Anal High Grade Squamous Intraepithelial LesionPhase 2anal HSIL HIV-NCT05555862
Artesunate Suppositories for the Treatment of HIV-negative Patients With Intra-anal HSIL
A Phase 2 study testing Artesunate in people with Anal High Grade Squamous Intraepithelial Lesion and Anal Precancerous Condition, run by Frantz Viral Therapeutics, LLC at 2 sites in United States.
Trial 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 more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Anal High Grade Squamous Intraepithelial Lesion, Anal Precancerous Condition, AIN 2/3
- What is being tested?
- Artesunate
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 17 participants
- Where
- 2 sitesUnited States
- Study dates
- 10 Feb 2023to 31 Dec 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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
- 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)
Show all 11Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Anal Dysplasia Clinic MidWest
Chicago, Illinois, 60614
Laser Surgery Care
New York, New York, 10011
About this study
Official title: A Phase II Double Blind, Placebo-controlled, Randomized Trial of Artesunate Suppositories for the Treatment of HIV-negative Patients With Anal High-grade Squamous Intraepithelial Lesions (Anal HSIL)
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.
- Conditions
- Anal High Grade Squamous Intraepithelial Lesion, Anal Precancerous Condition, AIN 2/3, HPV Infection, Anal Dysplasia, HPV Disease
- Being tested
- Artesunate, Placebo
- Keywords
- human papillomavirus, non-surgical, topical treatment, anal HSIL, anal cancer prevention, treatment study, cancer prevention, high-risk HPV, HPV16, suppository
- Medical subject headings
- Papillomavirus Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ArtesunateDrug
Also called Artesunic acid, dihydroartemisinin (DHA), artemisinin
artesunate formulated as intra-anal suppositories
PlaceboDrug
placebo intra-anal suppository
| Study group | Role | What they receive |
|---|---|---|
| Artesunate suppositories | Experimental | Four 5-day cycles of artesunate suppositories Receives: Drug: Artesunate |
| Placebo suppositories | Placebo comparator | Four 5-day cycles of placebo suppositories Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 18 weeks
Secondary outcomes (4)
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
Measured: 42 weeks
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)
Measured: 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
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
Measured: 42 weeks
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
Measured: 42 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Sep 2022
- Study start10 Feb 2023
- Primary completion28 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Anal High Grade Squamous Intraepithelial LesionStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Randomised
- Intervention model
- Parallel groups
- Model description
- Double blind, placebo controlled randomized trial
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Frantz Viral Therapeutics, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Amarex CRO, University of California, San Francisco, Anal Dysplasia Clinic MidWest, Laser Surgery Care
- Responsible party
- Sponsor
- Investigators
- Joel Palefsky, MD, Principal investigator, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05555862
- Sponsor's protocol ID
- ART-AIN IIB
- Acronym
- anal HSIL HIV-
- Status last verified
- May 2025
- First submitted
- 22 Sep 2022
- Last updated
- 18 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: No protected health information (PHI) will be shared with other researchers.
Study at a glance
From the registry record, last updated by the sponsor on 18 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Double Blind, Placebo-controlled, Randomized Trial of Artesunate Suppositories for the Treatment of HIV-negative Patients With Anal High-grade Squamous Intraepithelial Lesions (Anal HSIL)
- ClinicalTrials.gov ID
- NCT05555862
- Acronym / sponsor ID
- anal HSIL HIV- / ART-AIN IIB
- 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
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Frantz Viral Therapeutics, LLCIndustry (pharma, biotech, devices)
- Collaborators
- Amarex CRO, University of California, San Francisco, Anal Dysplasia Clinic MidWest, Laser Surgery Care
- Study start
- 10 Feb 2023 (actual)
- Primary completion
- 28 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 27 Sep 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
- Model description
- Double blind, placebo controlled randomized trial
| Arm | Type | Receives |
|---|---|---|
| Artesunate suppositories | Experimental | Drug: ArtesunateFour 5-day cycles of artesunate suppositories |
| Placebo suppositories | Placebo comparator | Drug: PlaceboFour 5-day cycles of placebo suppositories |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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:
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ArtesunateAlso: Artesunic acid, dihydroartemisinin (DHA), artemisininartesunate formulated as intra-anal suppositories | Drug | Artesunate suppositories |
| Placeboplacebo intra-anal suppository | Drug | Placebo suppositories |
Primary outcome
Number of subjects with complete and partial response by week 18
Time frame: 18 weeks
Number of participants who achieve complete or partial response after 4 5-day cycles of artesunate as determined by HRA-directed biopsy(ies)
4 secondary outcomes
Number of participants who have achieved clearance of detectable human papilloma virus (HPV) over the study window
Time frame: 42 weeks
Number of participants with complete and partial response after week 18 but over the study window
Time frame: 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
Time frame: 42 weeks
Number of participants who undergo complete response who maintain their response over the study window
Time frame: 42 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 May 2025.
- Individual participant dataSharing planned: No. No protected health information (PHI) will be shared with other researchers.
Related trials for Anal High Grade Squamous Intraepithelial Lesion
RecruitingPhase 2
CASCADE 2002: PROTECT Study, "PRObiotics to TrEat Anal preCancer Trial
HSIL, High Grade Squamous Intraepithelial Lesion, HPV-Related Squamous Cell Carcinoma
Active, not recruiting
Screening Algorithms for Cervical and Anal High-Grade Squamous Intraepithelial Lesions in People With HIV in Mexico and Puerto Rico
AIDS-Related Anal Carcinoma, AIDS-Related Cervical Carcinoma
Active, not recruitingObservational study
Anal High-risk HPV, HSIL, and Microbiome Among Hispanic Peoples Living With HIV (PLWH)
Human Immunodeficiency Virus, Human Papillomavirus-Related Anal Squamous Cell Carcinoma
RecruitingPhase 1
Safety of Anal Curcumin
Anal High Grade Squamous Intraepithelial Lesion
Not yet recruitingPhase 1
Use of ACU-D1 in HPV Associated Vulvar and Perianal Lesions in People With HIV
Human Papilloma Virus, Human Immunodeficiency Virus
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Joel Palefsky, MD principal investigator
University of California, San Francisco
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Artesunate in people with Anal High Grade Squamous Intraepithelial Lesion and Anal Precancerous Condition, run by Frantz Viral Therapeutics, LLC at 2 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 11 inclusion and 8 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: under way, not looking for participants, as of the sponsor's last update on 18 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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. Some participants in this study may receive a placebo. 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?
This study is still under way. Results are not available in the source record yet.
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?
Number of subjects with complete and partial response by week 18 (time frame: 18 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 18 May 2025.