Clinical Trials /

Trial of Encapsulated Rapamycin (eRapa) for Bladder Cancer Prevention

NCT04375813

Description:

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.

Related Conditions:
  • Bladder Carcinoma
  • Prostate Carcinoma
Recruiting Status:

Recruiting

Phase:

Phase 2

Trial Eligibility

Document

Title

  • Brief Title: Trial of Encapsulated Rapamycin (eRapa) for Bladder Cancer Prevention
  • Official Title: Phase II Trial of Encapsulated Rapamycin (eRapa) for Bladder Cancer Prevention

Clinical Trial IDs

  • ORG STUDY ID: ER-B01
  • NCT ID: NCT04375813

Conditions

  • Non-muscle Invasive Bladder Cancer

Interventions

DrugSynonymsArms
eRapaencapsulated rapamycinActive Study Drug Group
PlacebosPlacebo Group

Purpose

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, then
      annually following registration. 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 4 years
      following enrollment.
    

Trial Arms

NameTypeDescriptionInterventions
Active Study Drug GroupActive ComparatorPatients will be given 0.5mg eRapa (encapuslated rapamycin) orally each weekday (Monday-Friday) for one year.
  • eRapa
Placebo GroupPlacebo ComparatorPatients will be given a placebo (visually identical to the eRapa (encapsulated rapamycin)) orally each weekday (Monday-Friday) for one year.
  • Placebos

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.
             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.

          -  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
      
Maximum Eligible Age:N/A
Minimum Eligible Age:18 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Determine 1-year Recurrence Free Survival (RFS) rate
Time Frame:1 year
Safety Issue:
Description:The primary analysis will be the estimation of the RFS rate with the 95% confidence interval and comparison of the rate to the historical rate of 68% using the single arm log-rank test (one-sided alpha = 0.05). If the observed rate of RFS is 80% the expected number of events is about 20-28. Recurrence confirmed by pathologic examination of biopsied tissue.

Details

Phase:Phase 2
Primary Purpose:Interventional
Overall Status:Recruiting
Lead Sponsor:Rapamycin Holdings, Inc. dba Emtora Biosciences

Last Updated

June 12, 2020