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Could a cancer drug tame rare overgrowth disease?

NCT ID NCT03290092

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
The trial has finished. Results may not be published yet.
Stopped early This study
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This status has not been confirmed recently, so it may be out of date.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 2 times

Summary

This early-phase trial tested taselisib, a drug that blocks a faulty protein causing excessive growth in people with PIK3CA-related overgrowth. Nineteen participants took the drug daily for four weeks to see if it was safe and tolerable. The study was stopped early, so we have limited information, but it may help design future trials.

What this could mean

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

Active substance
Taselisib (a drug that blocks a protein called PI3K, which is overactive in this condition)
What this could lead to
If it works, this could point toward a drug treatment for PIK3CA-related overgrowth, potentially reducing the need for repeated surgeries.
What could go wrong
This trial was terminated early with only 19 participants, so results are very limited. The drug may cause side effects like high blood sugar or diarrhea, and it is not yet clear if it helps symptoms.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

19 people

The number who actually took part.

Started

Jul 2017

Finished

Nov 2019

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

16 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: * Have given, or their legal representative has given, written informed consent to participate * Aged 16 years to 65 years inclusive * Male or female * Post-zygotic PIK3CA mutation * Clinically stable in the opinion of the investigator * Participant Pregnancy and contraception: o Female participants of child bearing potential must use an effective method of contraception during treatment and for at least 3 months after the final dose of taselisib. Acceptable methods are: * True abstinence (this must be the participant's usual and preferred lifestyle, not just for the duration of the trial) * Oral contraceptive (either combined or progestogen alone) * Contraceptive implant, injections or patches * Vaginal ring * Intrauterine device (IUD, coil or intrauterine system) * Condom and cap * Diaphragm plus spermicide * A female participant of child bearing potential is defined as a sexually mature woman not surgically sterilized or not post-menopausal for at least 12 consecutive months if aged 55 years or older. * Men must use one of the following, reliable forms to contraception for the entire duration of treatment and for 3 months after the final dose of taselisib: * Condom plus spermicide even if female partner is using another method of contraception (Men should also use a condom to protect male partners, or female partners who are pregnant or breast feeding, from exposure to the Trial medicine in semen). * True abstinence (this must be the participant's usual and preferred lifestyle, not just for the duration of the Trial) Exclusion Criteria: * Pregnant or breastfeeding * HIV infection * Hypersensitivity to taselisib or any of its excipients * Any current medical disorder or medication likely to impair ability to follow the trial protocol safely and effectively * Are concurrently taking an mTOR inhibitor or any other small molecule inhibitor of the PI3K-AKT signalling pathway * Unable or unwilling to give informed consent * Sirolimus or taselisib treatment in 12 weeks prior to screening * Treatment with a strong inducer or inhibitor of CYP3A4 without the possibility to stop this medication within the week prior to the screening. This includes: * Macrolide Antibiotics: clarithromycin, telithromycin, erythromycin, troleandomycin * Gastrointestinal prokinetic agents: metoclopramide. * Antifungals: itraconazole, ketoconazole, fluconazole, voriconazole, clotrimazole * Calcium channel blockers: verapamil, diltiazem, nicardipine * Grapefruit containing foods/drinks * Anticonvulsants: carbamazepine, phenobarbital, phenytoin * Antibiotics: rifampicin, rifabutin, rifapentine * Herbal preparations: St. John's wort (Hypericum perforatum). Other drugs: bromocriptine, cimetidine, danazol, cyclosporine, lansoprazole, calcium containing antacids. * Inability to attend trial visits * If less than 3 months post- major surgery at screening * Any past medical history of inflammatory bowel disease or chronic diarrhoea of unknown aetiology * History of type 1 or type 2 diabetes mellitus requiring insulin, GLP-1 analogues or oral hypoglycaemic agents. * History of inflammatory bowel disease, ischemic colitis, or colitis of unknown origin. * Fasting blood glucose \> 6.9 mmol/l * HbA1C \> 6% * Long QT, congenital or acquired * Active pneumonitis * Patients who require daily supplemental oxygen * Inadequate renal function defined as creatinine clearance or radioisotope GFR \< 60ml/min/1.73 m² * Inadequate liver function defined as: * Total bilirubin \> 2.0 x ULN or conjugated bilirubin \> 2.0 x ULN for age, and * SGPT (ALT) or SGOT (AST) ≥ 1.5 x ULN for age, and * Serum albumin \< 30 g/L * Inadequate fasting LDL cholesterol \> 4.2 mmol/l * Deprived of freedom by an administrative or court order, or benefiting from a system of legal protection (tutorship, curatorship or safeguard of justice). * Not covered by health insurance

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

Contacts and locations

Locations

  • CHU Hôtel Dieu

    Nantes, 44093, France

  • CHU La Réunion - Site GHSR

    Saint-Pierre, 97448, France

  • CHU d'Angers

    Angers, 49933, France

  • CHU de Bordeaux - GH Pellegrin

    Bordeaux, 33076, France

  • CHU de Nancy

    Vandœuvre-lès-Nancy, 54511, France

  • CHU de Nancy - Hôpital de Brabois

    Vandœuvre-lès-Nancy, 54511, France

  • CHU de Nice - Hôpital l'Archet 2

    Nice, 06202, France

  • CHU de Saint-Etienne Hôpital Nord

    Saint-Priest-en-Jarez, 42270, France

  • Chu Dijon Bourgogne

    Dijon, 21000, France

  • HCL - Hôpital femme-mère-enfant

    Lyon, France

  • Hôpital Arnaud de Villeneuve

    Montpellier, 34090, France

  • Hôpital Jeanne de Flandre

    Lille, 59037, France

  • Hôpital Larrey

    Toulouse, 31059, France

  • Hôpital Necker-Enfants malades

    Paris, 75743, France

  • Hôpital Raymond Poincaré (AP-HP)

    Garches, 92380, France

  • Hôpital Saint Eloi

    Montpellier, 34295, France

  • Hôpital Sud

    Rennes, 35203, France

  • Hôpital Trousseau

    Tours, 37044, France