Artesunate Suppositories for the Treatment of HIV-negative Patients With Intra-anal HSIL

NCT05555862 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: ART-AIN IIB
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 2

Brief Summary

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

Conditions

Anal High Grade Squamous Intraepithelial Lesion; Anal Precancerous Condition; AIN 2/3; HPV Infection; Anal Dysplasia; HPV Disease

Keywords

human papillomavirus; non-surgical; topical treatment; anal HSIL; anal cancer prevention; treatment study; cancer prevention; high-risk HPV; HPV16; suppository

Outcome Measures

Primary Outcomes (1)

• Number of subjects with complete and partial response by week 18

  Number of participants who achieve complete or partial response after 4 5-day cycles of artesunate as determined by HRA-directed biopsy(ies)

  18 weeks

Secondary Outcomes (4)

• Number of participants who have achieved clearance of detectable human papilloma virus (HPV) over the study window

  42 weeks

• Number of participants with complete and partial response after week 18 but over the study window

  30 weeks

• Number of participants who achieve complete and partial response of peri-anal HSIL, following 4 cycles of intra-anal artesunate suppositories, as determined by HRA-directed biopsy

  42 weeks

• Number of participants who undergo complete response who maintain their response over the study window

  42 weeks

Other Outcomes (5)

• Number of participants with Treatment-Emergent Adverse Events (TEAE)

  12 weeks

• Number of participants who withdrew from the study due to TEAEs

  12 weeks

• Changes in vital signs over the study window

  42 weeks

Study Arms (2)

Artesunate suppositories

EXPERIMENTAL

Four 5-day cycles of artesunate suppositories

Drug: Artesunate

Placebo suppositories

PLACEBO COMPARATOR

Four 5-day cycles of placebo suppositories

Drug: Placebo

Interventions

Artesunate DRUG

artesunate formulated as intra-anal suppositories

Also known as: Artesunic acid, dihydroartemisinin (DHA), artemisinin

Artesunate suppositories

Placebo DRUG

placebo intra-anal suppository

Placebo suppositories

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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. Patients must have residual anal HSIL lesions after diagnostic biopsies, as documented by HRA. This includes patients who are newly diagnosed with anal HSIL as well as those who have recurrent anal HSIL after medical therapy or surgical therapy. Patients who have intra-anal HSIL and also have peri-anal HSIL may be enrolled in the study.
• Women of childbearing potential agree to use birth control for the duration of the study.
• Laboratory values at Screening of:
• Serum alanine transaminase (SGPT/ALT) \< 5 x upper limit of normal (ULN)
• Serum aspartate transaminase (SGOT/AST) \< 5 x ULN
• Serum Bilirubin (total) \< 2.5 x ULN
• Serum Creatinine ≤ 1.5 x ULN
• Electrocardiogram (ECG) with no clinically significant findings as assessed by the Investigator.
• Weight ≥ 50kg

You may not qualify if:

• 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)
• Concomitant use of strong Uridine glucuronyl transferases (UGT) inhibitors
• Concomitant use of imiquimod or 5-fluorouracil (5-FU) for the duration of the study

Sponsors & Collaborators

• Frantz Viral Therapeutics, LLC: lead
• Amarex CRO: collaborator
• University of California, San Francisco: collaborator
• Anal Dysplasia Clinic MidWest: collaborator
• Laser Surgery Care: collaborator

Study Sites (2)

Anal Dysplasia Clinic MidWest

Chicago, Illinois, 60614, United States

Location

Laser Surgery Care

New York, New York, 10011, United States

Location

MeSH Terms

Conditions

Papillomavirus Infections

Interventions

Artesunate; artesunic acid; artenimol; artemisinin

Condition Hierarchy (Ancestors)

Sexually Transmitted Diseases, Viral; Sexually Transmitted Diseases; Communicable Diseases; Infections; DNA Virus Infections; Virus Diseases; Tumor Virus Infections; Genital Diseases; Urogenital Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Artemisinins; Reactive Oxygen Species; Free Radicals; Inorganic Chemicals; Organic Chemicals; Sesquiterpenes; Terpenes; Hydrocarbons

Study Officials

• Joel Palefsky, MD

  University of California, San Francisco

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Double-blind, placebo-controlled
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Double blind, placebo controlled randomized trial

Study Record Dates

• First Submitted: September 22, 2022
• First Posted: September 27, 2022
• Study Start: February 10, 2023
• Primary Completion (Estimated): December 28, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: May 18, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)