Clinical Trials Register

Summary
EudraCT Number:	2019-002660-27
Sponsor's Protocol Code Number:	CINC280I12201
National Competent Authority:	France - ANSM
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2019-11-13
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

France - ANSM

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%

Etude de phase II, randomisée, en ouvert, multicentrique, évaluant l’efficacité et l’innocuité du capmatinib (INC280) associé au pembrolizumab versus pembrolizumab en première ligne chez des patients ayant un cancer bronchique non à petites cellules localement avancé ou métastatique, avec surexpression de 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

Etude du capmatinib et du pembrolizumab dans le cancer du poumon

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 Pharma AG
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	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 Pharma S.A.S
B.5.2	Functional name of contact point	Information&Communication Médicales
B.5.3	Address:
B.5.3.1	Street Address	8/10 rue Henry Sainte Claire Deville, CS 40150
B.5.3.2	Town/ city	Rueil-Malmaison
B.5.3.3	Post code	92563
B.5.3.4	Country	France
B.5.4	Telephone number	+33 1 5547 6600
B.5.5	Fax number	+33 1 5547 6100
B.5.6	E-mail	icm.phfr@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

cancer bronchique non à petites cellules

E.1.1.1

Medical condition in easily understood language

lung cancer

cancer du poumon

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

Evaluer l’efficacité du capmatinib + pembrolizumab par rapport au pembrolizumab seul

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

• Evaluer l’effet antitumoral du capmatinib + pembrolizumab par rapport au pembrolizumab seul
• Caractériser le profil de tolérance du capmatinib + pembrolizumab et du pembrolizumab seul
• Caractériser le profil PK du capmatinib et du pembrolizumab
• Evaluer la prévalence et l’incidence de l’immunogénicité du 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

• Patient atteint de CBNPC confirmé histologiquement, avec documentation, localement avancé de stade III (inéligible pour une résection chirurgicale ou une chimioradiothérapie à visée curative) ou métastatique (stade IV) selon les critères AJCC/IASLC v8, et candidat à un traitement de première intention.
- Les traitements par chimiothérapie et/ou radiothérapie adjuvante ou néo-adjuvante (autres que les immunothérapies) sont autorisés à condition que ce traitement ait été terminé au moins 6 mois avant le diagnostic de CBNPC avancé ou métastatique.
• Diagnostic de CBNPC confirmé histologiquement ou cytologiquement, qui présente un statut EGFR non muté et qui ne présente pas de réarrangement d’ALK :
- Les patients atteints de CBNPC purement épidermoïde peuvent commencer le processus de sélection sans tests ou résultats pour les statuts d’EGFR et d’ALK. Cependant, les patients atteints de CBNPC purement épidermoïde ayant des mutations activatrices d’EGFR connues (par ex. des mutations dans les exons 19, 20 ou 21) ou un réarrangement d’ALK seront exclus.
• Un échantillon tumoral archivé ou un nouvel échantillon tumoral prélevé par biopsie qui présente un niveau élevé d’expression de PD-L1 (TPS ≥ 50 %), déterminé dans un laboratoire local ou un laboratoire central désigné par Novartis par immunohistochimie à l’aide du test PD-L1 22C3 pharmDx approuvé par les Autorités de Santé américaines.
- L’échantillon archivé doit être le bloc FFPE disponible le plus récent ou des coupes de tissus issues de ce bloc. Les coupes de tissus ne doivent PAS dater de plus de 5 mois après la coupe. Les échantillons archivés prélevés avant tout traitement systémique (tels que des traitements adjuvants) ne sont PAS acceptables).
- Si les résultats de l’expression de PD-L1, comme spécifié ci-dessus, ne sont pas disponibles localement, un nouvel échantillon tumoral ou un échantillon archivé sera nécessaire pour confirmer l’éligibilité du patient.
• Indice de performance ECOG ≤ 1.
• Présence d’au moins une lésion mesurable selon les critères RECIST v1.1. Une lésion précédemment irradiée peut être considérée comme une lésion mesurable uniquement en cas de signes évidents de progression depuis l’irradiation.

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. Patients ayant précédemment reçu un inhibiteur de MET ou une thérapie ciblée contre HGF.
2. Patients ayant précédemment reçu une immunothérapie (par ex. anticorps anti-PD-1, anti-PD-L1, anti-PD-L2, anti-CTLA-4, ou tout autre anticorps ou thérapie ciblant les voies de co-activation ou de checkpoints des lymphocytes T).
3. Antécédents connus d’hypersensibilité à tout excipient du capmatinib (crospovidone, mannitol, cellulose microcristalline, povidone, sodium lauryl sulfate, stéarate de magnésium, dioxyde de silicium colloïdal et divers composants d’enrobage).
4. Antécédents de réactions d’hypersensibilité sévères à d’autres anticorps monoclonaux, qui pourrait augmenter le risque de réaction sévère à la perfusion.
5. Métastases symptomatiques non traitées du système nerveux central. Les patients sont éligibles si ces métastases ont été traitées de manière adéquate par radiothérapie ou chirurgie et si elles sont stables depuis > 2 semaines après le traitement. Les patients ne doivent plus recevoir de corticoïdes depuis au moins 7 jours avant le début du traitement à l’étude.
6. Patients atteints d’un cancer ou ayant des antécédents de cancer autre que le CBNPC qui a été diagnostiquée et/ou a nécessité un traitement dans les 3 ans précédant l’inclusion dans l’étude, à l’exception des carcinomes basocellulaires et épidermoïdes de la peau complètement réséqués et de tous les types de carcinomes in situ complètement réséqués.
7. Présence ou antécédents de pneumonite ou de pneumopathie interstitielle, y compris une pneumopathie radio-induite cliniquement significative (c’est-à-dire affectant les activités quotidiennes ou nécessitant un traitement).
8. Maladie cardiaque non contrôlée, cliniquement significative.
9. Radiothérapie palliative pour des lésions osseuses pratiquée dans les 2 semaines précédant le début du traitement à l’étude.
10. Chirurgie majeure (par ex. intra-thoracique, intra-abdominale ou intra-pelvienne) dans les 4 semaines précédant le début du traitement à l’étude, ou patient n’ayant pas récupéré des effets indésirables de cette chirurgie. Les chirurgies thoraciques assistées par vidéo et les médiastinoscopies ne sont pas considérées comme des chirurgies majeures et les patients peuvent être inclus dans l’étude ≥ 1 semaine après la procédure.
11. Troubles ou maladies gastro-intestinaux qui pourraient altérer significativement l’absorption du capmatinib (par ex. maladies ulcéreuses, nausées non contrôlées, vomissements, diarrhées ou syndrome de malabsorption).
12. Traitement(s) concomitant(s) ayant un risque connu de Torsades de pointes selon www.qtdrugs.org, qui ne peuvent être interrompus ou remplacés par un autre traitement plus sûr.
13. Patients recevant des inducteurs forts de CYP3A4 qui ne peuvent être interrompus au moins 1 semaine avant le début du traitement par capmatinib et pendant toute la durée de l’étude.
14. Traitement chronique systémique par des corticoïdes (> 10 mg/jour de prednisone ou équivalent) ou tout autre traitement immunosuppresseur dans les 7 jours précédant la première dose du traitement à l’étude. Les traitements topiques, inhalés, par voie ophtalmique et nasale, les minéralcorticoïdes (par ex. fludrocortisone) pour les patients atteints d’hypotension orthostatique ou les faibles doses de corticoïdes de remplacement pour une insuffisance surrénale sont autorisés.
15. Patients ayant participé à une autre étude clinique (évaluant un médicament ou un dispositif médical) dans les 30 jours précédant le début du traitement à l’étude ou ayant reçu un médicament expérimental dans une période correspondant à 5 demi-vies du traitement expérimental, selon la durée la plus longue, ou patients devant recevoir tout autre médicament ou dispositif médical expérimental au cours de l’étude

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

Survie sans progression (SSP) selon l’évaluation du médecin-investigateur selon les critères RECIST v1.1

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

au moment de l'analyse principal (lorsque 50 patients ont eu un événement de SSP) et à la fin de l'étude

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

• Taux de réponse objective (TRO), taux de contrôle de la maladie (TCM), temps de réponse (TR) et durée de la réponse (DR) selon l’évaluation du médecin-investigateur selon les critères RECIST v1.1, et survie globale (SG)
• Incidence et sévérité des effets indésirables, des effets indésirables graves, des effets indésirables conduisant à des interruptions de traitement, réductions de dose et arrêts du traitement
• Paramètres PK et concentrations sanguines
• Présence d’anticorps dirigés contre le pembrolizumab à la baseline et incidence au cours du traitement par 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

au moment de l'analyse principal (lorsque 50 patients ont eu un événement de SSP) et à la fin de l'étude

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.

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

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

2020-01-10

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-12-11

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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