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Artesunate in Treating Patients with High-Grade Anal Intraepithelial Neoplasia

Trial Status: Active

This phase I trial studies the side effects and best dose of artesunate in treating patients with high-grade anal intraepithelial neoplasia. Anal intraepithelial neoplasia are precancerous cells that may or may not become a cancer in the future. Most of the changes that lead to cancer are caused by human papillomavirus (HPV). Artesunate may kill cells infected with HPV.

Inclusion Criteria

  • Biopsy-confirmed high-grade anal dysplasia (AIN 2, AIN 3, high grade squamous intraepithelial neoplasia [HSIL]) by high resolution anoscopy (HRA). This includes patients who are newly diagnosed with AIN 2/3 as well as those who have recurrent AIN 2/3 after medical therapy or surgical therapy
  • Female of childbearing potential: negative urine pregnancy test
  • Adequate contraceptive use for males and females of reproductive potential
  • Able to provide informed consent
  • Patients who have the ability to collaborate with planned follow-up (transportation, compliance history, etc.)
  • Patients who have and have not been immunized with the HPV vaccine
  • Weight >= 50 kg

Exclusion Criteria

  • Diagnosis of low-grade anal dysplasia (AIN 1, low-grade squamous intraepithelial neoplasia [LSIL]) by HRA
  • Known anal, vulvar, cervical, or penile cancer
  • CD4 count < 200 at the time of consideration for entry into this study. Patients, whose CD4 counts drop below 200 at any timepoint in this study, will be pulled to the standard of care treatment arm of surgical ablation
  • Unable to provide informed consent
  • Currently receiving systemic chemotherapy or radiation therapy for another cancer
  • Patients who are on medical treatment with systemic immunosuppressants or steroids (e.g., active autoimmune disease)
  • Extensive anal condyloma precludes the ability for the clinician to visualize HSIL during HRA
  • Pregnant female
  • Weight < 50 kg

Maryland

Baltimore
Johns Hopkins University / Sidney Kimmel Cancer Center
Status: ACTIVE
Contact: Sandy Fang
Phone: 410-955-7323

Wisconsin

Madison
University of Wisconsin Hospital and Clinics
Status: ACTIVE
Contact: Evie H. Carchman
Phone: 608-263-1386

PRIMARY OBJECTIVE:

I. To evaluate the safety and tolerability of intra-anal administration of artesunate, administered via suppository with 3 different schedules, in patients with high-grade anal intraepithelial neoplasia (AIN 2/3).

SECONDARY OBJECTIVES:

I. To measure the effect of intra-anal topical artesunate administration determined by pathology, based on the regression of AIN2/3 at study weeks 16, 32, and 40.

II. To evaluate the clearance of HPV as assessed by HPV genotype testing of cytologic specimens.

III. To create a tissue bank for future studies.

OUTLINE: This is a dose-escalation study. Patients are sequentially assigned to 1 of 2 groups.

GROUP I: Patients receive artesunate transanally once daily (QD) on days 1-5. Treatment repeats every 2 weeks for up to 2 cycles in the absence of disease progression or unacceptable toxicity.

GROUP II: Patients receive artesunate transanally QD on days 1-5. Treatment repeats every 2 weeks for up to 3 cycles in the absence of disease progression or unacceptable toxicity.

After completion of study, patients are followed up at 16, 28, and 40 weeks.

Trial Phase Phase I

Trial Type Treatment

Lead Organization
Johns Hopkins University / Sidney Kimmel Cancer Center

Principal Investigator
Sandy Fang

  • Primary ID J16175
  • Secondary IDs NCI-2019-00829, CRMS-65288, IRB00090922
  • Clinicaltrials.gov ID NCT03100045