Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation. (EPIK-L1)

May 11, 2026 updated by: Novartis Pharmaceuticals

A Two-stage Double-blind, Randomized, Placebo-controlled Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Interventional

Enrollment (Estimated)

232

Phase

  • Phase 2
  • Phase 3

Expanded Access

Available outside the clinical trial. See expanded access record.

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Argentina
      • Capital Federal, Argentina, C1023AAB
        • Recruiting
        • Novartis Investigative Site
    • Buenos Aires
      • CABA, Buenos Aires, Argentina, C1181ACH
        • Recruiting
        • Novartis Investigative Site
      • CABA, Buenos Aires, Argentina, C1425BEA
        • Recruiting
        • Novartis Investigative Site
  • Australia
    • New South Wales
      • Sydney, New South Wales, Australia, 2010
        • Recruiting
        • Novartis Investigative Site
      • Sydney, New South Wales, Australia, 2031
        • Recruiting
        • Novartis Investigative Site
    • Queensland
      • Brisbane, Queensland, Australia, 4101
        • Recruiting
        • Novartis Investigative Site
  • Belgium
      • Brussels, Belgium, 1200
        • Recruiting
        • Novartis Investigative Site
  • France
      • Angers, France, 49933
        • Recruiting
        • Novartis Investigative Site
      • Bordeaux, France, 33076
        • Recruiting
        • Novartis Investigative Site
      • Bron, France, 69677
        • Recruiting
        • Novartis Investigative Site
      • Caen, France, 14033
        • Recruiting
        • Novartis Investigative Site
      • Dijon, France, 21000
        • Recruiting
        • Novartis Investigative Site
      • Lille, France, 59000
        • Recruiting
        • Novartis Investigative Site
      • Marseille, France, 13885
        • Recruiting
        • Novartis Investigative Site
      • Montpellier, France, 34295
        • Recruiting
        • Novartis Investigative Site
      • Paris, France, 75015
        • Recruiting
        • Novartis Investigative Site
      • Paris, France, 75010
        • Recruiting
        • Novartis Investigative Site
      • Toulouse, France, 31054
        • Recruiting
        • Novartis Investigative Site
      • Tours, France, 37044
        • Recruiting
        • Novartis Investigative Site
  • Germany
      • Berlin, Germany, 13353
        • Recruiting
        • Novartis Investigative Site
    • Baden-Wurttemberg
      • Freiburg im Breisgau, Baden-Wurttemberg, Germany, 79106
        • Recruiting
        • Novartis Investigative Site
      • Mannheim, Baden-Wurttemberg, Germany, 68305
        • Withdrawn
        • Novartis Investigative Site
    • North Rhine-Westphalia
      • Cologne, North Rhine-Westphalia, Germany, 50937
        • Recruiting
        • Novartis Investigative Site
    • Saxony
      • Leipzig, Saxony, Germany, 04103
        • Recruiting
        • Novartis Investigative Site
  • Italy
      • Naples, Italy, 80122
        • Recruiting
        • Novartis Investigative Site
    • BO
      • Bologna, BO, Italy, 40138
        • Recruiting
        • Novartis Investigative Site
    • MI
      • Milan, MI, Italy, 20122
        • Recruiting
        • Novartis Investigative Site
    • RM
      • Roma, RM, Italy, 00165
        • Recruiting
        • Novartis Investigative Site
      • Roma, RM, Italy, 00168
        • Recruiting
        • Novartis Investigative Site
    • TO
      • Torino, TO, Italy, 10126
        • Recruiting
        • Novartis Investigative Site
  • Netherlands
    • Gelderland
      • Nijmegen, Gelderland, Netherlands, 6500HB
        • Recruiting
        • Novartis Investigative Site
    • South Holland
      • Rotterdam, South Holland, Netherlands, 3015 GD
        • Recruiting
        • Novartis Investigative Site
  • Spain
      • A Coruña, Spain, 15006
        • Recruiting
        • Novartis Investigative Site
      • Barcelona, Spain, 08035
        • Recruiting
        • Novartis Investigative Site
      • Madrid, Spain, 28046
        • Recruiting
        • Novartis Investigative Site
      • Madrid, Spain, 28009
        • Recruiting
        • Novartis Investigative Site
    • Balearic Islands
      • Palma, Balearic Islands, Spain, 07120
        • Recruiting
        • Novartis Investigative Site
    • Barcelona
      • Esplugues, Barcelona, Spain, 08950
        • Recruiting
        • Novartis Investigative Site
      • L'Hospitalet de Llobregat, Barcelona, Spain, 08907
        • Recruiting
        • Novartis Investigative Site
  • Switzerland
      • Lausanne, Switzerland, 1011
        • Recruiting
        • Novartis Investigative Site
  • United States
    • California
      • Oakland, California, United States, 94609
        • Recruiting
        • UCSF Benioff Children s Hospital
        • Principal Investigator:
          • Beth Apsel Winger
        • Contact:
      • Palo Alto, California, United States, 94304
        • Recruiting
        • Lucile Packard Childrens Hosp
        • Principal Investigator:
          • Joyce Teng
        • Contact:
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20010-2970
    • Florida
      • Jacksonville, Florida, United States, 32207
        • Recruiting
        • Nemours Childrens Clinic
        • Contact:
        • Principal Investigator:
          • Anderson Collier
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Recruiting
        • Childrens Hosp Boston Dept of Heme
        • Principal Investigator:
          • Tishi Shah
        • Contact:
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Recruiting
        • WA Uni School Of Med
        • Principal Investigator:
          • Bryan Sisk
        • Contact:
    • Ohio
      • Cincinnati, Ohio, United States, 45206
        • Recruiting
        • Cinn Children Hosp Medical Center
        • Principal Investigator:
          • Adrienne Hammill
        • Contact:
      • Cleveland, Ohio, United States, 44195
        • Recruiting
        • Cleveland Clinic Foundation
        • Contact:
        • Principal Investigator:
          • Michael Kelly
      • Cleveland, Ohio, United States, 44106
        • Recruiting
        • Univ Hospital Of Cleveland
        • Principal Investigator:
          • Howard Wang
        • Contact:
      • Columbus, Ohio, United States, 43205
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Oregon Health Science University
        • Principal Investigator:
          • Melinda Wu
        • Contact:
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • CHOP Abramson Pediatric Resch Ctr
        • Principal Investigator:
          • Denise Adams
        • Contact:
      • Pittsburgh, Pennsylvania, United States, 15224
        • Recruiting
        • Childrens Hosp Pittsburgh UPMC
        • Contact:
        • Principal Investigator:
          • Julia Segal
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • Baylor College of Medicine
        • Principal Investigator:
          • Ionela Iacobas
        • Contact:
      • Houston, Texas, United States, 77030
        • Recruiting
        • U of TX Health Science Ct
        • Principal Investigator:
          • Autumn Atkinson
        • Contact:
    • Washington
      • Seattle, Washington, United States, 98105
        • Recruiting
        • Childrens Hospital and Regional Medical Center
        • Principal Investigator:
          • Jonathan A Perkins
        • Contact:
          • Phone Number: 206-526-2100

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Sequential Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Adult participants, alpelisib dose 1 (Stage 1)
Adult participants (≥18 years of age) who will receive dose 1 of alpelisib an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1)
Drug: 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

Other Names:
  • BYL719
Experimental: Adult participants, alpelisib dose 2 (Stage 1)
Adult participants (≥18 years of age) who will receive dose 2 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1).
Drug: 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

Other Names:
  • BYL719
Experimental: Pediatric participants (6-17 years of age), alpelisib dose 2 (Stage 1)
Pediatric participants 6-17 years of age who will receive dose 2 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1)
Drug: 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

Other Names:
  • BYL719
Experimental: Pediatric participants (6-17 years of age), alpelisib dose 3 (Stage 1)
Pediatric participants 6-17 years of age who will receive dose 3 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1).
Drug: 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

Other Names:
  • BYL719
Experimental: Adult participants, alpelisib (Stage 2)
Adult participants (≥18 years of age) who will receive alpelisib at the dose selected for confirmatory phase in adult participants (Stage 2)
Drug: 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

Other Names:
  • BYL719
Placebo Comparator: Adult participants, placebo (Stage 2)
Adult participants (≥18 years of age) who will receive matching placebo
Drug: Placebo
In Stage 2, participants will receive matching placebo for 24 weeks of the study
Experimental: Pediatric participants (6-17 years of age), alpelisib (Stage 2)
Pediatric participants (6-17 years of age) who will receive alpelisib at the dose selected for confirmatory phase in pediatric participants (Stage 2)
Drug: 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

Other Names:
  • BYL719
Placebo Comparator: Pediatric participants (6-17 years of age), placebo (Stage 2)
Pediatric participants (6-17 years of age) who will receive matching placebo
Drug: Placebo
In Stage 2, participants will receive matching placebo for 24 weeks of the study
Experimental: Pediatric participants (0-5 years of age), alpelisib (Stage 2)
Pediatric participants of 0-5 years who will dose 3 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier
Drug: 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

Other Names:
  • BYL719

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Stage 2:Radiological response rate at Week 24 of Stage 2 (adult and pediatric (6 - 17 years of age) participants)
Time Frame: 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

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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)
Time Frame: 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: Change from baseline in patient global impression of change (PGI-C) scale (adult and pediatric (6-17 years of age) participants)
Time Frame: 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 investigator global impression of change (IGIC) scale (adult and pediatric (6-17 years of age) participants)
Time Frame: 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)
Time Frame: 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 2: Percentage of participants with a radiological response at Week 24 of Stage 2 (pediatric participants 0-5 years of age)
Time Frame: 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-reported outcomes measurement information system (PROMIS) profile domains(adult and pediatric (6-17 years of age) participants)
Time Frame: 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 1 and 2: Duration of response (DOR) in adult and pediatric participants who receive alpelisib
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Investigators

  • Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 24, 2023

Primary Completion (Estimated)

May 31, 2028

Study Completion (Estimated)

May 2, 2033

Study Registration Dates

First Submitted

July 4, 2023

First Submitted That Met QC Criteria

July 4, 2023

First Posted (Actual)

July 17, 2023

Study Record Updates

Last Update Posted (Actual)

May 12, 2026

Last Update Submitted That Met QC Criteria

May 11, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CBYL719P12201
  • 2023-504146-60-00 (Registry Identifier: EU CTIS number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

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

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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