Sirolimus for Cowden Syndrome With Colon Polyposis

Plain-language summary

This study examines Sirolimus in patients with Cowden syndrome who have colon polyposis. Patients aged 18 and older with a confirmed PTEN germline mutation and many polyps found on colonoscopy could take part. It was conducted in Phase 2 and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 2

Status

Completed

Sponsor

Ohio State University

Intervention

Sirolimus

Start date

9/16/2019

Estimated completion

6/5/2025

Countries

United States

Related genes

Locations

• Columbus, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* Cowden syndrome or other PTEN hamartoma tumor syndrome spectrum disorder
* Confirmed pathogenic or likely pathogenic PTEN germline mutation on genetic testing
* Previous colonoscopy with colon polyposis. This is defined for the study as either too many polyps to remove endoscopically as judged by the performing physician at the last colonoscopy or findings indicating at least an InSIGHT stage 1 score (over 20 polyps).
* Age 18 or greater
* Capacity to consent to study

Exclusion Criteria:

* Pregnancy or plans for pregnancy while on treatment or within 3 months of stopping treatment (for both women and men)
* Chronic kidney disease
* Chronic renal disease
* History of colon cancer or colon adenoma with high grade dysplasia
* History of colectomy