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Sirolimus in Cowden Syndrome and PTEN Hamartoma Tumor Syndromes
NCT00971789Phase 2
Key facts
- Status
- Completed
- Start
- Jul 2008
- Primary completion
- Oct 2012
- Countries
- 1
- Sponsor
- National Cancer Institute
- Modality
- Small Molecules
- Drugs
- sirolimus
- Conditions
- Cowden syndrome (CS)PTEN hamartoma tumor syndrome (PHTS)
01
Overview
Phase 2, open-label, single-arm trial of sirolimus in Cowden syndrome (CS) and PTEN hamartoma tumor syndrome (PHTS), sponsored by National Cancer Institute. Primary endpoints: Biochemical Changes in Benign and Malignant Tumor Tissues as Assessed by Immunohistochemistry at Day 56 and Number of Participants With Adverse Events at 47 months. Started Jul 2008; status: completed.
02
Design
1 arm- Study design
- Non-randomizedOpen-labelSingle group
Sirolimus Patients
Patients receive sirolimus daily.
03
Who it enrols
Adult patients with documented germline PTEN mutations meeting criteria for Cowden Syndrome, with adequate organ function and no recent chemotherapy or prior mTOR inhibitor use.
04
Endpoints
Primary
Biochemical Changes in Benign and Malignant Tumor Tissues as Assessed by Immunohistochemistry
Number of Participants With Adverse Events
Secondary
Inhibition of the mTOR pathway in peripheral blood mononuclear cells
Changes and duration of change in benign or malignant tumor size
Changes in tumor metabolism
Changes in lymphocyte counts
Changes in neuropsychological testing
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