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.
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
- SECONDARY ID:
R01CA252057
- NCT ID:
NCT04375813
Conditions
- Non-muscle Invasive Bladder Cancer
Interventions
Drug | Synonyms | Arms |
---|
eRapa | encapsulated rapamycin | Active Study Drug Group |
Placebos | | Placebo 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, 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.
Trial Arms
Name | Type | Description | Interventions |
---|
Active Study Drug Group | Active Comparator | Patients will be given 0.5mg eRapa (encapuslated rapamycin) orally each weekday (Monday-Friday) for one year. | |
Placebo Group | Placebo Comparator | Patients will be given a placebo (visually identical to the eRapa (encapsulated rapamycin)) orally each weekday (Monday-Friday) for one year. | |
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
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
July 21, 2021