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New drug trial aims to shrink painful lymphatic growths in kids and adults

NCT ID NCT05948943

Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Sep 18, 2026 · Updated 7 times

Summary

This study tests alpelisib, a drug that targets a specific gene mutation (PIK3CA), in people with lymphatic malformations—abnormal growths of lymph vessels. The trial includes children and adults and compares alpelisib to a placebo to see if it can shrink the growths and ease symptoms. Researchers are recruiting 232 participants across multiple sites.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
alpelisib (a targeted drug that blocks a specific protein called PI3K, which is overactive in these growths)
What this could lead to
If successful, this could provide a new treatment option to shrink lymphatic malformations and improve symptoms for people with PIK3CA mutations.
What could go wrong
This is an early-to-mid-stage trial, so results are not yet proven. The drug may cause side effects, and not all participants may respond.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

About 232 people

The number the study aims to enrol. It can still change while the study runs.

Started

Nov 2023

Expected to finish

May 2033

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

0 to 100 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

cystic hygroma lymphangioma Lymphatic Abnormalities lymphatic malformation PIK3CA-related overgrowth spectrum

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Lymphatic malformations

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    60 sites in 12 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Baylor College Of Medicine

    RECRUITING

    Houston, Texas, 77030, United States

  • CHOP Abramson Pediatric Resch Ctr

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Childrens Hosp Boston Dept of Heme

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • Childrens Hosp Pittsburgh UPMC

    RECRUITING

    Pittsburgh, Pennsylvania, 15224, United States

  • Childrens Hospital and Regional Medical Center

    RECRUITING

    Seattle, Washington, 98105, United States

  • Childrens Hospital of Orange County

    RECRUITING

    Orange, California, 92868, United States

  • Childrens National Medical Center

    RECRUITING

    Washington D.C., District of Columbia, 20010-2970, United States

  • Cinn Children Hosp Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229-3039, United States

  • Cleveland Clinic Foundation

    RECRUITING

    Cleveland, Ohio, 44195, United States

  • Lucile Packard Childrens Hosp

    RECRUITING

    Palo Alto, California, 94304, United States

  • Nationwide Children s Hospital

    RECRUITING

    Columbus, Ohio, 43205, United States

  • Nemours Childrens Clinic

    RECRUITING

    Jacksonville, Florida, 32207, United States

  • Novartis Investigative Site

    RECRUITING

    CABA, Buenos Aires, C1181ACH, Argentina

  • Novartis Investigative Site

    RECRUITING

    CABA, Buenos Aires, C1425BEA, Argentina

  • Novartis Investigative Site

    RECRUITING

    Capital Federal, C1023AAB, Argentina

  • Novartis Investigative Site

    RECRUITING

    Sydney, New South Wales, 2010, Australia

  • Novartis Investigative Site

    RECRUITING

    Sydney, New South Wales, 2031, Australia

  • Novartis Investigative Site

    RECRUITING

    Brisbane, Queensland, 4101, Australia

  • Novartis Investigative Site

    RECRUITING

    Brussels, 1200, Belgium

  • Novartis Investigative Site

    RECRUITING

    Brno, 625 00, Czechia

  • Novartis Investigative Site

    RECRUITING

    Angers, 49933, France

  • Novartis Investigative Site

    RECRUITING

    Bordeaux, 33076, France

  • Novartis Investigative Site

    RECRUITING

    Bron, 69677, France

  • Novartis Investigative Site

    RECRUITING

    Caen, 14033, France

  • Novartis Investigative Site

    RECRUITING

    Dijon, 21000, France

  • Novartis Investigative Site

    RECRUITING

    Lille, 59000, France

  • Novartis Investigative Site

    RECRUITING

    Marseille, 13885, France

  • Novartis Investigative Site

    RECRUITING

    Montpellier, 34295, France

  • Novartis Investigative Site

    RECRUITING

    Paris, 75010, France

  • Novartis Investigative Site

    RECRUITING

    Paris, 75015, France

  • Novartis Investigative Site

    RECRUITING

    Toulouse, 31054, France

  • Novartis Investigative Site

    RECRUITING

    Tours, 37044, France

  • Novartis Investigative Site

    RECRUITING

    Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

  • Novartis Investigative Site

    WITHDRAWN

    Mannheim, Baden-Wurttemberg, 68305, Germany

  • Novartis Investigative Site

    RECRUITING

    Cologne, North Rhine-Westphalia, 50937, Germany

  • Novartis Investigative Site

    RECRUITING

    Leipzig, Saxony, 04103, Germany

  • Novartis Investigative Site

    RECRUITING

    Berlin, 13353, Germany

  • Novartis Investigative Site

    RECRUITING

    Ulm, 89081, Germany

  • Novartis Investigative Site

    RECRUITING

    Bologna, BO, 40138, Italy

  • Novartis Investigative Site

    RECRUITING

    Milan, MI, 20122, Italy

  • Novartis Investigative Site

    RECRUITING

    Roma, RM, 00165, Italy

  • Novartis Investigative Site

    RECRUITING

    Roma, RM, 00168, Italy

  • Novartis Investigative Site

    RECRUITING

    Torino, TO, 10126, Italy

  • Novartis Investigative Site

    RECRUITING

    Naples, 80122, Italy

  • Novartis Investigative Site

    RECRUITING

    Rotterdam, South Holland, 3015 GD, Netherlands

  • Novartis Investigative Site

    RECRUITING

    Nijmegen, 6525 GA, Netherlands

  • Novartis Investigative Site

    RECRUITING

    Palma, Balearic Islands, 07120, Spain

  • Novartis Investigative Site

    RECRUITING

    Esplugues, Barcelona, 08950, Spain

  • Novartis Investigative Site

    RECRUITING

    L'Hospitalet de Llobregat, Barcelona, 08907, Spain

  • Novartis Investigative Site

    RECRUITING

    A Coruña, 15006, Spain

  • Novartis Investigative Site

    RECRUITING

    Barcelona, 08035, Spain

  • Novartis Investigative Site

    RECRUITING

    Madrid, 28009, Spain

  • Novartis Investigative Site

    RECRUITING

    Madrid, 28046, Spain

  • Novartis Investigative Site

    RECRUITING

    Lausanne, 1011, Switzerland

  • Novartis Investigative Site

    RECRUITING

    London, WC1N 3JH, United Kingdom

  • Oregon Health Science University

    RECRUITING

    Portland, Oregon, 97239, United States

  • U of TX Health Science Ct

    RECRUITING

    Houston, Texas, 77030, United States

  • UCSF Benioff Children s Hospital

    RECRUITING

    Oakland, California, 94609, United States

  • Univ Hospital Of Cleveland

    RECRUITING

    Cleveland, Ohio, 44106, United States

  • University of North Carolina at Chapel Hill

    RECRUITING

    Chapel Hill, North Carolina, 27599, United States

  • WA Uni School Of Med

    RECRUITING

    St Louis, Missouri, 63110, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.