Clinical trial NCT03792516: Artesunate Ointment for the Treatment of High Grade Vulvar Intraepithelial Neoplasia (HSIL VIN2/3)
Official title: A Phase I Proof-of-Concept Study of Artesunate Ointment for the Treatment of Patients With High-Grade Vulvar Intraepithelial Neoplasia (HSIL VIN 2/3)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Vulvar Dysplasia
HPV-Related Vulvar Intraepithelial Neoplasia
Preinvasive Vulvar Disease
Vulva Intraepithelial Neoplasia
Vulvar Diseases
Keywords: artesunate
pre-invasive disease
treatment
alternative
non-surgical
cancer prevention
Lead sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Sponsor class: OTHER
Collaborators: name: Frantz Viral Therapeutics, LLC
First posted: 2019-01-03
Enrollment: 15
Sex: FEMALE
Minimum age (years): 18
Maximum age (years): 100
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: This is a Phase I, proof-of-concept treatment study to evaluate the safety, tolerability and feasibility of topical artesunate ointment to treat high grade vulvar intraepithelial neoplasia (HSIL VIN2/3).

Detailed description: Phase I open-label dose escalation study of topical artesunate, formulated as ointment, in the treatment of adult females with biopsy-confirmed HSIL VIN2/3. Fifteen (15) subjects will undergo up to a total of three cycles of topical artesunate. The first cycle will be initiated on Day 0, the second at Week 2, and the third and final cycle at week 4.

Primary Objective:

To evaluate the safety and tolerability of artesunate ointment applied topically on VIN2/3 lesions

To measure the effect of artesunate ointment on histologic regression of HSIL/VIN2/3 to VIN1 or less, confirmed by a colposcopically-directed biopsy, at study week 15.

Eligibility criteria: Inclusion Criteria:

* Colposcopically-directed biopsy diagnosis of high-grade vulvar dysplasia (VIN2, VIN3, VIN2/3, HSIL), including both new and recurrent disease.
* Females of childbearing potential: negative urine pregnancy test
* Ability to provide informed consent
* Ability to collaborate with planned follow-up (transportation, compliance history, etc.)
* Use of contraception through the study exit visit (week 28)

Exclusion Criteria:

* Concomitant use of any topical immune modulating agents e.g. imiquimod, Aldara ®
* Cluster of differentiation 4 (CD4) count \< 200 at the time of screening for eligibility.
* Unable to provide informed consent
* Currently receiving systemic chemotherapy or radiation therapy for another cancer.
* Pregnant females
* Concurrent dermatological disorders involving the vulva (i.e., herpes, ulceration secondary to Crohn's disease) or vulvar dermatosis (i.e., lichen planus, lichen sclerosus, or lichen simplex chronicus);
* Women weighing less than 50 kg

Primary outcomes:
measure: Safety and tolerability of artesunate ointment applied topically on VIN2/3 lesions as assessed by number of participants experiencing serious adverse events or dose-limiting toxicities
timeFrame: 15 weeks
Secondary outcomes:
measure: Effect of artesunate ointment as assessed by number of participants with regression to VIN1 or less at week 15
timeFrame: 15 weeks
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: Johns Hopkins Outpatient Center
state: Maryland
city: Cleveland
country: United States
facility: Cleveland Clinic Fairview Hospital
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation - Main Campus
state: Ohio
city: Mayfield Heights
country: United States
facility: Cleveland Clinic - Hillcrest Hospital
state: Ohio
Study officials:
affiliation: Johns Hopkins University
name: Cornelia L Trimble, MD
role: PRINCIPAL_INVESTIGATOR
References: