Could a cancer drug tame rare overgrowth disease?
NCT ID NCT03290092
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
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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19 people
The number who actually took part.
- Started
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Jul 2017
- Finished
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Nov 2019
- Lead sponsor
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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
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Anyone
- Healthy volunteers
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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 Hide 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
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CHU Hôtel Dieu
Nantes, 44093, France
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CHU La Réunion - Site GHSR
Saint-Pierre, 97448, France
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CHU d'Angers
Angers, 49933, France
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CHU de Bordeaux - GH Pellegrin
Bordeaux, 33076, France
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CHU de Nancy
Vandœuvre-lès-Nancy, 54511, France
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CHU de Nancy - Hôpital de Brabois
Vandœuvre-lès-Nancy, 54511, France
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CHU de Nice - Hôpital l'Archet 2
Nice, 06202, France
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CHU de Saint-Etienne Hôpital Nord
Saint-Priest-en-Jarez, 42270, France
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Chu Dijon Bourgogne
Dijon, 21000, France
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HCL - Hôpital femme-mère-enfant
Lyon, France
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Hôpital Arnaud de Villeneuve
Montpellier, 34090, France
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Hôpital Jeanne de Flandre
Lille, 59037, France
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Hôpital Larrey
Toulouse, 31059, France
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Hôpital Necker-Enfants malades
Paris, 75743, France
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Hôpital Raymond Poincaré (AP-HP)
Garches, 92380, France
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Hôpital Saint Eloi
Montpellier, 34295, France
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Hôpital Sud
Rennes, 35203, France
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Hôpital Trousseau
Tours, 37044, France