Everolimus for Social Difficulties in PTEN Germline Mutations
This study is testing if everolimus, a medication given as oral capsules, can improve social abilities in people with PTEN Hamartoma Tumor Syndrome (PHTS). PHTS is a genetic condition that can cause various health issues, including social difficulties. Researchers want to see if everolimus is safe and effective for adults and children aged 5 to 45 who have PHTS and experience at least moderate social challenges. You would need to have a confirmed diagnosis of PHTS through genetic testing and score above 60 on a social skills assessment. The study will measure changes in social skills over six months. The current status of this study is unclear, and it plans to enroll 60 participants.
- Study design
- This is a double-blind study, meaning neither you nor your doctor will know if you are receiving everolimus or a placebo (an inactive capsule). It plans to enroll 60 participants.
- What's involved
- You would take oral capsules once daily for six months. Your social skills will be measured at the start and then monthly for six months.
- Compensation
- Not stated in the trial record.
- Follow-up
- Your social skills will be measured for six months after starting treatment.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Double-Blind Trial of Everolimus for Improving Social Abilities in PTEN Germline Mutations
At a glance
Conditions
Where it's being run
3 sites across 3 statesWho to contact
Opens a ready-to-send draft in your own email app — review before sending.
What this trial measures
- Change from baseline in severity of social skill deficits based on Social Responsiveness Scale (SRS) total score over 6 months.Baseline, month 1, month 2, month 3, month 4, month 5, month 6
The SRS is a well-validated, reliable, and widely used measure for identifying and measuring impairments in social processing and associated behaviors. It is completed by an observer who knows the participant well (e.g. parent, care-giver, partner)