Trial results
AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
According to the results reported on ClinicalTrials.gov, this trial (NCT03094832) looked at a medicine called miransertib in people with two rare conditions — PROS (PIK3CA-related overgrowth spectrum) and PS (Proteus syndrome). The trial was run in two parts (Part A and Part B), with Part B divided into several smaller groups, including a compassionate use group for people who needed access outside the main study. In total, 50 people started the trial across all groups. The trial was primarily measuring how many participants experienced any unwanted medical event (called an "adverse event") while taking the medicine, and how many stopped taking the medicine because of such an event. The reported data shows that in Part A (17 participants with PROS or PS), all 17 experienced at least one adverse event, and 2 stopped taking the medicine because of one. In Part B's main cohort of 22 people with PROS, 20 experienced at least one adverse event, and none stopped treatment because of an adverse event. The single participant in the PS-only cohort (Cohort 2) had no adverse events reported. Of the 8 participants in the mixed PROS/PS cohort (Cohort 3), 6 experienced at least one adverse event, and none stopped treatment due to one. The one treated participant in the compassionate use group experienced one adverse event and did not stop treatment because of it. It is worth noting that only 4 participants across the entire trial were recorded as having fully completed the study. The reported data does not include results for any secondary outcome measures, so those figures cannot be described here. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 2 Peripheral Neuropathy Trial, Terminated
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You (or your child) must be at least 2 years old and have a body surface area large enough to meet the study's minimum size requirement (confirm with trial site)
- You must have a confirmed diagnosis of a PIK3CA-Related Overgrowth Spectrum (PROS) condition or Proteus Syndrome (PS), with a specific genetic change (mutation) identified through testing
- A tissue sample from the overgrowth area must be available to send to the study team (either a previously collected sample or a new one)
- Your condition must be getting worse, causing significant health problems, or have a poor outlook — for example, overgrowth lesions have increased in number or size recently
- At least one overgrowth lesion must be large enough to be measured using scans or physical measurements
- Your liver, kidneys, and other organs must be working well enough based on blood tests at screening
- If you are female and could become pregnant, you must have a negative pregnancy test before joining, and all participants who are sexually active must agree to use reliable contraception during the study and for 90 days after finishing treatment
- You (or your caregiver) must be able to fill in quality-of-life questionnaires during the study
- Depending on which group (cohort) you enter, there may be specific age ranges and additional diagnosis requirements (confirm with trial site)
Who may not be able to join:
- People with Type 1 diabetes, or uncontrolled Type 2 diabetes that requires certain medications, or with high fasting blood sugar levels at the screening visit
- People who have had a heart attack or moderate-to-severe heart failure within the 6 months before starting the study drug, or who have certain heart rhythm problems, or whose heart pumping function is below a certain level (confirm with trial site)
- People who have had major surgery, radiation therapy, or certain immune-based treatments within 4 weeks before starting the study drug
- People who have taken other experimental treatments for PROS or PS (such as sirolimus or everolimus) within 2 weeks before starting the study drug
- People who have previously had a serious bad reaction or could not tolerate a type of drug similar to the one used in this trial (called AKT inhibitors)
- People with another serious uncontrolled illness unrelated to PROS or PS, such as an active infection, HIV, a condition affecting how the body absorbs food, or mental health or substance use issues that would make it hard to follow the study rules
- People who are pregnant or breastfeeding
- People who are unable to follow the study schedule or take the study drug as directed
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
1 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Number of Participants Who Experienced an Adverse Event (AE); Number of Participants Who Discontinued Study Treatment Due to an AE
Can't join this trial?
Data last synced from ClinicalTrials.gov: 2 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.