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Drug shows promise for rare overgrowth disorder in long-term study

NCT ID NCT04980833

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study looks at the long-term safety and effectiveness of the drug alpelisib in people with PIK3CA-Related Overgrowth Spectrum (PROS), a rare condition causing abnormal tissue growth. It includes 41 children and adults who previously took alpelisib in an earlier trial. Researchers will track side effects and how well the drug controls overgrowth over time.

What this could mean

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

Active substance
alpelisib
What this could lead to
If successful, this could confirm alpelisib as a long-term treatment option to control overgrowth in PROS, improving quality of life.
What could go wrong
This is a small, phase 2 study with no placebo group, so results may not be definitive. Side effects like high blood sugar or rash are possible.

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

41 people

The number who actually took part.

Started

Jan 2022

Expected to finish

Aug 2027

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

2 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.

Inclusion Criteria: * Participants who had previously participated in the study EPIK-P1. * Signed informed consent form and assent (when applicable) from the participant, parent, or guardian must be obtained prior to any study related screening procedures being performed. * Participant is treated with at least one dose of alpelisib after the EPIK-P1 study data cut- off date of 09-Mar-2020. Exclusion Criteria: For participants in the retrospective period \- All EPIK-P1 participants who permanently discontinued the investigational drug on or prior to the cut-off date 09-Mar-2020. For participants in the prospective period * Previous alpelisib treatment discontinuation (after 09-Mar-2020) due to any of the following adverse events: * Grade 4 skin and subcutaneous tissue disorders * Stevens-Johnson-Syndrome (SJS)/ Toxic Epidermal Necrolysis (TEN) or other SJS/TEN-like severe skin reactions (any grade) * Grade 4 hyperglycemia without confounding factors * Pneumonitis (any grade) * Grade 4 stomatitis * Grade 4 pancreatitis * Recurrent grade 4 thrombocytopenia * Grade 3 or 4 serum creatinine increase * Grade 4 isolated total bilirubin elevation * Recurrent grade 3 or 4 QT interval corrected by Fridericia's formula prolongation (\>500 ms or \>60 ms change from baseline) * Known impairment of GI function due to concomitant disease that may significantly alter the absorption of the study drug (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection) at time of informed consent. * Participant with uncontrolled diabetes mellitus (Type I or II) at time of informed consent. 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

  • Boston Childrens Hospital

    Boston, Massachusetts, 02215, United States

  • Novartis Investigative Site

    Dijon, 21000, France

  • Novartis Investigative Site

    Montpellier, 34295, France

  • Novartis Investigative Site

    Paris, 75015, France

  • Novartis Investigative Site

    Dublin, 12, Ireland

  • Novartis Investigative Site

    Madrid, 28046, Spain

More trials for these conditions

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