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Not yet recruitingRare disease

Long‑term safety study of alpelisib alone or with fulvestrant in adults with breast cancer who benefited from previous alpelisib treatment

Verified siteRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

The study focuses on people with PROS (PIK3CA‑Related Overgrowth Spectrum), a rare condition that causes abnormal growth of body tissues, and on individuals with Breast Neoplasms, a term used for breast cancer. Participants receive the oral medication alpelisib (also known by the code name BYL719), which is taken as a tablet and may be given alone or together with other prescribed medicines.

The purpose of the study is to evaluate the long‑term safety of alpelisib. After completing a previous trial with this drug, participants continue to take the medication for an extended period while attending scheduled visits where doctors check for side effects, record any serious health problems (called adverse events), and assess whether the treatment is still providing a benefit, meaning the disease is stable or improving.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial Screening and Baseline Assessment

    After joining the study, you will attend a baseline visit where the investigator confirms that you are suitable for continued treatment with alpelisib.

    During this visit, basic health measurements, laboratory tests, and a review of any current medications are performed to create a reference point for future safety evaluations.

  2. Step 2

    Start of Study Medication

    You will begin taking alpelisib as a film‑coated tablet.

    The prescribed dose is 300 mg taken by mouth once each day.

    If your investigator decides that a combination therapy is appropriate, you may also receive an injection of fulvestrant at a dose of 500 mg.

  3. Step 3

    Daily Oral Medication Routine

    Each day you will swallow one 300 mg tablet of alpelisib with water.

    It is important to take the tablet at roughly the same time each day to keep the drug level steady in your body.

  4. Step 4

    Scheduled Clinic Visits

    You will return to the study site at regular intervals (typically every 4 to 8 weeks) for safety check‑ups.

    During each visit the investigator will record any adverse events (side effects) and may perform blood tests, physical examinations, and other assessments to monitor your health.

  5. Step 5

    Administration of Fulvestrant (If Required)

    When the combination with fulvestrant is part of your treatment plan, the injection will be given by a healthcare professional at the clinic.

    The injection dose is 500 mg; the exact frequency (for example, every 4 weeks) will be determined by the investigator and explained to you at the time of administration.

  6. Step 6

    Ongoing Safety Monitoring

    Throughout the study you will be asked to report any new symptoms, changes in health, or concerns immediately to the study staff.

    The investigator will assess the severity of any reported side effects and decide whether any adjustments to the medication are needed.

  7. Step 7

    Study Completion or Discontinuation

    The study continues until the investigator determines that you no longer benefit from the medication, you experience an unacceptable side effect, or the overall study ends.

    If the study ends while you are still receiving alpelisib, the investigator will discuss any further treatment options with you.

Who can join the trial?

7 criteria

  • You must sign a written informed consent (or give assent if you are a child) before joining the study.
  • You must already be taking the medicine alpelisib, either by itself or together with other drugs, as part of another Novartis study and have met all the requirements of that earlier study (called the parent study).
  • The doctor must think that you are currently getting a benefit from the treatment with alpelisib.
  • You need to have followed the visit schedule in the parent study and be willing and able to keep up with future visits, treatment plans, and study procedures (this is called compliance).
  • Both men and women are allowed to take part.
  • People of all ages, including children, teenagers, and adults, can join the study.
  • The study may include participants who are considered vulnerable (for example, those who may need extra protection).

Who cannot join the trial?

6 criteria

  • You cannot join if you have permanently stopped taking alpelisib in a previous study, for any reason including withdrawing consent.
  • You cannot join if you currently have serious side effects (called Grade 3 or higher adverse events) that have not resolved, or if any side effect caused your dose of alpelisib to be paused and has not improved enough to restart treatment.
  • You cannot join if you are already receiving a treatment for your condition that is approved and paid for (reimbursed) in your country.
  • You cannot join if you are taking part in any other experimental clinical trial besides the original study.
  • You cannot join if you are pregnant or nursing (breastfeeding).
  • You cannot join if you are a woman who could become pregnant and do not agree to use a highly effective birth‑control method, or if you are a man who does not agree to use a condom or another highly effective method of contraception, avoid sperm donation, and follow steps to prevent your partner’s exposure to the study drug during the trial and for at least one week after stopping it.
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Investigated drugs

  • Fulvestrant

    is given by injection and works as a hormone therapy for certain breast cancers. It blocks the body’s estrogen receptors, which can help stop the growth of cancer cells that need estrogen to multiply. In this trial, participants receive fulvestrant to see how safe it is when used over a longer period.

  • Alpelisib

    (also known by the code name BYL719) is taken as an oral tablet. It belongs to a group of medicines that block a specific pathway inside cancer cells, helping to slow down or stop their growth. In the study, alpelisib is tested both by itself and together with other treatments to understand its long‑term safety for people who have already finished a previous alpelisib study and may still benefit from continued use.

What is already known about the treatment

  • FULVESTRANT

    Fulvestrant is given as an injection that is slowly released under the skin. It is an approved medicine that has been used for many years to treat certain breast cancers. It works by attaching to the estrogen receptor in cancer cells and helping the body break down that receptor, which slows tumor growth. Pharmacologically it is classified as a selective estrogen receptor degrader.

  • ALPELISIB

    Alpelisib comes as a film‑coated tablet taken by mouth. It is an approved drug, listed as an orphan medication, used for breast cancers with a specific PIK3CA mutation and is also being studied for PIK3CA‑related overgrowth conditions. It blocks the PI3K‑alpha enzyme inside cells, which reduces signals that tell the cells to grow and divide. It belongs to the class of PI3K inhibitors.

Investigated diseases

  • PIK3CA‑Related Overgrowth Spectrum

    A group of rare disorders caused by activating mutations in the PIK3CA gene that lead to abnormal tissue growth. It usually becomes apparent at birth or in early childhood. Overgrowth can affect limbs, skin, blood vessels, and internal organs. The affected areas tend to enlarge progressively over time. Some individuals may experience pain or functional limitations as the tissue expands.

  • Breast cancer

    A malignant tumor that originates from the cells lining the milk ducts or lobules of the breast. It often begins as a small, localized growth and can spread to nearby lymph nodes. As it advances, cancer cells may invade surrounding tissue and travel through the bloodstream to distant sites such as bone, lung, or liver. The disease typically progresses from early to more advanced stages if not controlled. Growth may be driven by hormonal factors or other cellular changes.

Trial detailsLast updated 14 Oct 2026
Age18+ yearsPhasePhase IITrial ID2026-525890-38-00Protocol codeCBYL719F12003BEstimated enrolment8 patientsSponsorNovartis Pharma AG

sourced from the EU Clinical Trials Register and site verification

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