Эта страница была переведена автоматически, точность перевода не гарантируется. Пожалуйста, обратитесь к английской версии для исходного текста.

Алпелисиб у детей и взрослых с лимфатическими мальформациями, связанными с мутацией PIK3CA. (EPIK-L1)

8 октября 2026 г. обновлено: Novartis Pharmaceuticals

Двухэтапное двойное слепое рандомизированное плацебо-контролируемое исследование по оценке эффективности, безопасности и фармакокинетики алпелисиба у детей и взрослых пациентов с лимфатическими мальформациями, связанными с мутацией 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.

Обзор исследования

Статус

Рекрутинг

Условия

Вмешательство/лечение

Подробное описание

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.

Тип исследования

Интервенционный

Регистрация (Оцененный)

232

Фаза

  • Фаза 2
  • Фаза 3

Расширенный доступ

Доступный вне клинических испытаний. См. запись расширенного доступа.

Контакты и местонахождение

В этом разделе приведены контактные данные лиц, проводящих исследование, и информация о том, где проводится это исследование.

Контакты исследования

  • Имя: Novartis Pharmaceuticals
  • Номер телефона: 1-888-669-6682
  • Электронная почта: novartis.email@novartis.com

Учебное резервное копирование контактов

  • Имя: Novartis Pharmaceuticals
  • Номер телефона: +41613241111
  • Электронная почта: novartis.email@novartis.com

Места учебы

  • Австралия
    • New South Wales
      • Sydney, New South Wales, Австралия, 2010
        • Рекрутинг
        • Novartis Investigative Site
      • Sydney, New South Wales, Австралия, 2031
        • Рекрутинг
        • Novartis Investigative Site
    • Queensland
      • Brisbane, Queensland, Австралия, 4101
        • Рекрутинг
        • Novartis Investigative Site
  • Аргентина
      • Capital Federal, Аргентина, C1023AAB
        • Рекрутинг
        • Novartis Investigative Site
    • Buenos Aires
      • CABA, Buenos Aires, Аргентина, C1181ACH
        • Рекрутинг
        • Novartis Investigative Site
      • CABA, Buenos Aires, Аргентина, C1425BEA
        • Рекрутинг
        • Novartis Investigative Site
  • Бельгия
      • Brussels, Бельгия, 1200
        • Рекрутинг
        • Novartis Investigative Site
  • Германия
      • Berlin, Германия, 13353
        • Рекрутинг
        • Novartis Investigative Site
      • Ulm, Германия, 89081
        • Рекрутинг
        • Novartis Investigative Site
    • Baden-Wurttemberg
      • Freiburg im Breisgau, Baden-Wurttemberg, Германия, 79106
        • Рекрутинг
        • Novartis Investigative Site
      • Mannheim, Baden-Wurttemberg, Германия, 68305
        • Отозван
        • Novartis Investigative Site
    • North Rhine-Westphalia
      • Cologne, North Rhine-Westphalia, Германия, 50937
        • Рекрутинг
        • Novartis Investigative Site
    • Saxony
      • Leipzig, Saxony, Германия, 04103
        • Рекрутинг
        • Novartis Investigative Site
  • Испания
      • A Coruña, Испания, 15006
        • Рекрутинг
        • Novartis Investigative Site
      • Barcelona, Испания, 08035
        • Рекрутинг
        • Novartis Investigative Site
      • Madrid, Испания, 28046
        • Рекрутинг
        • Novartis Investigative Site
      • Madrid, Испания, 28009
        • Рекрутинг
        • Novartis Investigative Site
    • Balearic Islands
      • Palma, Balearic Islands, Испания, 07120
        • Рекрутинг
        • Novartis Investigative Site
    • Barcelona
      • Esplugues, Barcelona, Испания, 08950
        • Рекрутинг
        • Novartis Investigative Site
      • L'Hospitalet de Llobregat, Barcelona, Испания, 08907
        • Рекрутинг
        • Novartis Investigative Site
  • Италия
      • Naples, Италия, 80122
        • Рекрутинг
        • Novartis Investigative Site
    • BO
      • Bologna, BO, Италия, 40138
        • Рекрутинг
        • Novartis Investigative Site
    • MI
      • Milan, MI, Италия, 20122
        • Рекрутинг
        • Novartis Investigative Site
    • RM
      • Roma, RM, Италия, 00165
        • Рекрутинг
        • Novartis Investigative Site
      • Roma, RM, Италия, 00168
        • Рекрутинг
        • Novartis Investigative Site
    • TO
      • Torino, TO, Италия, 10126
        • Рекрутинг
        • Novartis Investigative Site
  • Нидерланды
      • Nijmegen, Нидерланды, 6525 GA
        • Рекрутинг
        • Novartis Investigative Site
    • South Holland
      • Rotterdam, South Holland, Нидерланды, 3015 GD
        • Рекрутинг
        • Novartis Investigative Site
  • Соединенное Королевство
      • London, Соединенное Королевство, WC1N 3JH
        • Рекрутинг
        • Novartis Investigative Site
  • Соединенные Штаты
    • California
      • Oakland, California, Соединенные Штаты, 94609
        • Рекрутинг
        • UCSF Benioff Children s Hospital
        • Главный следователь:
          • Beth Apsel Winger
        • Контакт:
          • Sophie Shean
          • Номер телефона: +1 510 428 3885 8227
          • Электронная почта: sophie.shean@ucsf.edu
      • Orange, California, Соединенные Штаты, 92868
        • Рекрутинг
        • Childrens Hospital of Orange County
        • Контакт:
          • Eliud Espinal
          • Номер телефона: 714-509-8810
          • Электронная почта: eliud.espinal@choc.org
        • Главный следователь:
          • Maria Buethe
      • Palo Alto, California, Соединенные Штаты, 94304
        • Рекрутинг
        • Lucile Packard Childrens Hosp
        • Главный следователь:
          • Joyce Teng
        • Контакт:
    • District of Columbia
      • Washington D.C., District of Columbia, Соединенные Штаты, 20010-2970
        • Рекрутинг
        • Childrens National Medical Center
        • Контакт:
        • Главный следователь:
          • Yaser Diab
    • Florida
      • Jacksonville, Florida, Соединенные Штаты, 32207
        • Рекрутинг
        • Nemours Childrens Clinic
        • Контакт:
        • Главный следователь:
          • Anderson Collier
    • Massachusetts
      • Boston, Massachusetts, Соединенные Штаты, 02115
        • Рекрутинг
        • Childrens Hosp Boston Dept of Heme
        • Главный следователь:
          • Tishi Shah
        • Контакт:
    • Missouri
      • St Louis, Missouri, Соединенные Штаты, 63110
        • Рекрутинг
        • WA Uni School Of Med
        • Главный следователь:
          • Bryan Sisk
        • Контакт:
    • North Carolina
      • Chapel Hill, North Carolina, Соединенные Штаты, 27599
        • Рекрутинг
        • University of North Carolina at Chapel Hill
        • Контакт:
        • Главный следователь:
          • Alexandra Borst
    • Ohio
      • Cincinnati, Ohio, Соединенные Штаты, 45229-3039
        • Рекрутинг
        • Cinn Children Hosp Medical Center
        • Главный следователь:
          • Adrienne Hammill
        • Контакт:
          • Sarah Price
          • Номер телефона: 513-636-6090
          • Электронная почта: Sarah.Price@cchmc.org
      • Cleveland, Ohio, Соединенные Штаты, 44195
        • Рекрутинг
        • Cleveland Clinic Foundation
        • Главный следователь:
          • Michael Kelly
        • Контакт:
      • Cleveland, Ohio, Соединенные Штаты, 44106
        • Рекрутинг
        • Univ Hospital Of Cleveland
        • Главный следователь:
          • Howard Wang
        • Контакт:
      • Columbus, Ohio, Соединенные Штаты, 43205
        • Рекрутинг
        • Nationwide Children s Hospital
        • Контакт:
        • Главный следователь:
          • Bhuvana Setty
    • Oregon
      • Portland, Oregon, Соединенные Штаты, 97239
        • Рекрутинг
        • Oregon Health Science University
        • Главный следователь:
          • Melinda Wu
        • Контакт:
    • Pennsylvania
      • Philadelphia, Pennsylvania, Соединенные Штаты, 19104
        • Рекрутинг
        • CHOP Abramson Pediatric Resch Ctr
        • Главный следователь:
          • Denise Adams
        • Контакт:
      • Pittsburgh, Pennsylvania, Соединенные Штаты, 15224
        • Рекрутинг
        • Childrens Hosp Pittsburgh UPMC
        • Контакт:
          • Alex Berkebile
          • Номер телефона: 412-692-5346
          • Электронная почта: berkebileak@upmc.edu
        • Главный следователь:
          • Julia Segal
    • Texas
      • Houston, Texas, Соединенные Штаты, 77030
        • Рекрутинг
        • U of TX Health Science Ct
        • Главный следователь:
          • Autumn Atkinson
        • Контакт:
      • Houston, Texas, Соединенные Штаты, 77030
        • Рекрутинг
        • Baylor College Of Medicine
        • Главный следователь:
          • Ionela Iacobas
        • Контакт:
    • Washington
      • Seattle, Washington, Соединенные Штаты, 98105
        • Рекрутинг
        • Childrens Hospital and Regional Medical Center
        • Главный следователь:
          • Jonathan A Perkins
        • Контакт:
  • Франция
      • Angers, Франция, 49933
        • Рекрутинг
        • Novartis Investigative Site
      • Bordeaux, Франция, 33076
        • Рекрутинг
        • Novartis Investigative Site
      • Bron, Франция, 69677
        • Рекрутинг
        • Novartis Investigative Site
      • Caen, Франция, 14033
        • Рекрутинг
        • Novartis Investigative Site
      • Dijon, Франция, 21000
        • Рекрутинг
        • Novartis Investigative Site
      • Lille, Франция, 59000
        • Рекрутинг
        • Novartis Investigative Site
      • Marseille, Франция, 13885
        • Рекрутинг
        • Novartis Investigative Site
      • Montpellier, Франция, 34295
        • Рекрутинг
        • Novartis Investigative Site
      • Paris, Франция, 75015
        • Рекрутинг
        • Novartis Investigative Site
      • Paris, Франция, 75010
        • Рекрутинг
        • Novartis Investigative Site
      • Toulouse, Франция, 31054
        • Рекрутинг
        • Novartis Investigative Site
      • Tours, Франция, 37044
        • Рекрутинг
        • Novartis Investigative Site
  • Чехия
      • Brno, Чехия, 625 00
        • Рекрутинг
        • Novartis Investigative Site
  • Швейцария
      • Lausanne, Швейцария, 1011
        • Рекрутинг
        • Novartis Investigative Site

Критерии участия

Исследователи ищут людей, которые соответствуют определенному описанию, называемому критериям приемлемости. Некоторыми примерами этих критериев являются общее состояние здоровья человека или предшествующее лечение.

Критерии приемлемости

Возраст, подходящий для обучения

От 1 секунда до 100 лет (Ребенок, Взрослый, Пожилой взрослый)

Принимает здоровых добровольцев

Нет

Полы, имеющие право на обучение

Все

Описание

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

Учебный план

В этом разделе представлена ​​подробная информация о плане исследования, в том числе о том, как планируется исследование и что оно измеряет.

Как устроено исследование?

Детали дизайна

  • Основная цель: Уход
  • Распределение: Рандомизированный
  • Интервенционная модель: Последовательное назначение
  • Маскировка: Четырехместный

Оружие и интервенции

Группа участников / Армия
Вмешательство/лечение
Экспериментальный: 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)
Лекарство: 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

Другие имена:
  • 719 бел.руб.
Экспериментальный: 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).
Лекарство: 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

Другие имена:
  • 719 бел.руб.
Экспериментальный: 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)
Лекарство: 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

Другие имена:
  • 719 бел.руб.
Экспериментальный: 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).
Лекарство: 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

Другие имена:
  • 719 бел.руб.
Экспериментальный: 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)
Лекарство: 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

Другие имена:
  • 719 бел.руб.
Плацебо Компаратор: Adult participants, placebo (Stage 2)

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

In Stage 2, participants will receive matching placebo for 24 weeks of the study
Экспериментальный: 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)
Лекарство: 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

Другие имена:
  • 719 бел.руб.
Плацебо Компаратор: Pediatric participants (6-17 years of age), placebo (Stage 2)

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

In Stage 2, participants will receive matching placebo for 24 weeks of the study
Экспериментальный: 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
Лекарство: 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

Другие имена:
  • 719 бел.руб.

Что измеряет исследование?

Первичные показатели результатов

Мера результата
Мера Описание
Временное ограничение
Stage 2:Radiological response rate at Week 24 of Stage 2 (adult and pediatric (6 - 17 years of age) participants)
Временное ограничение: 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

Вторичные показатели результатов

Мера результата
Мера Описание
Временное ограничение
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)
Временное ограничение: 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)
Временное ограничение: 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)
Временное ограничение: 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)
Временное ограничение: 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)
Временное ограничение: 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)
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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

Соавторы и исследователи

Здесь вы найдете людей и организации, участвующие в этом исследовании.

Спонсор

Novartis Pharmaceuticals

Следователи

  • Директор по исследованиям: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Даты записи исследования

Эти даты отслеживают ход отправки отчетов об исследованиях и сводных результатов на сайт ClinicalTrials.gov. Записи исследований и сообщаемые результаты проверяются Национальной медицинской библиотекой (NLM), чтобы убедиться, что они соответствуют определенным стандартам контроля качества, прежде чем публиковать их на общедоступном веб-сайте.

Изучение основных дат

Начало исследования (Действительный)

24 ноября 2023 г.

Первичное завершение (Оцененный)

31 мая 2028 г.

Завершение исследования (Оцененный)

2 мая 2033 г.

Даты регистрации исследования

Первый отправленный

4 июля 2023 г.

Впервые представлено, что соответствует критериям контроля качества

4 июля 2023 г.

Первый опубликованный (Действительный)

17 июля 2023 г.

Обновления учебных записей

Последнее опубликованное обновление (Действительный)

9 октября 2026 г.

Последнее отправленное обновление, отвечающее критериям контроля качества

8 октября 2026 г.

Последняя проверка

1 октября 2026 г.

Дополнительная информация

Термины, связанные с этим исследованием

Ключевые слова

Дополнительные соответствующие термины MeSH

Другие идентификационные номера исследования

  • CBYL719P12201
  • 2023-504146-60-00 (Идентификатор реестра: EU CTIS number)
  • 2023 (Грант/контракт NIH США)

Планирование данных отдельных участников (IPD)

Планируете делиться данными об отдельных участниках (IPD)?

ДА

Описание плана 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

Информация о лекарствах и устройствах, исследовательские документы

Изучает лекарственный продукт, регулируемый FDA США.

Да

Изучает продукт устройства, регулируемый Управлением по санитарному надзору за качеством пищевых продуктов и медикаментов США.

Нет

Эта информация была получена непосредственно с веб-сайта clinicaltrials.gov без каких-либо изменений. Если у вас есть запросы на изменение, удаление или обновление сведений об исследовании, обращайтесь по адресу register@clinicaltrials.gov. Как только изменение будет реализовано на clinicaltrials.gov, оно будет автоматически обновлено и на нашем веб-сайте. .

Подписаться