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Could a pill shrink abnormal growths in PROS? new trial hopes to find out.

NCT ID NCT04589650

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 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Aug 19, 2026 · Updated 3 times

Summary

This phase 2 study tests the drug alpelisib in 206 children and adults with PIK3CA-related overgrowth spectrum (PROS), a rare condition causing abnormal tissue growth. Participants receive either alpelisib or a placebo for 16 weeks to see if the drug can shrink growths by at least 20%. The goal is to find a safe and effective treatment for this condition.

What this could mean

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

Active substance
alpelisib (BYL719), a drug taken by mouth
What this could lead to
If it works, alpelisib could become the first approved treatment to shrink abnormal tissue growth and improve symptoms in people with PROS.
What could go wrong
This is a mid-stage trial with only 206 participants, so results may not apply to everyone. The drug may cause side effects like high blood sugar or rash, and not all patients 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

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

206 people

The number who actually took part.

Started

Apr 2021

Expected to finish

Jan 2031

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 days 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: * Signed informed consent and assent (when applicable) from the patient, parent, legal authorized representative, or guardian prior to any study-related screening procedures were performed. * Male or female patients age above 0 day at the time of informed consent: Group 1: ≥ 18 years old, Group 2: 6-17 years old, Group 3: ≥ 0-5 years old, Group 4: ≥ 2-5 years old, Group 5: 6-17 years old. * Patients with diagnosis of PROS with symptomatic and /or progressive overgrowth and at least one measurable PROS-related lesion confirmed by BIRC assessment who had syndromic disease or isolated features at the time of informed consent. Patients, who previously had been receiving systemic treatment for PROS, could enter the study. * Documented evidence of a somatic mutation(s) in the PIK3CA gene performed in local laboratories using a DNA-based test validated according to the local regulations at the time of informed consent. * A tissue sample (fresh or archival) was to be sent to a Novartis-designated central laboratory. * Karnofsky (in patients \> 16 years old at study entry)/Lansky (≤ 16 years of age at study entry) performance status index ≥ 50. * Adequate bone marrow and organ function as assessed by central laboratory for eligibility. * Presence of at least one PROS-related measurable lesion defined as a lesion with longest diameter ≥ 2 cm, when the volume could be accurately and reproducibly measured by MRI, and associated with complaints, clinical symptoms or functional limitations affecting the patient's everyday life. Measurability was confirmed by BIRC before randomization. * Able to swallow study drug (as assessed within 7 days before study treatment start): * Groups 1, 2, 4, and 5: FCT, or as drinkable suspension when applicable. * Group 3: granules. Drug administration via feeding tube is allowed. Key Exclusion Criteria: * Patient with only isolated macrodactyly, epidermal nevus/nevi and macroencephaly (the only clinical feature or a combination of any three of them), in absence of other PROS-related lesions at the time of informed consent. * Previous treatment with alpelisib and/or any other PI3K inhibitor(s). * Radiation exposure for PROS treatment purpose within the previous 12 months on those PROS areas, which were expected to qualify for target lesions (except lesion(s) progressing after completion of radiotherapy) at time of informed consent. * Debulking or other major surgery performed within 3 months at time of informed consent. * Clinically meaningful bleeding related to PROS: Grade 2 within 14 days or grade 3 and more within 28 days before study treatment start as per CTCAE v4.03. * Clinically meaningful PROS-related thrombotic event (grade 2 and more as per CTCAE v4.03) within 30 days before informed consent, and/or sclerotherapy/embolization for vascular complications performed within 6 weeks before informed consent. * History of prior and or ongoing malignancy or ongoing investigations or treatment for malignancy at time of informed consent. * Clinically significant heart disease at time of informed consent. * Patients in Groups 1, 2, and 5 with documented pneumonitis or interstitial lung disease at time of informed consent and with impaired lung function (e.g., FEV1 or DLCO ≤ 70% of predicted) that was not related to PROS. Patients in Groups 3 and 4 with documented or suspicious pneumonitis or interstitial lung disease based on MRI images at time of informed consent. * History of acute pancreatitis within 1 year before informed consent or past medical history of chronic pancreatitis at time of informed consent. * Patients with an established diagnosis of type I diabetes mellitus or uncontrolled type II diabetes mellitus at time of informed consent. * Known impairment of gastrointestinal (GI) function due to concomitant GI disease that may significantly alter the absorption of the study drug at time of informed consent. * History of hypersensitivity to any drugs or metabolites of PI3K inhibitor or any of the excipients of alpelisib at time of informed consent. * Known history of Steven Johnson's syndrome, erythema multiforme or toxic epidermal necrolysis at time of informed consent. * Known history of seizure, or epilepsy, regardless of relatedness to PROS spectrum at time of informed consent, when epilepsy was not controlled and/or the patient may not be switched to non-enzyme inducing antiepileptic drug(s) at time of informed consent. * Patient with other concurrent severe and/or uncontrolled medical conditions that could, in the Treating Physician's judgment, contraindicate administration of alpelisib at time of informed consent. Patient with an active documented COVID-19 infection at time of informed consent could be included only when completely recovered and had no symptoms for at least 28 days before first dose of study medication. * Pregnant or breastfeeding female patients at time of informed consent. * Female patients of child-bearing potential who did not consent to use a highly effective method of contraception and male patients who did not consent to use a condom and/or a highly effective method of contraception for the duration of the study and for one week following discontinuation of alpelisib. * Patient was receiving any of the following medications and could not discontinue 7 days prior to the start of the treatment: strong inducers of CYP3A4 or inhibitors of breast cancer resistance protein (BCRP). * Not able to understand and to comply with study instructions and requirements at time of informed consent. * Participation in a prior investigational study within 4 weeks prior to study treatment start or within 5 half-lives of the investigational product, whichever was longer. * Patients with clinically significant worsening of PROS-related laboratory anomalies, physical signs and symptoms indicating an uncontrolled condition during the screening phase, particularly if systemic treatment with any other inhibitor of the PI3K/AKT/mTOR pathway was stopped prior to the start of study treatment. This included but was not limited to hypercoagulability state in patients not receiving prophylactic treatment. Other inclusion/exclusion criteria may apply

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Conditions

The condition(s) this trial relates to.

PIK3CA-related overgrowth spectrum

As listed by the trial registrant

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

PIK3CA-related overgrowth spectrum (PROS)

Contacts and locations

Locations

  • Baylor College Of Medicine

    Houston, Texas, 77030, United States

  • CHOP Abramson Pediatric Resch Ctr

    Philadelphia, Pennsylvania, 19104, United States

  • Childrens Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Childrens Hospital and Regional Medical Center

    Seattle, Washington, 98105, United States

  • Cincinnati Children s Hospital Medical Center

    Cincinnati, Ohio, 45229-3039, United States

  • Cinn Children Hosp Medical Center

    Cincinnati, Ohio, 45229-3039, United States

  • Fink Childrens Ambulatory Care Ctr

    New York, New York, 10016, United States

  • Novartis Investigative Site

    Montreal, Quebec, H2X 1R9, Canada

  • Novartis Investigative Site

    Montreal, Quebec, H3T 1C5, Canada

  • Novartis Investigative Site

    Beijing, 100730, China

  • Novartis Investigative Site

    Shanghai, 200011, China

  • Novartis Investigative Site

    Bordeaux, 33076, France

  • Novartis Investigative Site

    Dijon, 21000, France

  • Novartis Investigative Site

    Paris, 75015, France

  • Novartis Investigative Site

    Tours, 37044, France

  • Novartis Investigative Site

    Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

  • Novartis Investigative Site

    Düsseldorf, North Rhine-Westphalia, 40225, Germany

  • Novartis Investigative Site

    Leipzig, Saxony, 04103, Germany

  • Novartis Investigative Site

    Hamburg, 22149, Germany

  • Novartis Investigative Site

    Heidelberg, 69120, Germany

  • Novartis Investigative Site

    Hong Kong, 999077, Hong Kong

  • Novartis Investigative Site

    Roma, RM, 00165, Italy

  • Novartis Investigative Site

    Torino, TO, 10126, Italy

  • Novartis Investigative Site

    Nijmegen, 6525 GA, Netherlands

  • Novartis Investigative Site

    Oslo, 0372, Norway

  • Novartis Investigative Site

    Esplugues, Barcelona, 08950, Spain

  • Novartis Investigative Site

    Madrid, 28046, Spain

  • Novartis Investigative Site

    Bern, 3010, Switzerland

  • Novartis Investigative Site

    Zurich, 8032, Switzerland

  • Novartis Investigative Site

    West Midlands, Birmingham, B4 6NH, United Kingdom

  • Novartis Investigative Site

    London, SW17 0QT, United Kingdom

  • Novartis Investigative Site

    Manchester, M13 9WL, United Kingdom

  • UCSF Birthmarks and Vascular Center

    San Francisco, California, 94158, United States

  • UNC Chapel Hill

    Chapel Hill, North Carolina, 27599, United States

  • Unv of TX Southwestern Medical Center

    Dallas, Texas, 75235, United States

  • Washington Univ School Of Medicine

    St Louis, Missouri, 63110, United States

More trials for these conditions

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