Clinical trial NCT03100045: Intra-Anally Administered Artesunate in Patients With High-Grade Anal Intraepithelial Neoplasia (AIN 2/3)
Official title: A Phase I Study of Intra-Anally Administered Artesunate in Patients With High-Grade Anal Intraepithelial Neoplasia (AIN 2/3)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: HPV-Related Anal Intraepithelial Neoplasia
AIN2/3
Artesunate
Alternative Treatment
Anal Dysplasia
Precancerous Conditions
Human Papilloma Virus
Keywords: HPV
suppository
Artesunate
artemisinin
anal dysplasia
treatment
alternative treatment
anal precancer
Johns Hopkins
Sandy Fang
Frantz Viral Therapeutics
Wisconsin
Evie Carchman
Lead sponsor: Johns Hopkins University
Sponsor class: OTHER
Collaborators: name: Frantz Viral Therapeutics, LLC
name: University of Wisconsin, Madison
First posted: 2017-04-04
Enrollment: 18
Sex: ALL
Minimum age (years): 18
Maximum age (years): 100
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Intervention model description: Subjects will be enrolled sequentially in each treatment cohort.
Primary purpose: TREATMENT

Brief summary: This open label study investigates a novel non-surgical approach to the treatment of HPV-associated anal intraepithelial neoplasia, using Artesunate suppositories.

Detailed description: Patients diagnosed with AIN 2/3 will be enrolled sequentially in treatment cohorts receiving different doses of Artesunate suppositories administered trans-anally. Doses of escalation will be 200 mg, 400 mg, and 600 mg. Treatment cohorts will consist of 2 or 3 cycles at each dose level. Suppositories will be administered daily for 5 days. Five days constitutes 1 cycle. Up to 3 cycles will be administered at Weeks 0, 2, and 4.

Eligibility criteria: Inclusion Criteria:

* Age ≥ 18 years
* Biopsy-confirmed high-grade anal dysplasia (AIN 2, AIN 3, HSIL) by high resolution anoscopy (HRA)
* Female of childbearing potential: negative urine pregnancy test
* Able to provide informed consent
* Patients who have the ability to collaborate with planned follow-up (transportation, compliance history, etc.).
* Weight ≥50 kg.

Exclusion Criteria:

* Diagnosis of low-grade anal dysplasia (AIN 1, LSIL) by high resolution anoscopy
* Known anal, vulvar, cervical, or penile cancer
* CD4 count \< 200 at the time of consideration for entry into this study
* 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

Primary outcomes:
measure: Incidence of treatment-emergent adverse events (Safety and tolerability) of Artesunate suppositories for the treatment of anal intraepithelial neoplasia (AIN2/3)
timeFrame: 6 weeks from the date of the first dosing
Secondary outcomes:
measure: Number of patients with histologic regression of anal intraepithelial neoplasia (AIN2/3) to AIN1 or less
timeFrame: 16 weeks
measure: Number of patients with histologic regression of anal intraepithelial neoplasia (AIN2/3) to AIN1 or less
timeFrame: 28 weeks
measure: Number of patients with viral clearance of human papillomavirus (HPV) virus measured by HPV genotyping
timeFrame: 40 weeks
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: Johns Hopkins Hospital
state: Maryland
city: Madison
country: United States
facility: University of Wisconsin Carbone Cancer Center
state: Wisconsin
Study officials:
affiliation: Johns Hopkins University
name: Sandy H Fang, MD
role: PRINCIPAL_INVESTIGATOR
References: