The following info and data is provided "as is" to help patients around the globe.
We do not endorse or review these studies in any way.
Brief Title: Sirolimus for Improving Social Abilities in People With PTEN Germline Mutations
Official Title: A Randomized Controlled Double-Blind Trial of Sirolimus for Improving Social Abilities in People With PTEN Germline Mutations
Study ID: NCT06080165
Brief Summary: The goal of this study is to examine the safety and treatment effects of sirolimus for targeting social communication deficits in people with genetic disorders associated with PTEN germline mutations, which are often referred to as PTEN Harmartoma Tumor Syndrome (PHTS). The mechanism of sirolimus in the body has shown promise for helping to improve social communication skills in case reports of people with PHTS. Everolimus, a closely related compound, also showed benefits in social communication skills in a previous pilot trial in people with PHTS. This is a 6 month double-blind trial followed by at 6 month open label extension trial.
Detailed Description:
Minimum Age: 5 Years
Eligible Ages: CHILD, ADULT
Sex: ALL
Healthy Volunteers: No
Stanford University, Stanford, California, United States
Boston Children's Hospital, Boston, Massachusetts, United States
Cleveland Clinic, Cleveland, Ohio, United States
Name: Antonio Hardan, MD
Affiliation: Stanford University
Role: PRINCIPAL_INVESTIGATOR