NCT03047213

Sapanisertib in Treating Patients With Locally Advanced or Metastatic Bladder Cancer With TSC1 and/or TSC2 Mutations

An Open Label, Multicenter, Single Arm Phase II Study to Evaluate the Activity and Tolerability of the Novel mTOR Inhibitor, MLN0128 (TAK-228), in Patients With Locally Advanced or Metastatic Transitional Cell Carcinoma of the Urothelial Tract Whose Tumors Harbor a TSC1 and/or a TSC2 Mutation

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: National Cancer Institute (NCI) · NIH
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This pilot phase II trial studies how well sapanisertib works in treating patients with bladder cancer that has spread from where it started to nearby tissue or lymph nodes (locally advanced) or other places in the body (metastatic) with tuberous sclerosis (TSC)1 and/or TSC2 mutations (changes in deoxyribonucleic acid \[DNA\]). Sapanisertib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Detailed description

PRIMARY OBJECTIVE: I. To determine the overall response rate (ORR) defined as complete response (CR) and partial response (PR) in patients with locally advanced or metastatic transitional cell carcinoma (TCC) harboring a TSC1 mutation. SECONDARY OBJECTIVES: I. To evaluate the safety and tolerability of sapanisertib (MLN0128) (TAK-228) in patients with locally advanced or metastatic TCC harboring a TSC1 or TSC2 mutation. II. To evaluate progression free survival (PFS) and overall survival (OS). EXPLORATORY OBJECTIVES: I. To determine the ORR in patients with locally advanced or metastatic TCC harboring a TSC2 mutation. II. To evaluate toxicity, PFS, and OS in TSC2 mutation patients. OUTLINE: Patients receive sapanisertib orally (PO) once daily (QD) on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up for 4 weeks and every 6 months thereafter.

Conditions

• Locally Advanced Bladder Urothelial Carcinoma
• Metastatic Transitional Cell Carcinoma
• Metastatic Urothelial Carcinoma
• Recurrent Bladder Carcinoma
• Stage III Bladder Urothelial Carcinoma AJCC v6 and v7
• Stage IV Bladder Urothelial Carcinoma AJCC v7

Interventions

Type	Name	Description
DRUG	Sapanisertib	Given PO

Timeline

• Start date: 2017-08-24
• Primary completion: 2020-10-06
• Completion: 2026-11-12
• First posted: 2017-02-08
• Last updated: 2026-04-13
• Results posted: 2025-02-11

Locations

20 sites across 1 country: United States

Regulatory

• FDA-regulated drug study