Sirolimus to Treat Cowden Syndrome and Other PTEN Hamartomatous Tumor Syndromes

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Arun Rajan, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier:
NCT00971789
First received: September 3, 2009
Last updated: July 18, 2013
Last verified: July 2013
Results First Received: May 3, 2013  
Study Type: Interventional
Study Design: Endpoint Classification: Pharmacodynamics Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Cowden's Disease
Hamartoma Syndrome, Multiple
Interventions: Radiation: fludeoxyglucose F 18
Drug: sirolimus
Other: Clinical Videography

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

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  



  Baseline Characteristics
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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.
No text entered.

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  
Number of Participants  
[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  



  Outcome Measures
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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 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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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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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Dr. Arun Rajan
Organization: National Cancer Institute, National Institutes of Health
phone: 301-594-5322
e-mail: rajana@mail.nih.gov


Publications:
Publications automatically indexed to this study:

Responsible Party: Arun Rajan, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier: NCT00971789     History of Changes
Obsolete Identifiers: NCT00722449
Other Study ID Numbers: 080151, 08-C-0151
Study First Received: September 3, 2009
Results First Received: May 3, 2013
Last Updated: July 18, 2013
Health Authority: United States: Federal Government