Clinical trial • Phase II/III • Haematology|Rare Disease
ALPELISIB for Lymphatic malformation associated with PIK3CA mutation|Lymphangioma
Phase II/III trial of ALPELISIB for Lymphatic malformation associated with PIK3CA mutation|Lymphangioma.
Overview
- Trial Therapeutic Area
- Haematology|Rare Disease
- Trial Disease
- Lymphatic malformation associated with PIK3CA mutation|Lymphangioma
- Trial Stage
- Phase II/III
- Drug Modality
- Small molecule
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 04-09-2023
- First CTIS Authorization Date
- 10-01-2024
Trial design
Randomised, open-label, placebo to byl719 (matching placebo to byl719 50 mg, 125 mg, 200 mg film-coated tablets) used in the double-blind, placebo-controlled confirmatory phase (stage 2 groups 3 and 4)., adaptive Phase II/III trial in France, Netherlands, Germany and others.
- Randomised
- Yes
- Open Label
- Yes
- Comparator
- Placebo to BYL719 (matching placebo to BYL719 50 mg, 125 mg, 200 mg film-coated tablets) used in the double-blind, placebo-controlled confirmatory phase (Stage 2 groups 3 and 4).
- Adaptive
- True, Stage 1 is a dose selection stage (select the dose(s) for the confirmatory phase, DSCP) and is used to identify dose(s) for Stage 2; Stage 2 includes blinded placebo-controlled confirmatory phase with switching of placebo participants to active treatment at Week 24.
- Biomarker Stratified
- True, biomarker: PIK3CA mutation (participants must have evidence of a somatic PIK3CA mutation prior to randomization).
- Single Multiple Or Escalation Dose Combined
- Yes
- Target Sample Size
- 162
- Trial Duration For Participant
- 168
Eligibility
Recruits 162 paediatric patients.
- Vulnerable Population
- Vulnerable populations include pediatric participants (age groups 0-5, 6-17). Consent/assent handling: "Signed informed consent and assent (when applicable) from the participant, parent, legal authorized representative or guardian." Age-specific informed consent and assent forms are provided (Main ICF - Adult, Parent Legal Guardian ICFs, Child Assent, Adolescent Assent). Documents available in multiple languages per country-specific submissions.
Inclusion criteria
- {"criterion_text":"- Signed informed consent and assent (when applicable) from the participant, parent, legal authorized representative or guardian."}
- {"criterion_text":"- Participant must be willing to remain at the clinical site as required by the protocol and to adhere to study restrictions and examination schedules."}
- {"criterion_text":"- Participant has a physician confirmed and documented diagnosis of a symptomatic LyM at the time of informed consent (Note: the physician must confirm that the LyM cannot be included under the PROS diagnostic criteria)."}
- {"criterion_text":"- Participant is not considered as a candidate for or is not willing to receive non-drug therapies including but not limited to sclerotherapy, embolization, and surgery until the completion of Week 24 at the time of informed consent."}
- {"criterion_text":"- Participant has evidence of a somatic mutation(s) in the PIK3CA gene prior to randomization."}
- {"criterion_text":"- Participant has at least one measurable LyM lesion confirmed by BIRC assessment prior to randomization."}
- {"criterion_text":"- Participants must be able to ingest study drug in tablet or as an oral suspension (Groups 1 to 4) or granules or as an oral suspension (Group 5) as assessed within 7 days before study treatment start. Drug administration via feeding tubes is allowed."}
Exclusion criteria
- {"criterion_text":"- Participant has a physician-confirmed and documented diagnosis of PROS at the time of informed consent."}
- {"criterion_text":"- Participant has a physician-confirmed and documented diagnosis of a Central Conducting Lymphatic Anomaly, General Lymphatic Anomaly, Gorham-Stout disease, Kaposiform lymphangiomatosis at the time of informed consent."}
- {"criterion_text":"- Participant has a known history of Stevens-Johnson syndrome, erythema multiforme, or toxic epidermal necrolysis at the time of informed consent."}
- {"criterion_text":"- Participant has an established diagnosis of type I diabetes mellitus or uncontrolled type II diabetes mellitus at the time of informed consent."}
- {"criterion_text":"- Participant had previous treatment with alpelisib and/or any other PI3K inhibitors with treatment duration longer than 2 weeks at the time of informed consent."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Response (yes/no) at Week 24 (confirmatory phase) of Stage 2, defined by achieving at least 20% reduction in the sum of target lesion volumes (1 to 3 lesions), assessed by MRI by a blinded independent review committee (BIRC) at Week 24, provided that none of the individual target lesions has at least 20% increase from baseline and in absence of progression of non-target lesions and without new lesions.","definition_or_measurement_approach":"Radiological response assessed by MRI by a Blinded Independent Review Committee (BIRC) at Week 24; response defined as ≥20% reduction in sum of target lesion volumes (1-3 lesions) with no individual target lesion showing ≥20% increase versus baseline, no progression of non-target lesions, and no new lesions."}
Secondary endpoints
- {"endpoint_text":"- Response (yes/no) at Week 24 (confirmatory phase) of Stage 2 defined by achieving at least a 1-point improvement compared to baseline on the patient global impression of severity (PGI-S) scale.","definition_or_measurement_approach":"Patient-reported outcome: PGI-S scale; response = ≥1-point improvement from baseline at Week 24 in the confirmatory phase of Stage 2."}
- {"endpoint_text":"- Response at Week 24 of Stage 2, defined by achieving at least 20% reduction in the sum of target lesion volumes (1 to 3 lesions), assessed by MRI by a BIRC at Week 24, provided that none of the individual target lesions has at least 20% increase from baseline and in absence of progression of non-target lesions and without new lesions.","definition_or_measurement_approach":"Radiological response assessed by MRI by a BIRC at Week 24; same definition as primary radiological response (≥20% reduction in sum of target lesion volumes with no individual lesion ≥20% increase, no progression of non-target lesions, no new lesions)."}
Recruitment
- Planned Sample Size
- 162
- Recruitment Window Months
- 112
- Consent Approach
- Informed consent is obtained from the participant or from a parent/legal authorised representative or guardian as applicable; assent is obtained when applicable. Age-specific ICFs and assent forms are provided (Main ICF - Adult; Parent Legal Guardian ICF; Child Assent; Adolescent Assent). Country- and language-specific ICFs are available (documents in English, French, Italian, Spanish, German, Dutch as indicated in submission documents).
Geography
- Total Number Of Sites
- 37
- Total Number Of Participants
- 162
France
- Earliest CTIS Part Ii Submission Date
- 11-12-2023
- Latest Decision Or Authorization Date
- 22-01-2026
- Processing Time Days
- 773
- Number Of Sites
- 14
- Number Of Participants
- 57
Sites
- Site Name
- Assistance Publique Hopitaux De Marseille
- Department Name
- 3105: CHU Timone, Département de Dermatologie
- Principal Investigator Name
- Stéphanie Mallet
- Principal Investigator Email
- Stephanie.MALLET@ap-hm.fr
- Contact Person Name
- Stéphanie Mallet
- Contact Person Email
- Stephanie.MALLET@ap-hm.fr
- Site Name
- Hospices Civils De Lyon
- Department Name
- 3101: Hôpital Femme Mère Enfant Département Médical
- Principal Investigator Name
- Laurent Guibaud
- Principal Investigator Email
- laurent.guibaud@chu-lyon.fr
- Contact Person Name
- Laurent Guibaud
- Contact Person Email
- laurent.guibaud@chu-lyon.fr
- Site Name
- Centre Hospitalier Universitaire De Toulouse
- Department Name
- 3109: Département de médecine vasculaire - Hôpital Rangueil
- Principal Investigator Name
- Julie Malloizel-Delaunay
- Principal Investigator Email
- Malloizel-delaunay.j@chu-toulouse.fr
- Contact Person Name
- Julie Malloizel-Delaunay
- Contact Person Email
- Malloizel-delaunay.j@chu-toulouse.fr
- Site Name
- Pellegrin Hospital
- Department Name
- 3102: Unité de Dermatologie Pédiatrique
- Principal Investigator Name
- Christine Labrèze
- Principal Investigator Email
- christine.labreze@chu-bordeaux.fr
- Contact Person Name
- Christine Labrèze
- Contact Person Email
- christine.labreze@chu-bordeaux.fr
- Site Name
- Centre Hospitalier Regional Universitaire De Tours
- Department Name
- 3100: Hôpital Clocheville, Département de Médecine Pédiatrique
- Principal Investigator Name
- Annabel Maruani
- Principal Investigator Email
- annabel.maruani@univ-tours.fr
- Contact Person Name
- Annabel Maruani
- Contact Person Email
- annabel.maruani@univ-tours.fr
- Site Name
- Centre Hospitalier Universitaire D'Angers
- Department Name
- 3111: Département Dermatologie
- Principal Investigator Name
- Ludovic MARTIN
- Principal Investigator Email
- Lumartin@chu-angers.fr
- Contact Person Name
- Ludovic MARTIN
- Contact Person Email
- Lumartin@chu-angers.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- 3103: Hôpital Lariboisière, Département de Neuroradiologie
- Principal Investigator Name
- Annouk Bisdorff
- Principal Investigator Email
- annouk.bisdorff@aphp.fr
- Contact Person Name
- Annouk Bisdorff
- Contact Person Email
- annouk.bisdorff@aphp.fr
- Site Name
- Hopital Necker Enfants Malades
- Department Name
- 3104: Département Néphrologie Adultes
- Principal Investigator Name
- Guillaume Canaud
- Principal Investigator Email
- Guillaume.canaud@inserm.fr
- Contact Person Name
- Guillaume Canaud
- Contact Person Email
- Guillaume.canaud@inserm.fr
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- 3106: Institut Coeur Poumon, Département Physiologie Cardio Vasculaire
- Principal Investigator Name
- Caroline Chopinet
- Principal Investigator Email
- Caroline.CHOPINET@chu-lille.fr
- Contact Person Name
- Caroline Chopinet
- Contact Person Email
- Caroline.CHOPINET@chu-lille.fr
- Site Name
- Centre Hospitalier Universitaire De Dijon
- Department Name
- 3110: Département Génétique Médicale
- Principal Investigator Name
- Laurence Olivier-Faivre
- Principal Investigator Email
- Laurence.faivre@chu-djon.fr
- Contact Person Name
- Laurence Olivier-Faivre
- Contact Person Email
- Laurence.faivre@chu-djon.fr
- Site Name
- Hopital Saint Eloi
- Department Name
- 3107: Service de Médecine Vasculaire
- Principal Investigator Name
- Isabelle Quere
- Principal Investigator Email
- i-quere@chu-montpellier.fr
- Contact Person Name
- Isabelle Quere
- Contact Person Email
- i-quere@chu-montpellier.fr
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- 3106: Sérvice d’ORL Pédiatrique Hôpital Jeanne de Flandre
- Principal Investigator Name
- Caroline Chopinet
- Principal Investigator Email
- Caroline.CHOPINET@chu-lille.fr
- Contact Person Name
- Caroline Chopinet
- Contact Person Email
- Caroline.CHOPINET@chu-lille.fr
- Site Name
- Centre Hospitalier Universitaire De Caen Normandie
- Department Name
- 3108: Département Dermatologie
- Principal Investigator Name
- Anne Dompmartin
- Principal Investigator Email
- dompmartin-a@chu-caen.fr
- Contact Person Name
- Anne Dompmartin
- Contact Person Email
- dompmartin-a@chu-caen.fr
- Site Name
- Centre Hospitalier Universitaire De Toulouse
- Department Name
- 3109: Département Dermatologie
- Principal Investigator Name
- Julie Malloizel-Delaunay
- Principal Investigator Email
- Malloizel-delaunay.j@chu-toulouse.fr
- Contact Person Name
- Julie Malloizel-Delaunay
- Contact Person Email
- Malloizel-delaunay.j@chu-toulouse.fr
Netherlands
- Earliest CTIS Part Ii Submission Date
- 03-04-2024
- Latest Decision Or Authorization Date
- 13-03-2026
- Processing Time Days
- 709
- Number Of Sites
- 2
- Number Of Participants
- 18
Sites
- Site Name
- Stichting Radboud University Medical Center
- Department Name
- #3400: Department of pediatrics
- Principal Investigator Name
- Bas Henk Verhoeven
- Principal Investigator Email
- bas.verhoeven@radboudumc.nl
- Contact Person Name
- Bas Henk Verhoeven
- Contact Person Email
- bas.verhoeven@radboudumc.nl
- Site Name
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Department Name
- #3401: Department of pediatric dermatology
- Principal Investigator Name
- E.M. (Elyse) Verboom
- Principal Investigator Email
- e.verboom@erasmusmc.nl
- Contact Person Name
- E.M. (Elyse) Verboom
- Contact Person Email
- e.verboom@erasmusmc.nl
Germany
- Earliest CTIS Part Ii Submission Date
- 28-11-2023
- Latest Decision Or Authorization Date
- 29-04-2026
- Processing Time Days
- 883
- Number Of Sites
- 6
- Number Of Participants
- 21
Sites
- Site Name
- Universitaet Leipzig
- Department Name
- 3201:Klinik und Poliklinik für Kinder- und Jugendmedizin
- Principal Investigator Name
- Susanne Wendt
- Principal Investigator Email
- Susanne.wendt@medizin.uni-leipzig.de
- Contact Person Name
- Susanne Wendt
- Contact Person Email
- Susanne.wendt@medizin.uni-leipzig.de
- Site Name
- Universitaetsklinikum Ulm AöR
- Department Name
- 3206: HNO-Klinik, Kopf- und Halschirurgie
- Principal Investigator Name
- Adrian von Witzleben
- Principal Investigator Email
- Adrian.vonWitzleben@uniklinik-ulm.de
- Contact Person Name
- Adrian von Witzleben
- Contact Person Email
- Adrian.vonWitzleben@uniklinik-ulm.de
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- 3205: Klinik für Pädiatrie mit Schwerpunkt Hämatologie/ Onkologie
- Principal Investigator Name
- Susanne Holzhauer
- Principal Investigator Email
- Susanne.holzhauer@charite.de
- Contact Person Name
- Susanne Holzhauer
- Contact Person Email
- Susanne.holzhauer@charite.de
- Site Name
- Universitat Heidelberg
- Department Name
- 3202:Klinik für Radiologie und Nuklearmedizin
- Principal Investigator Name
- Maliha Sadick
- Principal Investigator Email
- Maliha.sadick@medma.uni-heidelberg.de
- Contact Person Name
- Maliha Sadick
- Contact Person Email
- Maliha.sadick@medma.uni-heidelberg.de
- Site Name
- Medical Center - University Of Freiburg
- Department Name
- 3200: Zentrum für Kinder- und Jugendmedizin
- Principal Investigator Name
- Friedrich Kapp
- Principal Investigator Email
- Friedrich.kapp@uniklinik-freiburg.de
- Contact Person Name
- Friedrich Kapp
- Contact Person Email
- Friedrich.kapp@uniklinik-freiburg.de
- Site Name
- University Hospital Cologne AöR
- Department Name
- 3204:Klinik und Poliklinik für Kinder- und Jugendmedizin
- Principal Investigator Name
- Boris Decarolis
- Principal Investigator Email
- boris.decarolis@uk-koeln.de
- Contact Person Name
- Boris Decarolis
- Contact Person Email
- boris.decarolis@uk-koeln.de
Italy
- Earliest CTIS Part Ii Submission Date
- 21-11-2023
- Latest Decision Or Authorization Date
- 13-05-2026
- Processing Time Days
- 904
- Number Of Sites
- 6
- Number Of Participants
- 21
Sites
- Site Name
- Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico
- Department Name
- 3302: UOC Dermatologia Università degli Studi
- Principal Investigator Name
- Iria Neri
- Principal Investigator Email
- iria.neri@aosp.bo.it
- Contact Person Name
- Iria Neri
- Contact Person Email
- iria.neri@aosp.bo.it
- Site Name
- Ospedale Pediatrico Bambino Gesu'
- Department Name
- 3300: UOC Trials (Pad. Salviati 1° piano)
- Principal Investigator Name
- Andrea Diociaiuti
- Principal Investigator Email
- andrea.diociaiuti@opbg.ne
- Contact Person Name
- Andrea Diociaiuti
- Contact Person Email
- andrea.diociaiuti@opbg.ne
- Site Name
- L’Azienda Ospedaliera Di Rilievo Nazionale Santobono-Pausilipon
- Department Name
- 3303: UOC Pediatria Generale e Dermo-Immuno- Reumatologia
- Principal Investigator Name
- Luigi Martemucci
- Principal Investigator Email
- lmartemucci230@gmail.com
- Contact Person Name
- Luigi Martemucci
- Contact Person Email
- lmartemucci230@gmail.com
- Site Name
- Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
- Department Name
- 3305: Unità di Genetica Clinica Pediatrica
- Principal Investigator Name
- Alessandro Mussa
- Principal Investigator Email
- Alessandro.mussa@unito.it
- Contact Person Name
- Alessandro Mussa
- Contact Person Email
- Alessandro.mussa@unito.it
- Site Name
- Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
- Department Name
- 3304: S.S.D. Dermatologia Pediatrica Università degli Studi
- Principal Investigator Name
- Riccardo Cavalli
- Principal Investigator Email
- riccardo.cavalli@policlinico.mi.it
- Contact Person Name
- Riccardo Cavalli
- Contact Person Email
- riccardo.cavalli@policlinico.mi.it
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- 3301: UOSA Malattie Rare di Transizione Dip. Salute della Donna del Bambino e Sanità Pubblica
- Principal Investigator Name
- Chiara Leoni
- Principal Investigator Email
- chiara.leoni@policlinicogemelli.it
- Contact Person Name
- Chiara Leoni
- Contact Person Email
- chiara.leoni@policlinicogemelli.it
Spain
- Earliest CTIS Part Ii Submission Date
- 24-11-2023
- Latest Decision Or Authorization Date
- 13-05-2026
- Processing Time Days
- 901
- Number Of Sites
- 7
- Number Of Participants
- 27
Sites
- Site Name
- Hospital Universitari Vall D Hebron
- Department Name
- 3504: Medicina interna
- Principal Investigator Name
- Maria Camprodón Gómez
- Principal Investigator Email
- maria.camprodon@vallhebron.cat
- Contact Person Name
- Maria Camprodón Gómez
- Contact Person Email
- maria.camprodon@vallhebron.cat
- Site Name
- Complexo Hospitalario Universitario A Coruna
- Department Name
- 3503: Cirujano Plástico Pediátrico
- Principal Investigator Name
- Manuel Gómez Tellado
- Principal Investigator Email
- manuel.gomez.tellado@sergas.es
- Contact Person Name
- Manuel Gómez Tellado
- Contact Person Email
- manuel.gomez.tellado@sergas.es
- Site Name
- Hospital General Universitario Gregorio Maranon
- Department Name
- 3502: Dermatology
- Principal Investigator Name
- Minia Campos Dominguez
- Principal Investigator Email
- minia.campos@salud.madrid.org
- Contact Person Name
- Minia Campos Dominguez
- Contact Person Email
- minia.campos@salud.madrid.org
- Site Name
- Bellvitge University Hospital
- Department Name
- 3505: Dermatología
- Principal Investigator Name
- Anna Jucglà Serra
- Principal Investigator Email
- ajucgla@bellvitgehospital.cat
- Contact Person Name
- Anna Jucglà Serra
- Contact Person Email
- ajucgla@bellvitgehospital.cat
- Site Name
- Hospital Universitario La Paz
- Department Name
- 3500: Pediatric Surgery
- Principal Investigator Name
- Juan Carlos López Gutiérrez
- Principal Investigator Email
- queminfantil.hulp@salud.madrid.org
- Contact Person Name
- Juan Carlos López Gutiérrez
- Contact Person Email
- queminfantil.hulp@salud.madrid.org
- Site Name
- University Hospital Son Espases
- Department Name
- 3506: Cirugía pediátrica
- Principal Investigator Name
- Maria de los Angeles Muñoz Miguelsanz
- Principal Investigator Email
- mariadelosangeles.munozmiguelsanz@ssib.es
- Contact Person Name
- Maria de los Angeles Muñoz Miguelsanz
- Contact Person Email
- mariadelosangeles.munozmiguelsanz@ssib.es
- Site Name
- Sant Joan De Deu Barcelona Hospital
- Department Name
- 3501: Dermatology
- Principal Investigator Name
- Eulalia Baselga Torres
- Principal Investigator Email
- eulalia.baselga@sjd.es
- Contact Person Name
- Eulalia Baselga Torres
- Contact Person Email
- eulalia.baselga@sjd.es
Belgium
- Earliest CTIS Part Ii Submission Date
- 04-03-2024
- Latest Decision Or Authorization Date
- 22-04-2026
- Processing Time Days
- 779
- Number Of Sites
- 1
- Number Of Participants
- 14
Sites
- Site Name
- Cliniques Universitaires Saint-Luc
- Department Name
- #3001: Centre de malformations vasculaires congenitales
- Principal Investigator Name
- Emmanuel Seront
- Principal Investigator Email
- emmanuel.seront@saintluc.uclouvain.be
- Contact Person Name
- Emmanuel Seront
- Contact Person Email
- emmanuel.seront@saintluc.uclouvain.be
Czechia
- Earliest CTIS Part Ii Submission Date
- 11-03-2026
- Latest Decision Or Authorization Date
- 13-05-2026
- Processing Time Days
- 63
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Fakultni Nemocnice Brno
- Department Name
- 3700: Klinika detske onkologie
- Principal Investigator Name
- Peter Mudry
- Principal Investigator Email
- Mudry.Peter@fnbrno.cz
- Contact Person Name
- Peter Mudry
- Contact Person Email
- Mudry.Peter@fnbrno.cz
Sponsor
Primary sponsor
- Full Name
- Novartis Pharma AG
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Switzerland
Contract research organisations
- Name
- Parexel International (IRL) Limited
- Name
- Icon Clinical Research Limited
- Name
- Iqvia Rds Inc.
- Name
- Veeda Clinical Research Limited
- Responsibilities
- Pharmacokinetic sample analysis
Third parties
- {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Jumo Health USA Inc.","duties_or_roles":"Patient recruitment and retention","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United States","full_name":"Yprime LLC","duties_or_roles":"eCOA, providing the electronic tablet for patients","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Adelphi Values Limited","duties_or_roles":"Patient embedded interviews vendor","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"Independent central LyM image review and analysis","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"India","full_name":"Veeda Clinical Research Limited","duties_or_roles":"Pharmacokinetic sample analysis","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Qualitymetric Incorporated LLC","duties_or_roles":"Validation of selected PROs for LyM indication","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Iqvia Rds Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Patient Reimbursement and Transportation Support","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services S.a.r.l.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"Italy","full_name":"Opis S.r.l.","duties_or_roles":"TMF archive","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"RWS Life Sciences Inc.","duties_or_roles":"PRO translations and licensing","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- BYL719 (film-coated tablet, granules)
- Active Substance
- ALPELISIB
- Modality
- Small molecule
- Routes Of Administration
- Oral (ORAL USE)
- Route
- Oral
- Authorisation Status
- Authorised
- Orphan Designation
- Yes
- Dose Levels
- Product entries include max daily doses up to 250 mg for tablets and up to 50 mg for granules (as recorded in product data).
- Maximum Dose
- 250 mg (max daily dose amount recorded for some tablet presentations); 50 mg (max daily dose for some granule presentations)
- Investigational Product Name
- Placebo to BYL719 200 mg film-coated tablets
- Modality
- Other
- Investigational Product Name
- Placebo to BYL719 125 mg film-coated tablets
- Modality
- Other
- Investigational Product Name
- Placebo to BYL719 50 mg film-coated tablets
- Modality
- Other
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