Proteus syndrome Trials in Disease

Long-term Safety Study of Miransertib for Patients with PIK3CA-related Overgrowth Spectrum or Proteus Syndrome

This clinical trial is designed to evaluate the long-term safety of miransertib in patients with Proteus Syndrome and a related condition called PIK3CA-related overgrowth spectrum (PROS). Both conditions cause abnormal growth of body tissues, which can lead to pain, mobility problems, and other complications. Miransertib is a targeted therapy that works by blocking specific pathways in cells that contribute to abnormal growth.

Main inclusion criteria:

• Patients must have either PROS or Proteus Syndrome
• Patients must be between 2 and 120 years of age
• Patients must have previously participated in other miransertib studies or be currently receiving miransertib treatment as part of approved research
• Male patients must agree to use contraception during the study and for at least 90 days after the last dose, unless confirmed unable to produce sperm
• Female patients must not be pregnant or breastfeeding, and if able to have children, must agree to use highly effective contraception or abstain from sexual intercourse

Main exclusion criteria:

• Other serious medical conditions that could interfere with the study
• Pregnancy or breastfeeding
• Participation in another clinical trial within the last 30 days
• Known allergy to miransertib or its ingredients
• History of drug or alcohol abuse within the past year
• Certain heart conditions that could be affected by the study medication
• Use of medications that might interact with miransertib
• History of certain types of cancer
• Uncontrolled diabetes or high blood pressure
• Inability to comply with study procedures

Focus and goal of the trial:

The primary focus of this study is to monitor the long-term safety and tolerability of miransertib when used continuously over an extended period. Participants will continue taking miransertib in capsule form and will be regularly assessed by the study team. The trial will track any serious side effects, adverse events, or reasons that might require participants to stop taking the medication. Regular follow-up visits will be scheduled to ensure patient safety and to gather important data about how the medication affects individuals with these rare conditions over time. The study is expected to conclude by December 15, 2025.

Investigational drug:

Miransertib is the medication being tested in this trial. It is classified as a selective allosteric AKT inhibitor, which means it targets and blocks specific molecular pathways involved in cell growth and survival. The drug is taken orally in hard capsule form. While miransertib is still under investigation and not yet widely used in standard medical practice, it shows promise in managing conditions characterized by abnormal tissue growth. By targeting the molecular mechanisms that contribute to overgrowth, miransertib may help control symptoms associated with Proteus Syndrome and PROS.