Esta página foi traduzida automaticamente e a precisão da tradução não é garantida. Por favor, consulte o versão em inglês para um texto fonte.

Alpelisib em pacientes pediátricos e adultos com malformações linfáticas associadas a uma mutação PIK3CA. (EPIK-L1)

8 de outubro de 2026 atualizado por: Novartis Pharmaceuticals

Um estudo duplo-cego, randomizado e controlado por placebo em dois estágios para avaliar a eficácia, segurança e farmacocinética do alpelisibe em pacientes pediátricos e adultos com malformações linfáticas associadas a uma mutação 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.

Visão geral do estudo

Status

Recrutamento

Condições

Intervenção / Tratamento

Descrição detalhada

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 estudo

Intervencional

Inscrição (Estimado)

232

Estágio

  • Fase 2
  • Fase 3

Acesso expandido

Disponível fora do ensaio clínico. Consulte registro de acesso expandido.

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Contato de estudo

Estude backup de contato

Locais de estudo

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

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

1 segundo a 100 anos (Filho, Adulto, Adulto mais velho)

Aceita Voluntários Saudáveis

Não

Gêneros Elegíveis para o Estudo

Tudo

Descrição

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

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

  • Finalidade Principal: Tratamento
  • Alocação: Randomizado
  • Modelo Intervencional: Atribuição sequencial
  • Mascaramento: Quadruplicar

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
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)
Medicamento: 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

Outros nomes:
  • 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).
Medicamento: 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

Outros nomes:
  • 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)
Medicamento: 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

Outros nomes:
  • 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).
Medicamento: 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

Outros nomes:
  • 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)
Medicamento: 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

Outros nomes:
  • BYL719
Comparador de Placebo: Adult participants, placebo (Stage 2)

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

Outros nomes:
  • BYL719
Comparador de Placebo: Pediatric participants (6-17 years of age), placebo (Stage 2)

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

Outros nomes:
  • BYL719

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Stage 2:Radiological response rate at Week 24 of Stage 2 (adult and pediatric (6 - 17 years of age) participants)
Prazo: 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 resultados secundários

Medida de resultado
Descrição da medida
Prazo
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)
Prazo: 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)
Prazo: 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)
Prazo: 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)
Prazo: 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)
Prazo: 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)
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

Novartis Pharmaceuticals

Investigadores

  • Diretor de estudo: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Real)

24 de novembro de 2023

Conclusão Primária (Estimado)

31 de maio de 2028

Conclusão do estudo (Estimado)

2 de maio de 2033

Datas de inscrição no estudo

Enviado pela primeira vez

4 de julho de 2023

Enviado pela primeira vez que atendeu aos critérios de CQ

4 de julho de 2023

Primeira postagem (Real)

17 de julho de 2023

Atualizações de registro de estudo

Última Atualização Postada (Real)

9 de outubro de 2026

Última atualização enviada que atendeu aos critérios de controle de qualidade

8 de outubro de 2026

Última verificação

1 de outubro de 2026

Mais Informações

Termos relacionados a este estudo

Palavras-chave

Termos MeSH relevantes adicionais

Outros números de identificação do estudo

  • CBYL719P12201
  • 2023-504146-60-00 (Identificador de registro: EU CTIS number)
  • 2023 (Concessão/Contrato do NIH dos EUA)

Plano para dados de participantes individuais (IPD)

Planeja compartilhar dados de participantes individuais (IPD)?

SIM

Descrição do plano 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

Informações sobre medicamentos e dispositivos, documentos de estudo

Estuda um medicamento regulamentado pela FDA dos EUA

Sim

Estuda um produto de dispositivo regulamentado pela FDA dos EUA

Não

Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .

Se inscrever