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Anal NeoplasmPhase 2NCT03499795

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

A Phase 2 study testing VGX-3100 and CELLECTRA™ 5PSP in people with Anal Neoplasm, run by Inovio Pharmaceuticals at 3 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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)… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Anal Neoplasm
What is being tested?
VGX-3100 and CELLECTRA™ 5PSP
Could I receive a placebo?
No, everyone receives an active treatment
How many people
23 participants
Where
3 sitesin 2 countries
Study dates
15 May 2018to 26 May 2021
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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

  • 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).

Who cannot join

  • 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;
Show all 20Show fewer
  • 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;
  • Significant acute or chronic medical illness that could be negatively impacted by the electroporation treated as deemed by the Investigator;
  • Current or history of clinically significant, medically unstable disease which, in the judgment of the investigator, would jeopardize the safety of the subject, interfere with study assessments or endpoint evaluation, or otherwise impact the validity of the study results;
  • Prior major surgery within 4 weeks of Day 0;
  • Participation in an interventional study with an investigational compound or device within 4 weeks of signing the ICF;
  • Any illness or condition that in the opinion of the Investigator may affect the safety of the subject or the evaluation of any study endpoint.

Trial locations and contacts

3 sites in 2 countries.

3 sites in 2 countries

United States2 sites

  • Howard Brown Health (HBH)-Sheridan

    Chicago, Illinois, 60613

  • Laser Surgery Care

    New York, New York, 10011

Other trials in United States

Canada1 site

  • Clinique de Recherche en Sante

    Québec, Quebec, G1S2L6

Other trials in Canada

About this study

Official title: A Phase 2, Open Label, Study of 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), (AIN2, AIN3, PAIN2, PAIN3) in Individuals That Are Seronegative for Human Immunodeficiency Virus (HIV)-1/2

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
Being tested
VGX-3100, CELLECTRA™ 5PSP
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)
Medical subject headings
Anus Neoplasms, Squamous Intraepithelial Lesions

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • VGX-3100Biological

    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.

  • CELLECTRA™ 5PSPDevice

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

Study groups in NCT03499795
Study groupRoleWhat they receive
VGX-3100ExperimentalAdult 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.
Receives: Biological: VGX-3100; Device: CELLECTRA™ 5PSP

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

    Measured: Week 36

Secondary outcomes (10)

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

    An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can include any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. A TEAE is any AE either reported for the first time or worsening of a pre-existing event after the first dose of study drug.

    Measured: 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)

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

    An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can include any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.

    Measured: From first injection up to Week 88

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

    Measured: Week 36

  4. 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

    Measured: Week 36

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

    Measured: Week 36

  6. Percentage of Participants With no Evidence of Anal or Anal/Peri-Anal Low-Grade Squamous Intraepithelial Lesion (LSIL) or HSIL at Week 36

    Measured: Week 36

  7. Percentage of Participants With no Progression of Anal or Anal/Peri-Anal HSIL to Carcinoma From Baseline to Week 36

    Measured: From Baseline to Week 36

  8. Percent Change From Baseline in the Number of Intra-Anal and/or Peri-Anal Lesions as Determined by the Investigator at Weeks 36, 64, and 88

    Measured: From Baseline to Weeks 36, 64, and 88

  9. Mean Change From Baseline in Frequency of HPV-16/17 E6/E7-specific CD8+/CD137+ Peripheral Blood Mononuclear Cells (PBMCs) That Were Perforin Positive at Week 15

    PBMCs were isolated from whole blood samples. The assessment of cellular immune activity occurred via the application Flow Cytometry for the purposes of performing a Lytic Granule Loading Assay. The Lytic Granule Loading assay examined cluster of differentiation (CD)8/CD137 cellular markers and Perforin (proteins involved in lytic degranulation and cytotoxic potential) intracellular marker. The frequency was presented as number of perforin-positive CD8 i.e., CD8+/ CD137+ PBMCs cells per million CD8+/ CD137+ PBMCs cells.

    Measured: Baseline and Week 15

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

    Measured: Week 36

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Apr 2018
  2. Study start15 May 2018
  3. Primary completion16 Jun 2020
  4. Study completion26 May 2021

Resources

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
23 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03499795
Sponsor's protocol ID
HPV-203
Status last verified
Jul 2023
First submitted
9 Apr 2018
Last updated
14 Jul 2023
FDA-regulated drug
Yes
FDA-regulated device
Yes
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing VGX-3100 and CELLECTRA™ 5PSP in people with Anal Neoplasm, run by Inovio Pharmaceuticals at 3 sites in 2 countries.

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 8 inclusion and 20 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: finished, as of the sponsor's last update on 14 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States, Canada. 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. 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?

Yes. Results are posted in the registry (first posted 3 Jul 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Percentage of Participants With no Histologic Evidence of Anal or Anal/Peri-Anal HSIL and no Evidence of HPV-16/18 at Week 36 (time frame: Week 36). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03499795.

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 14 Jul 2023.

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