Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Boston Children's Hospital
Age
5–45
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
1. Diagnosis of PTEN Harmartoma Tumor Syndrom (PHTS), confirmed by genetic testing (the testing may be done as part of study screening)
2. Experiences at least moderate levels of social difficulties, based on SRS T score \> 60 (measured during screening Have at least a moderate impairment in social abilities, based on SRS score during study screening
3. Fluent in English
4. Females of child-bearing potential must have no plans to become pregnant and be using contraception during the study (if sexually active).
5. Availability of parent, care-giver, partner or other suitable individual who can provide observation reports and provide transportation to attend clinic visits
6. Adequate liver, kidney and bone-marrow function (checked during screening)
7. Medically stable
8. No plans to change school, behavioral therapies, home services or speech therapy during the study period
9. Ability to swallow medicine in pill form
You may not be if
1. Ongoing or planned treatment with any medication with known or possible ant-mTOR activity (e.g. sirolimus), or strong inducers or inhibitors of CYP3A, CYP2D6, P450 or PgP (e.g. cyclosporine, ketoconazole, erythromycin, rifampin, phenytoin, phenobarbital) or ACE inhibitors
2. Chronic treatment with systemic corticosteroids or other immunosuppressive treatments (topical or inhaled corticosteroids are allowed).
3. Major surgery or any anti-cancer therapies (including radiotherapy) within 4 weeks of enrollment
4. Neurosurgery within 6 months of enrollment
5. Uncontrolled diabetes defined as HbA1c \>8% despite treatment
6. Uncontrolled hyperlipidemia (defined as fasting serum cholesterol \> 300 mg/dL OR \>7.75 mmol/L AND fasting triglycerides \> 2.5 x ULN, assessed during screening)
7. History of Hepatitis B, Hepatitis C or HIV
8. Participation in a clinical trial in the 60 days prior to study entry
9. Known intolerance or hypersensitivity to everolimus or other rapamycin analogs (e.g. sirolimus, temsirolimus)
10. Patients who have a history of another primary malignancy, with the exceptions of non-melanoma skin cancer, and carcinoma in situ of the cervix, uteri, or breast from with the patient has been disease free for \> 3 years
