RAMSETE: RAD001 in Advanced and Metastatic Silent Neuro-endocrine Tumors in Europe

Official Title: “A Single Arm, Multicenter Single Stage Phase II Trial of RAD001 as Monotherapy in the Treatment of Metastatic Non Syndromic Neuro-endocrine Tumors”

To evaluate the preliminary efficacy and safety of RAD001 as monotherapy for first-line treatment of patients with metastatic papillary carcinoma of the kidney.

Intervention(s) in this Clinical Trial

• Drug: Everolimus

Arms, Groups and Cohorts in this Clinical Trial

• Experimental: Everolimus

Primary Measures

• To evaluate the efficacy of RAD001 as monotherapy in patients with non syndromic neuro-endocrine tumours (NET). Efficacy is defined as the proportion of patients with complete (CR) or partial response (PR) according to RECIST criteria.
  • Time Frame: At baseline, Every 12 weeks, at end of study treatment
    Safety Issue?: No

Secondary Measures

• To evaluate the disease control rate (CR + PR + SD)
  • Time Frame: Every 12 weeks
    Safety Issue?: No
• To evaluate the biochemical response rate based on the tumour marker CgA
  • Time Frame: At baseline, Every 28 days or every 12 weeks, at end of study treatment
    Safety Issue?: Yes
• To evaluate progression-free survival in this patient population
  • Time Frame: done as required at statisticla analysis
    Safety Issue?: No
• To evaluate overall survival in this patient population
  • Time Frame: at date of last contact or at death
    Safety Issue?: Yes
• To further characterize the safety profile of RAD001 (Incidence of AEs)
  • Time Frame: Every 28 days
    Safety Issue?: Yes

Inclusion criteria:

• 1. ≥ 18 years old
• 2. Patients with advanced (unresectable or metastatic) biopsy proven non-syndromic neuro-endocrine carcinoma, low or intermediate grade
• 3. Radiological documentation of disease progression within 12 months prior to study entry. If patients received anti-tumor therapy during the past 12 months, they must have radiological documentation of PD while on or after receiving the therapy
• 4. Patients may have received previous treatments (chemotherapy, biotherapy, peptide-receptor radionuclide therapy); an overall maximum of 3 systemic treatment is allowed
• 5. Patients with at least one measurable lesion
• 6. Patients with an ECOG Performance Status 0-2
• 7. Adequate bone marrow function
• 8. Adequate liver function
• 9. Adequate renal function
• 10. Adequate lipid profile

Exclusion criteria:

• 1. Patients with poorly differentiated neuroendocrine carcinoma, high-grade neuroendocrine carcinoma, adenocarcinoid, goblet cell carcinoid and small cell carcinoma
• 2. Patients with carcinoid with hormone related symptoms (diarrhea ≥ 4 stools per day and/or flushes)
• 3. Patients with Islet cell carcinomas or pancreatic NET
• 4. Patients who received prior therapy with VEGF pathway inhibitor within 4 weeks prior to study entry
• 5. Patients who entered PRRT within 3 months prior to study entry
• 6. Patients who received CT, biotherapy or radiotherapy within 4 weeks prior to study entry
• 7. Patients who have previously received systemic mTOR inhibitors
• 8. Patients with a known hypersensitivity to everolimus or other rapamycins or to its excipients
• 9. Patients with uncontrolled central nervous system (CNS) metastases
• 10. Patients receiving chronic systemic treatment with corticosteroids or another immunosuppressive agent
• 11. Patients with a known history of HIV seropositivity
• 12. Patients with autoimmune hepatitis
• 13. Patients with an active, bleeding diathesis
• 14. Patients who have any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study
• 15. Patients who have a history of another primary malignancy and off treatment ≤ 3 years, with the exception of non-melanoma skin cancer and carcinoma in situ of the uterine cervix
• 16. Female patients who are pregnant or breast feeding, or adults of reproductive potential who are not using effective birth control methods
• 17. Patients who are using other investigational agents or who had received investigational drugs ≤ 4 weeks prior to study treatment start
• 18. Patients unwilling to or unable to comply with the protocol

Gender Eligibility for this Clinical Trial: Both

Minimum Age for this Clinical Trial: 18 Years

Maximum Age for this Clinical Trial: N/A

Are Healthy Volunteers Accepted for this Clinical Trial?: No

Lead Investigator: Novartis Pharmaceuticals Industry

Overall Clinical Trial Officials and Contacts

Novartis Pharmaceuticals Study Director Novartis Pharmaceuticals  

Overall Contact: Novartis Pharmaceuticals +1-800-340-6843 

Information obtained from ClinicalTrials.gov on February 12, 2012

Link to the current ClinicalTrials.gov record. http://clinicaltrials.gov/show/NCT00688623

Study ID Number: CRAD001CDE16

ClinicalTrials.gov Identifier: NCT00688623

Health Authority: Germany: Bundesinstitut für Arzneimittel und Medizinprodukte (BfArM)