Clinical trial NCT06206564: Artesunate Ointment for the Treatment of Anal HSIL in HIV-negative Participants
Official title: A Phase II Double Blind, Placebo-controlled, Randomized Trial of Artesunate Ointment for the Treatment of HIV-negative Patients With Anal High-grade Squamous Intraepithelial Lesions (Anal HSIL)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Anal High-grade Squamous Intraepithelial Lesion
Anal HSIL
Anal HPV Infection
AIN
Keywords: intra-anal
topical
non-surgical
Lead sponsor: Frantz Viral Therapeutics, LLC
Sponsor class: INDUSTRY
Collaborators: name: Laser Surgery Care, LLC
name: Anal Dysplasia Clinic MidWest
First posted: 2024-01-16
Enrollment: 48
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Double blind, placebo-controlled randomized trial
Primary purpose: TREATMENT

Brief summary: This is a phase II double blind, placebo-controlled, randomized study of artesunate ointment for the treatment of HIV-negative men and women who have anal high grade squamous intraepithelial lesions (anal HSIL)

Detailed description: Eligible participants in this study are randomized 2:1 to receive either artesunate or placebo ointment for the treatment of anal HSIL. Both groups receive four 5-day cycles of ointment, at weeks 0, 2, 4, and 6. Participants are followed closely with anoscopy or high resolution anoscopy (HRA) at weeks 8, 18, 30, and 42. After the week 18 HRA visit, participants who have at least partial response will be followed with HRA at week 30.

Participants who are found to be non-responders at week 18 will undergo standard of care ablation.

Participants who are found to have anal HSIL at weeks 30 or 42 will be followed according to standard of care procedures.

Primary Objective: To evaluate the complete and partial histopathologic response to four 5-day cycles of artesunate ointment in adult patients with biopsy-proven HPV-associated intra-anal HSIL (18 weeks).

Secondary Objectives:

Efficacy:

To evaluate the viral clearance after four 5-day cycles of artesunate ointment To evaluate complete and partial intra-anal response To evaluate complete and partial peri-anal response To evaluate persistence of response throughout the study window

Safety:

To evaluate the safety of artesunate ointment for the treatment of intra-anal HSIL.

Eligibility criteria: Inclusion Criteria:

* Adult men and women age ≥ 18 years
* Capable of informed consent
* Biopsy-confirmed intra-anal high-grade dysplasia (AIN 2, AIN 3, anal HSIL) by HRA.
* Positive anal human papillomavirus (HPV) test.
* Women of childbearing potential agree to use birth control for the duration of the study.
* Laboratory values at Screening of:

  1. Serum alanine transaminase (SGPT/ALT) \< 5 x upper limit of normal (ULN)
  2. Serum aspartate transaminase (SGOT/AST) \< 5 x ULN
  3. Serum Bilirubin (total) \< 2.5 x ULN
  4. Serum Creatinine ≤ 1.5 x ULN
* Electrocardiogram (ECG) with no clinically significant findings as assessed by the Investigator.
* Weight ≥ 50kg

Exclusion Criteria:

* Pregnant and nursing women
* Diagnosis of low-grade anal dysplasia (AIN 1, LSIL), without the concomitant diagnosis of anal HSIL, by HRA
* Concurrent anal, vulvar, cervical, or penile cancer
* HIV-seropositivity
* 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). Participants using nasal steroid treatments for allergic conditions may participate in the study.
* Concomitant use of strong UGT (Uridine-diphosphate-glucuronosyltransferase) inhibitors
* Concomitant use of imiquimod or 5-fluorouracil (5-FU) for the duration of the study

Primary outcomes:
measure: Complete and partial response by week 18
timeFrame: 18 weeks
Secondary outcomes:
measure: HPV clearance
timeFrame: 42 weeks
measure: Complete and partial response after week 18
timeFrame: 30 weeks
measure: Complete and partial peri-anal response after intra-anal ointment application
timeFrame: 42 weeks
measure: Persistence of response
timeFrame: 42 weeks
Other outcomes:
measure: Safety of intra-anal artesunate ointment
timeFrame: 42 weeks
Locations:
city: Chicago
country: United States
facility: Anal Dysplasia Clinic MidWest
state: Illinois
city: New York
country: United States
facility: Laser Surgery Care
state: New York
Study officials:
References: