Study on the Long-Term Safety of Alpelisib for Patients with PIK3CA-Related Overgrowth Spectrum (PROS)

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What is this study about?

This clinical trial is focused on studying a condition known as PIK3CA-Related Overgrowth Spectrum (PROS). This is a rare disorder that causes abnormal growth in various parts of the body due to changes in the PIK3CA gene. The treatment being tested in this study is a medication called alpelisib, which is taken as a film-coated tablet. The purpose of the study is to evaluate the long-term safety and tolerability of alpelisib in patients who have previously participated in a related study.

Participants in this study will take alpelisib orally, which means it is swallowed in tablet form. The study will monitor patients over a period to see how they respond to the treatment and to check for any new or worsening side effects. The study will also look at other health aspects, such as changes in laboratory values, vital signs, and heart function. Additionally, the study will assess growth, bone and dental development, and sexual maturation in applicable age groups.

The study will gather information on the number of healthcare visits or hospitalizations due to PROS, as well as any other treatments or surgeries related to the condition. This information will help researchers understand the long-term effects of alpelisib and its impact on the symptoms and complications associated with PROS. The study is expected to continue until 2027, providing valuable insights into the management of this rare condition.

1 joining the study

Participation in this study requires a previous involvement in the EPIK-P1 study. A signed informed consent form is necessary before any study-related procedures begin.

2 medication administration

The study involves taking the medication alpelisib, also known as BYL719. This medication is provided in the form of a film-coated tablet and is taken orally.

The dosage and frequency of alpelisib will be determined by the study team based on individual needs and previous treatment history.

3 prospective period

During this period, the main objective is to assess the long-term safety and tolerability of alpelisib.

Participants will be monitored for any new or worsening side effects. Regular assessments will include laboratory tests, vital signs, and cardiac function evaluations.

Additional evaluations may include growth, bone and dental development, and sexual maturation for applicable ages.

4 retrospective period

This period involves reviewing past data to understand the incidence, type, and severity of any side effects experienced during previous treatments.

The study will also collect information on any healthcare visits or hospitalizations related to the condition.

5 end of study

The study is estimated to conclude by September 1, 2027. Participants will be informed about the outcomes and any further steps if necessary.

Who Can Join the Study?

The patient must have previously participated in a study called EPIK-P1.
The patient or their parent/guardian must sign an informed consent form. This is a document that explains the study and confirms that the patient agrees to participate.
The patient must have taken at least one dose of a medication called alpelisib after a specific date, March 9, 2020. Alpelisib is a drug used in the study.
If the patient stopped taking the medication for safety reasons before the first visit of the new study period, they can still participate by providing past data.
If the patient stopped taking the medication for other reasons, like worsening of their condition, they can restart the treatment if the study doctor thinks it will help. They can then participate in both parts of the study.
The study is open to both male and female patients.
The study includes patients from certain age groups, specifically children, teenagers, and adults.
The study may include patients who are considered vulnerable, meaning they might need extra protection or care.

Who Cannot Join the Study?

Patients who have a medical condition other than PIK3CA-Related Overgrowth Spectrum (PROS) that could interfere with the study.
Patients who are not within the specified age range for the study.
Patients who are not able to follow the study procedures or instructions.
Patients who are pregnant or breastfeeding.
Patients who have participated in another clinical trial recently.
Patients who have a history of allergic reactions to the study medication.
Patients who have a serious medical condition that could affect their safety during the study.
Patients who are taking medications that could interfere with the study drug.
Patients who have a history of drug or alcohol abuse.
Patients who have a mental health condition that could affect their ability to participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Hopital Necker Enfants Malades	Paris	France

Other Sites

Site Name	City	Country
Centre Hospitalier Universitaire De Montpellier	Montpellier	France
Children’s Health Ireland	Dublin	Ireland
Cfsyrg Hnwnmkijgpm Unhghconjnxmf Du Dhwxu	Dijon	France
Fxnmqtiqw Pyym Lq Imypumqnkrucf Bqfpumago Dhs Hiyrexzq Uihxqwaguxsry Lm Pox	Madrid	Spain

Trial status

Country	Status	Recruitment Start
France	Not recruiting	27.01.2022
Ireland	Not recruiting	27.01.2022
Spain	Not recruiting	27.01.2022

Trial locations

Investigated drugs:

Alpelisib

Alpelisib is a medication being studied for its long-term safety and effectiveness in patients with a condition known as PIK3CA Related-Overgrowth Spectrum (PROS). This medication is being tested to see how well it can be tolerated by patients over an extended period. Alpelisib works by targeting specific pathways in the body that are involved in the overgrowth of tissues, which is a characteristic of PROS. The goal is to determine if it can help manage the symptoms associated with this condition.

PIK3CA-Related Overgrowth Spectrum (PROS) – This is a group of rare disorders caused by mutations in the PIK3CA gene, leading to abnormal growth and development in various parts of the body. Individuals with PROS may experience overgrowth of tissues, which can affect the skin, bones, and other organs. The condition can vary widely in its presentation, with some people having mild symptoms and others experiencing significant physical changes. Over time, the overgrowth can lead to complications such as pain, mobility issues, and functional impairments. The progression of PROS is typically gradual, with symptoms potentially worsening as the individual ages. The specific areas of the body affected and the severity of symptoms can differ greatly from person to person.

Trial ID:

2023-508522-95-00

Protocol code:

CBYL719F12401

Trial Phase:

Therapeutic exploratory (Phase II)

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