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Alpelisib 用于治疗与 PIK3CA 突变相关的淋巴畸形的儿童和成人患者。 (EPIK-L1)

2026年10月8日 更新者：Novartis Pharmaceuticals

一项两阶段双盲、随机、安慰剂对照研究，旨在评估 Alpelisib 在患有 PIK3CA 突变相关淋巴畸形的儿童和成人患者中的疗效、安全性和药代动力学。

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.

研究概览

地位

招聘中

条件

干预/治疗

详细说明

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.

研究类型

介入性

注册 (估计的)

232

阶段

  • 阶段2
  • 第三阶段

扩展访问

可用的 查看扩展访问记录。

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习联系方式

研究联系人备份

学习地点

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

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

1秒 至 100年 (孩子、成人、年长者)

接受健康志愿者

不

有资格学习的性别

全部

描述

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

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 分配：随机化
  • 介入模型：顺序分配
  • 屏蔽：四人间

武器和干预

参与者组/臂
干预/治疗
实验性的：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)
药品：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

其他名称：
  • BYL719
实验性的：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).
药品：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

其他名称：
  • BYL719
实验性的：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)
药品：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

其他名称：
  • BYL719
实验性的：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).
药品：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

其他名称：
  • BYL719
实验性的：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)
药品：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

其他名称：
  • BYL719
安慰剂比较：Adult participants, placebo (Stage 2)

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

In Stage 2, participants will receive matching placebo for 24 weeks of the study
实验性的：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)
药品：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

其他名称：
  • BYL719
安慰剂比较：Pediatric participants (6-17 years of age), placebo (Stage 2)

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

In Stage 2, participants will receive matching placebo for 24 weeks of the study
实验性的：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
药品：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

其他名称：
  • BYL719

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Stage 2:Radiological response rate at Week 24 of Stage 2 (adult and pediatric (6 - 17 years of age) participants)
大体时间：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

次要结果测量

结果测量
措施说明
大体时间
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)
大体时间：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)
大体时间：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)
大体时间：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)
大体时间：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)
大体时间：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)
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

Novartis Pharmaceuticals

调查人员

  • 研究主任：Novartis Pharmaceuticals、Novartis Pharmaceuticals

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2023年11月24日

初级完成 (估计的)

2028年5月31日

研究完成 (估计的)

2033年5月2日

研究注册日期

首次提交

2023年7月4日

首先提交符合 QC 标准的

2023年7月4日

首次发布 (实际的)

2023年7月17日

研究记录更新

最后更新发布 (实际的)

2026年10月9日

上次提交的符合 QC 标准的更新

2026年10月8日

最后验证

2026年10月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • CBYL719P12201
  • 2023-504146-60-00 (注册表标识符：EU CTIS number)
  • 2023 (美国 NIH 拨款/合同)

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

是的

IPD 计划说明

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

药物和器械信息、研究文件

研究美国 FDA 监管的药品

是的

研究美国 FDA 监管的设备产品

不

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

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