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Clinical Trials/NCT04444921
NCT04444921
Active, Not Recruiting
Phase 3

A Randomized Phase III Study of Immune Checkpoint Inhibition With Chemotherapy in Treatment-Naive Metastatic Anal Cancer Patients

National Cancer Institute (NCI)1136 sites in 1 country205 target enrollmentNovember 17, 2020

Overview

Phase
Phase 3
Intervention
Paclitaxel
Conditions
Anal Basaloid Carcinoma
Sponsor
National Cancer Institute (NCI)
Enrollment
205
Locations
1136
Primary Endpoint
Progression-free survival (PFS)
Status
Active, Not Recruiting
Last Updated
yesterday

Overview

Brief Summary

This phase 3 trial compares the addition of nivolumab to chemotherapy (carboplatin and paclitaxel) versus usual treatment (chemotherapy alone) for the treatment of anal cancer that has spread to other places in the body (metastatic). Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Chemotherapy drugs, such as carboplatin and paclitaxel, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving nivolumab together with carboplatin and paclitaxel may help doctors find out if the treatment is better or the same as the usual approach.

Detailed Description

PRIMARY OBJECTIVE: I. To demonstrate that anti-PD-1 therapy in combination with carboplatin/weekly paclitaxel results in improved progression-free survival (PFS) versus systemic chemotherapy alone. SECONDARY OBJECTIVES: I. To demonstrate that anti-PD-1 therapy in combination with carboplatin/weekly paclitaxel results in improved overall survival (OS) versus systemic chemotherapy alone. II. To demonstrate that anti-PD-1 therapy in combination with carboplatin/weekly paclitaxel results in improved objective response using Response Evaluation Criteria in Solid Tumors (RECIST) version (v)1.1 versus systemic chemotherapy alone. III. To evaluate toxicity profiles of the two regimens. IV. To elucidate the role of the human papillomavirus (HPV) circulating cell-free deoxyribonucleic acid (cfDNA) viral load on PFS with the hypothesis that longer PFS will be associated with viral load. OUTLINE: Patients are randomized to 1 of 2 arms. Randomization will be 2:1 favoring the experimental regimen, Arm B. ARM A: Patients receive paclitaxel intravenously (IV) on days 1, 8, and 15 of each cycle, and carboplatin IV on day 1 of each cycle. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity. ARM B: Patients receive nivolumab IV over 30 minutes on days 1 and 15 of cycle 1 and then on day 1 only of subsequent cycles, paclitaxel IV on days 1, 8, and 15 of each cycle, and carboplatin on day 1 of each cycle. Treatment repeats every 28 days for up to 6 cycles for carboplatin and paclitaxel, and up to 2 years for nivolumab in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up at 1 month, then every 3 months for 2 years.

Registry
clinicaltrials.gov
Start Date
November 17, 2020
End Date
March 31, 2027
Last Updated
yesterday
Study Type
Interventional
Study Design
Parallel
Sex
All

Investigators

Responsible Party
Sponsor

Eligibility Criteria

Inclusion Criteria

  • Patient must have inoperable, recurrent, or metastatic disease not amenable to curative therapy
  • Patient must have histological or cytological confirmation of anal squamous cell carcinoma (includes basaloid and cloacogenic lesions) from the primary tumor or a newly diagnosed recurrent/metastatic lesion
  • Patient must be \>= 18 years of age
  • Patient must have Eastern Cooperative Oncology Group (ECOG) performance status =\< 0-1
  • Patients must have measurable disease according to Response Evaluation Criteria in Solid Tumors (RECIST) criteria version 1.1 and based on radiologic assessment performed \< 4 weeks prior to randomization
  • Patient receiving palliative (limited-field) radiation therapy is allowed, as long as the lesion treated for palliation is not a target lesion and is \> 7 days from completion from palliative radiation
  • Patients with brain metastasis are eligible if patient is asymptomatic and if treatment ended \> 3 months prior to randomization. Patients with treated brain metastases are eligible if follow-up brain imaging after central nervous system (CNS)-directed therapy shows no evidence of progression within 4 weeks prior to randomization
  • Patient must not be pregnant or breast-feeding due to the potential harm to an unborn fetus and possible risk for adverse events in nursing infants with the treatment regimens being used. All patients of childbearing potential must have a blood test or urine study with a minimum sensitivity of 25 IU/L or equivalent units of Bacille Calmette-Guerin (BCG), within 14 days prior to randomization to rule out pregnancy. A patient of childbearing potential is defined as anyone, regardless of whether they have undergone tubal ligation, who meets the following criteria: 1) has achieved menarche at some point, 2) has not undergone a hysterectomy or bilateral oophorectomy; or 2) has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months).
  • Patients of childbearing potential must not expect to conceive children by using accepted and effective method(s) of contraception or to abstain from sexual intercourse for at least one week prior to the start of treatment, and continue for 5 months after the last dose of protocol treatment
  • Absolute neutrophil count \>= 1,500/mcL (obtained \< 14 days prior to randomization)

Exclusion Criteria

  • Not provided

Arms & Interventions

Arm A (carboplatin, paclitaxel)

Patients receive paclitaxel IV on days 1, 8, and 15 of each cycle, and carboplatin IV on day 1 of each cycle. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity.

Intervention: Paclitaxel

Arm A (carboplatin, paclitaxel)

Patients receive paclitaxel IV on days 1, 8, and 15 of each cycle, and carboplatin IV on day 1 of each cycle. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity.

Intervention: Carboplatin

Arm B (carboplatin, paclitaxel, nivolumab)

Patients receive nivolumab IV over 30 minutes on days 1 and 15 of cycle 1 and then on day 1 only of subsequent cycles, paclitaxel IV on days 1, 8, and 15 of each cycle, and carboplatin on day 1 of each cycle. Treatment repeats every 28 days for up to 6 cycles for carboplatin and paclitaxel, and up to 2 years for nivolumab in the absence of disease progression or unacceptable toxicity.

Intervention: Nivolumab

Arm B (carboplatin, paclitaxel, nivolumab)

Patients receive nivolumab IV over 30 minutes on days 1 and 15 of cycle 1 and then on day 1 only of subsequent cycles, paclitaxel IV on days 1, 8, and 15 of each cycle, and carboplatin on day 1 of each cycle. Treatment repeats every 28 days for up to 6 cycles for carboplatin and paclitaxel, and up to 2 years for nivolumab in the absence of disease progression or unacceptable toxicity.

Intervention: Paclitaxel

Arm B (carboplatin, paclitaxel, nivolumab)

Patients receive nivolumab IV over 30 minutes on days 1 and 15 of cycle 1 and then on day 1 only of subsequent cycles, paclitaxel IV on days 1, 8, and 15 of each cycle, and carboplatin on day 1 of each cycle. Treatment repeats every 28 days for up to 6 cycles for carboplatin and paclitaxel, and up to 2 years for nivolumab in the absence of disease progression or unacceptable toxicity.

Intervention: Carboplatin

Outcomes

Primary Outcomes

Progression-free survival (PFS)

Time Frame: Up to 2 years

Defined as the first of progressive disease or death due to any cause. Analyzed using a stratified two-sided overall 0.05 level log-rank test. Will utilize standard Eastern Cooperative Oncology Group -American College of Radiology Imaging Network interim monitoring for efficacy evaluation.

Secondary Outcomes

  • Objective response rate (ORR)(Up to 2 years)
  • Overall survival(Time between treatment randomization and death by any cause, assessed up to 2 years)
  • Incidence of adverse events(Up to 2 years)
  • Association of PFS with human papillomavirus (HPV) circulating cell-free deoxyribonucleic acid (DNA) viral load(Up to 2 years)

Study Sites (1136)

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