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    Summary
    EudraCT Number:2016-004586-67
    Sponsor's Protocol Code Number:CBYL719X2402
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-07-21
    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.1Member State ConcernedFrance - ANSM
    A.2EudraCT number2016-004586-67
    A.3Full title of the trial
    A phase II, multicenter, open-label, two-cohort, noncomparative study to assess the efficacy and safety of alpelisib plus fulvestrant or letrozole in patients with PIK3CA mutant, hormone receptor (HR) positive, HER2-negative advanced breast cancer (aBC), who have progressed on or after CDK 4/6 inhibitor treatment
    Étude de phase II non comparative, multicentrique, en ouvert, à deux bras de traitement, évaluant l’efficacité et l’innocuité de
    l’alpelisib associé au fulvestrant ou au létrozole chez des patients atteints de cancer du sein à un stade avancé avec mutation du gène PIK3CA, récepteurs hormonaux positifs (RH+) et HER2 négatif, ayant progressé pendant ou après un traitement par inhibiteur de CDK 4/6
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Efficacy and safety of treatment with alpelisib plus endocrine therapy in patients with HR+, HER2- negative aBC, with PIK3CA mutations, whose disease has progressed on or after CDK 4/6 treatment with an aromatase inhibitor (AI) or fulvestrant
    Efficacité et innocuité du traitement avec l'alpelisib associé à la thérapie endocrinienne chez des patients avec RH+, HER2 négatif atteints de cancer du sein à un stade avancé avec mutation du gène PIK3CA, dont la maladie a progressé pendant ou après le traitement CDK 4/6 avec un inhibiteur de l'aromatase (IA) ou fulvestrant
    A.3.2Name or abbreviated title of the trial where available
    BYLieve
    A.4.1Sponsor's protocol code numberCBYL719X2402
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorNovartis Pharma AG
    B.1.3.4CountrySwitzerland
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportNovartis Pharma AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Pharma S.A.S
    B.5.2Functional name of contact pointInformation&Communication Médicales
    B.5.3 Address:
    B.5.3.1Street Address2 et 4 rue Lionel Terray
    B.5.3.2Town/ cityRueil-Malmaison
    B.5.3.3Post code92500
    B.5.3.4CountryFrance
    B.5.4Telephone number+33 1 5547 6600
    B.5.5Fax number+33 1 5547 6100
    B.5.6E-mailicm.phfr@novartis.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameAlpelisib
    D.3.2Product code BYL719
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNALPELISIB
    D.3.9.1CAS number 1217486-61-7
    D.3.9.2Current sponsor codeBYL719
    D.3.9.4EV Substance CodeSUB180707
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameAlpelisib
    D.3.2Product code BYL719
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNALPELISIB
    D.3.9.1CAS number 1217486-61-7
    D.3.9.2Current sponsor codeBYL719
    D.3.9.4EV Substance CodeSUB180707
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 3
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameFulvestrant
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNFulvestrant
    D.3.9.1CAS number 129453-61-8
    D.3.9.3Other descriptive nameFULVESTRANT
    D.3.9.4EV Substance CodeSUB13933MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 4
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameLetrozole
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNLETROZOLE
    D.3.9.1CAS number 112809-51-5
    D.3.9.4EV Substance CodeSUB08444MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2.5
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another 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.1.1Medical condition(s) being investigated
    Hormone receptor positive, HER2-negative advanced breast cancer
    Cancer du sein à un stade avancé avec récepteurs hormonaux positifs et HER2 négatif
    E.1.1.1Medical condition in easily understood language
    breast cancer
    Cancer du sein
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10072737
    E.1.2Term Advanced breast cancer
    E.1.2System Organ Class 100000020819
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the proportion of patients who are alive without disease progression at 6 months based on local investigator assessment per RECIST v1.1 in cohort A (alpelisib in combination with fulvestrant) and cohort B (alpelisib in combination letrozole) among patients with HR+, HER2-negative aBC harboring a PIK3CA mutation who have progressed on or after CDK 4/6 inhibitor combination with an AI or fulvestrant
    Evaluer la proportion de patients en vie et sans progression de la maladie au bout de 6 mois d’après l’évaluation du médecin-investigateur selon les critères RECIST v1.1 dans le groupe A (alpelisib associé au fulvestrant) et dans le groupe B (alpelisib associé au létrozole) parmi les patients atteints de CSa avec mutation de PIK3CA, RH+, HER2- et ayant progressé pendant ou après un traitement par un inhibiteur de CDK 4/6 associé à un IA ou au fulvestrant.
    E.2.2Secondary objectives of the trial
    To assess PFS based on local investigator assessment for each cohort

    To assess PFS on next-line treatment (PFS2) for each cohort

    To assess overall response rate (ORR) and clinical benefit rate (CBR) based on local investigator assessment for each cohort

    To assess duration of response (DOR) in patients with confirmed complete response (CR) or PR for each cohort.

    To evaluate the safety and tolerability of the combination for each cohort
    Evaluer la survie sans progression (SSP) d’après l’évaluation du médecininvestigateur pour chaque groupe.

    Evaluer la SSP sous la ligne suivante de traitement (SSP2) pour chaque groupe.

    Evaluer le taux de réponse objective (RO) et le taux de bénéfice Clinique (TBC) d’après l’évaluation du médecin-investigateur pour chaque groupe.

    Evaluer la durée de réponse globale (DRG) chez les patients ayant une réponse complète (RC) ou partielle (RP) au traitement confirmée pour chaque groupe.

    Evaluer l’innocuité et la tolérance de l’association pour chaque groupe.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Patient is male or female 18 years or older
    - Patient is identified PIK3CA mutant status
    - Patient has confirmed HER2-negative advanced breast cancer (aBC)
    -Patient must be diagnosed with aBC with documented progression on or after CDK 4/6 treatment (adjuvant or metastatic setting)
    - Patient has histological and/or cytological confirmed ER+ and/or PgR+ aBC
    - Patient has either measurable disease per RECIST v1.1 or at least one predominantly lytic bone lesion must be present
    - ECOG function of greater or equal to 2
    - Patient has adequate bone marrow function
    - Patient has adequate liver and renal function
    - Patient homme ou femme de 18 ans ou plus
    - Patient ayant une mutation du gène PIK3CA
    - Patient atteint d'un cancer du sein avancé (CSa) négatif pour HER2 confirmé
    - Le CSa avec progression documentée doit être diagnostiqué pendant ou après un traitement par inhibiteur de CDK 4/6 (comme traitement adjuvant ou comme traitement des metastases)
    - Patient ayant un diagnostic de cancer du sein positif pour les récepteurs aux oestrogènes et/ou à la progestérone confirmé histologiquement et/ou cytologiquement (RH+)
    - Patient ayant une maladie mesurable d’après les critères RECIST v1.1 ou au moins une lésion osseuse à prédominance lytique devra être présente
    - Indice de performance ECOG supérieur ou égal à 2
    - Patient ayant une fonction de la moelle osseuse adequate
    - Patient ayant une fonction hépatique et rénale adéquate
    E.4Principal exclusion criteria
    - patient has received prior treatment with any PI3K inhibitors
    - patient with clinically manifest diabetes mellitus, or documented steroid induced diabetes mellitus
    -Patient has a concurrent malignancy or malignancy within 3 years of study screening period, with the exception of adequately treated, basal or squamous cell carcinoma, non-melanoma skin cancer or curatively resected cervical cancer
    -Patient has received radiotherapy ≤ 4 weeks or limited field radiation for palliation ≤ 2 weeks prior to enrollment, and who has not recovered to grade 1 or better from related side effects of such therapy
    -History of acute pancreatitis within 1 year of screening or past medical history of pancreatitis
    -Bilateral diffuse lymphangitis carcinomatosis
    -Patients with central nervous system (CNS) involvement unless they meet ALL of the following criteria:
    • At least 4 weeks from prior therapy completion (including radiation and/or surgery) to starting the study treatment
    • Clinically stable CNS tumor at the time of screening untreated or without evidence of progressions for at least 4 weeks after treatment as
    determined by clinical examination and brain imaging (MRI or CT) during screening period and stable low dose of steroids for 2 weeks prior to initiating study treatment
    -Patient with severe liver impairment (Child Pugh score B/C)
    -Patient has impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of the study drugs
    -Patient has documented pneumonitis which is active and requiring treatment
    - Patient a reçu un traitement antérieur par un inhibiteur de PI3K, quel qu’il soit
    - Patient atteint de diabète manifeste ou diabète induit par corticostéroïdes documenté
    - Patient a un cancer simultané ou cancer survenu dans les 3 ans avant la période de sélection, à l’exception des cancers suivants correctement traités : carcinome à cellules basales ou squameuses, cancer de la peau non mélanomateux, ou cancer du col de l’utérus réséqué
    - Patient a reçu de la radiothérapie dans les 4 semaines ou radiothérapie palliative à champ limité dans les 2 semaines précédant l’inclusion, et absence de récupération jusqu’à un grade ≤ 1 des effets indésirables de cette thérapie
    - Antécédent de pancréatite aiguë dans l’année précédant la sélection ou antécédents de pancréatite
    - Carcinomatose diffuse lymphangitique bilatérale
    - Patient atteint du système nerveux central (SNC) sauf si le patient remplit tous les critères suivants :
    • Précédent traitement (y compris radiothérapie et/ou chirurgie) au moins 4 semaines avant de commencer le traitement à l’étude
    • Tumeur du SNC stable au moment de la sélection, non traitée ou sans signe de progression pendant au moins 4 semaines après son traitement comme déterminé par l’examen clinique et l’imagerie du cerveau (IRM ou scanner) pendant la période de sélection et par la prise de corticostéroïdes à faible doses stables pendant les 2 semaines précédant le début du traitement à l’étude.
    - Patient avec dysfonctionnement sévère du foie (Score de Child Pugh B/C)
    - Patient avec dysfonctionnement ou maladie gastro-intestinale qui pourrait significativement altérer l’absorption des traitements à l’étude
    - Patient ayant une pneumonite documentée au moment de la sélection qui est active et nécessite un traitement
    E.5 End points
    E.5.1Primary end point(s)
    The percentage of patients who are alive without disease progression
    Le pourcentage de patients en vie sans progression de la maladie
    E.5.1.1Timepoint(s) of evaluation of this end point
    Date of first dose to approximately 6 months
    Date de la première dose à environ 6 mois
    E.5.2Secondary end point(s)
    Progression free survival (PFS) for each cohort

    Progression free survival (PFS) on next line treatment PFS2) for each cohort

    Percentage of participants Overall response rate (ORR) for each cohort

    Percentage of participants with clinical benefit rate (CBR) for each cohort

    Duration of response (DOR)

    Further secondary objectives and details are described in the protocol
    Survie sans progression (SSP) pour chaque groupe

    Survie sans progression (SSP) sous la ligne suivante de traitement (SSP2) pour chaque groupe

    Taux de réponse objective (RO) pour chaque groupe

    Taux de bénéfice Clinique (TBC) pour chaque groupe

    Durée de réponse globale (DRG)

    D'autres objectifs secondaires et détails sont décrits dans le protocole
    E.5.2.1Timepoint(s) of evaluation of this end point
    date of first dose to up to approximately 25 months

    Date of first dose to date of first documented progression up to approximately 25 months

    Date of first dose and up to approximately 25 months

    Date of first dose and up to approximately 25 months

    Date of first documented response to first documented progression or death up to approximately 25 months
    Date de la première dose jusqu'à environ 25 mois

    Date de la première dose jusqu'au jour de la première progression documentée jusqu'à environ 25 mois

    Date de la première dose et jusqu'à environ 25 mois

    Date de la première dose et jusqu'à environ 25 mois

    Date de la première réponse documentée jusqu'à la première progression documentée ou à la mort jusqu'à environ 25 mois
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA31
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Argentina
    Belgium
    Bulgaria
    Canada
    Chile
    France
    Germany
    India
    Israel
    Italy
    Japan
    Mexico
    Netherlands
    Singapore
    Spain
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    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 primary analysis for each cohort will be performed when all patients included in the corresponding cohort experienced progression, discontinued early or 6 months after the last patient has started treatment. The final analysis will be performed 18 months after the last patient has started treatment. Unless otherwise mentioned, all analyses will be performed separately for each cohort. This applies to all countries
    L’analyse principale pour chaque groupe sera réalisée quand tous les patients inclus dans ce groupe auront une progression de leur maladie, auront arrêté prématurément l’étude ou auront terminé 6 mois dans l’étude. L’analyse finale sera réalisée quand tous les patients auront arrêté prématurément l’étude avant 18 mois ou auront terminé 18 mois dans l’étude. Sauf mention contraire, toutes les analyses seront faites séparément pour chaque groupe. Cela s'applique à tous les pays
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 30
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 130
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state23
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 84
    F.4.2.2In the whole clinical trial 160
    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)
    If alpelisib is not commercially available and reimbursed in a participating country by the time the study is completed (18 months after LPFV), Novartis will have a transition plan in place to ensure that patients have equivalent access without delays in treatment.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2017-07-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-06-20
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2024-11-12
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