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RAD001 in Previously Treated Patients With Metastatic Pancreatic Cancer

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ClinicalTrials.gov Identifier: NCT00409292
Recruitment Status : Completed
First Posted : December 8, 2006
Results First Posted : August 11, 2014
Last Update Posted : August 11, 2014
Sponsor:
Collaborators:
Massachusetts General Hospital
Beth Israel Deaconess Medical Center
Novartis
Information provided by (Responsible Party):
Brian Wolpin, MD, MPH, Dana-Farber Cancer Institute

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Pancreatic Cancer
Intervention Drug: RAD001
Enrollment 33
Recruitment Details  
Pre-assignment Details  
Arm/Group Title RAD001
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RAD001 was administered continuously at a dose of 10 mg daily by mouth until disease progression, unacceptable toxicity, or withdrawal of consent.

Four weeks of study drug was considered to be one cycle of treatment.

Period Title: Overall Study
Started 33
Completed 33
Not Completed 0
Arm/Group Title RAD001
Hide Arm/Group Description RAD001: Taken orally daily for as long as the participant continues to receive a benefit.
Overall Number of Baseline Participants 33
Hide Baseline Analysis Population Description
Our study design used a one-stage design with a target accrual of 35 eligible patients, with the assumption that an improvement in PFS at 2 months from 50% to 71% would warrant further study in this patient population.
Age, Customized  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 33 participants
61
(39 to 78)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 33 participants
Female
15
  45.5%
Male
18
  54.5%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 33 participants
33
1.Primary Outcome
Title To Assess Progression-free Survival of RAD001 at Two Months in Patients With Metastatic Pancreatic Cancer Whose Disease Has Progressed on Gemcitabine Chemotherapy.
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The Outcome Measure is reporting the number of participants experiencing Progression-free Survival at 2 months after treatment.

The study was designed with a primary end point of progression-free survival (PFS), defined as the time from study entry to documentation of progressive disease or death from any cause. On the basis of prior studies of second-line treatment in metastatic pancreatic cancer, we estimated that such treatment has been associated with a median PFS of 2 months. Our study design used a one-stage design with a target accrual of 35 eligible patients, with the assumption that an improvement in PFS at 2 months from 50% to 71% would warrant further study in this patient population.

Time Frame two months
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Hide Analysis Population Description
On the basis of prior studies of 2nd line treatment in metastatic pancreatic cancer, we estimated a median PFS of 2 months. Our study design used a one-stage design with a target accrual of 35 eligible patients, with the assumption that an improvement in PFS at 2 months from 50% to 71% would warrant further study in this population.
Arm/Group Title RAD001
Hide Arm/Group Description:
RAD001 was administered continuously at a dose of 10 mg daily by mouth until disease progression, unacceptable toxicity, or withdrawal of consent.
Overall Number of Participants Analyzed 29
Measure Type: Number
Unit of Measure: participants
23
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection RAD001
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.10
Comments [Not Specified]
Method binomial hypothesis test
Comments [Not Specified]
2.Secondary Outcome
Title To Assess the Safety of RAD001 in Patients With Metastatic Pancreatic Cancer
Hide Description Patients were followed for the duration of their time on treatment and 30 days after their last dose of RAD001. The number of patients with treatment-related adverse events are reported.
Time Frame Patients were followed for the duration of their time on treatment and 30 days after their last dose of RAD001.
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Hide Analysis Population Description
A total of 33 eligible patients received at least 1 week of study drug and are included in our toxicity analyses.
Arm/Group Title RAD001
Hide Arm/Group Description:
RAD001: Taken orally daily for as long as the participant continues to receive a benefit.
Overall Number of Participants Analyzed 33
Measure Type: Number
Unit of Measure: participants
33
3.Secondary Outcome
Title to Assess Response Rate Associated With RAD001 in This Patient Population.
Hide Description The secondary objectives of the study were to assess tumor response rate and overall survival. Patients were required to have measurable disease per Response Evaluation Criteria in Solid Tumors (RECIST). Per RECIST, for target lesions assessed by CT: Complete Response (CR) is Disappearance of all target lesions; Partial Response (PR) is >=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) equals CR + PR.
Time Frame 2 years
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Hide Analysis Population Description
Of the 33 patients enrolled, 29 reached restaging. Three patients who did not reach restaging were removed from study because of withdrawal of consent. One patient was removed from study after 8 days of treatment because of worsening of a preexisting perirectal fistula, requiring surgical intervention.
Arm/Group Title RAD001
Hide Arm/Group Description:
RAD001: Taken orally daily for as long as the participant continues to receive a benefit.
Overall Number of Participants Analyzed 29
Measure Type: Number
Unit of Measure: participants
Stable Disease as Best Response 7
Progressive Disease as Best Response 22
4.Secondary Outcome
Title to Assess Overall Survival Associated With RAD001 in This Patient Population.
Hide Description Overall survival was defined as the time from study entry until death from any cause.
Time Frame 2 years
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Hide Analysis Population Description
[Not Specified]
Arm/Group Title RAD001
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RAD001 was administered continuously at a dose of 10 mg daily by mouth until disease progression, unacceptable toxicity, or withdrawal of consent.

Four weeks of study drug was considered to be one cycle of treatment.

RAD001: Taken orally daily for as long as the participant continues to receive a benefit.

Overall Number of Participants Analyzed 29
Median (Full Range)
Unit of Measure: months
4.5
(0.97 to 15.43)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title RAD001
Hide Arm/Group Description [Not Specified]
All-Cause Mortality
RAD001
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
RAD001
Affected / at Risk (%)
Total   25/33 (75.76%) 
Blood and lymphatic system disorders   
Lymphocytes  2/33 (6.06%) 
Neutrophils  3/33 (9.09%) 
Platelets  4/33 (12.12%) 
Gastrointestinal disorders   
Nausea  1/33 (3.03%) 
Vomiting  1/33 (3.03%) 
Oral Mucositis/Stomatitis  1/33 (3.03%) 
General disorders   
Fatigue  3/33 (9.09%) 
Hepatobiliary disorders   
ALT  1/33 (3.03%) 
Metabolism and nutrition disorders   
Hypokalemia  2/33 (6.06%) 
Hyperglycemia  6/33 (18.18%) 
Hypercholesterolemia  1/33 (3.03%) 
1
Term from vocabulary, CTCAE (3.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
RAD001
Affected / at Risk (%)
Total   33/33 (100.00%) 
Blood and lymphatic system disorders   
Lymphocytes  8/33 (24.24%) 
Neutrophils  5/33 (15.15%) 
Hemoglobin  17/33 (51.52%) 
Platelets  14/33 (42.42%) 
Gastrointestinal disorders   
Nausea  14/33 (42.42%) 
Vomiting  4/33 (12.12%) 
Oral Mucositis/Stomatitis  9/33 (27.27%) 
Anorexia  5/33 (15.15%) 
Diarrhea  8/33 (24.24%) 
Constipation  5/33 (15.15%) 
General disorders   
Fatigue  16/33 (48.48%) 
Hepatobiliary disorders   
ALT  3/33 (9.09%) 
AST  10/33 (30.30%) 
Metabolism and nutrition disorders   
Hypokalemia  7/33 (21.21%) 
Hyperglycemia  16/33 (48.48%) 
Hypercholesterolemia  6/33 (18.18%) 
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: Brian Wolpin, MD
Organization: Dana-Farber Cancer Institute
Phone: 617-632-3000
Responsible Party: Brian Wolpin, MD, MPH, Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier: NCT00409292     History of Changes
Other Study ID Numbers: 06-197
First Submitted: December 6, 2006
First Posted: December 8, 2006
Results First Submitted: April 30, 2014
Results First Posted: August 11, 2014
Last Update Posted: August 11, 2014