Zopa Retreatment and Vector Shedding in Adults With RRP

December 11, 2025 updated by: Precigen, Inc

Open-Label Study of Zopapogene Imadenovec Retreatment and Vector Shedding Evaluation in Adult Patients With Recurrent Respiratory Papillomatosis

This open-label study evaluates safety, vector shedding, and retreatment efficacy of Zopapogene imadenovec (Zopa) in adults with recurrent respiratory papillomatosis (RRP). Two cohorts will be enrolled (n=30): Cohort 1 to assess the magnitude and duration of adenoviral vector shedding in urine, feces, skin, and nasal tissue; Cohort 2 to assess the complete response rate following retreatment.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

This is a single-arm, open-label confirmatory study of PRGN-2012 in RRP patients with a history of repeated surgical debulking for the management of their disease. Patients will be treated with PRGN-2012 and followed for at least 12 months to evaluate for efficacy and safety endpoints.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
30

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • United States
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Recruiting
        • Winship Cancer Institute, Emory University
        • Principal Investigator:
          • Nabil Saba, MD
        • Contact:
    • Maryland
      • Bethesda, Maryland, United States, 20892
        • Recruiting
        • National Institute of Health
        • Contact:
          • Scott Norberg, DO
        • Contact:
    • Ohio
      • Cincinnati, Ohio, United States, 45267
        • Recruiting
        • University of Cincinnati
        • Principal Investigator:
          • Aaron Friedman, MD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  • Age 18 years and older.
  • Clinical diagnosis of recurrent respiratory papillomatosis with histological confirmation of papilloma.
  • Cohort 1: Treatment-naïve with respect to Zopa.
  • Cohort 2: Received a minimum of four administrations of Zopa at 5 × 10^11 PU per injection and require clinically indicated debulking procedures.
  • Presence of laryngotracheal papillomas accessible for endoscopic cleanout.
  • ECOG performance status 0 or 1.
  • Sexually active participants of reproductive potential must agree to use contraception during treatment and for 120 days for males and 6 months for females after last dose.
  • Ability to understand and sign informed consent.

Key Exclusion Criteria:

  • Conditions or therapies that increase risk or interfere with participation per investigator judgment.
  • Systemic corticosteroids >10 mg prednisone equivalent or other immunosuppressive medications within 14 days prior to dosing.
  • Other systemic RRP treatments or investigational agents within 30 days.
  • History of heparin-induced thrombocytopenia or vaccine-induced thrombotic thrombocytopenia.
  • Active uncontrolled HIV, hepatitis B, or hepatitis C infection.
  • Pregnant or nursing women.
  • Known allergy to any study drug component.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Cohort 1 Vector Shedding
Zopa administered subcutaneously at 5 × 10^11 particle units on Day 1, Week 2, Week 6, and Week 12. Samples for vector shedding to be collected from urine, feces, skin, and nasal tissue.
Drug: Zopapogene imadenovec (Zopa)
Zopa will be administered as 4 subcutaneous administrations over a 12-week interval.
Experimental: Cohort 2 Retreatment
Patients with prior treatment with Zopa (5 × 10^11 PU per injection) and require clinically indicated debulking procedure
Drug: Zopapogene imadenovec (Zopa)
Zopa will be administered as 4 subcutaneous administrations over a 12-week interval.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Cohort 1 Vector shedding
Time Frame: Up to 6 weeks following last administration, with additional collections if needed until two consecutive measurements at or below limit of detection
Magnitude and duration of Zopa vector shedding measured in urine, fecal, skin, and nasal tissue
Up to 6 weeks following last administration, with additional collections if needed until two consecutive measurements at or below limit of detection
Cohort 2 Complete Response Rate
Time Frame: 12 months following last administration
Percentage of subjects achieving a complete response defined as no clinically indicated debulking procedures during the 12 months following completion of Zopa retreatment.
12 months following last administration

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Interval to First Debulking Procedure (Cohort 2)
Time Frame: 3 years following last administration
Determine the interval to the first debulking procedure following retreatment with Zopapogene imadenovec (Zopa).
3 years following last administration
Safety and Tolerability of Zopa (Cohort 1 and Cohort 2)
Time Frame: Up to 28 days after last administration, with extended monitoring during follow-up.
Assess the safety and tolerability of Zopa in adult subjects with recurrent respiratory papillomatosis (RRP), including incidence and severity of adverse events graded per CTCAE v5.0.
Up to 28 days after last administration, with extended monitoring during follow-up.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Precigen, Inc

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Amy Lankford, PhD, Precigen, Inc

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 11, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 2, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 2, 2028

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
August 1, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 1, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 5, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
December 15, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 11, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
December 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • PRGN-2012-301

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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