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BRIEF TITLE: Encapsulated Rapamycin (eRapa) for Bladder Cancer Prevention

Phase II Trial of Encapsulated Rapamycin (eRapa) for Bladder Cancer Prevention


  • Org Study ID: ER-B01
  • Secondary ID: R01CA252057
  • NCT ID: NCT04375813
  • NCT Alias:
  • Sponsor: Rapamycin Holdings, Inc. dba Emtora Biosciences - Industry
  • Source: Rapamycin Holdings Inc.

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.

Overal Status Start Date Phase Study Type
Recruiting January 25, 2021 Phase 2 Interventional

Primary Outcomes:

Primary Outcome 1 - Measure: Determine 1-year Recurrence Free Survival (RFS) rate

Primary Outcome 1 - Time Frame: 1 year

Primary Outcome 2 - Measure: Change in Urinary Quality of Life

Primary Outcome 2 - Time Frame: Scored at baseline and months 3, 6, 12 and 24. For any subject who has a recurrence after enrollment, but prior to completing a full year of dosing at Visit 6, this will also be measured at the End of Treatment visit.

Primary Outcome 3 - Measure: Change in Cognitive Function

Primary Outcome 3 - Time Frame: EXIT will be scored at baseline and on months 12 and 24.For any subject who has a recurrence after enrollment, but prior to completing a full year of dosing at Visit 6, this will also be measured at the End of Treatment visit.

Primary Outcome 4 - Measure: Change in Cognitive Function

Primary Outcome 4 - Time Frame: SLUMS will be scored at baseline and on months 12 and 24.For any subject who has a recurrence after enrollment, but prior to completing a full year of dosing at Visit 6, this will also be measured at the End of Treatment visit.

Primary Outcome 5 - Measure: Change in Cognitive Function

Primary Outcome 5 - Time Frame: TAPS will be scored at baseline and on months 6, 12, and 24. For any subject who has a recurrence after enrollment, but prior to completing a full year of dosing at Visit 6, this will also be measured at the End of Treatment visit.

Primary Outcome 6 - Measure: Change in cytoscopy consistent with recurrence (time to recurrence)

Primary Outcome 6 - Time Frame: Patients will undergo office-based cystoscopy with urine cytology every 3 months for the first 2 years, then every 6 months for 2 years, and at year 5, with biopsies per standard of care if necessary.

Primary Outcome 7 - Measure: Change in Short Physical Performance Battery

Primary Outcome 7 - Time Frame: SPPB will be scored at baseline and on months 6, 12, and 24. For any subject who has a recurrence after enrollment, but prior to completing a full year of dosing at Visit 6, this will also be measured at the End of Treatment visit.

Primary Outcome 8 - Measure: Change in Handgrip Strength

Primary Outcome 8 - Time Frame: Handgrip Strength will be scored at baseline and on months 6, 12, and 24. For any subject who has a recurrence after enrollment, but prior to completing a full year of dosing at Visit 6, this will also be measured at the End of Treatment visit.

Condition:

  • Non-muscle Invasive Bladder Cancer

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
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Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: No

Official Information

Name: Robert S Svatek, MD, MSCI

Role: Principal Investigator

Affiliation: UT Health San Antonio

Overall Contact

Name: Stephanie Althoff

Phone: 210-722-4936

Email: salthoff@cancerinsight.com

Locations

Facility Status Contact
UT Southwestern Medical Center
Dallas, Texas 75390
United States
Recruiting Donna Mitchell
214-645-8789
donna.mitchell@utsouthwestern.edu
South Texas Veterans Health Care System, Audie L. Murphy Veterans Hospital
San Antonio, Texas 78229
United States
Not yet recruiting Allison Wheeler, MS
210-567-1172
wheelera2@uthscsa.edu
UT Health San Antonio
San Antonio, Texas 78229
United States
Recruiting Kerri Kendrick
210-450-9600
Kendrick@uthscsa.edu