Trial of Encapsulated Rapamycin (eRapa) for Bladder Cancer Prevention

NCT ID: NCT04375813

Last Updated: 2025-05-15

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 166 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-01-25
• Study Completion Date: 2026-01-31

Brief Summary

eRapa (encapsulated rapamycin) will be investigated for secondary prevention in patients with diagnosed non-muscle invasive bladder cancer (NMIBC) through a phase II double-blind randomized controlled trial of long-term (one year) prevention with eRapa versus placebo. The primary hypothesis is that eRapa decreases the risk of cancer relapse for patients with NMIBC. Secondary hypotheses are that eRapa can improve certain immune parameters and improve cognition and physical function without adversely affecting patient-reported outcomes and quality of life.

Detailed Description

The study is a multi-site phase II double-blind randomized trial. Subjects will be randomized into placebo arm or intervention arm with low dose (0.5 mg) eRapa (encapsulated rapamycin) Monday-Friday for one year or until disease recurrence. Patients will undergo endoscopic evaluation of the bladder every 3 months for 2 years, then every 6 months for 2 years, and at year 5. Some patients may also concurrently receive BCG immune therapy maintenance (weekly for 6 weeks for induction period, weekly for 3 weeks at 3 months, 6 months, and then every 6 months for a total of 7 maintenance cycles following tumor removal) per standard of care. Patient-reported outcome (PRO) assessments, cognitive assessments, and physical assessments will be completed according to the study calendar. Research blood to assess safety, immune response and rapamycin level will be collected regularly throughout the study period. Participants will be followed for up to 5 years following enrollment.

Conditions

Non-muscle Invasive Bladder Cancer

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: PREVENTION
• Blinding Strategy: DOUBLE

Study Groups

Active Study Drug Group

Patients will be given 0.5mg eRapa (encapuslated rapamycin) orally each weekday (Monday-Friday) for one year.

Group Type: ACTIVE_COMPARATOR

eRapa

Intervention Type: DRUG

0.5mg eRapa (encapsulated rapamycin) oral capsules

Placebo Group

Patients will be given a placebo (visually identical to the eRapa (encapsulated rapamycin)) orally each weekday (Monday-Friday) for one year.

Group Type: PLACEBO_COMPARATOR

Placebos

Intervention Type: DRUG

placebo capsules visually identical to eRapa oral capsules

Interventions

eRapa

0.5mg eRapa (encapsulated rapamycin) oral capsules

Intervention Type: DRUG

Placebos

placebo capsules visually identical to eRapa oral capsules

Intervention Type: DRUG

Other Intervention Names

encapsulated rapamycin

Eligibility Criteria

Inclusion Criteria

• Pathologically (histologically) proven diagnosis of non-muscle invasive (Ta, Tis, or T1) bladder cancer within 90 days prior to enrollment
• Able to give informed consent
• 18 years or older
• Patients must not be taking oral glucocorticoids at the time of registration
• Not have active, uncontrolled infections
• No other prior non-bladder malignancy is allowed except for the following: adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, adequately treated Stage I or II cancer from which the patient is currently in complete remission, or any other cancer from which the patient has been disease free for five years.
• Patients with localized prostate cancer who are being followed by an active survelillance program are also eligible.
• Patients must not be pregnant or nursing, as the use of Intravesical BCG is not recommended during pregnancy. Women/ men of reproductive potential must have agreed to use an effective contraceptive method. A woman is considered to be of "reproductive potential" if she has had menses at any time in the preceding 12 consecutive months. Examples of effective contraception include hormonal contraception, double barrier method (condom with spermicidal cream, diaphragms with spermicidal cream, or condoms with diaphragms), Intrauterine device, and/or partner vasectomy. In addition to routine contraceptive methods, "effective contraception" also includes heterosexual celibacy and surgery intended to prevent pregnancy (or with a side-effect of pregnancy prevention) defined as a hysterectomy, bilateral oophorectomy, or bilateral tubal ligation. However, if at any point a previously celibate patient chooses to become heterosexually active during the time period for use of contraceptive measures outlined in the protocol, he/she is responsible for beginning contraceptive measures. Both male and female patients will be required to disclose contraception method during screening and agree to continue to use that contraception method through the end of their participation in the study.
• Patients must have had all grossly visible papillary tumors removed within 90 days prior to registration or cystoscopy confirming no grossly visible papillary tumors within 90 days prior to registration.
• Patients with T1 disease must have cross-sectional imaging of abdomen/pelvis demonstrating no evidence of nodal involvement or metastatic disease (MRI or CT scan) within 90 days prior to registration. Patients with T1 disease must have re-resection confirming ≤ T1 disease within 90 days prior to registration.
• Patients must no have received prior intravesical BCG

Exclusion Criteria

• Have muscle-invasive or higher (≥T2) bladder cancer
• Unable to give informed consent
• Age 17 or younger
• Taking oral glucocorticoids at the time of registration
• Another cancer requiring active treatment (except basal cell carcinoma or squamous cell carcinoma of the skin)
• Patients at risk of pregnancy that are unwilling or unable to take effective contraception during the study period, or patients that are nursing during the study period. Women/ Men of reproductive potential must have agreed to use an effective contraceptive method or will be considered ineligible for study participation.
• Evidence of nodal involvement or metastatic disease (MRI or CT scan) within 90 days prior to registration
• History of prior intravesical BCG
• History of prior Rapamycin treatment

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Cancer Institute (NCI)

NIH

Sponsor Role: collaborator

Robert Svatek

OTHER

Sponsor Role: lead

Responsible Party

Robert Svatek

Professor

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Robert S Svatek, MD, MSC

Role: PRINCIPAL_INVESTIGATOR

UT Health San Antonio

Locations

UT Southwestern Medical Center

Dallas, Texas, United States

Site Status: RECRUITING

UT Health San Antonio

San Antonio, Texas, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Fred Norton, BS

Role: CONTACT

nortonk1@uthscsa.edu

210 450 9630

Robert S Svatek, MD, MSC

Role: CONTACT

weaverb@uthscsa.edu

210 567 5643

Facility Contacts

Donna Mitchell

Role: primary

donna.mitchell@utsouthwestern.edu

214-645-8789

Jessie Williams

Role: backup

jessica.williams2@utsouthwestern.edu

214-648-9195

Fred Norton, BS

Role: primary

nortonk1@uthscsa.edu

210-450-9630

Other Identifiers

R01CA252057

Identifier Type: NIH

Identifier Source: secondary_id

View Link

STUDY00001422

Identifier Type: -

Identifier Source: org_study_id