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Trial record 6 of 6 for:    PTEN hamartoma tumor syndrome

Sirolimus to Treat Cowden Syndrome and Other PTEN Hamartomatous Tumor Syndromes

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ClinicalTrials.gov Identifier: NCT00971789
Recruitment Status : Completed
First Posted : September 4, 2009
Results First Posted : July 19, 2013
Last Update Posted : September 30, 2015
Sponsor:
Information provided by (Responsible Party):
Arun Rajan, M.D., National Institutes of Health Clinical Center (CC)

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Cowden's Disease
Hamartoma Syndrome, Multiple
Interventions Radiation: fludeoxyglucose F 18
Drug: sirolimus
Other: Clinical Videography
Enrollment 18
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Sirolimus Patients
Hide Arm/Group Description 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.
Period Title: Overall Study
Started 18
Completed 18
Not Completed 0
Arm/Group Title Sirolimus Patients
Hide Arm/Group Description 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.
Overall Number of Baseline Participants 18
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants
<=18 years
0
   0.0%
Between 18 and 65 years
15
  83.3%
>=65 years
3
  16.7%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 18 participants
43.54  (15.54)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants
Female
9
  50.0%
Male
9
  50.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
18
 100.0%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants
American Indian or Alaska Native
0
   0.0%
Asian
1
   5.6%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
17
  94.4%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 18 participants
18
1.Primary Outcome
Title Biochemical Changes in Benign and Malignant Tumor Tissues as Assessed by Immunohistochemistry.
Hide Description A biochemical change is defined as a decrease in certain protein levels (e.g. P-AKT (phosphorylated AKT), total S6, P-S6, and P-4E-BP1) important in cell growth. These are measured by collecting tissue samples which stained and protein levels are measured under the microscope. Scoring will be based on distribution and intensity of staining. Distribution will be scored as 0 (0%), 1 (1% to 50%), and 2 (51% to 100%) to indicate the percentage of positive cells of interest in a single core. The intensity of the signal will be scored as 1 (weak), 2 (moderate), and 3 (strong). The distribution score and intensity score will be summed into a total score (TS).
Time Frame Baseline, day 14, and day 56
Hide Outcome Measure Data
Hide Analysis Population Description
No patient underwent biopsy after the study treatment, so no such tissue analysis was done.
Arm/Group Title Sirolimus Patients
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
2.Primary Outcome
Title Number of Participants With Adverse Events
Hide Description Here is the number of participants with adverse events
Time Frame 47 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Sirolimus Patients
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 18
Measure Type: Number
Unit of Measure: Participants
17
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Sirolimus Patients
Hide Arm/Group Description 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.
All-Cause Mortality
Sirolimus Patients
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Sirolimus Patients
Affected / at Risk (%) # Events
Total   0/18 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Sirolimus Patients
Affected / at Risk (%) # Events
Total   17/18 (94.44%)    
Blood and lymphatic system disorders   
Blood/Bone Marrow - Other (anemia)  1  1/18 (5.56%)  1
CD4 count  1  1/18 (5.56%)  1
Edema: limb  1  2/18 (11.11%)  2
Leukocytes (total WBC)  1  2/18 (11.11%)  2
Lymphopenia  1  3/18 (16.67%)  5
Neutrophils/granulocytes (ANC/AGC)  1  1/18 (5.56%)  1
PTT (Partial Thromboplastin Time)  1  1/18 (5.56%)  2
Platelets  1  1/18 (5.56%)  1
Gastrointestinal disorders   
Constipation  1  4/18 (22.22%)  4
Dental: teeth  1  1/18 (5.56%)  1
Diarrhea  1  6/18 (33.33%)  9
Distension/bloating, abdominal  1  1/18 (5.56%)  1
Flatulence  1  1/18 (5.56%)  1
Gastritis (including bile reflux gastritis)  1  1/18 (5.56%)  1
Gastrointestinal - Other (bloating)  1  1/18 (5.56%)  1
Hemorrhage, GI::Colon  1  1/18 (5.56%)  1
Mucositis/stomatitis (clinical exam)::Oral cavity  1  3/18 (16.67%)  3
Nausea  1  2/18 (11.11%)  2
Pain::Abdomen NOS  1  2/18 (11.11%)  3
General disorders   
Fatigue (asthenia, lethargy, malaise)  1  7/18 (38.89%)  7
Insomnia  1  4/18 (22.22%)  5
Pain - Other (R/costal vert. angle; R/flank; shoulder)  1  2/18 (11.11%)  3
Rigors/chills  1  1/18 (5.56%)  1
Infections and infestations   
Infection with normal ANC or Grade 1 or 2 neutrophils::Eye NOS  1  1/18 (5.56%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils::Skin (cellulitis)  1  1/18 (5.56%)  1
Infection with unknown ANC::Sinus  1  1/18 (5.56%)  1
Metabolism and nutrition disorders   
ALT, SGPT (serum glutamic pyruvic transaminase)  1  7/18 (38.89%)  12
AST, SGOT(serum glutamic oxaloacetic transaminase)  1  3/18 (16.67%)  5
Albumin, serum-low (hypoalbuminemia)  1  7/18 (38.89%)  9
Alkaline phosphatase  1  5/18 (27.78%)  5
Bilirubin (hyperbilirubinemia)  1  2/18 (11.11%)  3
Calcium, serum-high (hypercalcemia)  1  2/18 (11.11%)  2
Cholesterol, serum-high (hypercholesteremia)  1  5/18 (27.78%)  5
Glucose, serum-high (hyperglycemia)  1  3/18 (16.67%)  4
Glucose, serum-low (hypoglycemia)  1  2/18 (11.11%)  2
Hemoglobin  1  7/18 (38.89%)  9
Lipase  1  1/18 (5.56%)  1
Magnesium, serum-high (hypermagnesemia)  1  4/18 (22.22%)  4
Magnesium, serum-low (hypomagnesemia)  1  3/18 (16.67%)  4
Phosphate, serum-low (hypophosphatemia)  1  3/18 (16.67%)  6
Proteinuria  1  1/18 (5.56%)  1
Sodium, serum-high (hypernatremia)  1  1/18 (5.56%)  1
Sodium, serum-low (hyponatremia)  1  2/18 (11.11%)  2
Triglyceride, serum-high (hypertriglyceridemia)  1  5/18 (27.78%)  5
Uric acid, serum-high (hyperuricemia)  1  1/18 (5.56%)  1
Musculoskeletal and connective tissue disorders   
Pain::Extremity-limb  1  1/18 (5.56%)  1
Pain::Joint  1  2/18 (11.11%)  2
Pain::Muscle  1  2/18 (11.11%)  3
Pain::Neck  1  1/18 (5.56%)  1
Nervous system disorders   
Memory impairment  1  1/18 (5.56%)  1
Mood alteration::Depression  1  1/18 (5.56%)  1
Neuropathy: sensory  1  2/18 (11.11%)  2
Pain::Head/headache  1  4/18 (22.22%)  4
Renal and urinary disorders   
Urine color change  1  1/18 (5.56%)  1
Reproductive system and breast disorders   
Irregular menses (change from baseline)  1  1/18 (5.56%)  1
Respiratory, thoracic and mediastinal disorders   
Cough  1  1/18 (5.56%)  1
Pain::Throat/pharynx/larynx  1  1/18 (5.56%)  1
Skin and subcutaneous tissue disorders   
Dry skin  1  3/18 (16.67%)  3
Rash/desquamation  1  1/18 (5.56%)  1
Rash: acne/acneiform  1  1/18 (5.56%)  1
Rash: hand-foot skin reaction  1  2/18 (11.11%)  2
Vascular disorders   
Vascular - Other (venous insufficiency)  1  1/18 (5.56%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
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
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
First Submitted: September 3, 2009
First Posted: September 4, 2009
Results First Submitted: May 3, 2013
Results First Posted: July 19, 2013
Last Update Posted: September 30, 2015