Sirolimus to Treat Cowden Syndrome and Other PTEN Hamartomatous Tumor Syndromes

Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups

	Description
Sirolimus Patients	sirolimus 6 mg by mouth loading dose and 2 mg by mouth daily in a 28 day treatment cycle. Patients who do not have cancer take the drug for a total of two cycles (56 days) unless they develop unacceptable side effects. Those who have cancer may continue sirolimus beyond cycle 2 until their disease worsens or they develop unacceptable side effects.

Participant Flow:   Overall Study

	Sirolimus Patients
STARTED	18
COMPLETED	18
NOT COMPLETED	0

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups

	Description
Sirolimus Patients	sirolimus 6 mg by mouth loading dose and 2 mg by mouth daily in a 28 day treatment cycle. Patients who do not have cancer take the drug for a total of two cycles (56 days) unless they develop unacceptable side effects. Those who have cancer may continue sirolimus beyond cycle 2 until their disease worsens or they develop unacceptable side effects.

Baseline Measures

	Sirolimus Patients
Overall Participants Analyzed  [Units: Participants]	18
Age  [Units: Participants]
<=18 years	0
Between 18 and 65 years	15
>=65 years	3
Age  [Units: Years] Mean (Standard Deviation)	43.54  (15.54)
Gender  [Units: Participants]
Female	9
Male	9
Ethnicity (NIH/OMB)  [Units: Participants]
Hispanic or Latino	0
Not Hispanic or Latino	18
Unknown or Not Reported	0
Race (NIH/OMB)  [Units: Participants]
American Indian or Alaska Native	0
Asian	1
Native Hawaiian or Other Pacific Islander	0
Black or African American	0
White	17
More than one race	0
Unknown or Not Reported	0
Region of Enrollment  [Units: Participants]
United States	18

1.  Primary:

Biochemical Changes in Benign and Malignant Tumor Tissues as Assessed by Immunohistochemistry.   [ Time Frame: Baseline, day 14, and day 56 ]

2.  Primary:

Number of Participants With Adverse Events   [ Time Frame: 47 months ]

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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