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Could a drug stop uncontrolled body growth? small study tests sirolimus

NCT ID NCT02443818

Disease control Sponsor: Centre Hospitalier Universitaire Dijon 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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026 · Updated 1 time

Summary

This pilot study tested the drug sirolimus in 16 people aged 3 to 65 who have excessive growth in parts of their body due to a change in the PIK3CA gene. The goal was to see if sirolimus can stop or slow that overgrowth. Researchers used MRI scans and other measures to track changes, and also looked at quality of life. This small study helps prepare for a larger international trial.

What this could mean

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

Active substance
Sirolimus (a drug that slows cell growth)
What this could lead to
If it works, this could point toward a treatment that stops or slows excessive growth in people with PIK3CA-related overgrowth disorders.
What could go wrong
This is a very small pilot study with only 16 people, so results may not apply to everyone. The drug may not stop growth or could cause side effects.

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

16 people

The number who actually took part.

Start date

May 2015

Finished

Nov 2016

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

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

Ages

3 to 65 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: * Post-zygotic PIK3CA mutation * Age: 3-65 years * Measurable overgrowth, in current progression or with clinical history of overgrowth progression * Patient clinically stable * Presence of a disability, social or cosmetic impairment requiring treatment from the patient perspective, * Written informed consent form signed and dated by the subject or by the patient's legal representative Exclusion Criteria: * Pregnancy or lactation * Women and men of reproductive age without any effective method of contraception (during treatment and up to 12 weeks after sirolimus discontinuation) * Hypersensitivity to the active substance (sirolimus) or to any of the excipients * Impossibility to obtain written informed consent form signed by the subject or the patient's legal representative, or vulnerable adults * Treatment with Sirolimus in the last 4 weeks before the trial * Personal history of malignancy or current investigations for suspected malignancy * Active skin infection requiring antibiotics or antiviral treatments * HIV or hepatitis B or C infection * Past history of Mycobacterium tuberculosis infection * Active pneumopathy * Uncontrolled infection * Chronic liver disease (ASAT or Alanine amino transférase (ALAT)\> 3 times upper normal limit) * Stage 3 (or more) chronic renal insufficiency (eGFR\< 60mls/min) * Neutropenia with neutrophiles \< 1.0 x10\^9/L * Uncontrolled dyslipidemia * Inability to attend study visits

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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.

Segmental overgrowth disorders due to PIK3CA

Contacts and locations

Locations

  • CHRU de Montpellier - Hôpital Saint-Eloi

    Montpellier, 34295, France

  • CHRU de Tours

    Tours, 37044, France

  • CHU Paris - Necker

    Paris, 75015, France

  • CHU d'Angers

    Angers, 49933, France

  • CHU de Bordeaux - GH Pellegrin

    Bordeaux, 33076, France

  • CHU de DIJON

    Dijon, 21079, France

  • HCL Lyon - CH Lyon Sud

    Pierre-Bénite, 69495, France

  • Hopitaux de Brabois Chu Nancy

    Nancy, 54511, France

  • Hôpital Jeanne de Flandre

    Lille, 59037, France

  • Hôpital mère-enfant de Nantes

    Nantes, 44093, France

  • Pôle Cardiovasculaire et Métabolique - Hôpital Larrey

    Toulouse, 31059, France