RecruitingPHASE2, PHASE3INTERVENTIONAL

Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.

This research study is looking at a new medicine called alpelisib for people, both children and adults, who have lymphatic malformations (LyM). These are abnormal growths in the lymphatic system, and for this study, they must be caused by a specific genetic change called a PIK3CA mutation. The main goal is to see if alpelisib can make these malformations smaller and help reduce the problems they cause, like pain or swelling, compared to a placebo (a dummy medicine with no active ingredients). The study will first find the best dose in a smaller group, then test that dose in a larger group to make sure it's both safe and effective. Participants will be closely monitored by a team of doctors and researchers.

At a glance

Status

Recruiting

Phase

PHASE2, PHASE3

Sponsor

Novartis Pharmaceuticals

Enrolment target

232

Start

24 Nov 2023

Estimated completion

02 May 2033

Lymphatic Malformations

What is this study about?

Lymphatic malformations (LyM) are unusual growths in the body's lymphatic system, which is part of your immune system. These can cause various health problems depending on where they are located. This study is specifically for people whose LyM are caused by a particular change in a gene called PIK3CA. Researchers believe that by targeting this gene, a new medicine called alpelisib might help shrink the malformations and improve symptoms.

This study will happen in two main stages. In the first stage, a small group of adults and children will receive different doses of alpelisib to help researchers figure out the safest and most effective dose. Once that dose is chosen, the second stage will begin. This larger stage will compare the chosen dose of alpelisib to a placebo, which looks like the real medicine but contains no active drug. This is done to clearly see if any improvements are truly due to alpelisib.

The overall aim is to gather strong evidence on whether alpelisib can be a helpful treatment option for people living with PIK3CA-mutated lymphatic malformations. The study will carefully monitor how the malformations change in size and how participants' symptoms improve, always making sure safety is the top priority.

Key takeaways

This study is testing alpelisib, a new medicine for lymphatic malformations caused by a PIK3CA gene change.
It aims to see if alpelisib can shrink malformations and reduce symptoms.
The study involves both children (0-17 years) and adults.
You might receive alpelisib or a placebo (dummy medicine).
Regular monitoring and medical check-ups are part of participating.
You can withdraw from the study at any time.

Who may be eligible?

To join this study, you must have a lymphatic malformation that has been confirmed by a doctor to be caused by a PIK3CA gene change. Your doctor must also confirm that your malformation isn't a type called PROS. You also need to be willing to follow the study's instructions and attend all appointments.

There are also some reasons why you might not be able to join. For example, if you have a different type of lymphatic condition like PROS, or certain other health conditions such as uncontrolled diabetes, you wouldn't be eligible. Also, if you've had certain severe skin reactions in the past, or if you've previously taken alpelisib or similar medicines, you won't be able to participate. Your study doctor will review all your medical information to see if this study is right for you.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Do you have a lymphatic malformation (LyM) diagnosed by a doctor?
Has a test confirmed your LyM is caused by a PIK3CA gene change?
Are you able to swallow tablets or liquid medicine?
Are you willing to attend regular clinic visits and follow study instructions?
Do you have any other serious medical conditions like uncontrolled diabetes or certain severe skin reactions?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll first have a screening visit to check if you're suitable. Once confirmed, you'll be given either the study medicine, alpelisib, or a placebo (a dummy medicine). You'll take this medicine, which could be a tablet, liquid, or granules, for at least 24 weeks. You'll have regular visits to the clinic for medical check-ups, scans to measure your lymphatic malformations, and to discuss any symptoms or side effects you might be experiencing. These visits are very important to make sure the medicine is safe and working as intended. After the initial 24 weeks, depending on the stage of the study, you might have the option to continue receiving the medicine in an 'open-label extension', meaning everyone knows they are receiving alpelisib. The exact number of visits and tests will be explained in detail by the study team.

Potential risks and benefits

Participating in this study might offer a potential benefit by giving you access to a new treatment that could help improve your lymphatic malformation and symptoms. Like all medicines, alpelisib may have side effects, and some are still unknown. The study team will carefully monitor you for any potential risks or side effects and will explain them fully before you join. Remember, you can choose to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (57)

UCSF Benioff Children s Hospital
Verified postcode
Oakland, United States· Recruiting
Lucile Packard Childrens Hosp
Verified postcode
Palo Alto, United States· Recruiting
Childrens National Medical Center
Verified postcode
Washington D.C., United States· Recruiting
Nemours Childrens Clinic
Verified postcode
Jacksonville, United States· Recruiting
Childrens Hosp Boston Dept of Heme
Verified postcode
Boston, United States· Recruiting
WA Uni School Of Med
Verified postcode
St Louis, United States· Recruiting
Cinn Children Hosp Medical Center
Verified postcode
Cincinnati, United States· Recruiting
Univ Hospital Of Cleveland
Verified postcode
Cleveland, United States· Recruiting
Cleveland Clinic Foundation
Verified postcode
Cleveland, United States· Recruiting
Nationwide Children s Hospital
Verified postcode
Columbus, United States· Recruiting
Oregon Health Science University
Verified postcode
Portland, United States· Recruiting
CHOP Abramson Pediatric Resch Ctr
Verified postcode
Philadelphia, United States· Recruiting

Common questions

What is a PIK3CA mutation?

It's a specific change in a gene called PIK3CA. This change can sometimes cause unusual growths like lymphatic malformations.

What is a placebo?

A placebo is a 'dummy' medicine that looks like the real drug but contains no active ingredients. It helps researchers fairly compare the new medicine's effects.

Will I know if I'm getting the active medicine or the placebo?

In some parts of the study, neither you nor your doctor will know if you're getting alpelisib or the placebo. This is called 'double-blind' and helps ensure unbiased results.

How long will I need to be in the study?

The main part of the study lasts at least 24 weeks. After that, you might have the option to continue in an 'extension' phase.

Can children of all ages join this study?

This study is open to children aged 0 to 17 years, as well as adults. The specific age groups might differ slightly depending on the study stage.

How to find out more

Novartis Pharmaceuticals

novartis.email@novartis.com 1-888-669-6682 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.