CClinicalTrials.gg
TerminatedNCT03498196Updated Aug 24, 2020Results posted

A Window of Opportunity Trial: Avelumab in Non-metastatic Muscle Invasive Bladder Cancer

A Phase 1/2 interventional study of Avelumab in Bladder Cancer, Metastatic Bladder Cancer and Invasive Bladder Cancer, sponsored by Baylor College of Medicine. Terminated at 3 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2020-08-24.

Sponsored by Baylor College of Medicine · Phase 1/2, Interventional, and Treatment

Why this study was terminated
The sponsor of this study has terminated the study due to poor enrollment.
Phase
Phase 1/2
Study type
Interventional
Enrollment
1
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Study summary

This is a pilot study of avelumab in patients with non-metastatic, muscle invasive bladder cancer who are eligible for radical cystectomy (RC), but are ineligible for cisplatin based neoadjuvant therapy. The target recruitment is 10 evaluable patients for this window of opportunity study. Pre- and post-treatment tumor samples from transurethral resection of the bladder tumor and RC will be used for study endpoints.

Read the detailed description

Avelumab is a fully human monoclonal PD-L1 antibody of the immunoglobulin G1 (IgG1) subclass. It works by binding to PD-L1 on tumor cells, immune cells and/or stromal cells. This prevents PD-L1 from interacting with PD-1. Inhibition of this interaction increases activation/survival of antitumor lymphocytes. It also increases innate immunity by resulting in decreased PD-1 suppression of NK cell function and bolsters antibody production by B cells due to less PD-L1 binding of PD-1 on B-cells. Additionally, avelumab has been suggested to have another mechanism involving antibody dependent cellular cytotoxicity (ADCC). ADCC in these cases involves NK cell recognition and lysis of tumor cells that have antibody bound to PD-L1. By blocking PD-L1, avelumab leads to less CD80 binding by PD-L1 and more CD80-CD28 binding in response to antigen presentation to T-cells. This results in increased costimulatory signaling and is another mechanism by which avelumab may enhance T-cell activation.

Avelumab has been shown to be efficacious across multiple metastatic tumor types, including urothelial cancer. The phase Ib study has reported survival and safety outcomes with >12 months followup using pooled data on 249 patients with metastatic UC (Apolo et al, ESMO Sept 2017). Patients had been treated with a median of 2 prior therapies in the metastatic setting and 13 patients who were cisplatin-ineligible were evaluated for safety alone. PD-L1 expression was not a criterion for enrollment. The confirmed objective response rate (ORR) was 16.1%, with 5% complete responses and 11.2% partial responses. The 6-month progression-free survival was 27%. The ORR was better than or comparable to chemotherapy in historical controls. Among responders, 70.3% were maintained > 12 months. Treatment-related adverse events (AE) occurred in 70%, with 10.7% of the total with AE's of grade >3. Immune-mediated AE's occurred in 18.5%, of which 4% were grade >3. There is currently a phase III clinical trial ongoing comparing avelumab to standard of care chemotherapy in the second line setting or beyond for metastatic UC. Two other checkpoint inhibitors have been approved in the last 2 years for first line treatment of patients with metastatic UC who are ineligible for cisplatin-based therapy.

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Conditions studied

  • Bladder Cancer
  • Metastatic Bladder Cancer
  • Invasive Bladder Cancer

Keywords

  • muscle invasive bladder cancer
  • non-muscle invasive bladder cancer
  • radical cystectomy
  • monoclonal antibodies
  • Avelumab
  • Invasive Resectable disease
  • MIBC
  • NMIBC
  • neoadjuvant chemotherapy
03

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Have undergone TURBT showing newly diagnosed muscle invasive UCB (mixed histology is allowed if the predominant histology is UCC) within 6 weeks prior to cycle 1, day 1 of treatment.
  • No prior systemic treatment for muscle invasive UCB
  • Clinical T2-T4a disease
  • No evidence of clinically positive lymph nodes or distant metastasis on computed tomography (CT) scans of chest and CT or magnetic resonance imaging (MRI) studies of the abdomen/pelvis. Imaging must be within 90 days of registration.
  • Male or female subjects aged ≥ 18 years old.
  • Must have adequate kidney, liver, and bone marrow function within 30 days of registration, as follows:
  • Absolute neutrophil count (ANC) ≥ 1.5 × 109/L
  • platelet count ≥ 100 × 109/L
  • hemoglobin ≥ 9 g/dL (may have been transfused)
  • Total bilirubin level ≤ 1.5 × ULN
  • AST and ALT levels ≤ 2.5 × ULN
  • Estimated creatinine clearance ≥ 30 mL/min according to the Cockcroft-Gault formula
  • Negative serum or urine pregnancy test at screening for women of childbearing potential (WOCBP), within 30 days of registration.
  • Both male and female subjects must agree to use highly effective contraception (see Section 6.1 Table 8) while receiving avelumab and for at least 60 days after last avelumab treatment if the risk of conception exists.

Female patients must agree to inform study coordinator or investigator immediately if they think they have become pregnant during the study.

  • Must have FFPE tissue available from the TURBT, and patient must consent to the use of tissue specimens from TURBT and RC for the study.
  • Patients must be ineligible for cisplatin-based NAC. Ineligibility criteria include: creatinine clearance \< 60 ml/min by Cockcroft-Gault formula, CTCAE grade ≥ 2 hearing loss, CTCAE grade ≥ 2 neuropathy, and at discretion of medical oncologist.
  • Must be eligible for RC in the opinion of the treating investigator, and willing to undergo this procedure.
  • ECOG performance status (PS) score of 0-2
  • Signed informed consent form.

Exclusion criteria

Exclusion Criteria:

Patients must not have any of the following:

  • IMMUNOSUPRESSANTS: Current use of immunosuppressive medication or within 4 weeks of C1D1, EXCEPT for the following: a. intranasal, inhaled, topical steroids, or local steroid injection (e.g., intra-articular injection); b. Systemic corticosteroids at physiologic doses ≤ 10 mg/day of prednisone or equivalent; c. Steroids as premedication for hypersensitivity reactions (e.g., CT scan premedication).
  • AUTOIMMUNE DISEASE: Active autoimmune disease that might deteriorate when receiving an immuno-stimulatory agent. Patients with diabetes type I, vitiligo, psoriasis, or hypo- or hyperthyroid diseases not requiring immunosuppressive treatment are eligible. Patients with type I diabetes or hypo- or hyperthyroidism should be on stable doses of medications for participation.
  • ORGAN TRANSPLANTATION: Prior organ transplantation including allogenic stem-cell transplantation.
  • INFECTIONS: Active infection requiring systemic therapy.
  • HIV/AIDS: Known history of testing positive for HIV or known acquired immunodeficiency syndrome.
  • HEPATITIS: Hepatitis B virus (HBV) or hepatitis C virus (HCV) infection at screening (positive HBV surface antigen or HCV RNA if anti-HCV antibody screening test positive)
  • VACCINATION: Vaccinate within 4 weeks of the first dose of avelumab and while on study drug is prohibited except for administration of inactivated vaccines
  • HYPERSENSITIIVTY TO STUDY DRUG: Known prior severe hypersensitivity to investigational product or any component in its formulations, including known severe hypersensitivity reactions to monoclonal antibodies (NCI CTCAE v4.03 Grade ≥ 3)
  • CARDIOVASCULAR DISEASE: Clinically significant (i.e., active) cardiovascular disease: cerebral vascular accident/stroke (\< 6 months prior to enrollment), myocardial infarction (\< 6 months prior to enrollment), unstable angina, congestive heart failure (≥ New York Heart Association Classification Class II), or serious cardiac arrhythmia requiring medication.
  • Other severe acute or chronic medical conditions including colitis, inflammatory bowel disease, pneumonitis, pulmonary fibrosis or psychiatric conditions including recent (within the past year) or active suicidal ideation or behavior; or laboratory abnormalities that may increase the risk associated with study participation or study treatment administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the patient inappropriate for entry into this study.
  • Relapsed MIBC (all patients participating in the study should be newly diagnosed)
  • Concomitant UCC outside the bladder (e.g., ureter, urethra or renal pelvis)
  • Underlying immune disorder (e.g., combined variable immunodeficiency syndrome)
  • Erythropoietin receptor agonists within 30 days prior to enrollment.
  • G-CSF, GM-CSF or TPO mimetics during the study period or within 3 weeks prior to study enrollment
  • Malignancies other than UCB within 5 years prior to Cycle 1, Day 1, with the exception of those with low risk of metastasis or death treated with expected curative outcome (such as, but not limited to, adequately treated carcinoma in situ of the cervix, basal or squamous cell skin cancer, localized prostate cancer treated with curative intent and absence of PSA relapse, or ductal carcinoma in situ of the breast treated surgically with curative intent) or incidental prostate cancer (T1a, Gleason score ≤ 6 and PSA \< 0.5 ng/ml)
  • Prior immunotherapy with T-cell co-stimulation or checkpoint targeted agents (e.g., CTLA-4 inhibitors, anti-PD1 antibodies or anti-PD-L1 antibodies)
  • Intravesical chemotherapy or biologic therapy within 6 weeks of Cycle 1, Day 1
  • Current participation in another clinical trial for MIBC
  • Nursing or pregnant woman
  • Uncontrolled cystitis, significant bladder pain or spasms, or gross hematuria that in the opinion of the principal investigator will preclude study participation
  • Major surgical procedures within 4 weeks of registration (other than for diagnosis) or anticipation that such a procedure will be needed during the study (other than RC)
04

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
1 participant (actual)

Study arms

  • Experimental
    Avelumab

    Avelumab 10 mg/kg intravenously, over 60 minutes every 2 weeks for 3 cycles or 42 days.

    Drug: Avelumab

Interventions

  • DrugAvelumab

    avelumab 10 mg/kg intravenously every 2 weeks for 3 cycles or 42 days.

    Also known as: MSB0010718C

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What researchers measure

Primary outcomes

  1. Change in T Cell Subpopulations

    The change in T cell subpopulations (CD8, CD4 and/or CD3) in tumor samples will be collected from FFPE tissue. FFPE tissue from the pre-study time point refers to tissue from the TURBT. FFPE tissue from the 2-3 week post-op time point refers to FFPE tissue from the RC

    Time frame: pre-study time and 2-3 week post-operation

Secondary outcomes

  1. Pathological Response Rate

    Number of patient with pathological response will be counted

    Time frame: pre-study time and 2-3 week post-operation

  2. 2 Year Disease Free Survival (DFS)

    A patient will be followed for recurrence and survival for up to 2 years after radical cystectomy.

    Time frame: 2 years after radical cystectomy

  3. Rate of High Grade(Grade 3-4) Adverse Event

    High grade adverse events are defined as grade of 3-4 on the CTCAE (Common terminology criteria for adverse events) grading scale.

    Time frame: 90 days post operation

06

Results

Posted Aug 24, 2020

Participant flow

Participant flow — Overall Study
MilestoneAvelumab
Started1
Completed1
Not completed0

Outcome measures

PrimaryChange in T Cell Subpopulations

The change in T cell subpopulations (CD8, CD4 and/or CD3) in tumor samples will be collected from FFPE tissue. FFPE tissue from the pre-study time point refers to tissue from the TURBT. FFPE tissue from the 2-3 week post-op time point refers to FFPE tissue from the RC

Time frame:
pre-study time and 2-3 week post-operation

No measurements were reported for this outcome.

SecondaryPathological Response Rate

Number of patient with pathological response will be counted

Time frame:
pre-study time and 2-3 week post-operation
Reported as:
Count of participants · Participants
Pathological Response Rate
ParticipantsAvelumab
Pathological Response Rate0
Secondary2 Year Disease Free Survival (DFS)

A patient will be followed for recurrence and survival for up to 2 years after radical cystectomy.

Time frame:
2 years after radical cystectomy
Reported as:
Number · months
2 Year Disease Free Survival (DFS)
monthsAvelumab
2 Year Disease Free Survival (DFS)5.48
SecondaryRate of High Grade(Grade 3-4) Adverse Event

High grade adverse events are defined as grade of 3-4 on the CTCAE (Common terminology criteria for adverse events) grading scale.

Time frame:
90 days post operation
Reported as:
Count of participants · Participants
Rate of High Grade(Grade 3-4) Adverse Event
ParticipantsAvelumab
Rate of High Grade(Grade 3-4) Adverse Event1

Adverse events

Collected over 90 days after radical cystectomy (RC) or end of the last cycle of Avelumab for those not receiving RC.. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Avelumab1/1 (100%)1/1 (100%)1/1 (100%)
Most frequent serious events
Most frequent serious events
EventAvelumab
SepsisInfections and infestations1/1
Urinary tract obstructionRenal and urinary disorders1/1
Most frequent other events
Most frequent other events
EventAvelumab
AnemiaBlood and lymphatic system disorders1/1
Creatinine increasedInvestigations1/1
Renal and urinary disorders - Other, specifyRenal and urinary disorders1/1

Baseline characteristics

Age, Continuous
Age, Continuous(years)Avelumab
Mean58 ± NA
Sex: Female, Male
Sex: Female, Male(Participants)Avelumab
Female0
Male1
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Avelumab
Hispanic or Latino0
Not Hispanic or Latino1
Unknown or Not Reported0
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Avelumab
American Indian or Alaska Native0
Asian0
Native Hawaiian or Other Pacific Islander0
Black or African American1
White0
More than one race0
Unknown or Not Reported0
Region of Enrollment
Region of Enrollment(participants)Avelumab
United States1
Current Smoking
Current Smoking(Participants)Avelumab
Count of participants1
07

Study locations

3 sites
  • Baylor College of Medicine Medical Center - McNair Campus
    Houston, Texas 77030, United States
  • Ben Taub General Hospital
    Houston, Texas 77030, United States
  • Michael E. DeBakey Veteran Affairs Medical Center
    Houston, Texas 77030, United States
08

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Study documents

  • Study protocol · Dec 11, 2017
  • Statistical analysis plan · Feb 23, 2018

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Undecided

09

Registry details

Key details

Study ID
NCT03498196
Lead sponsor
Baylor College of Medicine
Collaborators
Pfizer
Responsible party
Jennifer Taylor (Assistant Professor of Urology, Baylor College of Medicine) — Principal investigator
First posted
Apr 13, 2018
Start date
Dec 14, 2018
Primary completion
Dec 9, 2019
Completion
Dec 9, 2019
Results posted
Aug 24, 2020
Last update
Aug 24, 2020

Study contacts

Jennifer M. Taylor, MD
principal investigator · Baylor College of Medicine

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is terminated, as verified in Aug 2020. You cannot join it, but the record below documents what was studied.

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