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RecruitingPhase 2/Phase 3

Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.

Novartis Pharmaceuticals · NCT05948943

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 0 mo–100 · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 2, Phase 3
Target enrollment
232
US sites recruiting
18
Study start
November 24, 2023
Record last updated
October 9, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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.

Being studied: Alpelisib; Placebo

Conditions: Lymphatic Malformations

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full criteria

Key inclusion criteria

  • Signed informed consent and assent (when applicable) from the participant, parent, legal authorized representative or guardian.
  • 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.
  • 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).
  • 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.
  • Participant has evidence of a somatic mutation(s) in the PIK3CA gene prior to randomization.
  • Participant has at least one measurable LyM lesion confirmed by BIRC assessment prior to randomization.
  • 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

  • Participant has a physician-confirmed and documented diagnosis of PROS at the time of informed consent.
  • 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.
  • Participant has a known history of Stevens-Johnson syndrome, erythema multiforme, or toxic epidermal necrolysis at the time of informed consent.
  • Participant has an established diagnosis of type I diabetes mellitus or uncontrolled type II diabetes mellitus at the time of informed consent.
  • 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

Recruiting US locations

  • UCSF Benioff Children s Hospital

    Oakland, CA 94609

    Site contact: Sophie Shean · +1 510 428 3885 8227 · sophie.shean@ucsf.edu

  • Childrens Hospital of Orange County

    Orange, CA 92868

    Site contact: Eliud Espinal · 714-509-8810 · eliud.espinal@choc.org

  • Lucile Packard Childrens Hosp

    Palo Alto, CA 94304

    Site contact: Thomas Buschbacher · tbusch@stanford.edu

  • Childrens National Medical Center

    Washington D.C., DC 20010-2970

    Site contact: Regine Hyppolite · rhyppolite@childrensnational.org

  • Nemours Childrens Clinic

    Jacksonville, FL 32207

    Site contact: Rosita Almira · 904-697-3674 · rosita.almira@nemours.org

  • Childrens Hosp Boston Dept of Heme

    Boston, MA 02115

    Site contact: Kelsey Deemer · 617-355-5526 · kelsey.deemer@childrens.harvard.edu

  • WA Uni School Of Med

    St Louis, MO 63110

    Site contact: Alison Barnwell · abarnwell@wustl.edu

  • University of North Carolina at Chapel Hill

    Chapel Hill, NC 27599

    Site contact: Miriam Davis · miriam.davis@unc.edu

  • Cinn Children Hosp Medical Center

    Cincinnati, OH 45229-3039

    Site contact: Sarah Price · 513-636-6090 · Sarah.Price@cchmc.org

  • Univ Hospital Of Cleveland

    Cleveland, OH 44106

    Site contact: Erika Waites · 216-844-7028 · Erika.Waites@uhhospitals.org

  • Cleveland Clinic Foundation

    Cleveland, OH 44195

    Site contact: Natalie Mau · maun4@ccf.org

  • Nationwide Children s Hospital

    Columbus, OH 43205

    Site contact: Star Seum · star.seum@nationwidechildrens.org

  • Oregon Health Science University

    Portland, OR 97239

    Site contact: Annie Yang · yangann@ohsu.edu

  • CHOP Abramson Pediatric Resch Ctr

    Philadelphia, PA 19104

    Site contact: Melissa Casey · CASEYMA@chop.edu

  • Childrens Hosp Pittsburgh UPMC

    Pittsburgh, PA 15224

    Site contact: Alex Berkebile · 412-692-5346 · berkebileak@upmc.edu

  • Baylor College Of Medicine

    Houston, TX 77030

    Site contact: Gaylon Stevenson · 713-798-3701 · gnsteve1@texaschildrens.org

  • U of TX Health Science Ct

    Houston, TX 77030

    Site contact: Kelly Mulligan · 713-500-6852 · kelly.mulligan@uth.tmc.edu

  • Childrens Hospital and Regional Medical Center

    Seattle, WA 98105

    Site contact: Maya Younoszai · 206-526-2100 · maya.younosza@seattlechildrens.org

Study contacts (from the official record)

  • Novartis Pharmaceuticals · 1-888-669-6682 · novartis.email@novartis.com
  • Novartis Pharmaceuticals · +41613241111 · novartis.email@novartis.com
Alpelisib in Pediatric and Adult Patients With Lymphatic… (NCT05948943) | Bored Studies