Zopa Retreatment and Vector Shedding in Adults With RRP

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: Recruiting
• Conditions: Recurrent Respiratory Papillomatosis; Papillomavirus Infection; Papillomaviridae
• Intervention / Treatment: Drug: Zopapogene imadenovec (Zopa)

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: Interventional
• Enrollment (Estimated): 30
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Head of Clinical Operations; Phone Number: 301-556-9900; Email: clinicaltrials@precigen.com

Study Locations

• United States
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Recruiting
      • Winship Cancer Institute, Emory University
      • Principal Investigator: Nabil Saba, MD
      • Contact: Mosope Oyewole, CRC; Phone Number: 404-778-5351; Email: mdoyewo@emory.edu
  • Maryland
    • Bethesda, Maryland, United States, 20892
      • Recruiting
      • National Institute of Health
      • Contact: Scott Norberg, DO
      • Contact: Shannon Householder, RN; Phone Number: 240-656-8771; Email: Shannon.householder@nih.gov
  • Ohio
    • Cincinnati, Ohio, United States, 45267
      • Recruiting
      • University of Cincinnati
      • Principal Investigator: Aaron Friedman, MD
      • Contact: Rebecca Reinert; Phone Number: 513-558-1719; Email: gibsonrk@ucmail.uc.edu

Participation Criteria

Eligibility Criteria

• 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

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
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

Outcome Measure	Measure Description	Time Frame
Cohort 1 Vector shedding	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	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

Outcome Measure	Measure Description	Time Frame
Interval to First Debulking Procedure (Cohort 2)	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)	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

• Sponsor: Precigen, Inc
• Investigators: Study Director: Amy Lankford, PhD, Precigen, Inc

Study record dates

Study Major Dates

• Study Start (Actual): July 11, 2024
• Primary Completion (Estimated): December 2, 2027
• Study Completion (Estimated): December 2, 2028

Study Registration Dates

• First Submitted: August 1, 2024
• First Submitted That Met QC Criteria: August 1, 2024
• First Posted (Actual): August 5, 2024

Study Record Updates

• Last Update Posted (Actual): December 15, 2025
• Last Update Submitted That Met QC Criteria: December 11, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: Human Papilloma Virus; Retreatment; Viral Shedding; laryngotracheal disease; papillomatous disease; Zopapogene imadenovec
• Additional Relevant MeSH Terms: Urogenital Diseases; Genital Diseases; Pathologic Processes; Disease Attributes; Infections; Virus Diseases; Communicable Diseases; Sexually Transmitted Diseases, Viral; Sexually Transmitted Diseases; DNA Virus Infections; Tumor Virus Infections; Pathological Conditions, Signs and Symptoms; Papillomavirus Infections; Recurrent respiratory papillomatosis
• Other Study ID 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