NCT04929028

Brief Summary

This phase II trial studies the side effects of chemotherapy and intensity modulated radiation therapy in treating patients with low-risk HIV-associated anal cancer, and nivolumab after standard of care chemotherapy and radiation therapy in treating patients with high-risk HIV-associated anal cancer. Radiation therapy uses high energy x-rays to kill tumor cells and shrink tumors. Chemotherapy drugs, such as mitomycin, fluorouracil, and capecitabine, 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 chemotherapy with radiation therapy may kill more tumor cells. 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. Giving nivolumab after standard of care chemotherapy and radiation therapy may help reduce the risk of the tumor coming back.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
40

participants targeted

Target at P25-P50 for phase_2

Timeline
64mo left

Started Aug 2022

Longer than P75 for phase_2

Geographic Reach
1 country

14 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress42%
Aug 2022Sep 2031

First Submitted

Initial submission to the registry

June 16, 2021

Completed
2 days until next milestone

First Posted

Study publicly available on registry

June 18, 2021

Completed
1.1 years until next milestone

Study Start

First participant enrolled

August 9, 2022

Completed
9.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 15, 2031

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 15, 2031

Last Updated

May 13, 2026

Status Verified

April 1, 2026

Enrollment Period

9.1 years

First QC Date

June 16, 2021

Last Update Submit

May 12, 2026

Conditions

Outcome Measures

Primary Outcomes (2)

  • Incidence of adverse events (Low-risk stratum)

    Evaluated by Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. The adverse events will be summarized by overall, as well as by grade using frequency (percentage). A two-sided 95% confidence interval will be reported together with percentage estimates. The proportion of participants who experience grade 3-4 toxicities will be estimated using a binomial point estimate and its 95% confidence interval.

    Up to 5 years

  • Incidence of adverse events (High-risk stratum)

    Evaluated by CTCAE version 5.0. The adverse events will be summarized by overall, as well as by grade using frequency (percentage). A two-sided 95% confidence interval will be reported together with percentage estimates. The proportion of participants who experience grade 3-4 toxicities will be estimated using a binomial point estimate and its 95% confidence interval.

    Up to 5 years

Secondary Outcomes (5)

  • Disease free survival (High-risk stratum)

    Time from enrollment until progression of local disease, distant metastasis, secondary primary cancer or death, assessed at 2 years

  • Disease control rate (Low-risk stratum)

    Time from enrollment until first recurrence (locoregional or distant metastasis) or chemo-radiation-related death, assessed up to 5 years

  • Change in CD4+ cell counts (High-risk stratum)

    Baseline up to 5 years

  • Change in human immunodeficiency virus (HIV) viral load (Low-risk stratum)

    Baseline up to 5 years

  • Change in combination antiretroviral therapy (cART) adherence

    Baseline up to 5 years

Other Outcomes (5)

  • Relationship between specific human papillomavirus (HPV) subtypes and clinical response to reduced intensity chemo-radiation therapy (CRT) or nivolumab

    Up to 5 years

  • Relationship between expression of PD-1 in immune cells and PD-L1 in immune cells or cancer epithelial cells in the primary diagnostic tumor and clinical response to nivolumab or reduced intensity CRT

    Up to 5 years

  • Effect of reduced intensity CRT and nivolumab on viral HIV reservoirs

    Up to 5 years

  • +2 more other outcomes

Study Arms (2)

High-risk stratum (nivolumab)

EXPERIMENTAL

Patients receive nivolumab IV over 30 minutes on day 1. Treatment repeats every 4 weeks for up to 6 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo ECHO during screening as clinically indicated, sigmoidoscopy/colonoscopy, anoscopy/proctoscopy or digital rectal exam and CT throughout the study as well as blood sample collection during screening and EOT.

Procedure: AnoscopyProcedure: Biospecimen CollectionProcedure: ColonoscopyProcedure: Computed TomographyProcedure: Digital Rectal ExaminationProcedure: Echocardiography TestBiological: NivolumabProcedure: ProctoscopyOther: Questionnaire AdministrationProcedure: Sigmoidoscopy

Low-risk stratum (mitomycin

EXPERIMENTAL

Patients receive mitomycin IV on day 1 and either fluorouracil IV on day 1 or capecitabine PO BID on Monday-Friday until the completion of radiation therapy at the discretion of the treating physician. Patients also undergo IMRT QD for 20-23 treatment sessions over 6 weeks. Patients also undergo digital rectal exam, anoscopy/proctoscopy and CT throughout the study, receive FDG IV and undergo PET/CT, PET/MRI and /or MRI during screening and follow-up as well as blood sample collection during screening and EOT. Some patients undergo lymph node biopsy during screening at the discretion of the treating physician.

Procedure: AnoscopyProcedure: Biospecimen CollectionDrug: CapecitabineProcedure: ColonoscopyProcedure: Computed TomographyProcedure: Digital Rectal ExaminationProcedure: Echocardiography TestOther: Fludeoxyglucose F-18Drug: FluorouracilRadiation: Intensity-Modulated Radiation TherapyProcedure: Lymph Node BiopsyProcedure: Magnetic Resonance ImagingDrug: MitomycinProcedure: Positron Emission TomographyProcedure: ProctoscopyOther: Questionnaire AdministrationProcedure: Sigmoidoscopy

Interventions

AnoscopyPROCEDURE

Undergo anoscopy

High-risk stratum (nivolumab)Low-risk stratum (mitomycin

Undergo blood sample collection

Also known as: Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection
High-risk stratum (nivolumab)Low-risk stratum (mitomycin

Undergo FDG PET/CT or PET/MRI

Also known as: Medical Imaging, Positron Emission Tomography, PET, PET Scan, Positron emission tomography (procedure), Positron Emission Tomography Scan, Positron-Emission Tomography, PT
Low-risk stratum (mitomycin

Ancillary studies

High-risk stratum (nivolumab)Low-risk stratum (mitomycin
SigmoidoscopyPROCEDURE

Undergo sigmoidoscopy

Also known as: Proctosigmoidoscopy
High-risk stratum (nivolumab)Low-risk stratum (mitomycin

Undergo lymph node biopsy

Also known as: Biopsy of Lymph Node
Low-risk stratum (mitomycin

Undergo MRI or PET/MRI

Also known as: Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR, MR Imaging, MRI, MRI Scan, MRIs, NMR Imaging, NMRI, Nuclear Magnetic Resonance Imaging, sMRI, Structural MRI
Low-risk stratum (mitomycin

Given IV

Also known as: Ametycine, Jelmyto, MITO, Mito-C, Mito-Medac, Mitocin, Mitocin-C, Mitolem, Mitomycin C, Mitomycin pyelocalyceal, Mitomycin-C, Mitomycin-X, Mitomycine C, Mitosol, Mitozytrex, Mutamycin, Mutamycine, NCI-C04706
Low-risk stratum (mitomycin

Given PO

Also known as: Ro 09-1978/000, Xeloda
Low-risk stratum (mitomycin
ColonoscopyPROCEDURE

Undergo colonoscopy

High-risk stratum (nivolumab)Low-risk stratum (mitomycin

Undergo CT or FDG PET/CT

Also known as: CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography, Computerized axial tomography (procedure), Computerized Tomography, Computerized Tomography (CT) scan, CT, CT Scan, Diagnostic CAT Scan, Diagnostic CAT Scan Service Type, tomography
High-risk stratum (nivolumab)Low-risk stratum (mitomycin

Undergo digital rectal exam

Also known as: DRE
High-risk stratum (nivolumab)Low-risk stratum (mitomycin

Undergo ECHO

Also known as: EC, Echocardiography
High-risk stratum (nivolumab)Low-risk stratum (mitomycin

Receive FDG

Also known as: 18FDG, FDG, Fludeoxyglucose (18F), fludeoxyglucose F 18, Fludeoxyglucose F18, Fluorine-18 2-Fluoro-2-deoxy-D-Glucose, Fluorodeoxyglucose F18
Low-risk stratum (mitomycin

Given IV

Also known as: 5 Fluorouracil, 5 Fluorouracilum, 5 FU, 5-Fluoro-2,4(1H, 3H)-pyrimidinedione, 5-Fluorouracil, 5-Fluracil, 5-Fu, 5FU, AccuSite, Carac, Fluoro Uracil, Fluouracil, Flurablastin, Fluracedyl, Fluracil, Fluril, Fluroblastin, Ribofluor, Ro 2-9757, Ro-2-9757
Low-risk stratum (mitomycin

Undergo IMRT

Also known as: IMRT, Intensity modulated radiation therapy (procedure), Intensity Modulated RT, Intensity-Modulated Radiotherapy, Radiation, Intensity-Modulated Radiotherapy
Low-risk stratum (mitomycin
NivolumabBIOLOGICAL

Given IV

Also known as: ABP 206, BCD-263, BMS 936558, BMS-936558, BMS936558, CMAB819, MDX 1106, MDX-1106, MDX1106, NIVO, Nivolumab Biosimilar ABP 206, Nivolumab Biosimilar BCD-263, Nivolumab Biosimilar CMAB819, ONO 4538, ONO-4538, ONO4538, Opdivo
High-risk stratum (nivolumab)
ProctoscopyPROCEDURE

Undergo proctoscopy

High-risk stratum (nivolumab)Low-risk stratum (mitomycin

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • HIGH-RISK STRATUM: Participant is able to understand and willing to sign a written informed consent document
  • HIGH-RISK STRATUM: Participant must have histologically proven stage (T3-T4N0M0 OR T2-4N1M0) invasive squamous cell carcinoma (SCC) of the anus or anorectum as documented before CRT initiation, according to the American Joint Committee on Cancer (AJCC) 8th edition. Participants with squamous cell carcinoma of the anal margin are eligible if there is evidence of extension of the primary tumor into the anal canal. Participants with tumors of non-keratinizing histology such as basaloid, transitional cell or cloacogenic histology are permitted
  • HIGH-RISK STRATUM: HIV-positive. Documentation of HIV-1 infection by means of any one of the following:
  • Documentation of HIV diagnosis in the medical record by a licensed health care provider. If the record contains information that the patient is taking Food and Drug Administration (FDA)-approved combination therapy for HIV infection, then this can be part of the record substantiating the HIV positive diagnosis
  • HIV-1 ribonucleic acid (RNA) detection by a licensed HIV-1 RNA assay demonstrating \> 1000 RNA copies/mL
  • Any licensed HIV screening antibody and/or HIV antibody/antigen combination assay confirmed by a second licensed HIV assay such as a HIV-1 Western blot confirmation or HIV rapid multispot antibody differentiation assay.
  • NOTE: The term "licensed" refers to a kit that has been certified or licensed by an oversight body within the participating country and validated internally (e.g., United States \[U.S.\] FDA)
  • WHO (World Health Organization) and CDC (Centers for Disease Control and Prevention) guidelines mandate that confirmation of the initial test result must use a test that is different from the one used for the initial assessment. A reactive initial rapid test must be confirmed by either another type of rapid assay or an E/CIA that is based on a different antigen preparation and/or different test principle (e.g., indirect versus competitive), or a Western blot or a plasma HIV-1 RNA viral load
  • HIGH-RISK STRATUM: Age \>= 18 years
  • Because no dosing or adverse event data are currently available on the use of nivolumab in participants \< 18 years of age, children are excluded from this study
  • HIGH-RISK STRATUM: Eastern Cooperative Oncology Group (ECOG) performance status =\< 2 (Karnofsky \>= 50%)
  • HIGH-RISK STRATUM: Life expectancy of greater than 6 months
  • HIGH-RISK STRATUM: Hemoglobin \> 10 g/dL (within 2 weeks before enrollment)
  • HIGH-RISK STRATUM: Absolute neutrophil count: \>= 1,500/mm\^3 (within 2 weeks before enrollment)
  • HIGH-RISK STRATUM: Platelets: \>= 100,000/mm\^3 (within 2 weeks before enrollment)
  • +54 more criteria

You may not qualify if:

  • HIGH-RISK STRATUM: Any live vaccines within 30 days prior to enrollment
  • Examples of live vaccines include, but are not limited to, the following: measles, mumps, rubella, chicken pox, yellow fever, rabies, Bacillus Calmette-Guerin (BCG), and typhoid (oral) vaccine. Seasonal influenza vaccines for injection are generally killed virus vaccines and are allowed; however, intranasal influenza vaccines (e.g., Flu-Mist) are live attenuated vaccines and are not allowed
  • NOTE: No live vaccines may be administered while participating in the trial.
  • HIGH-RISK STRATUM: Participant has known interstitial lung disease that is symptomatic or may interfere with the detection or management of suspected drug-related pulmonary toxicity
  • HIGH-RISK STRATUM: Prior treatment with an immune checkpoint inhibitor (anti-PD-1, anti-PD-L1, anti-PD-L2, anti-CTLA4 monoclonal antibody)
  • HIGH-RISK STRATUM: Participant with an allogenic bone marrow/stem, cell or solid organ transplant
  • HIGH-RISK STRATUM: Participant is receiving any other investigational agents
  • HIGH-RISK STRATUM: History of allergic reactions attributed to compounds of similar chemical or biologic composition to nivolumab or other agents used in study
  • HIGH-RISK STRATUM: Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia or psychiatric illness/social situations that would limit compliance with study requirements
  • HIGH-RISK STRATUM: Participant has a history of a different malignancy, unless he/she have been disease-free for at least 2 years and are deemed by the investigator to be at low risk of recurrence
  • NOTE: Individuals with the following cancers are eligible if diagnosed and treated within the past 5 years: cervical cancer in situ, basal cell or squamous cell carcinoma of the skin, and stage I and IIA/IIB resected melanoma. In addition, participants on hormonal treatment for breast/gynecological and prostate tumors with no evidence of active disease are permitted, as well as participants with controlled Kaposi sarcoma (KS) not requiring systemic KS directed therapy
  • HIGH-RISK STRATUM: Pregnant or breastfeeding.
  • Pregnant women are excluded from this study because nivolumab is an anti-PD-1MAb agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with nivolumab, breastfeeding should be discontinued if the mother is treated with nivolumab
  • All FOCBP must have a blood test or urine study within 2 weeks prior to enrollment to rule out pregnancy
  • HIGH-RISK STRATUM: Participant has not recovered from adverse events due to CRT (i.e., have residual toxicity \> grade 1), excluding alopecia
  • +13 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (14)

Zuckerberg San Francisco General Hospital

San Francisco, California, 94110, United States

Location

George Washington University Medical Center

Washington D.C., District of Columbia, 20037, United States

Location

Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location

University of Illinois

Chicago, Illinois, 60612, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Mount Sinai West

New York, New York, 10019, United States

Location

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

Mount Sinai Hospital

New York, New York, 10029, United States

Location

Montefiore Medical Center-Einstein Campus

The Bronx, New York, 10461, United States

Location

Montefiore Medical Center - Moses Campus

The Bronx, New York, 10467, United States

Location

Pennsylvania Hospital

Philadelphia, Pennsylvania, 19107, United States

Location

Lyndon Baines Johnson General Hospital

Houston, Texas, 77026-1967, United States

Location

M D Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Virginia Mason Medical Center

Seattle, Washington, 98101, United States

Location

MeSH Terms

Conditions

Anus NeoplasmsHIV InfectionsRectal Neoplasms

Interventions

ProctoscopySpecimen HandlingCapecitabineColonoscopyFluorodeoxyglucose F18FluorouracildehydroftorafurRadiotherapy, Intensity-ModulatedSentinel Lymph Node BiopsyMagnetic Resonance SpectroscopyMitomycinMitozytrexNivolumabSigmoidoscopy

Condition Hierarchy (Ancestors)

Colorectal NeoplasmsIntestinal NeoplasmsGastrointestinal NeoplasmsDigestive System NeoplasmsNeoplasms by SiteNeoplasmsDigestive System DiseasesGastrointestinal DiseasesIntestinal DiseasesAnus DiseasesRectal DiseasesBlood-Borne InfectionsCommunicable DiseasesInfectionsSexually Transmitted Diseases, ViralSexually Transmitted DiseasesLentivirus InfectionsRetroviridae InfectionsRNA Virus InfectionsVirus DiseasesGenital DiseasesUrogenital DiseasesImmunologic Deficiency SyndromesImmune System Diseases

Intervention Hierarchy (Ancestors)

Endoscopy, GastrointestinalEndoscopy, Digestive SystemDiagnostic Techniques, Digestive SystemDiagnostic Techniques and ProceduresDiagnosisEndoscopyDiagnostic Techniques, SurgicalDigestive System Surgical ProceduresSurgical Procedures, OperativeMinimally Invasive Surgical ProceduresClinical Laboratory TechniquesInvestigative TechniquesDeoxycytidineCytidinePyrimidine NucleosidesPyrimidinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsUracilPyrimidinonesDeoxyribonucleosidesNucleosidesNucleic Acids, Nucleotides, and NucleosidesDeoxyglucoseDeoxy SugarsCarbohydratesRadiotherapy, ConformalRadiotherapy, Computer-AssistedRadiotherapyTherapeuticsBiopsyCytodiagnosisCytological TechniquesLymph Node ExcisionSpectrum AnalysisChemistry Techniques, AnalyticalMitomycinsIndolequinonesQuinonesOrganic ChemicalsAzirinesIndolesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingAntibodies, Monoclonal, HumanizedAntibodies, MonoclonalAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulins

Study Officials

  • Rafi Kabarriti

    AIDS Malignancy Consortium

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 16, 2021

First Posted

June 18, 2021

Study Start

August 9, 2022

Primary Completion (Estimated)

September 15, 2031

Study Completion (Estimated)

September 15, 2031

Last Updated

May 13, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will share

NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page

More information

Locations