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Could a cancer drug boost social abilities in rare genetic condition?

NCT ID NCT07218575

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests whether everolimus, a drug already approved for other conditions, can safely improve social abilities in people with PTEN Hamartoma Tumor Syndrome (PHTS). About 60 adults and children with PHTS and social difficulties will take either everolimus or a placebo for 6 months, then all will receive everolimus for another 6 months. The goal is to see if the drug helps with social skills and functioning.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
everolimus (Afinitor, Zortress)
What this could lead to
If it works, this could point toward a treatment for social difficulties in people with PTEN Hamartoma Tumor Syndrome.
What could go wrong
This is a small, early-phase trial with only 60 participants. The drug may not improve social abilities, and side effects from everolimus are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

About 60 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Sep 2026

An estimate. Start dates often move.

Expected to finish

Sep 2030

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

5 to 45 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 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 Exclusion Criteria: 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

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    3 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • Cleveland Clinic

    Cleveland, Ohio, 44195, United States

  • Stanford University

    Stanford, California, 94305, United States

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