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Alpelisib en pacientes pediátricos y adultos con malformaciones linfáticas asociadas a una mutación en PIK3CA. (EPIK-L1)

8 de octubre de 2026 actualizado por: Novartis Pharmaceuticals

Estudio de dos etapas, doble ciego, aleatorizado, controlado con placebo para evaluar la eficacia, la seguridad y la farmacocinética de alpelisib en pacientes pediátricos y adultos con malformaciones linfáticas asociadas con una mutación PIK3CA.

The main purpose of this study in participants with PIK3CA-mutated LyM is to assess the change in radiological response and symptom severity upon treatment with alpelisib film-coated tablets (FCT) as compared to placebo.

Descripción general del estudio

Estado

Reclutamiento

Condiciones

Intervención / Tratamiento

Descripción detallada

This is a phase II/III multi-center study with two stages:

  • Stage 1 is designed to select the dose(s) for the confirmatory phase (DSCP) for alpelisib in Stage 2 and will comprise a 24-week open-label core phase in adult (≥18 years of age) and pediatric participants (6-17 years of age) with PIK3CA-mutated LyM, followed by an extension. After eligibility has been confirmed at screening, participants will be randomized in a 1:1 ratio to the different alpelisib doses according to their age. Depending on the results at the end of Stage 1 core phase, the Stage 2 will be opened to adult and/or pediatric participants or the study may be stopped.
  • Stage 2 is designed to confirm the efficacy and assess safety of alpelisib at the DSCP in participants with PIK3CA-mutated LyM and will comprise a 24-week randomized, double blind, placebo-controlled confirmatory phase in adult (≥18 years of age) and pediatric participants 6-17 years of age followed by an open-label extension. After eligibility has been confirmed at screening participants will be randomized in a 2:1 ratio to alpelisib or placebo.

Additionally, in parallel, Stage 2 will include a 24-week open-label core phase in pediatric participants 0-5 years of age followed by an extension, if pediatric participants will be enrolling in Stage 2.

Based on the results of the 24-week open-label core phase of Stage 1, the dose(s) for Stage 2 will be selected by Novartis in consultation with the Steering Committee (SC). During the 24-week randomized, double blind, placebo-controlled core phase of Stage 2, an Independent Data Monitoring Committee (DMC) will conduct periodic safety and efficacy reviews to assess the risk benefit profile of the treatment.

Tipo de estudio

Intervencionista

Inscripción (Estimado)

232

Fase

  • Fase 2
  • Fase 3

Acceso ampliado

Disponible fuera del ensayo clínico. Ver registro de acceso ampliado.

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Estudio Contacto

Copia de seguridad de contactos de estudio

Ubicaciones de estudio

  • Alemania
      • Berlin, Alemania, 13353
        • Reclutamiento
        • Novartis Investigative Site
      • Ulm, Alemania, 89081
        • Reclutamiento
        • Novartis Investigative Site
    • Baden-Wurttemberg
      • Freiburg im Breisgau, Baden-Wurttemberg, Alemania, 79106
        • Reclutamiento
        • Novartis Investigative Site
      • Mannheim, Baden-Wurttemberg, Alemania, 68305
        • Retirado
        • Novartis Investigative Site
    • North Rhine-Westphalia
      • Cologne, North Rhine-Westphalia, Alemania, 50937
        • Reclutamiento
        • Novartis Investigative Site
    • Saxony
      • Leipzig, Saxony, Alemania, 04103
        • Reclutamiento
        • Novartis Investigative Site
  • Argentina
      • Capital Federal, Argentina, C1023AAB
        • Reclutamiento
        • Novartis Investigative Site
    • Buenos Aires
      • CABA, Buenos Aires, Argentina, C1181ACH
        • Reclutamiento
        • Novartis Investigative Site
      • CABA, Buenos Aires, Argentina, C1425BEA
        • Reclutamiento
        • Novartis Investigative Site
  • Australia
    • New South Wales
      • Sydney, New South Wales, Australia, 2010
        • Reclutamiento
        • Novartis Investigative Site
      • Sydney, New South Wales, Australia, 2031
        • Reclutamiento
        • Novartis Investigative Site
    • Queensland
      • Brisbane, Queensland, Australia, 4101
        • Reclutamiento
        • Novartis Investigative Site
  • Bélgica
      • Brussels, Bélgica, 1200
        • Reclutamiento
        • Novartis Investigative Site
  • Chequia
      • Brno, Chequia, 625 00
        • Reclutamiento
        • Novartis Investigative Site
  • España
      • A Coruña, España, 15006
        • Reclutamiento
        • Novartis Investigative Site
      • Barcelona, España, 08035
        • Reclutamiento
        • Novartis Investigative Site
      • Madrid, España, 28046
        • Reclutamiento
        • Novartis Investigative Site
      • Madrid, España, 28009
        • Reclutamiento
        • Novartis Investigative Site
    • Balearic Islands
      • Palma, Balearic Islands, España, 07120
        • Reclutamiento
        • Novartis Investigative Site
    • Barcelona
      • Esplugues, Barcelona, España, 08950
        • Reclutamiento
        • Novartis Investigative Site
      • L'Hospitalet de Llobregat, Barcelona, España, 08907
        • Reclutamiento
        • Novartis Investigative Site
  • Estados Unidos
    • California
      • Oakland, California, Estados Unidos, 94609
        • Reclutamiento
        • UCSF Benioff Children s Hospital
        • Investigador principal:
          • Beth Apsel Winger
        • Contacto:
      • Orange, California, Estados Unidos, 92868
        • Reclutamiento
        • Childrens Hospital of Orange County
        • Contacto:
        • Investigador principal:
          • Maria Buethe
      • Palo Alto, California, Estados Unidos, 94304
        • Reclutamiento
        • Lucile Packard Childrens Hosp
        • Investigador principal:
          • Joyce Teng
        • Contacto:
    • District of Columbia
      • Washington D.C., District of Columbia, Estados Unidos, 20010-2970
        • Reclutamiento
        • Childrens National Medical Center
        • Contacto:
        • Investigador principal:
          • Yaser Diab
    • Florida
      • Jacksonville, Florida, Estados Unidos, 32207
        • Reclutamiento
        • Nemours Childrens Clinic
        • Contacto:
        • Investigador principal:
          • Anderson Collier
    • Massachusetts
      • Boston, Massachusetts, Estados Unidos, 02115
        • Reclutamiento
        • Childrens Hosp Boston Dept of Heme
        • Investigador principal:
          • Tishi Shah
        • Contacto:
    • Missouri
      • St Louis, Missouri, Estados Unidos, 63110
        • Reclutamiento
        • WA Uni School Of Med
        • Investigador principal:
          • Bryan Sisk
        • Contacto:
    • North Carolina
      • Chapel Hill, North Carolina, Estados Unidos, 27599
        • Reclutamiento
        • University of North Carolina at Chapel Hill
        • Contacto:
        • Investigador principal:
          • Alexandra Borst
    • Ohio
      • Cincinnati, Ohio, Estados Unidos, 45229-3039
        • Reclutamiento
        • Cinn Children Hosp Medical Center
        • Investigador principal:
          • Adrienne Hammill
        • Contacto:
      • Cleveland, Ohio, Estados Unidos, 44195
        • Reclutamiento
        • Cleveland Clinic Foundation
        • Investigador principal:
          • Michael Kelly
        • Contacto:
      • Cleveland, Ohio, Estados Unidos, 44106
        • Reclutamiento
        • Univ Hospital Of Cleveland
        • Investigador principal:
          • Howard Wang
        • Contacto:
      • Columbus, Ohio, Estados Unidos, 43205
    • Oregon
      • Portland, Oregon, Estados Unidos, 97239
        • Reclutamiento
        • Oregon Health Science University
        • Investigador principal:
          • Melinda Wu
        • Contacto:
    • Pennsylvania
      • Philadelphia, Pennsylvania, Estados Unidos, 19104
        • Reclutamiento
        • CHOP Abramson Pediatric Resch Ctr
        • Investigador principal:
          • Denise Adams
        • Contacto:
      • Pittsburgh, Pennsylvania, Estados Unidos, 15224
        • Reclutamiento
        • Childrens Hosp Pittsburgh UPMC
        • Contacto:
        • Investigador principal:
          • Julia Segal
    • Texas
      • Houston, Texas, Estados Unidos, 77030
        • Reclutamiento
        • U of TX Health Science Ct
        • Investigador principal:
          • Autumn Atkinson
        • Contacto:
      • Houston, Texas, Estados Unidos, 77030
        • Reclutamiento
        • Baylor College Of Medicine
        • Investigador principal:
          • Ionela Iacobas
        • Contacto:
    • Washington
      • Seattle, Washington, Estados Unidos, 98105
        • Reclutamiento
        • Childrens Hospital and Regional Medical Center
        • Investigador principal:
          • Jonathan A Perkins
        • Contacto:
  • Francia
      • Angers, Francia, 49933
        • Reclutamiento
        • Novartis Investigative Site
      • Bordeaux, Francia, 33076
        • Reclutamiento
        • Novartis Investigative Site
      • Bron, Francia, 69677
        • Reclutamiento
        • Novartis Investigative Site
      • Caen, Francia, 14033
        • Reclutamiento
        • Novartis Investigative Site
      • Dijon, Francia, 21000
        • Reclutamiento
        • Novartis Investigative Site
      • Lille, Francia, 59000
        • Reclutamiento
        • Novartis Investigative Site
      • Marseille, Francia, 13885
        • Reclutamiento
        • Novartis Investigative Site
      • Montpellier, Francia, 34295
        • Reclutamiento
        • Novartis Investigative Site
      • Paris, Francia, 75015
        • Reclutamiento
        • Novartis Investigative Site
      • Paris, Francia, 75010
        • Reclutamiento
        • Novartis Investigative Site
      • Toulouse, Francia, 31054
        • Reclutamiento
        • Novartis Investigative Site
      • Tours, Francia, 37044
        • Reclutamiento
        • Novartis Investigative Site
  • Italia
      • Naples, Italia, 80122
        • Reclutamiento
        • Novartis Investigative Site
    • BO
      • Bologna, BO, Italia, 40138
        • Reclutamiento
        • Novartis Investigative Site
    • MI
      • Milan, MI, Italia, 20122
        • Reclutamiento
        • Novartis Investigative Site
    • RM
      • Roma, RM, Italia, 00165
        • Reclutamiento
        • Novartis Investigative Site
      • Roma, RM, Italia, 00168
        • Reclutamiento
        • Novartis Investigative Site
    • TO
      • Torino, TO, Italia, 10126
        • Reclutamiento
        • Novartis Investigative Site
  • Países Bajos
      • Nijmegen, Países Bajos, 6525 GA
        • Reclutamiento
        • Novartis Investigative Site
    • South Holland
      • Rotterdam, South Holland, Países Bajos, 3015 GD
        • Reclutamiento
        • Novartis Investigative Site
  • Reino Unido
      • London, Reino Unido, WC1N 3JH
        • Reclutamiento
        • Novartis Investigative Site
  • Suiza
      • Lausanne, Suiza, 1011
        • Reclutamiento
        • Novartis Investigative Site

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

1 segundo a 100 años (Niño, Adulto, Adulto Mayor)

Acepta Voluntarios Saludables

No

Géneros elegibles para el estudio

Todos

Descripción

Key inclusion criteria:

  1. Signed informed consent and assent (when applicable) from the participant, parent, legal authorized representative or guardian.
  2. Participant must be willing to remain at the clinical site as required by the protocol and be willing to adhere to study restrictions and examination schedules.
  3. 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).
  4. 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 in Stage 1 and 2.
  5. Participant has evidence of a somatic mutation(s) in the PIK3CA gene prior to randomization.
  6. Participant has at least one measurable LyM lesion confirmed by BIRC assessment prior to randomization.
  7. Participants must be able to ingest study drug (either in tablet form or as a drinkable 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.

Key exclusion criteria:

  1. Participant has a physician-confirmed and documented diagnosis of PROS at the time of informed consent.
  2. 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.
  3. Participant has a known history of Stevens-Johnson syndrome, erythema multiforme, or toxic epidermal necrolysis at the time of informed consent.
  4. Participant has an established diagnosis of type I diabetes mellitus or uncontrolled type II diabetes mellitus at the time of informed consent.
  5. 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.

Other inclusion/exclusion criteria may apply

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: Aleatorizado
  • Modelo Intervencionista: Asignación Secuencial
  • Enmascaramiento: Cuadruplicar

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: Adult participants, alpelisib dose 1 (Stage 1)

Adult participants (≥18 years of age) who will receive dose 1 of alpelisib an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1)
Droga: Alpelisib

In Stage 1: adult participants (≥18 years of age) will receive dose 1 or dose 2 of alpelisib; pediatric participants (6-17 years of age) will receive dose 2 or dose 3 of alpelisib.

In Stage 2: Adult participants will receive alpelisib at the dose selected for confirmatory phase in adult participants; pediatric participants (6-17 years of age) will will receive alpelisib at the dose selected for confirmatory phase in pediatric participants; and pediatric participants of 0-5 years of age will receive dose 3 of alpelisib

Otros nombres:
  • BYL719
Experimental: Adult participants, alpelisib dose 2 (Stage 1)

Adult participants (≥18 years of age) who will receive dose 2 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1).
Droga: Alpelisib

In Stage 1: adult participants (≥18 years of age) will receive dose 1 or dose 2 of alpelisib; pediatric participants (6-17 years of age) will receive dose 2 or dose 3 of alpelisib.

In Stage 2: Adult participants will receive alpelisib at the dose selected for confirmatory phase in adult participants; pediatric participants (6-17 years of age) will will receive alpelisib at the dose selected for confirmatory phase in pediatric participants; and pediatric participants of 0-5 years of age will receive dose 3 of alpelisib

Otros nombres:
  • BYL719
Experimental: Pediatric participants (6-17 years of age), alpelisib dose 2 (Stage 1)

Pediatric participants 6-17 years of age who will receive dose 2 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1)
Droga: Alpelisib

In Stage 1: adult participants (≥18 years of age) will receive dose 1 or dose 2 of alpelisib; pediatric participants (6-17 years of age) will receive dose 2 or dose 3 of alpelisib.

In Stage 2: Adult participants will receive alpelisib at the dose selected for confirmatory phase in adult participants; pediatric participants (6-17 years of age) will will receive alpelisib at the dose selected for confirmatory phase in pediatric participants; and pediatric participants of 0-5 years of age will receive dose 3 of alpelisib

Otros nombres:
  • BYL719
Experimental: Pediatric participants (6-17 years of age), alpelisib dose 3 (Stage 1)

Pediatric participants 6-17 years of age who will receive dose 3 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1).
Droga: Alpelisib

In Stage 1: adult participants (≥18 years of age) will receive dose 1 or dose 2 of alpelisib; pediatric participants (6-17 years of age) will receive dose 2 or dose 3 of alpelisib.

In Stage 2: Adult participants will receive alpelisib at the dose selected for confirmatory phase in adult participants; pediatric participants (6-17 years of age) will will receive alpelisib at the dose selected for confirmatory phase in pediatric participants; and pediatric participants of 0-5 years of age will receive dose 3 of alpelisib

Otros nombres:
  • BYL719
Experimental: Adult participants, alpelisib (Stage 2)

Adult participants (≥18 years of age) who will receive alpelisib at the dose selected for confirmatory phase in adult participants (Stage 2)
Droga: Alpelisib

In Stage 1: adult participants (≥18 years of age) will receive dose 1 or dose 2 of alpelisib; pediatric participants (6-17 years of age) will receive dose 2 or dose 3 of alpelisib.

In Stage 2: Adult participants will receive alpelisib at the dose selected for confirmatory phase in adult participants; pediatric participants (6-17 years of age) will will receive alpelisib at the dose selected for confirmatory phase in pediatric participants; and pediatric participants of 0-5 years of age will receive dose 3 of alpelisib

Otros nombres:
  • BYL719
Comparador de placebos: Adult participants, placebo (Stage 2)

Adult participants (≥18 years of age) who will receive matching placebo
Droga: Placebo

In Stage 2, participants will receive matching placebo for 24 weeks of the study
Experimental: Pediatric participants (6-17 years of age), alpelisib (Stage 2)

Pediatric participants (6-17 years of age) who will receive alpelisib at the dose selected for confirmatory phase in pediatric participants (Stage 2)
Droga: Alpelisib

In Stage 1: adult participants (≥18 years of age) will receive dose 1 or dose 2 of alpelisib; pediatric participants (6-17 years of age) will receive dose 2 or dose 3 of alpelisib.

In Stage 2: Adult participants will receive alpelisib at the dose selected for confirmatory phase in adult participants; pediatric participants (6-17 years of age) will will receive alpelisib at the dose selected for confirmatory phase in pediatric participants; and pediatric participants of 0-5 years of age will receive dose 3 of alpelisib

Otros nombres:
  • BYL719
Comparador de placebos: Pediatric participants (6-17 years of age), placebo (Stage 2)

Pediatric participants (6-17 years of age) who will receive matching placebo
Droga: Placebo

In Stage 2, participants will receive matching placebo for 24 weeks of the study
Experimental: Pediatric participants (0-5 years of age), alpelisib (Stage 2)

Pediatric participants of 0-5 years who will dose 3 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier
Droga: Alpelisib

In Stage 1: adult participants (≥18 years of age) will receive dose 1 or dose 2 of alpelisib; pediatric participants (6-17 years of age) will receive dose 2 or dose 3 of alpelisib.

In Stage 2: Adult participants will receive alpelisib at the dose selected for confirmatory phase in adult participants; pediatric participants (6-17 years of age) will will receive alpelisib at the dose selected for confirmatory phase in pediatric participants; and pediatric participants of 0-5 years of age will receive dose 3 of alpelisib

Otros nombres:
  • BYL719

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Stage 2:Radiological response rate at Week 24 of Stage 2 (adult and pediatric (6 - 17 years of age) participants)
Periodo de tiempo: Baseline, Week 24

Radiological response 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. The percentage of participants with a radiological response at Week 24 of Stage 2 in adult and pediatric (6-17 years of age) groups will be assessed
Baseline, Week 24

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Stage 2: Percentage of participants with at least a 1-point improvement compared to baseline based on patient global impression of severity (PGI-S) scale at Week 24 of Stage 2 (adult and pediatric (6 - 17 years of age) participants)
Periodo de tiempo: Baseline, Week 24

PGI-S is a single item measure to assess the overall severity of a patient's condition. This single item instrument uses a 5-point rating scale, which ranges from 1 (no symptoms) to 5 (very severe). Lower scores indicate better health status. The percentage of participants with at least a 1-point improvement compared to baseline at Week 24 of Stage 2 in adult and pediatric (6-17 years of age) groups will be assessed
Baseline, Week 24
Stage 2: Percentage of participants with a radiological response at Week 24 of Stage 2 (pediatric participants 0-5 years of age)
Periodo de tiempo: Baseline, Week 24

Radiological response 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. The percentage of participants with a radiological response at Week 24 of Stage 2 in pediatric participants 0-5 years of age will be assessed
Baseline, Week 24
Stage 2: Change from baseline in patient global impression of change (PGI-C) scale (adult and pediatric (6-17 years of age) participants)
Periodo de tiempo: Up to approximately 8 years

PGI-C is a single item measure to assess the change in overall symptoms severity since the start of study. This single item instrument uses a 7-point rating scale, which ranges from 1 (very much improved) to 7 (very much worse). Lower scores indicate better health status. The change from baseline in PGI-C score will be assessed in adult and pediatric (6-17 years of age) participants
Up to approximately 8 years
Stage 2: Change from baseline in patient-reported outcomes measurement information system (PROMIS) profile domains(adult and pediatric (6-17 years of age) participants)
Periodo de tiempo: Up to approximately 8 years

The PROMIS-29 plus 2 Profile v2.1 (completed by adult participant) includes 29 items across domains of depression, anxiety, physical function, pain interference, fatigue, sleep disturbance, ability to participant in social roles and activities, cognitive function abilities and pain intensity.

The PROMIS Pediatric-25 Profile v2.0 (completed by children over 8 years of age) and PROMIS Parent-Proxy-25 Profile v2.0 (completed by parents for children under 8 years of age) include 25 items across the domains of depressive symptoms, anxiety, physical function-mobility, pain interference, fatigue, and peer relationships and pain intensity All items (except the pain intensity item) use a 5-point Likert scale, which ranges from 1 (not at all) to 5 (very much). The pain intensity item is scored on a 0-10 numeric rating scale, where 0 represents "no pain" and 10 represents "worst imaginable pain".

The change from baseline in PROMIS domains will be assessed
Up to approximately 8 years
Stage 2: Change from baseline in investigator global impression of change (IGIC) scale (adult and pediatric (6-17 years of age) participants)
Periodo de tiempo: Up to approximately 8 years

The IGIC involves a single question that asks the investigator to rate the change in the patient's condition since the start of treatment or intervention, using a 7-point scale ranging from 1 (Very much improved) to 7 (Very much worse). The change from baseline in IGIC score will be assessed in adult and pediatric (6-17 years of age) participants
Up to approximately 8 years
Stage 2: Change from baseline in health utilities of the EuroQol 5-dimension (EQ-5D) (adult and pediatric (6-17 years of age) participants)
Periodo de tiempo: Up to approximately 8 years

The EQ-5D-5L (completed by adult participants) includes 5 items addressing dimensions of mobility, self-care, usual activities, pain/discomfort, and anxiety/depression), each with 5 response options, ranging from 1=no problems to 5=extreme problems The EQ-5D-Y (completed by children over 8 years of age) and EQ-5D-Y Proxy version (completed by parent for participants under 8 years of age or unable to record for themselves) includes 5 items addressing dimensions of mobility, self-care, usual activities, pain/discomfort, and anxiety/depression), each with 3 response options, ranging from 1= no problems to 3= a lot of problems
Up to approximately 8 years
Stage 1 and 2: Duration of response (DOR) in adult and pediatric participants who receive alpelisib
Periodo de tiempo: Up to approximately 8 years

DOR is defined as the time from first documented response until progression of LyM lesions by BIRC or death. This analysis only applies to participants who are on treatment with alpelisib (Stage 1 and 2) and who achieve response.
Up to approximately 8 years
Stage 1: Radiological response rate of alpelisib in adult and pediatric (6-17 years of age) participants
Periodo de tiempo: Baseline, Week 24

Radiological response 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.

The percentage of participants (adult and pediatric 6-17 years of age) with radiological response at Week 24 of Stage 1 will be assessed.
Baseline, Week 24
Stage 1 and 2: Radiological response rate of alpelisib in adult and pediatric participants
Periodo de tiempo: Up to approximately 8 years

Radiological response defined by achieving at least 20% reduction in the sum of target lesion volumes (1 to 3 lesions), assessed by MRI by a BIRC, 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.

The percentage of participants who receive alpelisib (Stage 1 and 2) with radiological response will be assessed.
Up to approximately 8 years
Stage 1 and 2: Alpelisib plasma concentrations
Periodo de tiempo: On Day 1 of Week 8, 16, 24, 48 and 120

Alpelisib plasma concentrations in adult and pediatric participants (Stage 1 and 2).
On Day 1 of Week 8, 16, 24, 48 and 120
Stage 1 and 2: Percentage of participants with of LyM-related symptoms, complications, and comorbidities on treatment with alpelisib in adult and pediatric participants at Week 24
Periodo de tiempo: Week 24

Percentage of participants with of LyM-related symptoms, complications, and comorbidities on treatment with alpelisib in adult and pediatric participants at Week 24 (Stage 1 and 2)
Week 24
Stage 1 and 2: Percentage of participants with of LyM-related symptoms, complications, and comorbidities on treatment with alpelisib in adult and pediatric participants
Periodo de tiempo: Up to approximately 8 years

Percentage of participants with of LyM-related symptoms, complications, and comorbidities on treatment with alpelisib in adult and pediatric participants (Stage 1 and 2)
Up to approximately 8 years
Stage 1 and 2: Change from baseline in LyM lesions in adult and pediatric participants at Week 24
Periodo de tiempo: Baseline, Week 24

Change from baseline in the sum of target lesion volumes, the sum of MRI-measurable non-target lesion (if applicable) volumes, and the sum of all MRI-measurable lesion (target and non-target) volumes as assessed by BIRC at Week 24 in adult and pediatric participants (Stage 1 and 2)
Baseline, Week 24
Stage 1 and 2: Change from baseline in LyM lesions in adult and pediatric participants
Periodo de tiempo: Up to approximately 8 years

Change from baseline in the sum of target lesion volumes, the sum of MRI-measurable non-target lesion (if applicable) volumes, and the sum of all MRI-measurable lesion (target and non-target) volumes as assessed by BIRC in adult and pediatric participants (Stage 1 and 2)
Up to approximately 8 years
Stage 1 and 2: Percentage of participants with changes in non-target lesions in adult and pediatric participants at Week 24
Periodo de tiempo: Baseline, Week 24

Percentage of participants with changes in non-target lesions as assessed by BIRC at Week 24 in adult and pediatric participants (Stage 1 and 2)
Baseline, Week 24
Stage 1 and 2: Percentage of participants with changes in non-target lesions in adult and pediatric participants
Periodo de tiempo: Up to approximately 8 years

Percentage of participants with changes in non-target lesions as assessed by BIRC in adult and pediatric participants (Stage 1 and 2)
Up to approximately 8 years
Stage 1 and 2: Percentage of participants with new lesions in adult and pediatric participants at Week 24
Periodo de tiempo: Baseline, Week 24

The percentage of participants with new lesions as assessed by BIRC at Week 24 in adult and pediatric participants (Stage 1 and 2)
Baseline, Week 24
Stage 1 and 2: Percentage of participants with new lesions in adult and pediatric participants
Periodo de tiempo: Up to approximately 8 years

The percentage of participants with new lesions as assessed by BIRC in adult and pediatric participants (Stage 1 and 2)
Up to approximately 8 years

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Novartis Pharmaceuticals

Investigadores

  • Director de estudio: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

24 de noviembre de 2023

Finalización primaria (Estimado)

31 de mayo de 2028

Finalización del estudio (Estimado)

2 de mayo de 2033

Fechas de registro del estudio

Enviado por primera vez

4 de julio de 2023

Primero enviado que cumplió con los criterios de control de calidad

4 de julio de 2023

Publicado por primera vez (Actual)

17 de julio de 2023

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

9 de octubre de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

8 de octubre de 2026

Última verificación

1 de octubre de 2026

Más información

Términos relacionados con este estudio

Palabras clave

Términos MeSH relevantes adicionales

Otros números de identificación del estudio

  • CBYL719P12201
  • 2023-504146-60-00 (Identificador de registro: EU CTIS number)
  • 2023 (Subvención/contrato del NIH de EE. UU.)

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

SÍ

Descripción del plan IPD

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

Sí

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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