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Active, not recruitingNCT05555862anal HSIL HIV-Updated May 18, 2025

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

A Phase 2 interventional study of Artesunate and Placebo in Anal High Grade Squamous Intraepithelial Lesion, Anal Precancerous Condition and AIN 2/3, sponsored by Frantz Viral Therapeutics, LLC. Active, not recruiting at 2 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-05-18.

Sponsored by Frantz Viral Therapeutics, LLC · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
17
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study 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)

Read the detailed description

Eligible participants in this study are randomized 2:1 to receive either artesunate or placebo suppositories for the treatment of anal HSIL. Both groups receive four 5-day cycles of suppositories, at weeks 0, 2, 4, and 6. At least two of the dosing visits are done in person. The remaining dosing visits may be done as telehealth visits (suppositories are mailed to the patient's home). 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 suppositories 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 suppositories in adults with biopsy-proven HPV-associated intra-anal anal HSIL over the study window (42 weeks)
  • To evaluate complete and partial intra-anal histopathologic response after the week 18 time point but over the study window (30 weeks).
  • To evaluate complete and partial peri-anal histopathologic response following artesunate suppositories over the course of the study (42 weeks).
  • To evaluate persistence of response throughout the study window (42 weeks)

Safety:

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

02

Conditions studied

  • 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
03

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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. 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:

    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

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)
  • Concomitant use of strong Uridine glucuronyl transferases (UGT) inhibitors
  • Concomitant use of imiquimod or 5-fluorouracil (5-FU) for the duration of the study
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Double (Participant, Investigator)
Enrollment
17 participants (actual)

Study arms

  • Experimental
    Artesunate suppositories

    Four 5-day cycles of artesunate suppositories

    Drug: Artesunate

  • Placebo comparator
    Placebo suppositories

    Four 5-day cycles of placebo suppositories

    Drug: Placebo

Interventions

  • DrugArtesunate

    artesunate formulated as intra-anal suppositories

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

  • DrugPlacebo

    placebo intra-anal suppository

05

What researchers measure

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)

    Time frame: 18 weeks

Secondary outcomes

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

    Number of participants who had HPV strains detected at study entry which become undetectable within the study window

    Time frame: 42 weeks

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

    Number of participants who achieve complete or partial response after week 18, as documented by HRA-directed biopsy(ies)

    Time frame: 30 weeks

  3. 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

    Number of participants who had both peri-anal and intra-anal HSIL, whose peri-anal HSIL achieves complete or partial response following 4 cycles of intra-anal artesunate suppositories

    Time frame: 42 weeks

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

    Number of participants who complete or partial response who maintain this response over the study window

    Time frame: 42 weeks

Other outcomes

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

    Number of participants who had adverse events, as defined by CTCAE v5.0, related to the study intervention

    Time frame: 12 weeks

  2. Number of participants who withdrew from the study due to TEAEs

    Number of participants who were withdrawn from the study due to adverse events related to the study drug

    Time frame: 12 weeks

  3. Changes in vital signs over the study window

    Number of participants who had clinically significant changes in vital signs

    Time frame: 42 weeks

  4. Changes in physical examination abnormalities

    Number of participants who had clinically significant changes in physical examination abnormalities

    Time frame: 42 weeks

  5. Clinically significant changes in ECG between baseline and post dosing

    Number of participants who had clinical significant changes in ECG between pre-dosing and post dosing

    Time frame: 8 weeks

06

Study locations

2 sites
  • Anal Dysplasia Clinic MidWest
    Chicago, Illinois 60614, United States
  • Laser Surgery Care
    New York, New York 10011, United States
07

References and documents

Individual participant data

Plan to share: No — No protected health information (PHI) will be shared with other researchers.

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT05555862
Lead sponsor
Frantz Viral Therapeutics, LLC
Collaborators
Amarex CRO, University of California, San Francisco, Anal Dysplasia Clinic MidWest, Laser Surgery Care
Responsible party
Sponsor
First posted
Sep 27, 2022
Start date
Feb 10, 2023
Primary completion
Dec 28, 2026 (estimated)
Completion
Dec 31, 2026 (estimated)
Last update
May 18, 2025

Study contacts

Joel Palefsky, MD
principal investigator · University of California, San Francisco

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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