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Alpelisib hos pediatriske og voksne pasienter med lymfatiske misdannelser assosiert med en PIK3CA-mutasjon. (EPIK-L1)

8. oktober 2026 oppdatert av: Novartis Pharmaceuticals

En to-trinns dobbeltblind, randomisert, placebokontrollert studie for å vurdere effektiviteten, sikkerheten og farmakokinetikken til Alpelisib hos pediatriske og voksne pasienter med lymfatiske misdannelser assosiert med en PIK3CA-mutasjon.

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.

Studieoversikt

Status

Status

Indikerer gjeldende rekrutteringsstatus eller utvidet tilgangsstatus.
Rekruttering

Forhold

Forhold

Sykdommen, lidelsen, syndromet, sykdommen eller skaden som studeres. På ClinicalTrials.gov kan tilstander også inkludere andre helserelaterte problemer, som levetid, livskvalitet og helserisiko.

Intervensjon / Behandling

Intervensjon / Behandling

En prosess eller handling som er fokus for en klinisk studie. Intervensjoner inkluderer legemidler, medisinsk utstyr, prosedyrer, vaksiner og andre produkter som enten er undersøkende eller allerede er tilgjengelige. Intervensjoner kan også inkludere ikke-invasive tilnærminger, som utdanning eller modifisering av kosthold og trening.

Detaljert beskrivelse

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.

Studietype

Studietype

Beskriver arten av en klinisk studie. Studietyper inkluderer intervensjonsstudier (også kalt kliniske studier), observasjonsstudier (inkludert pasientregistre) og utvidet tilgang.
Intervensjonell

Registrering (Antatt)

Registrering

Antall deltakere i en klinisk studie. Den "forventede" påmeldingen er måltallet for deltakere som forskerne trenger for studien.
232

Fase

Fase

Stadiet av en klinisk studie som studerer et medikament eller biologisk produkt, basert på definisjoner utviklet av U.S. Food and Drug Administration (FDA). Fasen er basert på studiens målsetting, antall deltakere og andre egenskaper. Det er fem faser: Tidlig fase 1 (tidligere oppført som fase 0), fase 1, fase 2, fase 3 og fase 4. Ikke relevant brukes til å beskrive studier uten FDA-definerte faser, inkludert utprøving av enheter eller atferdsintervensjoner.
  • Fase 2
  • Fase 3

Utvidet tilgang

Utvidet tilgang

En måte for pasienter med alvorlige sykdommer eller tilstander som ikke kan delta i en klinisk studie for å få tilgang til et medisinsk produkt som ikke er godkjent av U.S. Food and Drug Administration (FDA). Også kalt medfølende bruk. Det finnes forskjellige utvidede tilgangstyper.
Tilgjengelig

Tilgjengelig

  • Tilgjengelig: Utvidet tilgang er for øyeblikket tilgjengelig for denne undersøkelsesbehandlingen, og pasienter som ikke er deltakere i den kliniske studien kan kanskje få tilgang til stoffet, biologisk eller medisinsk utstyr undersøkes.
  • Ikke lenger tilgjengelig: Utvidet tilgang var tilgjengelig for denne intervensjonen tidligere, men er ikke tilgjengelig for øyeblikket og vil ikke være tilgjengelig i fremtiden.
  • Midlertidig ikke tilgjengelig: Utvidet tilgang er for øyeblikket ikke tilgjengelig for denne intervensjonen, men forventes å være tilgjengelig i fremtiden.
  • Godkjent for markedsføring: Intervensjonen har blitt godkjent av U.S. Food and Drug Administration for bruk av publikum.
 utenfor den kliniske utprøvingen. Se utvidet tilgangspost.

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiekontakt

Studiekontakt

Navn og kontaktinformasjon for personen som kan svare på påmeldingsspørsmål for en klinisk studie. Hvert sted hvor studien gjennomføres kan også ha en spesifikk kontakt, som kan være bedre i stand til å svare på disse spørsmålene.

Studer Kontakt Backup

Studiesteder

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

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Kvalifikasjonskriterier

Nøkkelkravene som personer som ønsker å delta i en klinisk studie må oppfylle eller egenskapene de må ha. Kvalifikasjonskriterier består av både inklusjonskriterier (som kreves for at en person skal delta i studien) og eksklusjonskriterier (som hindrer en person i å delta). Typer kvalifikasjonskriterier inkluderer om en studie godtar friske frivillige, har alders- eller aldersgruppekrav, eller er begrenset av kjønn.

Alder som er kvalifisert for studier

1 sekund til 100 år (Barn, Voksen, Eldre voksen)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Beskrivelse

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

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Randomisert
  • Intervensjonsmodell: Sekvensiell tildeling
  • Masking: Firemannsrom

Våpen og intervensjoner

Deltakergruppe / Arm

Deltakergruppe / Arm

En gruppe eller undergruppe av deltakere i en klinisk studie som mottar en spesifikk intervensjon/behandling, eller ingen intervensjon, i henhold til studiens protokoll.
Intervensjon / Behandling

Intervensjon / Behandling

En prosess eller handling som er fokus for en klinisk studie. Intervensjoner inkluderer legemidler, medisinsk utstyr, prosedyrer, vaksiner og andre produkter som enten er undersøkende eller allerede er tilgjengelige. Intervensjoner kan også inkludere ikke-invasive tilnærminger, som utdanning eller modifisering av kosthold og trening.
Eksperimentell: 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)
Legemiddel: 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

Andre navn:
  • BYL719
Eksperimentell: 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).
Legemiddel: 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

Andre navn:
  • BYL719
Eksperimentell: 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)
Legemiddel: 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

Andre navn:
  • BYL719
Eksperimentell: 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).
Legemiddel: 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

Andre navn:
  • BYL719
Eksperimentell: 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)
Legemiddel: 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

Andre navn:
  • BYL719
Placebo komparator: Adult participants, placebo (Stage 2)

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

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

Andre navn:
  • BYL719
Placebo komparator: Pediatric participants (6-17 years of age), placebo (Stage 2)

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

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

Andre navn:
  • BYL719

Hva måler studien?

Primære resultatmål

Primære resultatmål

I en klinisk studies protokoll er det planlagte utfallsmålet som er det viktigste for å evaluere effekten av en intervensjon/behandling. De fleste kliniske studier har ett primært utfallsmål, men noen har mer enn ett.
Resultatmål
Tiltaksbeskrivelse
Tidsramme
Stage 2:Radiological response rate at Week 24 of Stage 2 (adult and pediatric (6 - 17 years of age) participants)
Tidsramme: 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

Sekundære resultatmål

Sekundære resultatmål

I en klinisk studies protokoll, et planlagt utfallsmål som ikke er like viktig som det primære utfallsmålet for å evaluere effekten av en intervensjon, men som fortsatt er av interesse. De fleste kliniske studier har mer enn ett sekundært utfallsmål.
Resultatmål
Tiltaksbeskrivelse
Tidsramme
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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Sponsor

Organisasjonen eller personen som initierer studien og som har myndighet og kontroll over studien.
Novartis Pharmaceuticals

Etterforskere

Etterforskere

En forsker involvert i en klinisk studie. Beslektede termer inkluderer stedets hovedetterforsker, stedsunderetterforsker, studieleder, studieleder og studiehovedetterforsker.
  • Studieleder: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

Studiestart

Den faktiske datoen da den første deltakeren ble registrert i en klinisk studie. Den "forventede" studiestartdatoen er datoen som forskerne tror vil være studiestartdatoen.
24. november 2023

Primær fullføring (Antatt)

Primær fullføring

Datoen da den siste deltakeren i en klinisk studie ble undersøkt eller mottok en intervensjon for å samle inn endelige data for det primære utfallsmålet. Hvorvidt den kliniske studien ble avsluttet i henhold til protokollen eller ble avsluttet, påvirker ikke denne datoen. For kliniske studier med mer enn ett primært utfallsmål med ulike fullføringsdatoer, refererer dette begrepet til datoen da datainnsamlingen er fullført for alle de primære utfallsmålene. Den "forventede" primære fullføringsdatoen er datoen som forskerne tror vil være den primære fullføringsdatoen for studien.
31. mai 2028

Studiet fullført (Antatt)

Studiet fullført

Datoen da den siste deltakeren i en klinisk studie ble undersøkt eller mottok en intervensjon/behandling for å samle inn endelige data for de primære utfallsmålene, sekundære utfallsmålene og uønskede hendelsene (det vil si siste deltakers siste besøk). Den "forventede" studiens fullføringsdato er datoen som forskerne tror vil være studiens fullføringsdato.
2. mai 2033

Datoer for studieregistrering

Først innsendt

Først innsendt

Datoen da studiesponsoren eller etterforskeren først sendte inn en studiepost til ClinicalTrials.gov. Det er vanligvis en forsinkelse på noen dager mellom første innsendte dato og postens tilgjengelighet på ClinicalTrials.gov (den første postedatoen).
4. juli 2023

Først innsendt som oppfylte QC-kriteriene

Først innsendt som oppfylte QC-kriteriene

Datoen da studiesponsoren eller etterforskeren først sender inn en studiepost som er i samsvar med gjennomgangskriteriene for National Library of Medicine (NLM) kvalitetskontroll (QC). Sponsoren eller etterforskeren må kanskje revidere og sende inn en studiepost én eller flere ganger før NLMs kvalitetskontrollkriterier oppfylles. Det er sponsorens eller etterforskerens ansvar å sikre at studieposten er i samsvar med NLM QC-gjennomgangskriteriene.
4. juli 2023

Først lagt ut (Faktiske)

Først lagt ut

Datoen da studieposten først var tilgjengelig på ClinicalTrials.gov etter at National Library of Medicine (NLM) kvalitetskontroll (QC) gjennomgang har konkludert. Det er vanligvis en forsinkelse på noen dager mellom datoen studiesponsoren eller etterforskeren sendte inn studieposten og den første postedatoen.
17. juli 2023

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

Sist oppdatering lagt ut

Den siste datoen da endringer i en studiepost ble gjort tilgjengelig på ClinicalTrials.gov. Det kan være en forsinkelse mellom når endringene ble sendt til ClinicalTrials.gov av studiens sponsor eller etterforsker (datoen for siste innsending av oppdatering) og datoen for siste oppdatering.
9. oktober 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

Siste oppdatering sendt inn som oppfylte QC-kriteriene

Den siste datoen da studiesponsoren eller etterforskeren sendte inn endringer i en studiepost som er i samsvar med gjennomgangskriteriene for National Library of Medicine (NLM) kvalitetskontroll (QC). Det er sponsorens eller etterforskerens ansvar å sikre at studieposten er i samsvar med NLM QC-gjennomgangskriteriene.
8. oktober 2026

Sist bekreftet

Sist bekreftet

Den siste datoen da studiesponsoren eller etterforskeren bekreftet at informasjonen om en klinisk studie på ClinicalTrials.gov var nøyaktig og oppdatert. Hvis en studie med rekrutteringsstatus for rekruttering; ikke rekrutterer ennå; eller aktiv, ikke rekruttering har ikke blitt bekreftet de siste 2 årene, studiens rekrutteringsstatus vises som ukjent.
1. oktober 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

Andre studie-ID-numre

Identifikatorer eller ID-numre andre enn NCT-nummeret som er tildelt en klinisk studie av studiens sponsor, finansierere eller andre. Disse tallene kan inkludere unike identifikatorer fra andre prøveregistre og National Institutes of Health-stipendnumre.
  • CBYL719P12201
  • 2023-504146-60-00 (Registeridentifikator: EU CTIS number)
  • 2023 (U.S. NIH-stipend/kontrakt)

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

JA

IPD-planbeskrivelse

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

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

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