Recurrent Respiratory Papillomatosis (RRP)Phase 3NCT07901738
Zopapogene Imadenovec for the Treatment of Recurrent Respiratory Papillomatosis in Children
A Phase 3 study testing Zopapogene imadenovec-drba in people with Recurrent Respiratory Papillomatosis (RRP) and Juvenile-onset Recurrent Respiratory Papillomatosis, run by Precigen, Inc at 1 site in United States.
Trial summary
Recurrent respiratory papillomatosis (RRP) is a rare disease in which wart-like growths caused by human papillomavirus (HPV) types 6 or 11 form in the airway. These growths can affect the voice and breathing and often come back after they are removed, meaning many children will need repeated surgeries to keep their… Read more about this study
- Who is it for?
- Ages 1 year to 17 years
- Conditions the study is for
- Recurrent Respiratory Papillomatosis (RRP), Juvenile-onset Recurrent Respiratory Papillomatosis, Papillomavirus Infections
- What is being tested?
- Zopapogene imadenovec-drba
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Dec 2026to Jan 2033
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 1 year to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age 1 to 17 years
- Clinical diagnosis of RRP with histologically confirmed papilloma
- Laryngotracheal papillomas accessible for endoscopic debulking
- At least 3 lifetime debulking procedures (the Day -1 procedure counts)
- Active RRP, shown by at least 1 clinically indicated debulking procedure within the 12 months before treatment, including the Day -1 procedure. For participants who received systemic bevacizumab, the qualifying procedure must show recurrence of active disease after bevacizumab was stopped; if the Day -1 procedure is used, it must be clinically indicated for recurrent disease.
- Willing to undergo endoscopic evaluation, operative interventions, and biopsies per protocol
Who cannot join
- Any condition, therapy, or lab abnormality that would increase participation risk, impair receipt of protocol therapy, or confound interpretation of results, per the Investigator
- Systemic corticosteroids (>2 mg/kg/day or >10 mg/day prednisone equivalent) or other immunosuppressants within 14 days of dosing (inhaled, topical, intranasal, intra-ocular, and adrenal replacement doses permitted)
- Any systemic or intralesional RRP treatment, including bevacizumab and investigational agents, within 30 days or 3 half-lives of the prior treatment, whichever is longer
- Any vaccine within 30 days before the first dose
- Persisting Grade >1 toxicity from prior therapy (alopecia and Grade ≤2 events not posing a safety risk are acceptable)
- History of heparin-induced thrombocytopenia (HIT) or vaccine-induced immune thrombotic thrombocytopenia (VITT)
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
Children's National Hospital
Washington D.C., District of Columbia, 20010
Pamela Mudd, MD MBA FAAP, 202-476-4852, PMudd@childrensnational.org
Pamela Mudd, MD MBA FAAP
About this study
Official title: An Open-Label Study of Zopapogene Imadenovec for the Treatment of Recurrent Respiratory Papillomatosis in Pediatric Patients
Recurrent respiratory papillomatosis (RRP) is a rare disease in which wart-like growths caused by human papillomavirus (HPV) types 6 or 11 form in the airway. These growths can affect the voice and breathing and often come back after they are removed, meaning many children will need repeated surgeries to keep their airway open. There is no medicine approved to treat RRP in children.
This study is testing zopapogene imadenovec, an investigational immunotherapy designed to help the immune system recognize and control cells infected with HPV 6 and 11. Zopapogene imadenovec is approved in the United States for the treatment of adults with recurrent respiratory papillomatosis.
Read more
All participants in this study will receive zopapogene imadenovec; there is no placebo group. The study drug is given as four injections under the skin over about three months. Before the first injection, participants have a standard surgical procedure to remove visible growths. Participants are then followed for at least 12 months to see how many need further surgery, and are contacted periodically for up to five years after their last injection.
The study will enroll children in two age groups, starting with older children and opening to younger children only after safety has been reviewed.
- Conditions
- Recurrent Respiratory Papillomatosis (RRP), Juvenile-onset Recurrent Respiratory Papillomatosis, Papillomavirus Infections
- Being tested
- Zopapogene imadenovec-drba
- Medical subject headings
- Recurrent respiratory papillomatosis, Papillomavirus Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Zopapogene imadenovec-drbaBiological
Zopapogene imadenovec
| Study group | Role | What they receive |
|---|---|---|
| Zopapogene imadenovec | Experimental | Participants receive four subcutaneous administrations of zopapogene imadenovec on Day 1, Week 2, Week 6, and Week 12, each in a different limb. Participants aged ≥6 to <18 years receive 5 x 10^11 particle units per injection; participants aged ≥1 to ≤5 years begin at 2.5 x 10^11 particle units per injection with sentinel dosing. All participants undergo a standard-of-care debulking procedure on Day -1. Receives: Biological: Zopapogene imadenovec-drba |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete response rate
Percentage of participants with a complete response, defined as no requirement for any clinically indicated debulking intervention during the 12 months following the last administration of zopapogene imadenovec.
Measured: 12 months following last administration of study drug
Secondary outcomes (3)
Number of participants with adverse events (AEs) and treatment-related AEs
Incidence and severity of all AEs and of AEs assessed as related to zopapogene imadenovec, graded by NCI CTCAE version 6.0.
Measured: From first administration through 12 months after the last administration (treatment-related AEs through 28 days after the last administration)
Time to first clinically indicated debulking intervention (duration of response)
Interval from completion of zopapogene imadenovec treatment until the first clinically indicated debulking intervention, estimated by the Kaplan-Meier method.
Measured: From the last administration (Week 12) up to 5 years
Objective response rate (ORR)
Percentage of participants with a complete response or a partial response. Partial response is defined as at least a 50% decrease in the number of clinically indicated debulking interventions during the 12 months after treatment compared with the 12 months before treatment.
Measured: 12 months before the first administration and 12 months after the last administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Oct 2026
- Study start31 Dec 2026 (estimated)
- Primary completionJan 2029 (estimated)
- Study completionJan 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Recurrent Respiratory Papillomatosis (RRP)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Precigen, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07901738
- Sponsor's protocol ID
- PRGN-2012-302
- Status last verified
- Oct 2026
- First submitted
- 21 Oct 2026
- Last updated
- 28 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Study of Zopapogene Imadenovec for the Treatment of Recurrent Respiratory Papillomatosis in Pediatric Patients
- ClinicalTrials.gov ID
- NCT07901738
- Acronym / sponsor ID
- PRGN-2012-302
- Conditions
- Recurrent Respiratory Papillomatosis (RRP), Juvenile-onset Recurrent Respiratory Papillomatosis, Papillomavirus Infections
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Precigen, IncIndustry (pharma, biotech, devices)
- Study start
- 31 Dec 2026 (estimated)
- Primary completion
- Jan 2029 (estimated)
- Study completion
- Jan 2033 (estimated)
- First posted
- 28 Oct 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Recurrent respiratory papillomatosis (RRP) is a rare disease in which wart-like growths caused by human papillomavirus (HPV) types 6 or 11 form in the airway. These growths can affect the voice and breathing and often come back after they are removed, meaning many children will need repeated surgeries to keep their airway open. There is no medicine approved to treat RRP in children.
This study is testing zopapogene imadenovec, an investigational immunotherapy designed to help the immune system recognize and control cells infected with HPV 6 and 11. Zopapogene imadenovec is approved in the United States for the treatment of adults with recurrent respiratory papillomatosis.
All participants in this study will receive zopapogene imadenovec; there is no placebo group. The study drug is given as four injections under the skin over about three months. Before the first injection, participants have a standard surgical procedure to remove visible growths. Participants are then followed for at least 12 months to see how many need further surgery, and are contacted periodically for up to five years after their last injection.
The study will enroll children in two age groups, starting with older children and opening to younger children only after safety has been reviewed.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Zopapogene imadenovec | Experimental | Biological: Zopapogene imadenovec-drbaParticipants receive four subcutaneous administrations of zopapogene imadenovec on Day 1, Week 2, Week 6, and Week 12, each in a different limb. Participants aged ≥6 to <18 years receive 5 x 10^11 particle units per injection; participants aged ≥1 to ≤5 years begin at 2.5 x 10^11 particle units per injection with sentinel dosing. All participants undergo a standard-of-care debulking procedure on Day -1. |
Who may qualify
- Ages
- 1 year to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Age 1 to 17 years
- Clinical diagnosis of RRP with histologically confirmed papilloma
- Laryngotracheal papillomas accessible for endoscopic debulking
- At least 3 lifetime debulking procedures (the Day -1 procedure counts)
- Active RRP, shown by at least 1 clinically indicated debulking procedure within the 12 months before treatment, including the Day -1 procedure. For participants who received systemic bevacizumab, the qualifying procedure must show recurrence of active disease after bevacizumab was stopped; if the Day -1 procedure is used, it must be clinically indicated for recurrent disease.
Key exclusion criteria (6 listed)
- Any condition, therapy, or lab abnormality that would increase participation risk, impair receipt of protocol therapy, or confound interpretation of results, per the Investigator
- Systemic corticosteroids (>2 mg/kg/day or >10 mg/day prednisone equivalent) or other immunosuppressants within 14 days of dosing (inhaled, topical, intranasal, intra-ocular, and adrenal replacement doses permitted)
- Any systemic or intralesional RRP treatment, including bevacizumab and investigational agents, within 30 days or 3 half-lives of the prior treatment, whichever is longer
- Any vaccine within 30 days before the first dose
- Persisting Grade >1 toxicity from prior therapy (alopecia and Grade ≤2 events not posing a safety risk are acceptable)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Zopapogene imadenovec-drbaZopapogene imadenovec | Biological | Zopapogene imadenovec |
Primary outcome
Complete response rate
Time frame: 12 months following last administration of study drug
Percentage of participants with a complete response, defined as no requirement for any clinically indicated debulking intervention during the 12 months following the last administration of zopapogene imadenovec.
3 secondary outcomes
Number of participants with adverse events (AEs) and treatment-related AEs
Time frame: From first administration through 12 months after the last administration (treatment-related AEs through 28 days after the last administration)
Time to first clinically indicated debulking intervention (duration of response)
Time frame: From the last administration (Week 12) up to 5 years
Objective response rate (ORR)
Time frame: 12 months before the first administration and 12 months after the last administration
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2026.
- Individual participant dataSharing planned: Undecided
Related trials for Recurrent Respiratory Papillomatosis (RRP)
Not yet recruitingPhase 2
Combination Bevacizumab and PRGN-2012 in Adults With Recurrent Respiratory Papillomatosis (RRP)
Respiratory Recurrent Papillomatosis (RRP), Human Papillomavirus (HPV)
Not yet recruitingPhase 1/2
Efficacy of Booster Injection With Gardasil-9 Vaccine on Intersurgical Interval* for Adults With Recurrent Respiratory Papillomatosis*
Recurrent Respiratory Papillomatosis (RRP)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Zopapogene imadenovec-drba in people with Recurrent Respiratory Papillomatosis (RRP) and Juvenile-onset Recurrent Respiratory Papillomatosis, run by Precigen, Inc at 1 site in United States.
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 6 inclusion and 6 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: opening soon, as of the sponsor's last update on 28 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Eliza Argonza-Aviles (240-961-9869, clinicaltrials@precigen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Complete response rate (time frame: 12 months following last administration of study drug). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 28 Oct 2026; AzTrials last refreshed its copy on 8 Nov 2026.