The purpose of this study is to determine if alpelisib can help reduce the size of tissue overgrowth in people with PROS. During the study, participants will receive either alpelisib or placebo. The medication will be given daily for 16 weeks, with the possibility of continued treatment. The study will measure changes in the size of affected tissue areas using Magnetic Resonance Imaging (MRI) scans.

The treatment will be evaluated based on whether it can reduce the size of abnormal tissue growths by at least 20%. Participants will be monitored throughout the study to check how well the medication works and to watch for any side effects. The study includes both adults aged 18 and older, and children between 6 and 17 years old.

The purpose of this study is to determine how well alpelisib works in treating both adults and children (age 2 and older) who have PROS. The medication will be given daily at doses up to 250 milligrams for a treatment period lasting up to 168 weeks.

During the study, participants will have their tissue growths measured using MRI scans to see if they shrink in response to the treatment. The study will also monitor pain levels, overall symptoms, and any side effects that may occur. Participants will be checked regularly for changes in their condition, including how the medication affects their daily activities and quality of life.

The purpose of the study is to determine the best dose of RLY-2608 for different groups of participants and to assess its safety and effectiveness. The study will be conducted in several parts. Initially, participants will receive RLY-2608 to find the most suitable dose and to monitor any side effects. In a later part of the study, the effectiveness of RLY-2608 will be compared to a placebo to see how well it works in reducing the size of the overgrowths or malformations.

Participants in the study will be monitored over a period of time to observe any changes in their condition and to ensure their safety. The study aims to provide valuable information on how RLY-2608 can be used to treat individuals with conditions driven by the PIK3CA mutation, potentially leading to improved treatment options in the future.

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.

The purpose of this study is to evaluate how safe and tolerable miransertib is when used on its own over a long period. Participants in this study are those who are already receiving miransertib in other studies. The study will monitor participants to see if they experience any serious side effects or if they need to stop taking the medication due to any adverse effects. The study is designed to provide valuable information about the safety of miransertib for individuals with PROS or Proteus Syndrome.

Participants will continue their treatment with miransertib and will be regularly assessed by the study team. The study aims to ensure that the medication is safe for long-term use and to gather data on any potential side effects. This information will help in understanding the long-term impact of miransertib on individuals with these rare conditions.

Participants in the study will receive either Alpelisib or a placebo, which looks like the medication but does not contain the active ingredient. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual medication or the placebo. This helps ensure that the results are not influenced by expectations. The main goal is to see if there is an improvement in adaptive behavior, which refers to how well a person can adjust to changes and manage daily life activities.

Throughout the study, various assessments will be conducted, including changes in brain structure using MRI scans, monitoring of any side effects, and evaluations of quality of life. The study will also look at changes in neuropsychological functions such as attention, cognition, and motor skills. The trial will last for up to 24 months, with regular check-ins to monitor progress and any changes in the participants’ condition. The ultimate aim is to determine if Alpelisib can provide a meaningful benefit to those living with MCAP.