Trial Outcomes & Findings for RAD001 in Previously Treated Patients With Metastatic Pancreatic Cancer (NCT NCT00409292)

Last Updated: 2014-08-11

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

33 participants

Primary outcome timeframe

two months

Results posted on

2014-08-11

Participant Flow

Participant milestones

Participant milestones
Measure	RAD001 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.
Overall Study STARTED	33
Overall Study COMPLETED	33
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

RAD001 in Previously Treated Patients With Metastatic Pancreatic Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	RAD001 n=33 Participants RAD001: Taken orally daily for as long as the participant continues to receive a benefit.
Age, Customized	61 years n=99 Participants
Sex: Female, Male Female	15 Participants n=99 Participants
Sex: Female, Male Male	18 Participants n=99 Participants
Region of Enrollment United States	33 participants n=99 Participants

PRIMARY outcome

Timeframe: two months

Population: 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.

Outcome measures

Outcome measures
Measure	RAD001 n=29 Participants RAD001 was administered continuously at a dose of 10 mg daily by mouth until disease progression, unacceptable toxicity, or withdrawal of consent.
To Assess Progression-free Survival of RAD001 at Two Months in Patients With Metastatic Pancreatic Cancer Whose Disease Has Progressed on Gemcitabine Chemotherapy.	23 participants

SECONDARY outcome

Timeframe: Patients were followed for the duration of their time on treatment and 30 days after their last dose of RAD001.

Population: A total of 33 eligible patients received at least 1 week of study drug and are included in our toxicity analyses.

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.

Outcome measures

Outcome measures
Measure	RAD001 n=33 Participants RAD001 was administered continuously at a dose of 10 mg daily by mouth until disease progression, unacceptable toxicity, or withdrawal of consent.
To Assess the Safety of RAD001 in Patients With Metastatic Pancreatic Cancer	33 participants

SECONDARY outcome

Timeframe: 2 years

Population: 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.

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.

Outcome measures

Outcome measures
Measure	RAD001 n=29 Participants RAD001 was administered continuously at a dose of 10 mg daily by mouth until disease progression, unacceptable toxicity, or withdrawal of consent.
to Assess Response Rate Associated With RAD001 in This Patient Population. Stable Disease as Best Response	7 participants
to Assess Response Rate Associated With RAD001 in This Patient Population. Progressive Disease as Best Response	22 participants

SECONDARY outcome

Timeframe: 2 years

Overall survival was defined as the time from study entry until death from any cause.

Outcome measures

Outcome measures
Measure	RAD001 n=29 Participants RAD001 was administered continuously at a dose of 10 mg daily by mouth until disease progression, unacceptable toxicity, or withdrawal of consent.
to Assess Overall Survival Associated With RAD001 in This Patient Population.	4.5 months Interval 0.97 to 15.43

Adverse Events

RAD001

Serious events: 25 serious events

Other events: 33 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	RAD001 n=33 participants at risk
Blood and lymphatic system disorders Lymphocytes	6.1% 2/33
Blood and lymphatic system disorders Neutrophils	9.1% 3/33
Blood and lymphatic system disorders Platelets	12.1% 4/33
Metabolism and nutrition disorders Hypokalemia	6.1% 2/33
Metabolism and nutrition disorders Hyperglycemia	18.2% 6/33
Hepatobiliary disorders ALT	3.0% 1/33
Metabolism and nutrition disorders Hypercholesterolemia	3.0% 1/33
General disorders Fatigue	9.1% 3/33
Gastrointestinal disorders Nausea	3.0% 1/33
Gastrointestinal disorders Vomiting	3.0% 1/33
Gastrointestinal disorders Oral Mucositis/Stomatitis	3.0% 1/33

Other adverse events

Other adverse events
Measure	RAD001 n=33 participants at risk
Blood and lymphatic system disorders Lymphocytes	24.2% 8/33
Blood and lymphatic system disorders Neutrophils	15.2% 5/33
Blood and lymphatic system disorders Hemoglobin	51.5% 17/33
Blood and lymphatic system disorders Platelets	42.4% 14/33
Metabolism and nutrition disorders Hypokalemia	21.2% 7/33
Metabolism and nutrition disorders Hyperglycemia	48.5% 16/33
Hepatobiliary disorders ALT	9.1% 3/33
Hepatobiliary disorders AST	30.3% 10/33
Metabolism and nutrition disorders Hypercholesterolemia	18.2% 6/33
General disorders Fatigue	48.5% 16/33
Gastrointestinal disorders Nausea	42.4% 14/33
Gastrointestinal disorders Vomiting	12.1% 4/33
Gastrointestinal disorders Oral Mucositis/Stomatitis	27.3% 9/33
Gastrointestinal disorders Anorexia	15.2% 5/33
Gastrointestinal disorders Diarrhea	24.2% 8/33
Gastrointestinal disorders Constipation	15.2% 5/33

Additional Information

Brian Wolpin, MD

Dana-Farber Cancer Institute

Phone: 617-632-3000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place