Sirolimus for Cowden Syndrome with Colon Polyposis
What You Need to Know Before You Apply
What is the purpose of this trial?
Colon polyposis (the presence of multiple colon polyps) is very common with Cowden syndrome, as over 60% of patients have 50 or more polyps. In a previous clinical trial, some participants had reduction in the number of colon polyps with the use of the medication sirolimus for a very short time period. This study is investigating sirolimus and its effect on the number of colon polyps in patients with Cowden syndrome and polyposis over a 1 year period.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is sirolimus safe for humans?
How is the drug Sirolimus unique for treating Cowden Syndrome with Colon Polyposis?
What data supports the effectiveness of the drug Sirolimus for Cowden Syndrome with Colon Polyposis?
Who Is on the Research Team?
Peter P. Stanich
Principal Investigator
Ohio State University
Are You a Good Fit for This Trial?
This trial is for adults over 18 with Cowden syndrome or related disorders, who have a genetic mutation called PTEN and more than 50 colon polyps that can't be removed endoscopically. It's not for those with chronic kidney disease, past high-grade colon tumors, colectomy history, or current/pending pregnancy.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive sirolimus 2 mg by mouth daily for 1 year. Colonoscopy with polyp estimation at trial entrance and study completion.
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Sirolimus
Sirolimus is already approved in United States, European Union, Canada, Japan for the following indications:
Find a Clinic Near You
Who Is Running the Clinical Trial?
Ohio State University
Lead Sponsor
PTEN Research
Collaborator
Pfizer
Industry Sponsor
Albert Bourla
Pfizer
Chief Executive Officer since 2019
PhD in Biotechnology of Reproduction, Aristotle University of Thessaloniki
Patrizia Cavazzoni
Pfizer
Chief Medical Officer
MD from McGill University