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Study Summary
No. 2006-0334:.......Endocrine; Gastrointestinal......James Yao......Gastrointestinal Medical Oncology
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Study Summary Title
Study Summary
Number:
2006-0334
Study Title:An open label, stratified, single-arm phase II study of RAD001 in patients with advanced pancreatic neuroendocrine tumor (NET) after failure of cytotoxic chemotherapy (CRAD001C2239)
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Physician New Patient Referral
Name:James YaoPatients Call:800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Dept:Gastrointestinal Medical OncologyReferring MD
Call:
800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Phone:713-792-2828
Contact us about clinical trials
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General Information
Disease Group:Endocrine
Gastrointestinal
Supported By:N/A
Phase of Study:Phase IIReturn
Visit:
During cycle one, patients will return every 2 weeks, then prior to every cycle
thereafter.
Treatment
Agents:
RAD001
Sandostatin LAR
Home Care:Since RAD001 is an oral medication, patients may self administer it at home.
Treatment Loc:Independent Multicenter Arrangements
Estimated
Length of Stay
in Houston:
Patients will not be hospitalized for treatment on this study
Description/
Intervention:
The goal of this clinical research study is to learn if RAD001 can help to
control the disease in patients with advanced pancreatic neuroendocrine
tumors. The safety of RAD001 and RAD001 in combination with Sandostatin LAR®
Depot (octreotide LAR) will also be studied.
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Study Objectives / Outcomes
Primary Objective:

To determine the objective response rate (ORR) (including complete response and partial response) of RAD001 10 mg po daily monotherapy in patients with advanced (unresectable or metastatic) pancreatic NETs after the failure of cytotoxic chemotherapy (stratum 1).

Secondary Objectives:
  • To determine the response duration of RAD001 10 mg po daily monotherapy (stratum 1).
  • To determine the objective response rate (ORR) and response duration of RAD001 10 mg po daily plus Sandostatin LAR® Depot therapy in patients whose tumors have progressed while receiving Sandostatin LAR® Depot therapy and after the failure of cytotoxic chemotherapy (stratum 2).
  • To determine the safety and tolerability of RAD001 monotherapy (10 mg/d) in patients with Pancreatic NET (stratum 1).
  • To determine the safety and tolerability of the combination of RAD001 (10 mg/d) plus Sandostatin LAR® Depot in patients with Pancreatic NET (stratum 2).
  • To determine the progression free survival (PFS) and the overall survival (OS) of patients receiving RAD001 10 mg per day in combination with Sandostatin LAR® and of patients receiving RAD001 10 mg per day monotherapy.
  • To assess the steady state exposure to RAD001 and to estimate the effect of coadministering Sandostatin LAR® Depot on RAD001 exposure.

Exploratory Objectives:
  • To determine the effects of RAD001 on plasma angiogenic molecules such as VEGF, and basic FGF.
  • To determine the effects of RAD001 on serum Lactate Dehydrogenase (LDH) isozymes.
  • To characterize pre-treatment tumor samples by immunohistochemical and genetic analyses indicating activation of the mTOR pathway.
  • To assess the change in octreotide levels after treatment with RAD001.
  • To assess the relationship between RAD001 steady state levels, tumor response, and CgA response (50% decrease).
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Study Status Information
Study Activation / Registration Date:06/21/2006
IRB Review and Approval Date:05/17/2006
Study Type:Phase Ii Or Phase I/Ii
Recruitment Status:Closed
Projected Accrual:144
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Enrollment Eligibility
If you do not meet the enrollment eligibility, there may be other treatment options for you. Please Contact the Referral Office for more information.

Inclusion Criteria:1) Adult male or female patients (>/= 18 years of age).

2) Advanced (unresectable or metastatic) biopsy-proven pancreatic NET documented as follows: 1- Radiologic, operative, or pathology reports should document a pancreatic location of tumor at some point in the patient's history; 2- Patients must have confirmed low-grade or intermediate-grade neuroendocrine carcinoma. Pathology report should state one of the following: carcinoid, islet cell carcinoma, pancreatic endocrine tumor, low-grade or well-differentiated neuroendocrine carcinoma, atypical carcinoid, intermediate-grade or moderately differentiated neuroendocrine carcinoma.

3) Documented objective progression of disease by RECIST criteria while receiving cytotoxic chemotherapy or documented progression at any time after receiving an adequate course of cytotoxic chemotherapy (i.e., at least 3 consecutive cycles or months of treatment with the same cytotoxic drug or regimen). Previous therapy with alpha interferon or tyrosine kinase inhibitors are allowed but would not be considered to be prior chemotherapy.

4) Objective progression of disease must be documented by RECIST criteria. Any of the following would be sufficient according to RECIST: a 20% increase in the sum of unidimensionally measured target lesions; a new lesion; unequivocal increase in non-measurable disease.

5) At screening, a triphasic CT or MRI scan must demonstrate measurable disease by RECIST criteria, i.e., the presence of at least one measurable lesion. Measurable disease lesions must be accurately measured in at least one dimension with longest diameter >/= 20 mm using conventional techniques or >/= 10 mm with spiral CT scan (with minimum lesion size no less than double the slice thickness).

6) Adequate bone marrow function as shown by: ANC >/= 1.5 x 10^9/L, Platelets >/= 100 x 10^9/L, Hb >9 g/dL.

7) Adequate liver function as shown by:serum bilirubin </= 1.5 x ULN; INR < 1.3 (or < 3 on anticoagulants); ALT and AST </= 2.5x ULN ( </= 5x ULN in patients with liver metastases).

8) Adequate renal function: serum creatinine </= 1.5 x ULN.

9) Fasting serum cholesterol </=300 mg/dL OR </= 7.75 mmol/L AND fasting triglycerides </= 2.5 x ULN. NOTE: In cases where one or both of these thresholds are exceeded, the patient can only be included after initiation of appropriate lipid lowering medication.

10) Women of childbearing potential must have a negative serum pregnancy test within 14 days of enrollment and/or a urine pregnancy test 48 hours prior to administration of the first study treatment.

11) Appropriate contraception is defined as surgical sterilization (e.g., bilateral tubal ligation, hysterectomy, vasectomy), hormonal contraception (implantable, patch, oral), and doublebarrier methods (any double combination of: IUD, male or female condom with spermicidal gel, diaphragm, sponge, cervical cap).

12) WHO Performance Status 0-2.

13) Signed informed consent to participate in the study must be obtained from patients after they have been fully informed of the nature and potential risks by the investigator (or his/her designee) with the aid of written information.

14) Inclusion criteria for Stratum 2 only: Meet all inclusion criteria defined above for both strata

15) Inclusion criteria for Stratum 2 only: Receiving treatment (at least 3 consecutive months) with Sandostatin LAR® Depot.

16) Inclusion criteria for Stratum 2 only: In addition to documentation of progressive disease on or after chemotherapy, patients in stratum 2 must have documented objective progression of disease while receiving Sandostatin LAR® Depot. The initial and follow-up scans documenting progression must have been obtained during continual monthly therapy with Sandostatin LAR® Depot, with at least 2 months separating the initial and follow-up scans. Progression must be documented by RECIST criteria as described above.

Exclusion Criteria:1) Anticancer therapy within 3 weeks of enrollment including chemotherapy, hormonal therapy, immunotherapy, surgery or radiotherapy. Investigational agents used solely for imaging purposes are permissible if deemed safe and acceptable by local review authorities. Sandostatin LAR® Depot is allowed in stratum 2.

2) Hepatic artery embolization within the last 6 months (1 month if there are other sites of measurable disease), or cryoablation of hepatic metastasis within 2 months of enrollment.

3) Prior therapy with RAD001 (everolimus) or other rapamycins (sirolimus, temsirolimus).

4) Patients with uncontrolled diabetes mellitus as defined by fast blood sugar > 1.5 x ULN.

5) Liver disease such as cirrhosis, chronic active hepatitis or chronic persistent hepatitis.

6) Patients with acute or chronic, active infectious disorders or patients with nonmalignant medical illnesses that are uncontrolled, or whose control may be jeopardized by the complications of initiating RAD001.

7) No other prior or concurrent malignancy is allowed except for the following: adequately treated basal cell or squamous cell skin cancer, or other adequately treated in situ cancer, or any other cancer from which the patient has been disease free for five years.

8) Known history of immunocompromise, including a positive HIV test. An HIV test will not be required; however, previous medical history will be reviewed.

9) Female patients who are pregnant or nursing (lactating), or adults of reproductive potential who are not using effective birth control methods. If barrier contraceptives are being used, these must be continued throughout the trial by both sexes.

10) Concomitant medications known to inhibit, induce or be a substrate to isoenzyme CYP3A are excluded unless the drugs are medically necessary and no substitutes are available. If there are no acceptable substitutes, special precautions should be taken in these patients.

11) Additional Exclusion Criteria for Stratum 1 only: Received treatment with Sandostatin LAR® Depot or any other long-acting somatostatin analogue in the 60 days prior to enrollment or any short-acting somatostatin analogue in the two weeks prior to enrollment.

12) Patients with poorly differentiated neuroendocrine carcinoma, high-grade neuroendocrine carcinoma, adenocarcinoid, goblet cell carcinoid and small cell carcinoma are not eligible.

13) Patients with severely impaired lung function.

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Links
Registration Number: NCT00363051
Study Information on Clinical Trials Registry (clinicaltrials.gov)

Other Links:
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Results


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