Clinical Trials Register

Summary
EudraCT Number:	2019-002660-27
Sponsor's Protocol Code Number:	CINC280I12201
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2019-12-20
Trial results	View 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

2019-002660-27

A.3

Full title of the trial

A randomized, open label, multicenter phase II study evaluating the efficacy and safety of capmatinib (INC280) plus pembrolizumab versus pembrolizumab alone as first line treatment for locally advanced or metastatic non-small cell lung cancer with PD-L1>/=50%

Estudio de fase II, multicéntrico, aleatorizado y abierto que evalúa la eficacia y seguridad de capmatinib (INC280) más pembrolizumab frente a pembrolizumab en monoterapia como tratamiento de primera línea para cáncer de pulmón de células no pequeñas localmente avanzado o metastásico con PD-L1 >/=50 %

A.3.1

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

Study of capmatinib and pembrolizumab in lung cancer

Estudio de capmatinib en combinación con pembrolizumab en sujetos con cáncer de pulmón

A.4.1

Sponsor's protocol code number

CINC280I12201

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	+34933064464
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	INC280
D.3.2	Product code	INC280
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	capmatinib
D.3.9.2	Current sponsor code	INC280
D.3.9.3	Other descriptive name	INC280
D.3.9.4	EV Substance Code	SUB31645
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	150
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	Keytruda
D.2.1.1.2	Name of the Marketing Authorisation holder	Merck Sharp & Dohme 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.3.4	Pharmaceutical form	Concentrate for 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	PEMBROLIZUMAB
D.3.9.3	Other descriptive name	KEYTRUDA
D.3.9.4	EV Substance Code	SUB167136
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	25
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.IMP: 3
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	INC280
D.3.2	Product code	INC280
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	capmatinib
D.3.9.2	Current sponsor code	INC280
D.3.9.3	Other descriptive name	INC280
D.3.9.4	EV Substance Code	SUB31645
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.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

non-small cell lung cancer

Cáncer de pulmón de células no pequeñas

E.1.1.1

Medical condition in easily understood language

lung cancer

Cáncer de pulmón

E.1.1.2

Therapeutic area

Diseases [C] - Cancer [C04]

MedDRA Classification

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 capmatinib plus pembrolizumab in comparison to pembrolizumab alone

Evaluar la eficacia de la combinación de capmatinib con pembrolizumab frente a pembrolizumab en monoterapia

E.2.2

Secondary objectives of the trial

• To evaluate the anti-tumor activity of capmatinib plus pembrolizumab in comparison to pembrolizumab alone
• To characterize the safety profile of capmatinib plus pembrolizumab and pembrolizumab alone
• To characterize the pharmacokinetics of capmatinib and pembrolizumab
• To evaluate the prevalence and incidence of immunogenicity of pembrolizumab

- evaluar la actividad antitumoral de capmatinib más pembrolizumab en comparación con pembrolizumab en monoterapia
- caracterizar el perfil de seguridad de capmatinib más pembrolizumab y pembrolizumab en monoterapia
- caracterizar la farmacocinética (PK) de capmatinib y pembrolizumab
- evaluar la prevalencia e incidencia de inmunogenicidad de pembrolizumab

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

• Histologically confirmed and documented locally advanced stage III (not candidates for surgical resection or definitive chemo-radiation) or stage IV (metastatic) NSCLC (per AJCC/IASLC v.8) for treatment in the first-line setting
- Treatment with chemotherapy and/or radiation as part of neoadjuvant/adjuvant therapy (other than immunotherapies) is allowed as long as therapy was completed at least 6 months prior to the diagnosis of advanced or metastatic disease.
• Histologically or cytologically confirmed diagnosis of NSCLC that is both EGFR wild type status and ALK- negative rearrangement status:
- Patients with NSCLC of pure squamous cell histology can enter screening without EGFR mutation or ALK rearrangement testing or result; however, patients with pure squamous cell histology who are known to have EGFR mutations in exons 19 or 21 or ALK rearrangements will be excluded.
• Have an archival tumor sample or newly obtained tumor biopsy with high PD-L1 expression (TPS≥ 50%) determined by IHC using FDA approved PD-L1 IHC 22C3 PharmDx assay at a local laboratory or at a Novartis designated central laboratory.
- The archival samples must be most recently available FFPE block or cut tissue sections from the block. Tissue sections must NOT be older than 5 months from the time of sectioning. Archival samples obtained prior to any systemic anti-neoplastic therapy (such as adjuvant therapy) will NOT be acceptable.
- If local laboratory testing of PD-L1 as described above is not available, a newly obtained tumor biopsy or an archival tumor sample is required to confirm the eligibility.
• ECOG performance status ≤ 1
• Have at least 1 measurable lesion by RECIST 1.1; a previously irradiated lesion may only be counted as a target lesion if there is clear sign of progression since the irradiation

- NSCLC localmente avanzado en estadio III (que no sean candidatos para resección quirúrgica o quimiorradiación definitiva) o estadio IV (metastásico) histológicamente confirmado y documentado (según AJCC/IASLC v.8) para el tratamiento de primera línea.
El tratamiento con quimioterapia o/y radiación como parte de la terapia neoadyuvante/adyuvante (salvo inmunoterapias) está permitido siempre y cuando la terapia se hubiera completado al menos 6 meses antes que el diagnóstico de la enfermedad avanzada o metastásica.
- Diagnóstico de NSCLC histológica o citológicamente confirmado que tenga un estado no mutado de EGFR y un estado de reordenamiento de ALK negativo.
Los sujetos con NSCLC de histología de células plenamente escamosas pueden entrar en la selección sin pruebas o resultados de mutación del EGFR o reordenamiento de ALK; sin embargo, quedarán excluidos los pacientes con histología de células plenamente escamosas que tengan mutaciones sensibilizantes del EGFR (como las identificadas en los exones 19, 20 o 21, entre otras) conocidas o reordenamientos de ALK conocidos.
- Sujetos con una muestra tumoral archivada o biopsia tumoral recién obtenida con expresión alta de PD-L1 (TPS >/=50 %) determinada mediante IHC utilizando la prueba PD-L1 IHC 22C3 PharmDx aprobada por la FDA, en un laboratorio local o en un laboratorio central designado por Novartis.
Las muestras archivadas deben ser el bloque FFPE más reciente o cortes de tejido del bloque. Los cortes de tejido NO deben tener más de 5 meses de antigüedad desde el momento de corte. Las muestras archivadas obtenidas antes de cualquier terapia antineoplásica sistémica (como la terapia adyuvante) NO serán aceptables.Si las pruebas analíticas locales de PD-L1 descritas anteriormente no están disponibles, será necesaria otra biopsia tumoral recién obtenida o una muestra tumoral archivada para confirmar la elegibilidad.
- Puntuación del estado funcional ECOG </=1.
- Sujetos con al menos una lesión medible según RECIST 1.1; una lesión previamente irradiada solo se podrá considerar una lesión diana en caso de existir signos claros de progresión desde la irradiación.

E.4

Principal exclusion criteria

1. Prior treatment with a MET inhibitor or HGF-targeting therapy
2. Prior immunotherapy (e.g. anti-PD-1, anti-PD-L1, anti-PD-L2, anti-CTLA-4 antibody, or any other antibody or drug specifically targeting T-cell co-stimulation or immune checkpoint pathways).
3. Have known hypersensitivity to any of the excipients of capmatinib (crospovidone, mannitol, microcrystalline cellulose, povidone, sodium lauryl sulfate, magnesium stearate, colloidal silicon dioxide, and various coating premixes).
4. History of severe hypersensitivity reactions to other monoclonal antibodies, which in the opinion of the investigator may pose an increased risk of serious infusion reaction.
5. Have untreated symptomatic central nervous system (CNS) metastases. Subjects are eligible if CNS metastases have been adequately treated with radiotherapy or surgery and remained stable for >2 weeks after treatment. The subjects must have been off steroids 7 days prior to study treatment
6. Presence or history of a malignant disease, other than NSCLC, that has been diagnosed and/or required therapy within the past 3 years. Exceptions to this exclusion include the following: completely resected basal cell and squamous cell skin cancers, and completely resected carcinoma in situ of any type.
7. Presence or history of interstitial lung disease or interstitial pneumonitis, including clinically significant radiation pneumonitis (i.e. affecting activities of daily living or requiring therapeutic intervention).
8. Clinically significant, uncontrolled heart diseases.
9. Prior palliative radiotherapy for bone lesions ≤ 2 weeks prior to starting study treatment.
10. Major surgery (e.g., intra-thoracic, intra-abdominal or intra-pelvic) within 4 weeks prior (2 weeks for resection of brain metastases) to starting study treatment or who have not recovered from side effects of such procedure. Video-assisted thoracic surgery (VATS) and mediastinoscopy are not counted as major surgery and subjects can be enrolled in the study ≥ 1 week after the procedure.
11. Impairment of GI function or GI disease that may significantly alter the absorption of capmatinib (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, or malabsorption syndrome).
12. Concomitant medication(s) with a “Known Risk of Torsades de Point” per www.qtdrugs.org that cannot be discontinued or replaced by safe alternative medication.
13. Receiving treatment with strong inducers of CYP3A4 that cannot be discontinued at least 1 week prior to the start of treatment with capmatinib and for the duration of the study.
14. Systemic chronic steroid therapy (>10 mg/day prednisone or equivalent) or any immunosuppressive therapy 7 days prior to planned date of first dose of study treatment. Topical, inhaled, nasal and ophthalmic steroids, mineralocorticoids (e.g., fludrocortisone) for patients with orthostatic hypotension, and low-dose supplemental corticosteroids for adrenocortical insufficiency are allowed.
15. Participation in a prior investigational study (drug or device) within 30 days prior to first dose of study treatment or within 5-half lives of the investigational product, whichever is longer, or those who are expected to receive any other investigational drug or device during the conduct of the study.

1. Tratamiento previo con un inhibidor de MET o terapia contra el HGF.
2. Inmunoterapia previa (p. ej., anticuerpo anti-PD-1, anti-PD-L1, anti-PD-L2 o anti-CTLA-4, o cualquier otro anticuerpo o fármaco dirigido específicamente a las vías de coestimulación de células T o de puntos de control inmunitario).
3. Hipersensibilidad conocida a cualquiera de los excipientes de capmatinib (crospovidona, manitol, celulosa microcristalina, povidona, lauril sulfato sódico, estearato de magnesio, dióxido de silicio coloidal y diferentes mezclas de recubrimiento).
4. Antecedentes de reacciones graves de hipersensibilidad a otros anticuerpos monoclonales, que según la opinión del investigador puedan suponer un mayor riesgo de reacción grave a la perfusión.
5. Metástasis sintomáticas no tratadas en el sistema nervioso central (SNC). Los sujetos son elegibles si las metástasis del SNC se han tratado adecuadamente con radioterapia o cirugía y se han mantenido estables durante >2 semanas después del tratamiento.
Los sujetos deben haber suspendido el tratamiento con esteroides 7 días antes del tratamiento del estudio.
6. Presencia o antecedentes de enfermedad maligna, salvo NSCLC, que haya sido diagnosticada y/o que haya requerido tratamiento durante los últimos 3 años. Las excepciones a esta exclusión son cánceres de piel de células escamosas y basales completamente resecados y carcinoma completamente resecado in situ de cualquier tipo.
7. Presencia o antecedentes de enfermedad pulmonar intersticial o neumonitis intersticial, incluida neumonitis de radiación clínicamente significativa (es decir, que afecte a actividades de la vida diaria o que requieran de tratamiento).
8. Enfermedades cardíacas no controladas clínicamente significativas.
9. Radioterapia paliativa previa para lesiones óseas </=2 semanas antes de iniciar el tratamiento del estudio.
10. Cirugía mayor (por ejemplo, intratorácica, intraabdominal o intrapélvica) durante las 4 semanas anteriores (2 semanas de resección de metástasis cerebrales) al inicio del tratamiento del estudio o que no se haya recuperado de los efectos secundarios derivados de dicho procedimiento. La cirugía torácica asistida por vídeo (VATS) y la mediastinoscopia no se consideran cirugía mayor, por lo que los sujetos se pueden incluir en el estudio >/=1 semana después del procedimiento.
11. Alteración de la función GI o enfermedad GI que pueda alterar significativamente la absorción de capmatinib (p. ej., enfermedades ulcerosas, náuseas incontroladas, vómitos, diarrea o síndrome de mala absorción).
12. Medicación concomitante con «riesgo de Torsades de Pointe (TdP)» según www.qtdrugs.org que no pueda discontinuarse ni sustituirse por medicación alternativa segura.
13. Sujetos que estén recibiendo tratamiento con inductores potentes de CYP3A4 que no puedan discontinuarse al menos una semana antes de iniciar el tratamiento con capmatinib ni durante el estudio.
14. Tratamiento crónico con esteroides sistémicos (> 10 mg/día de prednisona o equivalente) o cualquier tratamiento inmunosupresor durante los 7 días anteriores a la fecha prevista de la primera dosis del tratamiento del estudio. Se permite el uso de esteroides tópicos, inhalados, nasales y oftalmológicos, mineralocorticoides (p. ej.,
fludrocortisona) en pacientes con hipotensión ortostática, y corticosteroides complementarios en dosis bajas para la insuficiencia adrenocortical.
15. Sujetos que hayan participado en un estudio de investigación previo (fármaco o dispositivo) durante los 30 días anteriores a la primera dosis del tratamiento del estudio o durante las 5 vidas medias anteriores al producto en investigación, aquel periodo que sea más largo, o aquellos sujetos que esperen recibir cualquier fármaco o dispositivo en investigación durante el desarrollo del estudio.

E.5 End points

E.5.1

Primary end point(s)

Progression-free survival (PFS) based on local investigator assessment as per RECIST 1.1

Supervivencia libre de progresión (PFS) basándose en la evaluación del investigador local según RECIST 1.1

E.5.1.1

Timepoint(s) of evaluation of this end point

at primary analysis (once 50 subjects have experienced a PFS event) and at final analysis after completion of the study

En el análisis principal (una vez que un total de 50 sujetos hayan experimentado un acontecimiento de PFS) y en el análisis final tras la finalización del estudio

E.5.2

Secondary end point(s)

• Objective response rate (ORR), disease control rate (DCR), time-to-response (TTR) and duration of response (DOR) based on local investigator assessment as per RECIST 1.1 and overall survival (OS)
• Incidence and severity of AEs and SAEs, AEs leading to dose interruption, dose reduction and dose discontinuation
• Pharmacokinetic parameters and concentration
• Antidrug antibodies (ADA) prevalence at baseline and ADA incidence on treatment of pembrolizumab

- Tasa de respuesta objetiva (TRO), tasa de control de la enfermedad (DCR), tiempo hasta la respuesta (TTR) y duración de respuesta (DOR) basándose en la evaluación del investigador local según RECIST 1.1 y la supervivencia global (OS).
- incidencia e intensidad de los AA y AAG, AA que den lugar a la interrupción, la reducción y la discontinuación de la dosis.
- Caracterizar la farmacocinética (PK) midiendo los parámetros PK y la concentración
- Prevalencia de anticuerpos contra el fármaco (ADA) en la basal y la incidencia de ADA durante el tratamiento con pembrolizumab.

E.5.2.1

Timepoint(s) of evaluation of this end point

at primary analysis (once 50 subjects have experienced a PFS event) and at final analysis after completion of the study

En el análisis principal (una vez que un total de 50 sujetos hayan experimentado un acontecimiento de PFS) y en el análisis final tras la finalización del estudio

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)

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)

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

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

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

Belgium

Canada

Czech Republic

France

Germany

Greece

Hong Kong

India

Italy

Japan

Malaysia

Netherlands

Singapore

Spain

Taiwan

Thailand

United Kingdom

United States

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

The end of the study is defined as 30 months after the last subject’s date of randomization or when all enrolled subjects have died, withdrawn consent or been lost to follow up, whichever occurs earlier.
The disease and survival follow-up evaluations might not be completed if Novartis decides to stop enrollment prematurely. In such cases, end of study will be when the treatment period and the safety follow-up have been completed for all subjects.

30 meses después de la fecha de randomización del último paciente o cuando todos los pacientes hayan fallecido, retirado consentimiento o se haya perdido su seguimiento, lo que ocurra antes. Las evaluaciones de seguimiento de la enfermedad y de supervivencia podrían no completarse si NVS decide parar el reclutamiento prematuramente. Entonces, se considerará el final del estudio cuando se haya completado el periodo de tto y de seguimiento de seguridad para todos los pacientes.

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:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

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

2020-01-24

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-12-13

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2023-02-07

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