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Alpelisib u dětských a dospělých pacientů s lymfatickými malformacemi spojenými s mutací PIK3CA. (EPIK-L1)

8. října 2026 aktualizováno: Novartis Pharmaceuticals

Dvoustupňová dvojitě zaslepená, randomizovaná, placebem kontrolovaná studie k posouzení účinnosti, bezpečnosti a farmakokinetiky Alpelisibu u pediatrických a dospělých pacientů s lymfatickými malformacemi spojenými s mutací 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.

Přehled studie

Postavení

Nábor

Podmínky

Intervence / Léčba

Detailní popis

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.

Typ studie

Intervenční

Zápis (Odhadovaný)

232

Fáze

  • Fáze 2
  • Fáze 3

Rozšířený přístup

Dostupný mimo klinické hodnocení. Viz rozšířený záznam o přístupu.

Kontakty a umístění

Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.

Studijní kontakt

Studijní záloha kontaktů

Studijní místa

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

Kritéria účasti

Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.

Kritéria způsobilosti

Věk způsobilý ke studiu

1 sekunda až 100 let (Dítě, Dospělý, Starší dospělý)

Přijímá zdravé dobrovolníky

Ne

Pohlaví způsobilá ke studiu

Všechno

Popis

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

Studijní plán

Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.

Jak je studie koncipována?

Detaily designu

  • Primární účel: Léčba
  • Přidělení: Randomizované
  • Intervenční model: Sekvenční přiřazení
  • Maskování: Čtyřnásobek

Zbraně a zásahy

Skupina účastníků / Arm
Intervence / Léčba
Experimentální: 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)
Lék: 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

Ostatní jména:
  • BYL719
Experimentální: 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).
Lék: 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

Ostatní jména:
  • BYL719
Experimentální: 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)
Lék: 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

Ostatní jména:
  • BYL719
Experimentální: 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).
Lék: 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

Ostatní jména:
  • BYL719
Experimentální: 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)
Lék: 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

Ostatní jména:
  • BYL719
Komparátor placeba: Adult participants, placebo (Stage 2)

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

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

Ostatní jména:
  • BYL719
Komparátor placeba: Pediatric participants (6-17 years of age), placebo (Stage 2)

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

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

Ostatní jména:
  • BYL719

Co je měření studie?

Primární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Stage 2:Radiological response rate at Week 24 of Stage 2 (adult and pediatric (6 - 17 years of age) participants)
Časové okno: 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

Sekundární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
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)
Časové okno: 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)
Časové okno: 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)
Časové okno: 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)
Časové okno: 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)
Časové okno: 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)
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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

Spolupracovníci a vyšetřovatelé

Zde najdete lidi a organizace zapojené do této studie.

Sponzor

Novartis Pharmaceuticals

Vyšetřovatelé

  • Ředitel studie: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Termíny studijních záznamů

Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.

Hlavní termíny studia

Začátek studia (Aktuální)

24. listopadu 2023

Primární dokončení (Odhadovaný)

31. května 2028

Dokončení studie (Odhadovaný)

2. května 2033

Termíny zápisu do studia

První předloženo

4. července 2023

První předloženo, které splnilo kritéria kontroly kvality

4. července 2023

První zveřejněno (Aktuální)

17. července 2023

Aktualizace studijních záznamů

Poslední zveřejněná aktualizace (Aktuální)

9. října 2026

Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality

8. října 2026

Naposledy ověřeno

1. října 2026

Více informací

Termíny související s touto studií

Klíčová slova

Další relevantní podmínky MeSH

Další identifikační čísla studie

  • CBYL719P12201
  • 2023-504146-60-00 (Identifikátor registru: EU CTIS number)
  • 2023 (Grant/smlouva NIH USA)

Plán pro data jednotlivých účastníků (IPD)

Plánujete sdílet data jednotlivých účastníků (IPD)?

ANO

Popis plánu 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

Informace o lécích a zařízeních, studijní dokumenty

Studuje lékový produkt regulovaný americkým FDA

Ano

Studuje produkt zařízení regulovaný americkým úřadem FDA

Ne

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