboring the KRAS G12C mutation

A.3.1

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

Study of JDQ443 , in combination with trametinib or ribociclib or cetuximab, in advanced cancer with a specific mutation (changes in a gene) called KRAS G12C

A.4.1

Sponsor's protocol code number

CJDQ443E12101

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	Novartis Farmacéutica, 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	Novartis Pharma AG
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	Novartis Farmacéutica, S.A.
B.5.2	Functional name of contact point	Trial Monitoring Organization (TMO)
B.5.3	Address:
B.5.3.1	Street Address	Gran vía de les Corts Catalanes, 764
B.5.3.2	Town/ city	Barcelona
B.5.3.3	Post code	08013
B.5.3.4	Country	Spain
B.5.4	Telephone number	+34930353036
B.5.5	Fax number	+34932479903
B.5.6	E-mail	eecc.novartis@novartis.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	JDQ443
D.3.2	Product code	JDQ443
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	JDQ443
D.3.9.2	Current sponsor code	JDQ443
D.3.9.3	Other descriptive name	JDQ443
D.3.9.4	EV Substance Code	SUB218494
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	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	Yes
D.2.1.1.1	Trade name	Mekinist
D.2.1.1.2	Name of the Marketing Authorisation holder	Novartis Europharm 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	TMT212
D.3.2	Product code	TMT212
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	trametinib
D.3.9.2	Current sponsor code	TMT212
D.3.9.3	Other descriptive name	TRAMETINIB DIMETHYL SULFOXIDE
D.3.9.4	EV Substance Code	SUB167251
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	0.5
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	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	Yes
D.2.1.1.1	Trade name	Mekinist
D.2.1.1.2	Name of the Marketing Authorisation holder	Novartis Europharm 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	TMT212
D.3.2	Product code	TMT212
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	trametinib
D.3.9.2	Current sponsor code	TMT212
D.3.9.3	Other descriptive name	TRAMETINIB DIMETHYL SULFOXIDE
D.3.9.4	EV Substance Code	SUB167251
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	2
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	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	Yes
D.2.1.1.1	Trade name	Kisqali
D.2.1.1.2	Name of the Marketing Authorisation holder	Novartis Europharm 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	ribociclib
D.3.2	Product code	LEE011
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	ribociclib
D.3.9.2	Current sponsor code	LEE011
D.3.9.3	Other descriptive name	RIBOCICLIB SUCCINATE
D.3.9.4	EV Substance Code	SUB184164
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	200
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	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	Yes
D.2.1.1.1	Trade name	Erbitux
D.2.1.1.2	Name of the Marketing Authorisation holder	Merck 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	cetuximab
D.3.4	Pharmaceutical form	Solution for 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	Cetuximab
D.3.9.1	CAS number	205923-56-4
D.3.9.2	Current sponsor code	N/A
D.3.9.3	Other descriptive name	N/A
D.3.9.4	EV Substance Code	SUB01178MIG
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	5
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	Yes
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

adult patients with advanced (metastatic or unresectable) KRAS G12C mutant solid tumors

Pacientes adultos con tumores sólidos avanzados (metastásicos o irresecables) con mutación KRAS G12C

E.1.1.1

Medical condition in easily understood language

advanced cancer with a specific mutation (G12C) in the KRAS gene

cáncer avanzado con una mutación específica (G12C) en el gen KRAS

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	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

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	10061873
E.1.2	Term	Non-small cell lung cancer
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.0
E.1.2	Level	PT
E.1.2	Classification code	10061451
E.1.2	Term	Colorectal cancer
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

E.1.2 Medical condition or disease under investigation

E.1.2	Version	22.0
E.1.2	Level	LLT
E.1.2	Classification code	10069759
E.1.2	Term	KRAS mutation
E.1.2	System Organ Class	100000004850

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

Dose escalation
To characterize safety and tolerability of each treatment arm tested and identify the recommended doses and regimens for future studies.
Phase II
To assess the anti-tumor activity of each treatment arm.

Escalada de dosis
Caracterizar la seguridad y tolerabilidad de cada brazo de tratamiento evaluado e identificar las dosis recomendadas y las pautas posológicas para estudios futuros.
Fase II
Evaluar la actividad antitumoral de cada brazo de tratamiento.

E.2.2

Secondary objectives of the trial

Dose escalation
To assess the preliminary anti-tumor activity of each treatment arm
To characterize the PK of JDQ443 and corresponding combination
Phase II
To assess the preliminary anti-tumor activity of each treatment arm
To assess safety and tolerability of each treatment arm tested
To characterize the PK of JDQ443 and corresponding combination partner(s), as applicable, for each treatment arm

Escalada de dosis
Evaluar la actividad antitumoral preliminar de cada brazo de tratamiento.
Caracterizar la PK de JDQ443 y la combinación correspondiente.
Fase II
Evaluar la actividad antitumoral preliminar de cada brazo de tratamiento.
Evaluar la seguridad y la tolerabilidad de cada brazo de tratamiento.
Caracterizar la PK de JDQ443 y el (los) fármaco(s) correspondiente(s) con los que se combina, según proceda, para cada brazo de tratamiento

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Dose Escalation:
-Patients with advanced (metastatic or unresectable) KRAS G12C mutant solid tumors who have received standard of care therapy or are ineligible to receive such therapy.
Phase II:
-Patients with advanced (metastatic or unresectable) KRAS G12C mutant non-small cell lung cancer who have received one platinum-based chemotherapy regimen and immune checkpoint inhibitor therapy, unless patient was ineligible to receive such therapy
-Patients with advanced (metastatic or unresectable) KRAS G12C mutant colorectal cancer who have received fluropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, unless patient was ineligible to such therapy.
All patients:
-ECOG performance status of 0 or 1.
-Patients must have a site of disease amenable to biopsy and be a candidate for tumor biopsy according to the treating institution’s guidelines.

Escalada de dosis:
- Pacientes con tumores sólidos avanzados (metastásicos o irresecables) con mutación KRAS G12C que hayan recibido el tratamiento estándar o que no sean elegibles para recibirlo.
Fase II:
- Pacientes con cáncer de pulmón de células no pequeñas avanzado (metastásico o irresecable) con mutación KRAS G12C que hayan recibido una pauta posológica de quimioterapia basada en platino y terapia con un inhibidor de puntos de control inmunitario, a menos que no sean elegibles para recibir dicho tratamiento.
- Pacientes con cáncer colorrectal avanzado (metastásico o irresecable) con mutación KRAS G12C que hayan recibido quimioterapia basada en fluropirimidina, oxaliplatino e irinotecán, a menos que no sean elegibles para recibir dicho tratamiento.
Todos los pacientes:
- Estado funcional del ECOG de 0 o 1.
- Los pacientes deben tener un lugar de enfermedad en el que se pueda realizar una biopsia y ser candidatos para una biopsia del tumor según las pautas del centro encargado del tratamiento.

E.4

Principal exclusion criteria

Key exclusion applicable to all arms
-Tumors harboring driver mutations that have approved targeted therapies, with the exception of KRAS G12C mutations.
-Prior treatment with a KRAS G12C inhibitor is excluded for patients in a subset of groups in Phase II.
-Active brain metastases, including symptomatic brain metastases or known leptomeningeal disease
-Clinically significant cardiac disease or risk factors at screening
-Insufficient bone marrow, hepatic or renal function at screening

Criterios de exclusión principales que aplican a todos los brazos
- Tumores con mutaciones conductoras que cuenten con tratamientos dirigidos aprobados, con la excepción de las mutaciones KRAS G12C.
- Pacientes con tratamiento previo con un inhibidor de KRAS G12C en un subconjunto de los grupos de fase II.
- Metástasis cerebrales activas, incluidas metástasis cerebrales sintomáticas o enfermedad leptomeníngea conocida.
- Enfermedad cardíaca clínicamente significativa o factores de riesgo en la selección.
- Función medular, hepática o renal insuficiente en la selección.

E.5 End points

E.5.1

Primary end point(s)

1) Dose escalation: Incidence and severity of dose limiting toxicities (DLTs) of each combination treatment.
2) Dose escalation: Incidence and severity of adverse events (AEs) and serious adverse events (SAEs) by treatment
3) Dose escalation: Frequency of dose interruptions and reductions, by treatment
4) Dose Escalation: Dose intensity by treatment
5) Phase II: Overall Response Rate by Blinded Independent Review Committee (BIRC) per RECIST 1.1

1)Escalado de dosis: incidencia e intensidad de toxicidades limitantes de dosis (DLT) de cada tratamiento de combinación
2)Escalado de dosis: incidencia e intensidad de los acontecimientos adversos (AA) y los acontecimientos adversos graves (AAG) por tratamiento
3)Escalado de dosis: frecuencia de las interrupciones de dosis y reducciones de dosis por tratamiento
4)Escalado de dosis: intensidad de las dosis por tratamiento
5) Fase II: respuesta tumoral evaluada por el BIRC según RECIST 1.1

E.5.1.1

Timepoint(s) of evaluation of this end point

1) 28 days
2) to 6) 24 months

1) 28 días
2) a 6) 24 meses

E.5.2

Secondary end point(s)

1) Dose escalation and Phase II: ORR by local review per RECIST 1.1
2) Dose escalation and Phase II: Disease Control Rate (DCR) by local review per RECIST 1.1
3) Dose escalation and Phase II: Duration of Response (DoR) by local review per RECIST 1.1
4) Dose escalation and Phase II: Progression-Free Survival (PFS) by local review per RECIST 1.1
5) Phase II: DCR by BIRC per RECIST 1.1
6) Phase II: DoR by BIRC per RECIST 1.1
7) Phase II: PFS by BIRC per RECIST 1.1
8) Phase II: Overall survival (OS)
9) Dose escalation and Phase II: PK parameters - Maximum Concentration (Cmax), as applicable per arm
10) Dose escalation and Phase II: PK parameters - Minimum Concentration (Cmin), as applicable per arm
11) Dose escalation: Time to achieve Cmax - Tmax, as applicable per arm
12) Dose escalation and Phase II: Plasma or serum concentration vs time profiles - AUCtau, as applicable per arm
13) Dose escalation and Phase II: Plasma or serum concentration vs time profiles - AUCinf, as applicable per arm
14) Phase II: Incidence and severity of adverse events (AEs) and serious adverse events (SAEs) by treatment
15) Phase II: Frequency of dose interruptions and reductions, by treatment
16) Phase II: Dose intensity by treatment

1)Escalado de dosis y Fase II: ORR determinada mediante revisión local según RECIST 1.1
2) Escalado de dosis y Fase II: Tasa de control de enfermedades (TCE) por revisión local según RECIST 1.1
3) Escalado de dosis y Fase II: Duración de la respuesta (DoR) por revisión local según RECIST 1.1
4) Escalado de dosis y Fase II: Supervivencia libre de progresión (PFS) por revisión local según RECIST 1.1
5)Fase II: DCR por BIRC según RECIST 1.1
6)Fase II: DoR por BIRC según RECIST 1.1
7)Fase II: PFS por BIRC según RECIST 1.1
8)Fase II: Supervivencia global (SG)
9)Escalado de dosis y Fase II: parámetros farmacocinéticos - Concentración máxima (Cmax), según corresponda por brazo
10) Escalado de dosis y Fase II: parámetros farmacocinéticos - Concentración mínima (Cmin), según corresponda por brazo
11) Escalado de dosis: tiempo para lograr Cmax - Tmax, según corresponda por brazo
12) Escalado de dosis y Fase II: Concentración plasmática o sérica frente a perfiles de tiempo - AUCtau, según corresponda por brazo
13) Escalado de dosis y Fase II: perfiles de concentración plasmática o sérica frente a tiempo - AUCinf, según corresponda por brazo
14)Fase II: incidencia e intensidad de los acontecimientos adversos (AA) y los acontecimientos adversos graves (AAG) por tratamiento
15)Fase II: frecuencia de las interrupciones de dosis y reducciones de dosis por tratamiento
16)Fase II: intensidad de las dosis por tratamiento

E.5.2.1

Timepoint(s) of evaluation of this end point

1) to 8) and 14) to 16) 24 months
9) to 13) 5 months

1) a 8) y 14) a 16) 24 meses
9) a 13) 5 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

Yes

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)

Yes

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

Yes

E.7.1.3.1

Other trial type description

Dose escalation study

Estudio escalada de dosis

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

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

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

Information not present in EudraCT

E.8.2.2

Placebo

Information not present in EudraCT

E.8.2.3

Other

Information not present in EudraCT

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the 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

Japan

Korea, Republic of

Singapore

United States

France

Spain

Germany

Italy

Belgium

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

LVLS

LPLV

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

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:

275

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

175

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

300

F.4.2.2

In the whole clinical trial

450

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

2022-09-29

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-09-21

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

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

Austria
Belgium
Bulgaria
Croatia
Cyprus
Czechia
Denmark
Estonia
Finland
France
Germany
Greece
Hungary
Iceland
Ireland
Italy
Latvia
Liechtenstein
Lithuania
Luxembourg
Malta
Netherlands
Norway
Poland
Portugal
Romania
Slovakia
Slovenia
Spain
Sweden
United Kingdom
Outside EU/EEA

Clinical trials