Active not recruitingPHASE2INTERVENTIONAL

Study Assessing Long-term Safety and Efficacy of Alpelisib in Patients With PIK3CA-Related Overgrowth Spectrum (PROS) Who Previously Participated in Study CBYL719F12002 (EPIK-P1)

This study, called EPIK-P1, is for children and adults with a condition called PIK3CA-related Overgrowth Spectrum, or PROS. PROS causes parts of the body to grow too much. The study is designed to carefully watch how safe and helpful a medicine called Alpelisib is over a long period. All participants in this study have previously taken part in an earlier study and have continued to receive Alpelisib. Researchers want to understand the long-term effects of this treatment, both in terms of how well it works and any side effects people might experience. It's an important step in learning more about managing PROS effectively.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Novartis Pharmaceuticals

Enrolment target

Start

27 Jan 2022

Estimated completion

20 Aug 2027

What is this study about?

This study is designed to learn more about a medicine called Alpelisib for people, including children and adults, who have a condition called PIK3CA-related Overgrowth Spectrum (PROS). PROS is a rare condition that causes certain parts of the body to grow more than they should.

The main goal of this study is to look closely at how safe Alpelisib is and how well it works over a long period. All the people taking part in this study have already been receiving Alpelisib as part of an earlier research study (called EPIK-P1). This new study helps gather more information about the effects of the medicine beyond that first study.

Researchers will be gathering information in two main ways. First, they will look back at medical records from the past to see how participants were doing. Then, they will follow participants forward in time, collecting new information about their health, any changes in their PROS, and any side effects from the medication. This long-term monitoring is crucial to understand the full picture of Alpelisib's benefits and risks for people with PROS.

Key takeaways

This study aims to understand the long-term effects of Alpelisib for PROS.
It's for people who were already in an earlier study (EPIK-P1) and continued Alpelisib.
Researchers will look at past health records and collect new information for at least 5 years.
The study monitors both the safety and effectiveness of the treatment.
It includes children and adults aged 2 to 100 years old.
Participation is voluntary, and you can stop at any time.

Who may be eligible?

To join this study, you must have previously taken part in the EPIK-P1 study. You also need to have continued taking the study medicine, Alpelisib, after March 9, 2020. Everyone joining, or their parent/guardian if they're a child, will need to agree to participate by signing a consent form.

There are also some reasons why you might not be able to join the study. For example, if you stopped Alpelisib in the previous study for certain serious side effects like severe skin reactions, severe problems with blood sugar (diabetes) that were hard to control, or serious issues with your kidneys or liver, you wouldn't be able to continue in this particular study. Also, if you have certain digestive problems that could stop the medicine from working properly, or if you have uncontrolled diabetes, you might not be eligible.

The study is open to both males and females, aged 2 years old up to 100 years old.

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.

Were you in the previous EPIK-P1 study?
Have you been taking Alpelisib continuously since March 9, 2020?
Do you have any severe, uncontrolled medical conditions like diabetes or serious digestive issues?
Did you stop Alpelibib in the past due to very serious side effects, such as severe skin reactions or serious organ problems?
Are you, or your child, aged 2 to 100 years old?

Answer every question to see your result.

What does participation involve?

If you join this study, you'll be taking the medicine Alpelisib. Initially, the researchers will look back at your health records from previous treatments received after March 9, 2020. This initial part won't involve any new hospital visits or specific treatments just for the study. After this, the study will move into a "forward-looking" stage. During this stage, you'll have regular check-ups and tests, just like you would for your usual medical care, but they will be more structured to collect consistent information for the study. These visits will help doctors monitor your health, how your PROS is progressing, and any side effects you might experience. The total length of your participation in this forward-looking part of the study will be at least five years, unless you stop the treatment earlier.

Potential risks and benefits

Taking part in any study has potential benefits and risks. The main potential benefit for you is continued access to Alpelisib, which may help manage your PROS condition. The study also contributes valuable information that could help others with PROS in the future. Potential risks include side effects from the Alpelisib medication itself, which will be closely monitored by your care team. There's also the chance that the treatment might not be effective for you. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (6)

Boston Childrens Hospital
Verified postcode
Boston, United States
Novartis Investigative Site
Verified postcode
Dijon, France
Novartis Investigative Site
Verified postcode
Montpellier, France
Novartis Investigative Site
Verified postcode
Paris, France
Novartis Investigative Site
Verified postcode
Dublin, Ireland
Novartis Investigative Site
Verified postcode
Madrid, Spain

Common questions

What is PIK3CA-related Overgrowth Spectrum (PROS)?

PROS is a condition where certain parts of the body grow more than they should, caused by a change in a specific gene.

What is Alpelisib?

Alpelisib is a medicine being studied to see if it can help stop or slow down the overgrowth seen in PROS.

How long will I be in the study?

After an initial review of past records, you'll be actively followed in the study for at least five years, or until you decide to stop treatment earlier.

Will I have to pay for the medication or visits?

Study-related medication and visits are typically covered by the study sponsors. Always ask your doctor for specifics regarding costs.

Do I have to live in a specific place to join?

This is a "multi-centre" study, meaning it takes place in various locations. Your doctor can tell you if there's a study site near you.