Secondary Prevention Trial of Rapamycin in Patients With Resected Non-muscle Invasive Bladder Cancer

NCT03298958 | Withdrawn Phase 3 DMC FDA Drug

• 1 other identifier: HSC20170277H
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The study is a multi-site Phase 3 double-blinded randomized placebo-controlled trial. Subjects are randomly assigned to receive either a placebo or oral Sirolimus: 0.5 mg daily. All subjects will be treated for 2 years or until disease recurrence. Patients will undergo endoscopic evaluation of the bladder every 3 months for the first 2 years following enrollment and then every 6 months for an additional 2 years on study. Selection of BCG immune therapy is at the discretion of the treating urologist but in general is reserved for high-risk patients. Patients concurrently receiving BCG immune therapy will receive standard BCG therapy including induction (weekly for 6 weeks) and maintenance (weekly for 3 weeks at 3 months, 6 months, and then every 6 months following tumor resection).

Conditions

Bladder Cancer

Outcome Measures

Primary Outcomes (1)

• Test the hypothesis that rapamycin 0.5 mg daily increases recurrence-free survival for patients with non-muscle invasive bladder cancer

  Recurrence is defined as histologically-proven bladder cancer (stage CIS, Ta, T1 or ≥T2)

  Patients are treated for 2 years or until disease relapse

Secondary Outcomes (2)

• To determine effects of rapamycin on recurrence-free survival (RFS) for subgroup of patients with non-muscle invasive bladder cancer concurrently receiving BCG immune therapy.

  Patients are treated for 2 years or until disease relapse

• To compare the effects of rapamycin on BCG-specific immunity during treatment of non-muscle invasive bladder cancer with maintenance BCG.

  baseline and 6 months after registration

Study Arms (2)

Placebo Oral Tablet

PLACEBO COMPARATOR

Subject will be randomized to take the Placebo once daily for 2 years or until disease recurrence

Drug: Placebo Oral Tablet

Sirolimus (Rapamycin) 0.5 mg/day for 2 years

ACTIVE COMPARATOR

Subject will be randomized to take Sirolimus (Rapamycin) 0.5mg once daily for 2 years or until disease recurrence

Drug: Rapamycin

Interventions

Rapamycin DRUG

Subject will be randomized to one of the 2 arms

Also known as: Sirolimus

Sirolimus (Rapamycin) 0.5 mg/day for 2 years

Placebo Oral Tablet DRUG

Subject will be randomized to one of the 2 arms

Placebo Oral Tablet

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Pathologically (histologically) proven diagnosis of non-muscle invasive (Ta, Tis or T1) bladder cancer within 60 days prior to enrollment
• Be able to give informed consent
• Be age 18 or older
• Not be in an immunosuppressed state (e.g. HIV, use of chronic steroids)
• Not have active, uncontrolled infections
• Not be on agents known to alter rapamycin metabolism significantly
• Not have a reported history of liver disease (e.g. cirrhosis)
• Not have a prior history of non-bladder cancer unless the cancer is clinically stable and not requiring active treatment except basal cell carcinoma or squamous cell carcinoma of the skin.
• Not pregnant, or taking effective contraception before rapamycin therapy, during therapy and for 12 weeks after discontinuation of therapy.

You may not qualify if:

• Have muscle-invasive (≥T2) bladder cancer
• Unable to give informed consent
• Age \< 18
• Immunosuppressed state (e.g. HIV, use of chronic steroids)
• Active, uncontrolled infections
• On agents known to alter rapamycin metabolism significantly
• Another cancer requiring active treatment (except basal cell carcinoma or squamous cell carcinoma of the skin)
• Patients at risk of pregnancy who are unwilling or unable to take effective contraception before rapamycin therapy, during therapy, and for 12 weeks after discontinuation of therapy.
• Individuals with a reported history of liver disease (e.g. cirrhosis)

Sponsors & Collaborators

• The University of Texas Health Science Center at San Antonio: lead

Study Sites (1)

University of Texas Health Science Center at San Antonio

San Antonio, Texas, 78229, United States

Location

MeSH Terms

Conditions

Urinary Bladder Neoplasms

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Urologic Neoplasms; Urogenital Neoplasms; Neoplasms by Site; Neoplasms; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Urinary Bladder Diseases; Urologic Diseases; Male Urogenital Diseases

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Double-blinded for study staff (investigator) and patient
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: When it is determined that you are eligible for the study, you will be assigned by chance (like flipping a coin) to one of 2 study groups. * Sirolimus 0.5 mg/day for 2 years or until disease recurrence * Placebo

Study Record Dates

• First Submitted: September 26, 2017
• First Posted: October 2, 2017
• Study Start: November 1, 2018
• Primary Completion: February 1, 2022
• Study Completion: May 1, 2022
• Last Updated: June 12, 2019

Record last verified: 2019-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)