Pharmacodynamics, Safety/Tolerability and Efficacy of Topical Omiganan in Patients With uVIN

Study Overview
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Brief Summary
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To assess the pharmacodynamics, safety/tolerability and efficacy of topical Omiganan (CLS001) in patients with usual type vulvar intraepithelial neoplasia (uVIN).

Official Title
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A Phase 2, Randomized, Double-Blind, Parallel-Group Study to Assess the Pharmacodynamics, Safety/Tolerability and Efficacy of Topical Omiganan in Patients With Usual Type Vulvar Intraepithelial Neoplasia

Conditions
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Usual Type Vulval Intraepithelial Neoplasia (uVIN)

Intervention / Treatment
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* Drug: Omiganan (CLS001) topical gel
* Drug: Vehicle topical gel


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Women ≥ 18 years Biopsy proven uVIN, biopsies to have been taken within the last three months Written informed consent to participate in the trial At least one lesion that can be accurately measured (using RECIST criteria) in at least one dimension with longest diameter ≥ 20mm OR in two perpendicular dimensions that when multiplied together give a surface area of ≥ 120mm2 (e.g. 15mm x 8mm or 12mm x 10mm) This is to ensure that 4x4mm biopsies can be performed on this lesion. Exclusion Criteria: Has any concomitant disease or significant medical conditions that would, in the opinion of the Investigator, potentially compromise the safety or compliance of the patient or may preclude the patient's successful completion of the clinical trial. Clinically significant abnormalities, as judged by the Investigator, in laboratory test results (including hepatic and renal panels, complete blood count, chemistry panel and urinalysis) or ECG. In the case of uncertain or questionable results, tests performed during screening may be repeated before randomization to confirm eligibility or judged to be clinically irrelevant for healthy subjects. Indication of a current active infectious disease of the vulva, other than HPV Pregnant, breast feeding or trying to conceive Active treatment for uVIN (i.e. surgical excision, lasertherapy, imiquimod, photodynamic therapy) within the previous month Patients receiving immunosuppressive therapy HIV positive or transplant patients Any condition that in the opinion of the investigator could interfere with the conduct of the study

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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Female

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Omiganan (CLS001)<br>CLS001 topical gel, 2.5% | Drug: Omiganan (CLS001) topical gel<br> <br> |
| Placebo Comparator: Vehicle<br>Vehicle topical gel | Drug: Vehicle topical gel<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pharmacodynamics (HPV Viral Load Assessment) | Assessment of target lesions by quantitative PCR including HPV genotyping in swabs and biopsies | 24 Weeks | 
| Pharmacodynamics (Local Immunity Status) | Histological changes in immune cells in the mucosa/submucosa | 24 Weeks | 
| Clinical Assessment (Lesions by RECIST) | Efficacy assessment of lesions by RECIST | 24 Weeks | 
| Clinical Assessment (Percent clearance of Lesions) | Efficacy assessment of percent clearance of lesions | 24 Weeks | 
| Clinical Assessment (Sum of the longest diameter (SLD)) | Efficacy assessment of the sum of the longest diameter (SLD)) | 24 Weeks | 
| Clinical Assessment (Histology) | Efficacy assessment of the histology (regression of uVIN to no dysplasia) | 24 Weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and Tolerability (Adverse Events) | Adverse Events will be collected throughout the study | 48 Weeks | 
| Safety and Tolerability (Laboratory Safety Testing) | Laboratory Samples will be collected throughout the study | 48 Weeks | 
| Safety and Tolerability (12-Lead ECGs) | 12-Lead ECGs will be performed throughout the study | 48 Weeks | 
| Safety and Tolerability (Vital Signs) | Vital Signs will be collected throughout the study | 48 Weeks | 
| Pharmacokinetics (Area Under the Curve) | AUC will be computed | 12 Weeks | 
| Pharmacokinetics (Maximum Plasma Concentration) | Cmax will be determined | 12 Weeks | 
| Pharmacokinetics (Tmax) | Tmax will be determined | 12 Weeks |