Status:
NOT_YET_RECRUITING
Double-Blind Trial of Everolimus for Improving Social Abilities in PTEN Germline Mutations
Lead Sponsor:
Boston Children's Hospital
Conditions:
Cowden's Disease
Cowden's Syndrome
Eligibility:
All Genders
5-45 years
Phase:
PHASE2
PHASE3
Brief Summary
The goal of this study is to examine the safety and treatment effects of everolimus in adults and children with PTEN Hamartoma Tumor Syndrome (PHTS) who experience social difficulties. The study will ...
Eligibility Criteria
Inclusion
- Diagnosis of PTEN Harmartoma Tumor Syndrom (PHTS), confirmed by genetic testing (the testing may be done as part of study screening)
- 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
- Fluent in English
- Females of child-bearing potential must have no plans to become pregnant and be using contraception during the study (if sexually active).
- Availability of parent, care-giver, partner or other suitable individual who can provide observation reports and provide transportation to attend clinic visits
- Adequate liver, kidney and bone-marrow function (checked during screening)
- Medically stable
- No plans to change school, behavioral therapies, home services or speech therapy during the study period
- Ability to swallow medicine in pill form
Exclusion
- 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
- Chronic treatment with systemic corticosteroids or other immunosuppressive treatments (topical or inhaled corticosteroids are allowed).
- Major surgery or any anti-cancer therapies (including radiotherapy) within 4 weeks of enrollment
- Neurosurgery within 6 months of enrollment
- Uncontrolled diabetes defined as HbA1c \>8% despite treatment
- 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)
- History of Hepatitis B, Hepatitis C or HIV
- Participation in a clinical trial in the 60 days prior to study entry
- Known intolerance or hypersensitivity to everolimus or other rapamycin analogs (e.g. sirolimus, temsirolimus)
- 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
Key Trial Info
Start Date :
April 1 2026
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
April 1 2030
Estimated Enrollment :
60 Patients enrolled
Trial Details
Trial ID
NCT07218575
Start Date
April 1 2026
End Date
April 1 2030
Last Update
October 20 2025
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