Recruitment

Recruitment Status
Active, not recruiting
Estimated Enrollment
22

Summary

Conditions
Kidney Cancer
Type
Interventional
Phase
Phase 2
Design
Allocation: N/AIntervention Model: Single Group AssignmentMasking: None (Open Label)Primary Purpose: Treatment

Participation Requirements

Age
Between 18 years and 125 years
Gender
Both males and females

Description

Background: Papillary RCC is the second most common histologic subtype of kidney cancer, accounting for approximately 10-15% of cases Type 1 papillary RCC occurs in both sporadic and hereditary forms, which are histologically identical. Non familial type 1 papillary RCC can present as both solitary ...

Background: Papillary RCC is the second most common histologic subtype of kidney cancer, accounting for approximately 10-15% of cases Type 1 papillary RCC occurs in both sporadic and hereditary forms, which are histologically identical. Non familial type 1 papillary RCC can present as both solitary renal tumors and as bilateral, multifocal disease There are no standard agents of proven efficacy for patients with advanced papillary RCC. Patients with disease localized to the kidney are managed surgically while patients with advanced/unresectable disease are usually managed in the community with VEGF pathway antagonists or mTOR inhibitors. Activating mutations of MET were identified in the germline of affected HPRC patients, who have a predilection for the development of bilateral, multifocal type 1 papillary RCC. Somatic MET mutations have been found in a subset of patients with non-inherited, sporadic papillary renal carcinoma The investigational agent INC280 is a selective MET inhibitor lacking activity against the VEGF pathway This is a proof-of-concept study using INC280 in patients with papillary RCC to test the idea that effectively blocking the HGF/MET pathway will lead to clinical activity in patients with papillary renal cell cancer Objectives: Primary Objective: -To determine the overall response rate (RECIST 1.1) in patients with papillary renal cell carcinoma treated with single agent INC280 Eligibility: Diagnosis of hereditary papillary renal carcinoma (HPRC) or sporadic papillary renal cell carcinoma (RCC) Patients with bilateral multifocal disease can have tumors localized to the kidney or have metastatic disease Patients with sporadic papillary RCC (but without multifocal disease) should have advanced disease that is considered unresectable ECOG 0-2 Measurable disease Adequate organ function No active brain metastases Prior therapy No more than 3 prior lines of systemic therapy Prior therapy with a MET inhibitor is allowed as long as the patient has not had progressive disease while receiving the agent Design: This is a phase 2 single center non-randomized trial. The study will be conducted using a Simon 2 stage minimax design. Initially 13 evaluable subjects will be recruited. If there are no responses to therapy, the study will be terminated. If there is at least 1 response an additional 7 evaluable subjects will be accrued. The two-stage minimax design is based on assuming an ineffective response rate of 5% and a targeted effective response rate of 25%. We also assume that the probability of accepting an ineffective treatment and the probability of rejecting an effective treatment are each 10%. Subjects will be dosed orally at a starting dose of 400 mg twice daily. The overall response rate (complete response + partial response) will be determined.

Tracking Information

NCT #
NCT02019693
Collaborators
Not Provided
Investigators
Principal Investigator: Ramaprasad Srinivasan, M.D. National Cancer Institute (NCI)