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Anal Squamous Cell CarcinomaPhase 2NCT02437851

Surgery in Treating Patients With Early Stage Anal Canal or Perianal Cancer and HIV Infection

A Phase 2 study testing Therapeutic Conventional Surgery in people with Anal Squamous Cell Carcinoma and HIV Infection, run by AIDS Malignancy Consortium at 5 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This phase II trial studies surgery in treating patients with anal canal or perianal cancer that is small and has not spread deeply into the tissues and human immunodeficiency virus (HIV) infection. Local surgery may be a safer treatment with fewer side effects than bigger surgery or radiation and chemotherapy. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Therapeutic Conventional Surgery
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
16 participants
Where
5 sitesUnited States
Study dates
Apr 2015to 15 Apr 2026
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • A single, biopsy-proven SISCCA as defined by the LAST criteria (=< 3mm depth of invasion, horizontal spread of =< 7 mm, and completely excised with at least 1 mm margin clear of cancer irrespective of the amount of HSIL) documented per investigator assessment in combination with the pathology report within 12 months before Segment B enrollment.
  • No evidence of any lymph node spread or distant metastases as determined by PET CT imaging within 16 weeks before Segment B enrollment. Alternatively, for those without PET CT capability, an MRI or CT of the abdomen and pelvis and a chest x-ray confirming no evidence of metastatic disease is acceptable.
  • Clinician believes that eradication of concomitant HSIL is reasonable and feasible based on the extent of disease and overall medical condition of the subject
  • HIV-1 infection, as documented by one of the following: 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, Documentation of HIV diagnosis in the medical record by a licensed HIV rapid test health care provider, HIV-1 RNA detection by a licensed HIV-1 RNA assay demonstrating >1000 RNA copies/mL, or Documentation of receipt of ART by a licensed health care provider.
  • Prior to Segment B enrollment, patients on combination anti-retroviral therapy (cART) will be required to have a minimum cluster of differentiation (CD)4 count of >= 200 and patients not on cART will be required to have a minimum CD4 count of >=350 to be eligible for the study; patients not currently on cART who have a CD4 count > 200 and who agree to start cART immediately will be eligible for participation; laboratory data should be obtained within 16 weeks prior to Segment B enrollment
  • Participants must have Eastern Cooperative Oncology Group (ECOG) performance status 0-2
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  • Participants must have a life expectancy of 2 years or more
  • Participants must not have any other concurrent malignancy
  • Participants must be age 18 years old or older.
  • Leukocytes: >= 3,000/mm^3
  • Absolute neutrophil count: >= 1,500/mm^3
  • Platelets: >= 100,000/mm^3
  • Women of childbearing potential must have a negative urine pregnancy test within 7 days prior to randomization enrollment; female participants enrolled in the treatment arm are advised to not become pregnant during study participation; all women of childbearing potential must agree to either commit to continued abstinence from heterosexual intercourse or use a reliable birth control method (oral contraceptive pills, intrauterine device, Nexplanon, DepoProvera, or bilateral tubal ligation, etc., or another acceptable method as determined by the investigator) during the entire period of the trial (5 years or more), and must not intend to become pregnant during study participation and for 3 months after treatment is discontinued if the participant is enrolled in the treatment arm.
  • Men should not father a child while in this study; men who could father a child must agree to use at least one form of birth control or continued abstinence from heterosexual intercourse if receiving topical treatment during during the study and for 2 weeks after stopping topical treatment
  • Participants must be able to understand and willing to sign a written informed consent document
  • Participants must, in the opinion of the Investigator, be capable of complying with the requirements of this protocol

Who cannot join

  • Anal cancer that cannot be completely excised with a >=1 mm clear margin from surrounding tissue or where excision to obtain a clear margin would compromise sphincter function or anal canal diameter
  • Concurrent anal canal or perianal HSIL or condyloma that in the judgment of the clinician cannot be cleared or can only be cleared with undue morbidity to the patient
  • No prior history of anal cancer, including SISCCA
  • Ongoing use of anticoagulant therapy other than aspirin or non-steroidal anti-inflammatory drugs (NSAIDS) that cannot be stopped for surgical procedures
  • Acute treatment for an infection (excluding fungal infection of the skin and sexually transmitted infections) or other serious medical illness within 2 weeks before enrollment
  • Current systemic chemotherapy or radiation therapy that potentially causes bone marrow suppression that would preclude safe treatment of HSIL; Note: Kaposi's sarcoma limited to the skin is not exclusionary unless requiring systemic chemotherapy
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  • The participant's SISCCA must not have been ablated.
  • Participants who are receiving any other investigational agents within 4 weeks prior to enrollment. Investigational antiretroviral agents for HIV are acceptable.
  • Participant plans to relocate away from the study site during study participation.

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

United States5 sites

  • Grady Health System

    Atlanta, Georgia, 30303

  • Boston Medical Center

    Boston, Massachusetts, 02118

  • Laser Surgery Care

    New York, New York, 10011

  • University of California, San Francisco

    San Francisco, California, 94115

  • Montefiore Medical Center

    The Bronx, New York, 10461

Other trials in United States

About this study

Official title: A Multicenter Observational and Feasibility Study of Excision of Superficially Invasive Squamous Cell Carcinoma (SISCCA) of the Anal Canal and Perianus in HIV-Infected Persons

This phase II trial studies surgery in treating patients with anal canal or perianal cancer that is small and has not spread deeply into the tissues and human immunodeficiency virus (HIV) infection. Local surgery may be a safer treatment with fewer side effects than bigger surgery or radiation and chemotherapy.

Read the detailed description

PRIMARY OBJECTIVES:

  1. To determine the proportion of participants who develop treatment failure by 3 years is less than 25%, defined as the occurrence of distant or any nodal metastases or recurrence of cancer requiring chemotherapy (CMT), defined as a cancer that no longer meets the definition of superficially invasive squamous cell carcinoma (SISCCA) or a cancer that cannot be excised with a clear margin or preservation of sphincter function, or those who develop SISCCA recurrence but elect to undergo CMT rather than repeat excision in patients originally treated with excision of anal canal and perianal SISCCA.

II. To define the 1-year proportion of participants who develop incident anal squamous cancers at sites other than the location of the index SISCCA in patients treated with excision of anal canal and perianal SISCCA.

SECONDARY OBJECTIVES:

  1. To determine morbidities associated with local excision of SISCCA and treatment of concomitant HSIL, including non-healing ulcer, fissure, persistent pain and bleeding, stricture, incontinence, and colostomy at 3 years after enrollment.

EXPLORATORY OBJECTIVES:

  1. To determine the human papillomavirus (HPV) type in cancer and compare to that of overlying high-grade squamous intraepithelial lesions (HSIL) and HSIL biopsies collected concurrently that did not progress to cancer.

II. To determine and compare the HPV integration site in the anal cancer as well as in HSIL overlying or contiguous with the cancer and HSIL biopsies collected concurrently that did not progress to cancer.

III. Perform gene expression array analysis comparing expression in anal cancer with HSIL overlying or contiguous with the cancer.

IV. Perform gene expression array analysis comparing expression in HSIL biopsies that progressed to cancer with non-progressing HSIL biopsies at other locations.

  1. Characterize genetic changes in anal cancers compared with HSIL overlying or contiguous with the cancer.

VI. Characterize genetic changes in HSIL biopsies that progressed to cancer compared with non-progressing HSIL biopsies at other locations.

VII. Perform gene expression array analysis and characterize genetic changes of SISCCAs that were cured with wide local excision for comparison with SISCCAs that progressed after wide local excision.

OUTLINE:

Patients undergo surgery to remove anal or perianal cancer. Any HSIL remaining is treated with the goal for complete eradication in accordance with clinician and participant preference.

After completion of study treatment, patients are followed up every 3 months for 36 months.

Medical subject headings
Anus Neoplasms, HIV Infections, Anal Canal Carcinoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT02437851
Study groupRoleWhat they receive
Treatment (surgery)ExperimentalParticipants undergo therapeutic conventional surgery to remove anal or perianal cancer (SISCCA). Any HSIL remaining is treated with the goal for complete eradication in accordance with clinician and participant preference.
Receives: Procedure: Therapeutic Conventional Surgery

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. To determine if the proportion of participants who develop treatment failure by 3 years is less than 25%.

    Treatment failure is specifically defined as the occurrence of distant or any nodal metastases or recurrence of cancer that no longer meets the definition of SISCCA and that cannot be excised with a clear margin or preservation of sphincter function and requires CMT, or those who develop SISCCA recurrence but elect to undergo CMT rather than repeat excision in patients originally treated with excision of anal canal and perianal SISCCA.

    Measured: 3 years after surgery to remove SISCCA

Secondary outcomes (2)

  1. The cumulative proportion of study participants who have experienced treatment failure by 3 years will be estimated using the product-limit estimate and its 95% confidence interval using Greenwood's formula.

    To determine morbidities associated with local excision of SISCCA and treatment of concomitant HSIL

    Measured: 3 years after surgery to remove SISCCA

  2. The rate of treatment related adverse events including non-healing ulcer, fissure, persistent pain and bleeding, stricture, incontinence, and colostomy 6 months after excision of SISCCA.

    To determine morbidities associated with local excision of SISCCA and treatment of concomitant HSIL

    Measured: 6 months after surgery to remove SISCCA

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 May 2015 (estimated)
  2. Study startApr 2015
  3. Primary completion15 Apr 2026
  4. Study completion15 Apr 2026

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
Surgical removal of SISCCA with quarterly monitoring for recurrence and treatment of anal high-grade squamous intraepithelial lesions.
Masking
None (open label)
Enrolment
16 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02437851
Sponsor's protocol ID
AMC-092
Other IDs
NCI-2014-02056, AMC-092, AMC-092, U01CA121947
Status last verified
Apr 2026
First submitted
14 Jan 2015
Last updated
20 Apr 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided

Publications

  • Fuchshuber PR, Rodriguez-Bigas M, Weber T, Petrelli NJ. Anal canal and perianal epidermoid cancers. J Am Coll Surg. 1997 Nov;185(5):494-505. doi: 10.1016/s1072-7515(97)00094-x.

    PubMed 9358099

  • Steele SR, Varma MG, Melton GB, Ross HM, Rafferty JF, Buie WD; Standards Practice Task Force of the American Society of Colon and Rectal Surgeons. Practice parameters for anal squamous neoplasms. Dis Colon Rectum. 2012 Jul;55(7):735-49. doi: 10.1097/DCR.0b013e318255815e. No abstract available.

    PubMed 22706125

  • Benson AB, Venook AP, Al-Hawary MM, Cederquist L, Chen YJ, Ciombor KK, Cohen S, Cooper HS, Deming D, Engstrom PF, Garrido-Laguna I, Grem JL, Grothey A, Hochster HS, Hoffe S, Hunt S, Kamel A, Kirilcuk N, Krishnamurthi S, Messersmith WA, Meyerhardt J, Miller ED, Mulcahy MF, Murphy JD, Nurkin S, Saltz L, Sharma S, Shibata D, Skibber JM, Sofocleous CT, Stoffel EM, Stotsky-Himelfarb E, Willett CG, Wuthrick E, Gregory KM, Freedman-Cass DA. NCCN Guidelines Insights: Colon Cancer, Version 2.2018. J Natl Compr Canc Netw. 2018 Apr;16(4):359-369. doi: 10.6004/jnccn.2018.0021.

    PubMed 29632055

  • Leonard D, Beddy D, Dozois EJ. Neoplasms of anal canal and perianal skin. Clin Colon Rectal Surg. 2011 Mar;24(1):54-63. doi: 10.1055/s-0031-1272824.

    PubMed 22379406

  • Oehler-Janne C, Huguet F, Provencher S, Seifert B, Negretti L, Riener MO, Bonet M, Allal AS, Ciernik IF. HIV-specific differences in outcome of squamous cell carcinoma of the anal canal: a multicentric cohort study of HIV-positive patients receiving highly active antiretroviral therapy. J Clin Oncol. 2008 May 20;26(15):2550-7. doi: 10.1200/JCO.2007.15.2348. Epub 2008 Apr 21.

    PubMed 18427149

  • Wexler A, Berson AM, Goldstone SE, Waltzman R, Penzer J, Maisonet OG, McDermott B, Rescigno J. Invasive anal squamous-cell carcinoma in the HIV-positive patient: outcome in the era of highly active antiretroviral therapy. Dis Colon Rectum. 2008 Jan;51(1):73-81. doi: 10.1007/s10350-007-9154-7. Epub 2007 Dec 8.

    PubMed 18066626

  • Darragh TM, Colgan TJ, Cox JT, Heller DS, Henry MR, Luff RD, McCalmont T, Nayar R, Palefsky JM, Stoler MH, Wilkinson EJ, Zaino RJ, Wilbur DC; Members of LAST Project Work Groups. The Lower Anogenital Squamous Terminology Standardization Project for HPV-Associated Lesions: background and consensus recommendations from the College of American Pathologists and the American Society for Colposcopy and Cervical Pathology. J Low Genit Tract Dis. 2012 Jul;16(3):205-42. doi: 10.1097/LGT.0b013e31825c31dd.

    PubMed 22820980

  • Beahrs OH, Wilson SM. Carcinoma of the anus. Ann Surg. 1976 Oct;184(4):422-8. doi: 10.1097/00000658-197610000-00004.

    PubMed 189707

  • Klas JV, Rothenberger DA, Wong WD, Madoff RD. Malignant tumors of the anal canal: the spectrum of disease, treatment, and outcomes. Cancer. 1999 Apr 15;85(8):1686-93. doi: 10.1002/(sici)1097-0142(19990415)85:83.0.co;2-7.

    PubMed 10223561

  • Chang GJ, Berry JM, Jay N, Palefsky JM, Welton ML. Surgical treatment of high-grade anal squamous intraepithelial lesions: a prospective study. Dis Colon Rectum. 2002 Apr;45(4):453-8. doi: 10.1007/s10350-004-6219-8.

    PubMed 12006924

  • Pineda CE, Berry JM, Jay N, Palefsky JM, Welton ML. High-resolution anoscopy targeted surgical destruction of anal high-grade squamous intraepithelial lesions: a ten-year experience. Dis Colon Rectum. 2008 Jun;51(6):829-35; discussion 835-7. doi: 10.1007/s10350-008-9233-4. Epub 2008 Mar 25.

    PubMed 18363070

  • Zilli T, Schick U, Ozsahin M, Gervaz P, Roth AD, Allal AS. Node-negative T1-T2 anal cancer: radiotherapy alone or concomitant chemoradiotherapy? Radiother Oncol. 2012 Jan;102(1):62-7. doi: 10.1016/j.radonc.2011.09.015. Epub 2011 Oct 10.

    PubMed 21993403

  • Ortholan C, Ramaioli A, Peiffert D, Lusinchi A, Romestaing P, Chauveinc L, Touboul E, Peignaux K, Bruna A, de La Roche G, Lagrange JL, Alzieu C, Gerard JP. Anal canal carcinoma: early-stage tumors < or =10 mm (T1 or Tis): therapeutic options and original pattern of local failure after radiotherapy. Int J Radiat Oncol Biol Phys. 2005 Jun 1;62(2):479-85. doi: 10.1016/j.ijrobp.2004.09.060.

    PubMed 15890590

  • Ortholan C, Resbeut M, Hannoun-Levi JM, Teissier E, Gerard JP, Ronchin P, Zaccariotto A, Minsat M, Benezery K, Francois E, Salem N, Ellis S, Azria D, Champetier C, Gross E, Cowen D. Anal canal cancer: management of inguinal nodes and benefit of prophylactic inguinal irradiation (CORS-03 Study). Int J Radiat Oncol Biol Phys. 2012 Apr 1;82(5):1988-95. doi: 10.1016/j.ijrobp.2011.02.010. Epub 2011 May 11.

    PubMed 21570207

  • Goldstone SE, Hundert JS, Huyett JW. Infrared coagulator ablation of high-grade anal squamous intraepithelial lesions in HIV-negative males who have sex with males. Dis Colon Rectum. 2007 May;50(5):565-75. doi: 10.1007/s10350-006-0874-x.

    PubMed 17380365

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Therapeutic Conventional Surgery in people with Anal Squamous Cell Carcinoma and HIV Infection, run by AIDS Malignancy Consortium at 5 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 16 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 20 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

To determine if the proportion of participants who develop treatment failure by 3 years is less than 25%. (time frame: 3 years after surgery to remove SISCCA). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02437851, which also lists 50 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Apr 2026.

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