VGX-3100 Delivered Intramuscularly (IM) Followed by Electroporation (EP) for the Treatment of HPV-16 and/or HPV-18 Related Anal or Anal/Peri-Anal, High Grade Squamous Intraepithelial Lesion (HSIL) in Individuals Seronegative for Human Immunodeficiency Virus (HIV)-1/2

NCT03499795 | Completed Phase 2 Results FDA Drug FDA Device

• 1 other identifier: HPV-203
• Study Type: interventional
• Target: 23
• Locations: 2 countries
• Sites: 3

Brief Summary

This is a phase 2, open-label efficacy study of VGX-3100 administered by intramuscular (IM) injection followed by electroporation (EP) in adult men and women who are human immunodeficiency virus (HIV) negative with histologically confirmed anal or anal/peri-anal high-grade squamous intraepithelial lesion (HSIL) associated with human papilloma virus (HPV)-16 and/or HPV-18. Approximately 24 participants will receive at least 3 doses of VGX-3100.

Conditions

Anal Neoplasm

Keywords

High-grade squamous intraepithelial lesions (HSIL); Human papillomavirus (HPV); HPV-16; HPV-18; Anal intraepithelial neoplasia 2 (AIN2); Anal intraepithelial neoplasia 3 (AIN3); Peri-anal intraepithelial neoplasia 2 (PAIN2); Peri-anal intraepithelial neoplasia 3 (PAIN3)

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants With no Histologic Evidence of Anal or Anal/Peri-Anal HSIL and no Evidence of HPV-16/18 at Week 36

  Week 36

Secondary Outcomes (10)

• Number of Participants With at Least One Local and Systemic Treatment-emergent Adverse Event (TEAE) During 7 Days Following Each Dose

  7 days following each dose: Day 0 (Days 0 to 7), Week 4 (Days 22 to 28), Week 12 (Days 78 to 84), and Week 40 (Days 274 to 280)

• Number of Participants With at Least One Adverse Event (AE)

  From first injection up to Week 88

• Percentage of Participants With no Evidence of Anal or Anal/Peri-Anal HSIL at Week 36

  Week 36

• Percentage of Participants With no Evidence of HPV-16/18 From Intra-Anal and/or Peri-Anal Tissue by Type-Specific HPV Testing at Week 36

  Week 36

• Percentage of Participants With no Evidence of HPV-16/18 From Intra-Anal Swab by Specific HPV Testing at Week 36

  Week 36

Other Outcomes (1)

• Percent Change From Baseline in the Size of Peri-Anal Lesions as Determined by the Investigator at Weeks 36, 64, and 88

  From Baseline to Weeks 36, 64, and 88

Study Arms (1)

VGX-3100

EXPERIMENTAL

Adult participants who were HIV negative with histologically confirmed anal or anal/peri-anal HSIL associated with HPV-16 and/or 18, received four 6 mg doses of VGX-3100 as an IM injection on Day 0, Week 4, Week 12, and Week 40 followed immediately by EP using the CELLECTRA™ 5PSP device.

Biological: VGX-3100; Device: CELLECTRA™ 5PSP

Interventions

VGX-3100 BIOLOGICAL

One milliliter (1 mL) VGX-3100 (deoxyribonucleic acid \[DNA\] plasmids encoding E6 and E7 proteins of HPV types 16 and 18) will be injected IM and delivered by EP using CELLECTRA™ 5PSP on Day 0, Week 4 and Week 12, and potentially Week 40.

VGX-3100

CELLECTRA™ 5PSP DEVICE

IM injection of VGX-3100 is followed by EP with the CELLECTRA™ 5PSP device.

VGX-3100

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Negative screening test for HIV-1/2 within 30 days of Dose 1;
• Confirmed anal or anal/peri-anal HPV-16/18 infection at Screening by polymerase chain reaction (PCR) from HSIL specimen;
• Anal tissue specimen/slides for diagnosis must be collected within 10 weeks of first dose of VGX-3100;
• At least one anal or anal/peri-anal (AIN2/3 and/or PAIN2/PAIN3) lesion that is histologically-confirmed as HSIL at Screening;
• Appropriate candidate for histology collection procedures (i.e. excision or biopsy) as judged by the Investigator;
• Female subjects must be post-menopausal, surgically sterile or agree to avoid pregnancy by continued abstinence or use of a contraceptive method with failure rate of less than 1% per year from Screening to one month after last dose of study medication (Week 12 or Week 40)
• Men who could father a child must agree to use at least one form of birth control during or continued abstinence from heterosexual intercourse prior to the study, for the duration of study participation and one month after last dose of study medication.
• Normal Screening electrocardiogram (ECG).

You may not qualify if:

• Untreated micro invasive or invasive cancer;
• Biopsy-proven Vaginal Intraepithelial Neoplasia (VAIN) and not undergoing medical care and/or treatment for VAIN;
• Biopsy-proven Vulvar Intraepithelial Neoplasia (VIN) and not undergoing medical care and/or treatment for VIN;
• Biopsy-proven Cervical Intraepithelial Neoplasia (CIN) 2/3 and not undergoing medical care and/or treatment for CIN;
• Biopsy-proven Penile Intraepithelial Neoplasia (PIN) and not undergoing medical care and/or treatment for PIN;
• Anal or anal/peri-anal HSIL that is not accessible for sampling by biopsy instrument;
• Intra-anal and/or peri-anal lesion(s) that cannot be fully visualized at Screening;
• Inability to have complete and satisfactory high resolution anoscopic exams (HRAs)
• Any treatment for anal or anal/peri-anal HSIL (e.g. surgery) within 4 weeks of Screening;
• Pregnant, breast feeding or considering becoming pregnant within one month following the last dose of study medication;
• Presence of any abnormal clinical laboratory values greater than Grade 1 per Common Toxicity Criteria for Adverse Events (CTCAE) version 4.03 within 45 days prior to Day 0 or less than Grade 1 but deemed clinically significant by the Investigator;
• Immunosuppression as a result of underlying illness or treatment;
• History of previous therapeutic HPV vaccination;
• Receipt of any non-study related non-live vaccine within 2 weeks of any VGX-3100 dose;
• Receipt of any non-study related live vaccine (e.g. measles vaccine) within 4 weeks of any VGX-3100 dose;

Sponsors & Collaborators

• Inovio Pharmaceuticals: lead

Study Sites (3)

Howard Brown Health (HBH)-Sheridan

Chicago, Illinois, 60613, United States

Location

Laser Surgery Care

New York, New York, 10011, United States

Location

Clinique de Recherche en Sante

Québec, Quebec, G1S2L6, Canada

Location

MeSH Terms

Conditions

Anus Neoplasms; Squamous Intraepithelial Lesions

Interventions

VGX-3100

Condition Hierarchy (Ancestors)

Rectal Neoplasms; Colorectal Neoplasms; Intestinal Neoplasms; Gastrointestinal Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Neoplasms; Digestive System Diseases; Gastrointestinal Diseases; Intestinal Diseases; Anus Diseases; Rectal Diseases; Morphological and Microscopic Findings; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Study Director
• Organization: Inovio Pharmaceuticals

clinical.trials@inovio.com

267-440-4237

Study Officials

• Jeffrey Skolnik, MD

  Inovio Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 9, 2018
• First Posted: April 17, 2018
• Study Start: May 15, 2018
• Primary Completion: June 16, 2020
• Study Completion: May 26, 2021
• Last Updated: July 14, 2023
• Results First Posted: July 3, 2023

Record last verified: 2023-07

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1); US (2)